Avalik konsultatsioon vere, kudede ja rakkude Euroopa Liidu regulatsiooni ülevaatamise kohta

SELETUSKIRI Avalik konsultatsioon vere, kudede ja rakkude E uroopa Li idu regulatsiooni ülevaatamise kohta Sissejuhatus Euroopa Komisjon (edaspidi komisjon ) algatas 21. jaanuaril 202 1 . a avaliku konsultatsiooni vere, kudede ja rakkude Euroopa Liidu regulatsiooni ülevaatamise kohta. EL-is reguleerivad valdkonda peamiselt direktiiv 2002/98/EÜ , millega kehtestatakse inimvere ja verekomponentide kogumise, uurimise, töötlemise, säilitamise ja jaotamise kvaliteedi- ja ohutusnõuded ning muudetakse direktiivi 2001/83/EÜ , ning direktiiv 2004/23/EÜ i nimkudede ja -rakkude annetamise, hankimise, uurimise, töötlemise, säilitamise, ladustamise ja jaotamise kvaliteedi- ja ohutusstandardite kehtestamise kohta . Lisaks on täpsemad nõuded kehtestatud erinevate rakendusdirektiividega (nt tehnilised nõuded jälgitavusele ja tõsiste kõrvaltoimete teavitamisele, kudede ja rakkude kodeerimisele). Õigusaktides on sätestatud kvaliteedi- ja ohutusnõuded inimpäritolu vere, kudede ja rakkude töötlemise kõikidele etappidele alates annetamisest kuni kliinilise kasutamiseni patsientide ravis ( nt elundite ja kudede siirdamine, vere ülekanne, kunstlik viljastamine) . Ühtlasi on nende ainete doonorlus kriitilise tähtsusega teatavate ravimite ja meditsiiniseadmete tootmisel. K una õigusaktide vastuvõtmisest on möödas peaaegu 20 aastat, siis ei vasta need enam uutele teadus e ja tehnoloogia arengutele ega toeta innovatsiooni . Puudujääkidele EL i õigusaktides on viida t ud ka 2019.a komisjoni tellimusel läbi viidud hindami se aruandes . 2020.a novembris avaldas komisjon mõjuhindamise teekaardi , mille kohaselt ettepanekud õigusaktide ülevaatamise kohta on kavas esitada 2021.a lõpuks. Teekaardis on peamiste väljakutsetena välja toodud: P atsiendid ei ole piisavalt kaitstud välditavate riskide eest - ELis kehtivad ohutuse- ja kvaliteedinõuded on jäänud ajale jalgu. Kvaliteedi ja ohutuse alaseid soovitusi annavad erinevad organisatsioonid, see võib tekitada segadust ja ebaühtlast rakendamist. Samuti on paljud uued ravimeetodid jäänud reguleerimata, kuna ei ole selge milline regulatsioon nendele peaks kohalduma. Ebavõrdne kvaliteedi- ja ohutuse tase ning takistused piiriüleselt rakkude, kudede, ja vere kättesaadavaks tegemisel – nõuete erinev tõlgendamine ja rakendamine EL riikides toob kaasa barjäärid riikidevahelises koostöös, mille eesmärgiks on parandada juurdepääsu nendele ravimeetoditele. Näiteks puuduvad ühtsed nõuded inspektsioonide läbiviimisele, inspektoritele esitatavatele oskus- ja sõltumatusenõuetele, tegevus- ja kasutuslubade andmisele, ohutusjärelevalvele. D oonorite kaitse ja kunstlikust viljastamisest sündinud laste kaitse on ebapiisav – EL õigusaktides on minimaalselt reguleeritud doonorite kaitse ja jälgimine, nt kõrvalnähtudest teavitamine on ebapiisavalt reguleeritud, samuti munaraku ja spermadoonorite geneetiliste riskide tuvastamise nõuded ei vasta kaasaja tehnilistele võimalustele. Samas on kasvav nõudlus erasektori doonorpankadelt, mis tekitab survet rakkude, kudede ja vere annetamiseks. Õigusraamistik on innovatsioonile jalgu jäänud – kehtivad loamenetlused ei võta arvesse uute teraapiate riski ja kasu suhet. Usalduse puudumine ei toeta innovaatiliste lahenduste ja protse d uuride kasutuselevõtmist. Probleeme on ka meditsiiniseadmete ja ravimite regulatsioonide piirialadele jäävatele teraapiate ja neile kohalduva regulatsiooni määratlemisega. Õigusselguse puudumise ulatus ja selle tagajärjed vajavad põhjalikumat analüüsi . EL on haavatav vere-, kudede-, rakkudega varustamise häirete tõttu – teatud hädavajalike teraapiate osas on EL sõltuvuses impordist, näiteks plasmaderivaatide st ravimite tootmine sõltub peamiselt USA-st imporditavast plasma s t . EL on lähtunud tasuta doonorluse põhimõttest, kuid ei ole ette näinud muid mehhanisme varustatuse tagamiseks. Monitooringusüsteemide puudumise tõttu on keeruline häireid varustamises ette näha ja sellest tulenevaid riske leevendada. Välja on pakutud kolm peamist poliitikavalikut , olenevalt sellest kes ja millisel tasandil kehtestab detailsemad ohutus- ja kvaliteedinõuded : 1) peamiselt vereteenistuste ja koepankade eneseregulatsioonile tuginev lähenemine tugevdatud riikliku järelevalve ja EL audititega 2) tuginemine peamiselt E uroopa tasandi pädevate asutuste (nt ECDC, EDQM ) kehtestatavatele detailsematele nõuetele, koos tugevdatud riikliku järelevalve ja EL audititega 3) detailsete kvaliteedi- ja ohutusnõuete (sh tehnilised reeglid ja spetsifikatsioonid) kehtestamine EL tasandi regulatsiooniga koos tugevdatud riikliku järelevalvega. Poliitikavaliku i d on võimalik omavahel ka kombineerida , olenevalt valdkonnast ja sellest mida reguleeritakse. Valikud võtavad arvesse liikmesriikide pädevust otsustada eetilistes küsimustes, nt vabatahtliku tasuta annetuse põhimõte, doonorite anonüümsus, geneetiline skriinimine jms. Konsultatsiooni eesmärk on koguda huvigruppidelt ja pädevatel asutustelt tagasisidet peamiste probleemkohtade ja võimalike poliitikavalikute kohta. Konsultatsiooni põhiteemadeks on vere, kudede ja rakkude ohutus- ja kvaliteedinõuete kaasajastamine, sellega seotud järelevalve parandamine, innovatsiooni toetamine ning vere, kudede ja rakkude piisava varusta mise tagamine. Avalik konsultatsioon hõlmab kahte erinevat küsitlust, millest üldisem on suunatud kõikidele huvigruppidele ja teine, spetsiifilisem küsitlus , valdkonnas tegutsevatele organisatsioonidele ja selle eest vastutavatele pädevatele asutustele . Eesti seisukohad kujundatakse mõlemale küsimustikule vastamiseks. Vastused küsimustikule on toodud seletuskirja lisas. S eletuskirja koostasid S otsiaalministeeriumi ravimiosakonna nõunik Madis Tõns ( [email protected] , 626 9138), Silja Elunurm ( [email protected] , 626 9735 ) ning E uroopa L iidu ja väliskoostöö osakonna nõunik Elen Ohov ( [email protected] , 626 924 1 ). Ekspertidena olid kaasatud Ravimiameti bioloogil i ste preparaatide osakonna juhata Triin Suvi ( [email protected] ) ning spetsialistid Siim Suutre ( [email protected] ) ja Pille Säälik ( [email protected] ) . Valdkonna eest vastutab terviseala asekantsler Maris Jesse. K onsultatsioonile vastamise üldpõhimõtted Eestis reguleerivad valdkonda peamiselt viis seadust ja nende alusel antud rakendusaktid : Vereseadus (RT I 2005, 13, 63; jõustumine 01.05.2005, osaliselt 01.01.2008) Isiku doonoriks sobivuse kriteeriumid, hindamise tingimused ja kord ning vere loovutamist välistavate või piiravate haiguste ja muude tegurite loetelu (RTL 2005, 50, 707; jõustumine 15.05.2005) Verekomponentide valmistamise eeskiri (RT I, 29.07.2016, 6; jõustumine 01.08.2016) Verekomponentide kvaliteedi nõuded, verekomponentide kvaliteedi kontrollimise ja mikrobioloogiliste uuringute tingimused ja kord (RT I, 22.07.2016, 4; jõustumine 25.07.2016) Vereülekande tingimused ja kord (RTL 2005, 50, 704; jõustumine 15.05.2005) Immunohematoloogiliste uuringute tegemise tingimused ja kord (RTL 2005, 50, 703 , jõustumine 15.05.2005) Verevalvsuse ning verekomponentide tagasikutsumise tingimused ja kord (RTL 2005, 111, 1712; jõustumine 13.11.2005) Rakkude, kudede ja elundite hankimise, käitlemise ja siirdamise seadus (RT I, 26.02.2015, 1 ; jõustumine 01.03.2015) Rakkude, kudede ja elundite hankimise ja käitlemise eeskiri (RT I, 13.03.2015, 11; jõustumine 16.03.2015) Raku-, koe- ja elundidoonori valimise kriteeriumid, rakkude, kudede või elundi annetamist välistavate asjaolude loetelu, doonorile ettenähtud kohustuslike laboratoorsete uuringute loetelu ning uuringute tegemise tingimused ja kord (RT I, 20.03.2015, 3; jõustumine 23.03.2015) Rakkude, kudede ja elundite hankimise, käitlemise ja siirdamise aruannete koostamise nõuded, andmete koosseis ning esitamise kord (RT I, 20.03.2015, 4; jõustumine 23.03.2015) Kunstliku viljastamise ja embrüokaitse seadus (RT I 1997, 51, 824 ; jõustumine 17.07.1997) Ravimiseadus (plasmaderivaatide regulatsioon, verekeskuse pädeva isiku nõuded , i nimpäritolu rakkude, kudede ja elundite sisse- või väljave du, Ravimiameti tegevuslubade register ) Nõuded pädeva isiku kvalifikatsioonile ja kvalifikatsiooni tõendavate dokumentide loetelu (RTL 2005, 32, 459; jõustumine 25.03.2005) Ravimiameti eriluba nõudva kauba sisse- ja väljaveo ning ravimite isiklikuks tarbimiseks kaasavõtmise või saatmise tingimused ja kord, erilubade vormid ning Ravimiameti eriluba nõudvate kaupade loetelu ( RTL 2005, 23, 316 , jõustumine 01.03.2005 ) Ravimiameti tegevuslubade registri põhimäärus ( RT I, 21.12.2017, 16 , jõustumine 01.01.2018 ) Nakkushaiguste ennetamise ja tõrje seadus (v eredoonorluse nakkusohutuse tagamine , r akkude, kudede ja elundite hankimise, käitlemise ja siirdamise nakkusohutuse tagamine ) ( RT I 2003, 26, 160 , jõustumine 01.11. 2003 ) Nakkustekitajate suhtes doonorivere ja verekomponentide uurimise kord ( RT I, 27.10.2015, 8 , jõustumine 30.10.2015 ) Raku-, koe- ja elundidoonori valimise kriteeriumid, rakkude, kudede või elundi annetamist välistavate asjaolude loetelu, doonorile ettenähtud kohustuslike laboratoorsete uuringute loetelu ning uuringute tegemise tingimused ja kord ( RT I, 20.03.2015, 3 , jõustumine 23.03.2015 ). Valdkonna peamiste arengusuundade aluseks on rahvastiku tervise arengukava 2030 (RTA) . RTA annab visiooni ja eesmärgid, kuhu poole liikuda järgmise kümnendi jooksul ehk aastaks 2030. RTA üldine suund on toetada nii inimeste tervena elatud eluaastate kui ka eluea pikenemist. Samuti soovitakse jõuda mõtte- ja tegutsemisviisi muutumiseni, kus teenuseid või tooteid kasutavad inimesed on võrdsed partnerid tervishoiusüsteemis, vähendada ebavõrdsust tervises , kaasata enam kogukondi, tõhustada koostöö d valdkondade vahel ning pöörata tähelepanu ka võimalustele, mida pakub innovatsioon ning teadus- ja arendustegevus järgmisel kümnendil. Tervisesüsteem peab tagama teenustele tervikliku lähenemise, mis hõlmab nii tervise edendamist, haiguste ennetamist, integreeritud ravi ning teenusepakkumise korraldust tervishoiu- ja sotsiaalsüsteemi koostöös kui ka teenuseosutajate, asutuste ja süsteemide koordineerimist, olenemata nende kuuluvusest avalikku või erasektorisse. RTA-s tuuakse välja , et inimpäritolu bioloogilise materjali ( rakkude, kudede, elundite ) siirdamine on teatud juhtudel ainus elupäästev ravi viis . Samas on nende kliiniline kasutamine meditsiiniliselt keerukas ning tõsiste riskidega protseduur. Siirdamiseks sobivate rakkude, kudede ja elundite puudus on ülemaailmne probleem, seda nii kliinilises tegevuses kui ka teadusuuringute tegemise eesmärgil. RTA-s tuuakse ühe prioriteedina sekkumiste puhul välja t eadlikkuse suurendamine rakkude, kudede ja elundite doonorlusest. Eestis teostatakse näiteks vereloome tüvirakkude ja silma sarvkesta siirdamisi , vaskulaarkoe ja luukoe käitlemist. Elundite siirdamisel ja elundidoonorlusel on Eestis pikk ajalugu. Eestis teostatakse neeru-, maksa- ja kopsusiirdamist. Euroopa Liidus teevad liikmesriigid koostööd pädevate asutuste võrgustik e kaudu. Eesti t esindab vere, kudede ja rakkude valdkonna võrgustikes pädeva asutuse na Ravimiamet. Ravimiamet kontrolli b , kas verd käitlevad verekeskused järgivad vere seaduses ja selle alusel kehtestatud õigusaktides sätestatud nõudeid. Ravimiamet teeb järelevalvet r akkude, kudede ja elundite hankimise, käitlemise ja siirdamise seadus e ja selle alusel kehtestatud õigusaktides sätestatud rakkude, kudede ja elundite hankimise ja käitlemise, sealhulgas rakkude, kudede ja elundite kvaliteedi ja ohutuse nõuete järgimise üle. Samas tuleb aga arvestada, et riigisiseselt on osa ülesandeid ka Terviseametil. Terviseameti ülesanne on teha järelevalvet rakkude, kudede ja elundite siirdamise nõuete järgimise üle . Samuti peab Terviseamet kontrolli ma , kas verekabinetid järgivad vereseaduses ja selle alusel kehtestatud õigusaktides sätestatud nõudeid, mida kohaldatakse verekomponentide tellimisele, säilitamisele, haiglasisesele väljastamisele, immunohematoloogilistele uuringutele ja vereülekannete raviks kasutamisele ning erakorralistel asjaoludel vere kogumisele. Tulenevalt Terviseameti ülesannetest nakkushaiguste ennetamisel ja tõrjel nakkushaiguste ennetamise ja tõrje seaduse alusel, osaleb koostöös ECDC- ga Terviseamet (sh ka doonormaterjali nakkusohutuse tagamise küsimused). Euroopa Komisjonil on tihe koostöö ka teiste rahvusvaheliste organisatsioonidega nagu Euroopa Nõukogu, millel on samuti olu l ine roll rakkude ja kudede kvaliteedi tehniliste nõuete kehtestamisel ja kaasajastamisel. Lisaks annab ECDC praktilisi juhiseid EL õigusaktide ga kehtestatud nõuete rakendamisel , viib läbi riskihindamisi ja annab soovitusi seoses nakkushaiguste puhangutega, mis võivad mõjutada siirdamiseks mõeldud vere, kudede, rakkude ja elundite kvaliteeti ja ohutust. S eisukohad Euroopa Komisjoni peamiste küsimuste kohta Leiame, et EL vere, rakkude ja kudede alane regulatsioon on oluline, et tagada patsientide ohutuse ühtlane tase kõikides EL riikides. Kehtiv EL regulatsioon on üldjoontes täitnud eesmärki, kuid vajab ajakohastamist teadusliku ja tehnoloogilise arenguga kohandamiseks. Selgitus: Komisjoni läbiviidud ELi õigusaktide hindamise käigus leiti, et mitmed kehtivad ohutus- ja kvaliteedinõuded on aegunud ning tehniliste arengutega sammu pidamine selle s kiiresti muutuva s sektori s on olnud keeruline. Muu tused hõlmavad teaduse ja tehnika arengut (nt paremad doonoritestid, uued töötlemismeetodid, sealhulgas patogeenide inaktiveerimine), digitaalsete vahendite kättesaadavus (seireks), suurenenud turustamine ja sagenevad kliimasoojenemisega seotud epidemioloogilised puhangud (nt Lääne-Niiluse viirus) ning ülemaailmne liikuvus. Mitmed inimpäritolu ained jäävad praegu ver d , kudesid ja rakke käsitlevate EL õigusaktide reguleerimisalast välja , kuid nendega kaasnevad samaväärsed riskid nagu annetatud kudede ja rakkudega. Kui Haiguste Ennetamise ja Tõrje Euroopa Keskus (ECDC) või Euroopa Nõukogu kohandavad oma suuniseid selles valdkonnas regulaarselt, on ELi õiguse s kehtestatud tehniliste nõuete õigeaegne ajakohastamine osutunud ressursimahukaks ja mõnel juhul liiga aeglaseks. Toetame kudede ja rakkude EL regulatsiooni kohaldamisalasse teatud uudse kasutusega inimpäritolu materjalide lisamist ja vajadusel definitsioonide ajakohastamist. Toetame kõigi inimpäritolu materjalide käsitlemist ühtsete põhimõtete alusel , olenemata kas neid kasutatakse inimesel transfusiooniks , transplantatsiooniks või muu l raviotstarbel . Samas ei pea me vajalikuks ainult diagnostikaks, mitte-kliiniliseks teadustööks või õppetööks kasutatava vere, rakkude ja kudede hõlmamist kohaldamisalasse. Selgitus: EL õigusaktide reguleerimisala peaks hõlmama kõiki kliinilisel eesmärgil tehtavaid verekäitlemise etappe ja tegevusi, välja arvatud otseselt tervishoiuteenuse korraldamisega seotud küsimused, mis peavad jääma jätkuvalt liikmesriigi pädevusse. M itmed uudset kasutus leidnud inimpäritolu materjalid on praegu õiguslikult n.ö hallis alas ja nende õiguslik staatus vajab täpsustamist. Vajadus on tingitud tehnoloogilisest arengust ja kliinilistest parktikatest inimpäritolu materjali kasutamises. Inimpäritolu materjali ohutust reguleerivate EL regulatsioonide kohaldamisalasse peaks kuuluma lisaks transfusiooni eesmärgile ka muul kliinilisel eesmärgil kasutatav ad verest valmistatud preparaadid (nt trombotsüütide rikas plasma või se e rum itilgad ). Jätkuvalt ei peaks reguleerimisalasse kuuluma ainult diagnostilisel eesmärgil kogutavad proovid tervishoiuteenuse raames. Sam uti ei pea me vajal ikuks mitte-kliiniliseks teadustööks või õppetööks kasutatava vere, rakkude ja kudede hõlmamist kohaldamisalasse. See on ka praegu kehtiv põhimõte , mis aitab vältida põhjendamatu t halduskoormust. Toetame ka selliste inimpäritolu materjalide hõlmamist, mis praegu ei ole hõlmatud vere, kudede ja rakkude definitsiooniga (nt rinnapiim ja soolestiku mikrobioom ). Arvestades, et EL riikides on neid teemasid erinevalt EL õiguse raames tõlgendatud, oleks vajalik õigusselgus. Tulevikus oleks vaja ette näha õiguslik mehhanism, mis võimaldaks EL õigusaktide kohaldamisala paindlikult laiendada uudsetele toodetele, näiteks pädevate asutuste või EL tasandi ekspertasutuste (näiteks ECDC ) ja Euroopa Nõukogu as u tuste (näiteks EDQM) otsuse alusel. See aitaks vältida nn halli ala tekkimist ning toetaks innovatsiooni. Direktiivides toodud definitsioonid on üldiselt endiselt asjakohased. Siiski peame võimalikuks, et EL õigusaktide ülevaatamise käigus võib tekkida vajadus mõne mõiste täpsustamiseks. Vajadusel ja tehnoloogilistest arengutest lähtuvalt tuleb EL õigusesse lisada uusi definitsioone. Näiteks võiks täpsustada „töötlemise“ ( processing ) mõistet ja välistada mõiste alt juhtumid, kui kudesid ja rakkusid töödeldakse sama kirurgilise protseduuri osana (kudede ja rakkude mehaaniline töötlemine operatsioonisaalis, kui materjali kasutatakse samal patsiendil sama protseduuri käigus). Kui tegemist on aga komplekssema töötlemisega, mis hõlmab näiteks keemiliste ainete või ensüümide kasutamist, siis see peaks olema EL regulatsiooni kohaldamisalas. Vajadusel tuleks kasutada pädeva asutuse õigust otsustada juhtumipõhiselt. Eesti käitlejad on avaldanud muret , et praeguste regulatsioonide juures on üsna raskesti arusaadav, milliseid tegevusi loetakse rakkude hankimiseks ja käitlemiseks direktiivi 2004/23/EC tähenduses ning milliseid tegevusi rakkudel põhinevate uudsete ravimite tootmiseks. Peame vajalikuks EL tasandil täpsustada erinevate regulatsioonide piirialadel olevatele inimpäritolu aine tele ja materjalidele reeglite kohaldamist ning toetame selleks EL tasandi l nõuandva mehhanismi loomist. Selgitus: Paljud inimpäritolu ained ja materjalid (nt seerumtilgad, põlve süstitavad rasvarakud, luuimplantaadid) võivad jääda mitme valdkonna regulatsioonide kohaldamisalasse (meditsiiniseadmed, ravimid, uudsed ravimid ). Sel juhul on oluline, et kõigil osapooltel oleks õigusselgus milliseid nõudeid konkreetsel juhul tuleb kohaldada. Seetõttu on vajalik luua EL tasandi l mehhanism, mis nõustaks pädevaid asutusi ja kõiki teisi osapooli (sh erasektori ettevõtteid) kas aine/toode peaks kuuluma vere-kudede-rakkude EL regulatsiooni kohaldamisalasse ja milliseid nõudeid sellele peaks kohaldama . Sellisesse nõuandvasse kogusse peaksid kuuluma pädevate asutuste ja tervisetehnoloogia hindamise asutuste esindajad, teadus- ja kliinilised eksperdid. Oluline on seejuures tihe koostöö teiste valdkondlike EL regulatsioonide all tegutsevate nõuandvate ekspertgruppidega (nt uudsete ravimite komitee, meditsiiniseadmete klassifitseerimise ja piirialade määratlemise grupp). Oluline on koordineerida piirialade toodetele antavaid soovitusi kohaldatavate nõuete osas ja kokku leppida kriteeriumid, mida kasutatakse õigusliku staatuse otsustamisel Leiame, et doonorite kaitse üld põhimõtted ning üldised kvaliteedi- ja ohutusnõuded tuleks jätkuvalt reguleerida EL õigusakti des . Samas teatud detailsete tehnilis te nõuete ja reeglite kehtestamine võiks tulevikus toimuda senisest enam ka eksperttasandil EL pädeva te asutus te ja võrgustike kaudu, mis võimaldab suuremat paindlikkust nende ajakohastamisel . Peame oluliseks EL õigusaktides kvaliteedi ja ohutusnõuete regulaarset ajakohastamist ning õigusliku mehhanismi loomist nende regulaarse ks ja süsteemse ks ülevaatamise ks . Selgitus: EL ja riigisisestes õigusaktides tuleks jätkuvalt reguleerida olulisemad üldpõhimõtted, mis puudutavad näiteks d oonori tele esitatavaid nõudeid, doonori või tema pereliikmete nõusolek ut , doonorite kaitse ja jälgimise tagamist ning kompensatsiooni reegl e id . Samuti peaks õigusaktides reguleerima vere-, kudede- ja rakkude jälgitavuse nõuded (sh kodeerimine ja märgistamine) ning tõsiste kõrvaltoimete teavitamise ja hindamise nõuded. Oluline on EL õigusaktide läbivaatamise käigus põhjalikumalt analüüsida ka vere, kudede ja rakkude hädaolukorras varustamise tagamiseks ja hädaolukorraks valmisoleku tugevdamiseks vajalikke meetmeid, sh näiteks kohustuslike hädaolukorra plaanide koostamine. Tagamaks verekomponentide ohutust ja kvaliteedisüsteemide toimimist, peab verevalvsuse süsteem jätkuvalt hõlmama kõiki verekäitlemise etappe ja tegevusi (sh peab reguleerimisalasse kuuluma ka tervishoiuteenuse osutamise raames avaldunud rasked kõrvaltoimed). Verevalvsuse paremaks toimimiseks oleks mõistlik, kui ka haiglates aset leidvad ohtlikud kõrvalekalded registreeritakse ning analüüsitakse nende tekkepõhjuseid, rakendatakse vajadusel asjakohaseid korrigeerivaid meetmeid ning juhtumitest teavitataks pädevat asutust, vajadusel ka verekeskust. Selline läbimõeldud tegevus aitaks kaasa vereülekannete oh u tumaks muutmiseks ning tagaks parema kvaliteedisüsteemi toimimise kõikidel vere käitlejatel. EL õigusaktide regulaarseks ajakohastamiseks tuleb ette näha protsess, mis oleks piisavalt paindlik ja ei oleks liiga ajamahukas. Seejuures on oluline, et ajakohastamise protsessi oleks kaasatud valdkonna eksper did ja liikmesriikide pädevad asutused ning tagatud oleks erinevate valdkondade omavaheline koostöö (ravimid, meditsiiniseadmed, inimpäritolu materjalid). Osade praegu EL õ igusakti de s kehtestatud detailsemate nõuete puhul võiks samas kaaluda edaspidi seda, et need nõuded ja reeglid jääksid EL tasandi pädeva te a sutuste (näiteks ECDC, või Euroopa Nõukogu asutuste (näiteks EDQM ) otsustada. See annaks juurde paindlikkust ning nõudeid oleks võimalik operatiivsemalt kohandada vastavalt tehnika ja teaduse arengule. N äiteks võiks EL pädevad asutused kehtestada nõuded töötlemise keskkonnale (nt õhukvaliteet, hoiustamise temperatuur jms ). Samuti detailsemad doonorite meditsiinil is e skriinimise , nakkushaiguste testimise nõuded ja doonorluse välistamise kriteeriumid ei pea olema kehtestatud õigusaktiga. Oluline roll standardite kehtestamisel on EDQM- il . Vere käitlejad on lähtunud kvaliteedisüsteemi ülesehitamisel EDQM-i juhendi „Verekomponentide valmistamise, kasutamise ja kvaliteedi tagamise juhend” hea tava nõuetest ning selles toodud soovitustest. Vajadusel on juhendist lähtutud ka riigisiseste täiendavate nõuete kehtestamisel. Euroopa Liidu õigusesse lisandus alles 2016. aastal viide EDQM-i verekomponentide valmistamise, kasutamise ja kvaliteedi tagamise hea tava nõuetele (kohustusele neid järgida). Kindlasti pole mõistlik, et paralleelselt oleks Euroopas kasutusel mitmeid erinevaid hea tava juhendeid, sest sellega kaasneks lihtsalt ebamõistlik ressursikasutus. EDQMi vere valdkonna juhendeid jälgitakse ka väljapool EL-i, mis teeb olulise lt lihtsamaks vajadusel ka inimpäritolu materjalide liikumise EL-i ja kolmandate riikide vahel. EDQM-i juhendeid (lisaks verele on juhendid ka rakkude ja elundite valdkonnas) on regulaarselt lähtuvalt teaduse ja tehnoloogia arengust ning kliinilisest vajadusest uuendatud. Sellega omakorda on suudetud tagada juhendite ajakohasus. Samuti annab juhend parema võimaluse erinevate tehniliste üksikasjade selgitamiseks ja on seetõttu heaks abiks käitlejatele oma kvaliteedisüsteemi ülesehitamiseks. Lisaks on ECDC-l oluline roll nakkushaiguste ennetamisega seoses , sh doonormaterjali testimisega ning doonoritele kehtestatavate piirangutega seotud küsimustes. Liikmesriikide koostöö selles valdkonnas tagab laiema kompetentsi, mis omakorda aitab kaasa verekomponentide nakkusohutuse tagamisele. Eesti käitlejad on avaldanud arvamust, et direktiivid vajaks süsteemselt kaasajastamist ja ühtlustamist ka andmekaitse aspektist. Ühelt poolt on nõutav, et kõik kogutavad andmed oleksid muudetud anonüümseks nii, et doonorid ega retsipiendid poleks enam identifitseeritavad (2004/23/EC art 14), samas rakkude tõsistest kõrvaltoimetest teatamise kord (2006/86/EÜ lisa III) näeb ette, et pädevale asutusele tuleb edastada info ohustatud isiku (retsipient või doonor) kohta ja vere kõrvaltoimetest teatamise kord (2005/61/EÜ lisa II), et pädevale asutusele tuleb edastada info retsipiendi vanuse ja soo kohta. Analoogseid küsitavusi, millisel ajahetkel ja millise eesmärgiga on koepanga välisel isikul (sh järelevalve teostajal, pädeval asutusel) õigus juurdepääsuks doonorite ja retsipientide isiku- ja terviseandmetele, millal kodeeritud ( pseudonümiseeritud ) andmetele ja millal vaid anonüümsetele andmetele, on direktiivides rohkem. Omaette alateema on koepankade koostöö, sh nii riiklikud, riikidevahelised, üle- euroopalised kui ülemaailmsed registrid, milliseid andmeid võib nendes koguda ja töödelda ning millised turvanõuded peavad seejuures tagatud olema. Toetame liikmesriikide järel e valve tegevuste ühtlustamist ning järelevalvealase pädevate asutuste vahelise koostöö tugevdamist. Leiame, et ELi õigusaktides sätestatud loanõuded on piisavad ja ei toeta tegevusmahu arvesse võtmist tegevusloa andmise kriteeriumina. Samas tuleks õigusaktides täpsustada riiklike tegevuslubade vastastikuse tunnustamisega seonduvat. Selgitus: Usalduse suurendamiseks on vajalik liikmesriikide järelevalveasutuste tegevuse ühtlustamine ja koostöö , sh ühiste inspektsioonide läbiviimine ja vastastikused auditid . Vajalik on ka teatud üldpõhimõtete sätestamine EL õigusaktides, näiteks inspektorite sõltumatus ja huvide konflikti vältimine , inspektorite oskuste ja pädevuse ja piisava haldusvõimekuse tagamine (nt EL auditite läbiviimine haldussuutlikkuse hindamiseks) ning läbipaistvuse suurendamine (näiteks aasta-aruannete avaldamine inspektsioonide, loamenetluste ja ohutusjärelevalve kohta). Pädevate asutuste ametnike oskuste ja teadmiste ajakohasuse tagamiseks on vajalikud nii regulaarsed koolitused (sh rahvusvaheliste ja EL koolituste korraldamine) kui ka osalemine ühiste mitut liikmesriiki hõlmavate inspektsioonide ja auditite läbiviimisel. Üheks võimaluseks on ka EL pädevuste hindamise süsteemi sisseviimine. EL tasandil tegevusloa nõuete täpsustamisega seoses ei pea me põhjendatuks seada autoriseerimise eelduseks tegevuse mahu limi it e . Minimaalse mahu kehtestamine võib tekitada surve kasutada rohkem inimpäritolu materjali kui tegelikkuses oleks kliiniliselt näidustatud . S amuti võib see takistada spetsiifilisele valdkonnale spetsialiseerunud käitlejate tegevust. Lisaks tuleb siinkohal arvestada ka riikide suuruste erinevusega, mis võib löögi alla seada just väiksemate riikide käitlejad. Samuti ei ole vajalik tegevusloa nõude laiendamine sellistele üksustele nagu näiteks luuüdi registrid ja muud registrid, mida kasutatakse kliiniliste tulemuste hindamiseks . Registrite tegevus on reguleeritud muude õigusaktidega, sealhulgas andmekaitsenõuetest lähtuvalt. Eraldi loamenetlust ei ole vaja ka v ahendustegevuseks, mis ei ole otseselt seotud hankimise ja käitlemisega (nt kommertsalustel tegutsevad levitajad ja vahendajad ), kuna see tooks kaasa asjatut halduskoormus t . Samas tuleks õigusaktides täpsustada riiklike tegevuslubade vastastikuse tunnustamisega seonduvat , mis aita ks vähendada halduskoormust ning võimalda ks kiiremat asjaajamist liikmesriikide vahel . Arvestades inimpäritolu materjali iseärasusi, on kiire asjaajamine kriitilise tähtsusega. Toetame Euroopa enesevarustamise printsiipi ja sellele suunatud tegevusi, järgides tasustamata ja vabatahtliku doonorluse põhimõtteid ning doonori tervise kaitset. Selgitus: Toetame põhimõtet, et inimpäritolu materjalid (kaasa arvatud vereplasma) ei ole kaup . Peame oluliseks tasustamata elundi- ja vere doonorluse põhimõtte säilitamist , samas võib olla vajalik diferentseeritud lähenemine eri tüüpi kudedele ja rakkudele (nt sugurakkude annetus) . Selleks, et tagada piisavat varustamist vere, kudede ja rakkudega, tuleks EL õigusaktide läbivaatamisel põhjalikumalt analüüsida ka meetmeid, mis näiteks aitaksid pakkumist planeerida. Selleks võib olla vajalik näiteks monitooringu süsteemide sisseviimine EL tasandil või riigisiseselt, mis võimaldaks nõudluse ja pakkumise andmeid koguda ja analüüsida. Seejuures on oluline vältida liigse halduskoormuse tekkimist , näiteks andmete ja aruannete esitamise kohustustega seoses . Eesti käitlejad on toonud välja , et ühtlustamist vajaksid nõuded ja tingimused doonormaterjali piiriüleseks (õhu)transpordiks . T eema on muutunud eriti aktuaalseks Covid-19 pandeemia tingimustes . Näiteks on küsimusi tekitanud k as ja millal on nõutav turva- ja tollikontroll, millal ja millistel tingimustel võib tooteid transportida regulaar(tsiviil)lennuga, kas tellimuslend doonormaterjali transpordiks kvalifitseerub erakorraliseks meditsiinilennuks , mill e teenindamiseks ollakse valmis lennujaama erakorraliselt avama, kas piiriületus- ja lennujaamatoimingute eest on õigus küsida eritasusid jms. Praegu on nõuded doonormaterjali transpordile riikide, lennujaamade ja lennufirmade lõikes erinevad ja see on toonud kaasa takistusi doonormaterjali piiriülesel transpordil. Arvamuse saamine ja kooskõlastamine Seisukoha koostamise l on arvestatud Ravimiamet i ja valdkonna ekspertide arvamusega. Online Public Consultation on the Revision of the EU Legislation on Blood , Tissues and Cells Fields marked with * are mandatory . Fields marked with * are mandatory . Introduction The European Commission has conducted a comprehensive evaluation of the blood , tissues and cells (BTC) legislation , examining its functioning across the EU and published its findings in October 2019. In particular the evaluation assessed the extent to which the Main Directives met their original objectives and whether they remain fit for purpose , given all that has changed in the intervening period . The evaluation of the legislation , published in October 2019 , confirmed that the legislation had improved safety and quality of blood , tissues and cells used for transfusion , transplantation or medically assisted reproduction . The evaluation also highlighted a number of gaps and short-comings which will be addressed by a revision of the legislation to ensure the framework is up-to-date , fit for purpose and futurep r o o f . The Commission has launched an initiative to revise the legislation , addressing the identified shortcomings . The initiative aims to : update the legislation to provide a more flexible alignment with scientific and technological developments tackle the ( re -) emergence of communicable diseases , including lessons learnt from the COVID-19 pandemic focus on the increasing commercialisation and globalisation of the sector . This public consultation will be an important source of information for the process that will lead to the revision . The consultation does not address changes to other EU legal frameworks but it does explore if there are specific products that do not fall clearly under the blood , tissues and cells framework or the medicines and/ or medical device frameworks . Please note that a more in- depth and technical consultation is open in parallel to this one , for organisations that are directly involved in or impacted by these activities and have a good knowledge of the current legislation . If you are such an organisation , you should complete both this consultation and the targeted one , available on the Santé w eb pages . An external contracted study will also gather evidence and views to support the Impact Assessment . About you * Language of my contribution Bulgarian Croatian Czech Danish Dutch English Estonian - X Finnish French German Greek Hungarian Irish Italian Latvian Lithuanian Maltese Polish Portuguese Romanian Slovak Slovenian Spanish Swedish * I am giving my contribution as Academic / research institution Business association Company/ business organisation Consumer organisation EU citizen Environmental organisation Non-EU citizen Non- governmental organisation (NGO) Public authority - X Trade union Other * First name * Surname * Email ( this won't be published ) * Scope International Local National - X Regional * Level of governance Local Authority Local Agency * Level of governance Parliament Authority X Agency X * Organisation name 255 character (s) maximum * Organisation size Micro (1 to 9 employees ) Small (10 to 49 employees ) Medium (50 to 249 employees ) - X Large (250 or more ) Transparency register number 255 character (s) maximum Check if your organisation is on the transparency register . It's a voluntary database for organisations seeking to influence EU decision-making . Don’t know . * Does your organisation work in any of the following fields ? between 1 and 12 choices Blood collection and/ or blood banking Plasma collection for manufacture of medicinal products Tissue or cell donation or banking for transplantation Tissue or cell donation or banking for assisted reproduction Transfusion of blood and blood components Clinical application of tissues or cells - transplantation Clinical application of tissues or cells - assisted reproduction Government oversight of blood or tissue establishments ( inspection , authorisation , vigilance ) - X Medical ethics Pharmaceutical industry – plasma derived medicinal products Pharmaceutical industry – other BTC derived medicinal products Non- industrial developers of blood , tissue or cell based medicinal products Representation of donors of blood , tissues or cells Representation of patients treated with blood tissues or cells or products manufactured from them Government oversight of medicinal products - X Government oversight of medical devices Research using blood , tissues or cells Other field relevant to this consultation - X No direct activity in this field Please describe the relevant work of your organisation to this consultation 1 character (s) minimum Please describe any personal experience you have that is relevant to this consultation Blood donation Plasma donation Donation of bone marrow or cord blood Donation of sperm or eggs Donation of tissues by a relative after death Other relevant type of donation Being transfused Treatment with medicines made from blood , tissue or cell donations Receiving a tissue or cell transplant Medically assisted reproduction (in vitro fertilisation or intra-uterine insemination ) Seeking treatment by transfusion , transplantation or medically assisted reproduction Other - X Please describe your personal experience of relevance to this consultation Text of 1 to 1000 characters will be accepted Country of origin Please add your country of origin , or that of your organisation . Afghanistan Djibouti Libya Saint Martin Åland Islands Dominica Liechtenstein Saint Pierre and Miquelon Angola Equatorial Malawi Saudi Arabia Guinea Anguilla Eritrea Malaysia Senegal Antarctica Estonia - X Maldives Serbia Antigua and Barbuda Eswatini Mali Seychelles Argentina Ethiopia Malta Sierra Leone Albania Dominican Republic Lithuania Saint Vincent and the Grenadines Algeria Ecuador Luxembourg Samoa American Samoa Egypt Macau San Marino Andorra El Salvador Madagascar São Tomé and Príncipe 895350 1267333 895350 1546860 895350 2086991 Armenia Falkland Islands Marshall Singapore Islands Aruba Faroe Islands Martinique Sint Maarten Australia Fiji Mauritania Slovakia Austria Finland Mauritius Slovenia Azerbaijan France Mayotte Solomon Islands Bahamas French Guiana Mexico Somalia Bahrain French Micronesia South Africa Polynesia Bangladesh French Moldova South Georgia Southern and Antarctic Lands and the South Sandwich Islands Barbados Gabon Monaco South Korea Belarus Georgia Mongolia South Sudan Belgium Germany Montenegro Spain Belize Ghana Montserrat Sri Lanka Benin Gibraltar Morocco Sudan Bermuda Greece Mozambique Suriname Bhutan Greenland Myanmar / Burma Svalbard and Jan Mayen Bolivia Grenada Namibia Sweden Bonaire Saint Eustatius and Saba Guadeloupe Nauru Switzerland Bosnia and Herzegovina Guam Nepal Syria Botswana Guatemala Netherlands Taiwan Bouvet Island Guernsey New Caledonia Tajikistan Brazil Guinea New Zealand Tanzania British Indian Ocean Territory Guinea-Bissau Nicaragua Thailand British Virgin Islands Guyana Niger The Gambia Brunei Haiti Nigeria Timor-Leste Bulgaria Heard Island and McDonald Islands Niue Togo Burkina Faso Honduras Norfolk Island Tokelau Burundi Hong Kong Northern Tonga Mariana Islands Cambodia Hungary North Korea Trinidad and Tobago Cameroon Iceland North Tunisia Macedonia Canada India Norway Turkey Cape Verde Indonesia Oman Turkmenistan Cayman Islands Iran Pakistan Turks and Caicos Islands Central African Republic Iraq Palau Tuvalu Chad Ireland Palestine Uganda Chile Isle of Man Panama Ukraine 895350 1006856 895350 1286383 895350 2086991 China Israel Papua New Guinea United Arab Emirates Christmas Island Italy Paraguay United Kingdom Clipperton Jamaica Peru United States Cocos (Keeling) Islands Japan Philippines United States Minor Outlying Islands Colombia Jersey Pitcairn Islands Uruguay Comoros Jordan Poland US Virgin Islands Congo Kazakhstan Portugal Uzbekistan Cook Islands Kenya Puerto Rico Vanuatu Costa Rica Kiribati Qatar Vatican City Côte d’Ivoire Kosovo Réunion Venezuela Croatia Kuwait Romania Vietnam Cuba Kyrgyzstan Russia Wallis and Futuna Curaçao Laos Rwanda Western Sahara Cyprus Latvia Saint Barthélemy Yemen Czechia Lebanon Saint Helena Ascension and Tristan da Cunha Zambia Democratic Republic of the Congo Lesotho Saint Kitts and Nevis Zimbabwe Denmark Liberia Saint Lucia The Commission will publish all contributions to this public consultation . You can choose whether you would prefer to have your details published or to remain anonymous when your contribution is published . Fo r the purpose of transparency , the type of respondent ( for example , ‘ business association , ‘ consumer association ’, ‘EU citizen ’) country of origin , organisation name and size , and its transparency register number, are always published . Your e-mail address will never be published . Opt in to select the privacy option that best suits you . Privacy options default based on the type of respondent selected * Contribution publication privacy settings The Commission will publish the responses to this public consultation . You can choose whether you would like your details to be made public or to remain anonymous . Anonymous The type of respondent that you responded to this consultation as, your country of origin and your contribution will be published as received . Your name will not be published . Please do not include any personal data in the contribution itself . Public - X Your name , the type of respondent that you responded to this consultation as, your country of origin and your contribution will be published . * Contribution publication privacy settings The Commission will publish the responses to this public consultation . You can choose whether you would like your details to be made public or to remain anonymous . Anonymous Only organisation details are published : The type of respondent that you responded to this consultation as, the name of the organisation on whose behalf you reply as well as its transparency number, its size , its country of origin and your contribution will be published as received . Your name will not be published . Please do not include any personal data in the contribution itself if you want to remain anonymous . Public -X Organisation details and respondent details are published : The type of respondent that you responded to this consultation as, the name of the organisation on whose behalf you reply as well as its transparency number, its size , its country of origin and your contribution will be published . Your name will also be published . I agree with the personal data protection provisions The BTC evaluation findings An evaluation of the BTC legislation was published on 11 October 2019. Although the evaluation concluded that the legislation had increased safety and quality of blood , tissues and cells in the EU, a number of shortcomings and gaps were identified . Q1 To what extent are the findings of the evaluation still valid one year since the publication of the evaluation ? at most 8 answered row (s) 3829050 3423919 0 0 3819524 4185920 0 0 3143250 2090420 0 0 3819525 6296660 3140075 5350510 3870325 2861310 3787775 1318260 3743325 619760 Do you have additional insights on any of these topics that were not adequately highlighted in the evaluation ? 193675 205740 at most 8 choice (s) Technical requirements for safety and quality are not up-to-date There are substances of human origin that should be in the scope of the legislation but currently are not ( breast milk , fecal microbiota , serum eye drops etc.) Divergent national approaches to oversight by authorities leads to unequal protection and lack of inter- Member State trust Donors of blood , tissues and cells are not adequately protected by the legislation Children born from medically assisted reproduction techniques are not adequately protected The requirements for authorising new ways of preparing and using blood , tissues and cells are not adequate , particularly because demonstration of efficacy and safety in the recipient is not required . 226060 662305 There are sometimes difficulties in defining the borderlines for novel BTC ( used in transfusion , transplantation or assisted reproduction ) with other regulatory frameworks Current legislation has not proven adequate to protect EU patients from the risk of shortages or sudden supply disruption Please describe the additional information , specifying which problem you refer to 238760 352425 In regulatory perspective the technical requrements for safety and quality of tissues and cells need to be tailored to the specific products , but at the same time be clearly and univocally understandable and consider the practical needs and possibilities to carry out such processes in both laboratory and hospital operating room settings . Risk of shortages and supply disruptions needs additional analysis , in view of the COVID-19 pandemic lessons learned . 0 0 In regulatory perspective the technical requrements for safety and quality of tissues and cells need to be tailored to the specific products , but at the same time be clearly and univocally understandable and consider the practical needs and possibilities to carry out such processes in both laboratory and hospital operating room settings . Risk of shortages and supply disruptions needs additional analysis , in view of the COVID-19 pandemic lessons learned . 1500 character (s) maximum 1500 character (s) maximum Q2 Select up to 4 problems to which you would give highest priority 123825 158115 at most 4 choice (s) Technical requirements for safety and quality are not up-to-date 219075 646430 There are substances of human origin that should be in the scope of the legislation but currently are not ( breast milk , fecal microbiota , serum eye drops etc.) 190500 443230 0 0 Divergent national approaches to oversight by authorities leads to unequal protection and lack of inter- Member State trust Donors of blood , tissues and cells are not adequately protected by the legislation Children born from medically assisted reproduction techniques are not adequately protected 200025 691515 0 0 The requirements for authorising new ways of preparing and using blood , tissues and cells are not adequate , particularly because demonstration of efficacy and safety in the recipient is not required . There are sometimes difficulties in defining the borderlines for novel BTC ( used in transfusion , transplantation or assisted reproduction ) with other regulatory frameworks Current legislation has not proven adequate to protect EU patients from the risk of shortages or sudden supply disruption Q3 How did , in your view , the Covid-19 pandemic influence the evaluation conclusions ? at most 8 answered row (s) 3543300 6214110 0 0 4400550 5042535 0 0 4391025 4099560 0 0 4410074 2623185 0 0 3495675 2032635 0 0 4346575 3197225 4333875 1298575 4403725 606425 Current legislation has not proven adequate to protect EU patients from the risk of shortages or sudden supply disruption Current legislation has not proven adequate to protect EU patients from the risk of shortages or sudden supply disruption Q4 Are there other lessons learned from the Covid-19 pandemic that should be taken into account in the revision of the BTC legislation ? If so, please describe . 314578 243205 We have experienced some transport issues with SoHo products because of COVID-19 pandemic . Some of them have been related to organs , but we have couple of cases related to PBSC and gametes . We are not sure how this issue could be solved during the revision of the BTC legislation , but maybe a principles of centralised approach on SoHo transport could be formed during the revision . We have experienced some transport issues with SoHo products because of COVID-19 pandemic . Some of them have been related to organs , but we have couple of cases related to PBSC and gametes . We are not sure how this issue could be solved during the revision of the BTC legislation , but maybe a principles of centralised approach on SoHo transport could be formed during the revision . 1500 character (s) maximum 1500 character (s) maximum Keeping EU technical requirements up to date with scientific and medical knowledge and practice The EU legislation includes many rules regarding technical issues such as who can donate , what tests must be carried out on donors , what quality criteria should be met for the blood , tissues and cells that are supplied to hospitals and clinics , which types of adverse occurrences should be notified to authorities , etc. According to the evaluation , many of these rules are currently out of date . The evaluation also concluded that the rules should be extended to include donor protection and the protection of children born from medically assisted reproduction . The Commission is considering three possible options for setting and updating these technical rules : By professionals : the blood and tissue centres would conduct their own risk assessments and establish rules based on the conclusions , together with professional society guidance . This process would be reviewed for approval by inspectors from the national authority . EU law would require that professionals follow the rules and guidance of named expert bodies such as ECDC and EDQM , in consultation with professional associations . All detailed technical requirements would be described in EU legislation and kept up-to-date with regular amendments . Q5 Who should set out these technical rules to effectively achieve up-to-date safety and quality rules , based on good science ? ( Consider the time required to update the rules , including during crises , their quality as well as whether EU harmonisation is essential or not ) 4943474 561975 0 0 4943474 971550 0 0 Professionals Expert bodies EU law No answer Rules on donor suitability and testing * Professionals Expert bodies EU law No answer Rules on donor suitability and testing * 4942840 499389 0 0 4977572 1117600 0 0 4937125 2249857 4956175 1750060 Rules on donation frequency and donor monitoring . Rules on quality management by providers of blood , tissues and cells ( air quality requirements , documentation , quality control testing , training etc.) Rules on the technical characteristics of blood , tissues and cells provided for patients ( e.g . volume , cell numbers , labelling ) Criteria and templates for reporting and investigation of adverse reactions and events to authorities . Rules for the development of new processing methods or new clinical uses of blood , tissues and cells * * * * Rules on donation frequency and donor monitoring . Rules on quality management by providers of blood , tissues and cells ( air quality requirements , documentation , quality control testing , training etc.) Rules on the technical characteristics of blood , tissues and cells provided for patients ( e.g . volume , cell numbers , labelling ) Criteria and templates for reporting and investigation of adverse reactions and events to authorities . Rules for the development of new processing methods or new clinical uses of blood , tissues and cells * * * * Q6 In general , which of these options , in your view , would overall be most costeffective ? 4181475 969010 0 0 4197350 658495 4219575 368935 0 0 Very Quite Rather not Not at all No answer Professionals Expert bodies EU law * * * Very Quite Rather not Not at all No answer Professionals Expert bodies EU law * * * Cannot assess cost-effectiveness. Requirements should come from expert bodies, but tissue est ablishments should have some fle xibility depending on the specific situation and urgency. Regulation should also consider the practical possibilities of MS health system to ensure the required qu a lity levels. The BTC regulation evaluation showed that, over time, new substances of human origin , that do not fall within the scope of the BTC legislation , are put in use . Some fall wholly or partially under other frameworks nationally and some are unregulated at the EU level. Q7 In which of the following cases do you think that technical rules for safety and quality should be included in the scope of the BTC legislation ? 5165725 1533525 0 0 4257675 1200150 0 0 5158740 895985 0 0 4283075 2353310 5184775 1870710 Please provide a description of the other substances you consider should be included in this legislation and explain why . 307975 225425 Platelet rich plasma ( PRP ) could be included in the scope of the BTC from donation to distribution . For serum eye drops a specific guidance on home distribution would be desirable . Platelet rich plasma ( PRP ) could be included in the scope of the BTC from donation to distribution . For serum eye drops a specific guidance on home distribution would be desirable . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted Q8 If you have further comments on the technical rules for safety and quality of blood , tissues and cells and other substances of human origin , please enter them here . 352425 219710 The new legislation should consider that a significant amount of tissue processing ( during procurement and separately ) is still done in the operating rooms , where e.g . GMP level A air cleanliness cannot be achieved . At least not in operation conditions . It should be taken into account that in some cases the appropriate control of the operating room conditions with controlled air filtration , pressure and cleaning protocols are sufficient to ensure the quality and safety of the tissues . The new legislation should consider that a significant amount of tissue processing ( during procurement and separately ) is still done in the operating rooms , where e.g . GMP level A air cleanliness cannot be achieved . At least not in operation conditions . It should be taken into account that in some cases the appropriate control of the operating room conditions with controlled air filtration , pressure and cleaning protocols are sufficient to ensure the quality and safety of the tissues . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted Improving oversight of blood , tissue and cell activities The evaluation indicated that variable national approaches to oversight of blood , tissue and cell activities in Member States results in a lack of trust and creates barriers to the exchange of blood , tissues and cells between Member States . Q9 What would be the impact of introducing oversight principles for authorities in EU legislation . The principles might address independence of inspectors , conflicts of interest , and competency requirements for staff in authorities . Oversight principles for authorities would ensure quality and harmonised approach in both inspections and the work in BEs and TEs . Q10 Would audits by the European Commission of Member State competent authority control systems ( inspection , vigilance , reporting ) improve trust and interMember State exchange of blood , tissues and cells ? We think general audits would definitely improve trust as they do now in medicinal products field . Q11 Would greater collaboration between Member State competent authorities ( e. g. joint inspections , peer audits of inspections improve effectiveness of oversight and increase inter- Member state exchange of blood , tissues and cells ? It would increase trust and harmonisation . Q12 Would an EU programme of training of staff in national / regional authorities to agreed guidelines improve effectiveness of oversight and increase inter- Member state exchange of blood , tissues and cells ? EU programme of training of inspectors is always welcome and couple of such trainings have already taken place . Training would improve effectiveness of oversight . Increase of exchange of blood , tissues and cells cannot be predicted . * Q13 For questions 9 to 12, do you see any risks or potential negative impacts ? Yes No X No answer Please describe the risk or negative impact , specifying which question you refer to . 1500 character (s) maximum 1500 character (s) maximum Q14 If you have further comments on oversight of the blood , tissues and cells sector , please enter them here . – No further comments Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted Supporting innovation for patient benefit The BTC evaluation found that innovation was not facilitated optimally . In particular , only laboratory validation of new processing methods is required (no animal or clinical studies to demonstrate safety and efficacy in the patient ). * Q15 Should legal requirements be introduced in EU legislation for demonstrating safety , quality and efficacy when blood , tissues or cells are prepared or used in new ways ? Yes - X No No answer Please explain why 1500 character (s) maximum 354330 5715 This really depends on the area and activity in question . Also the „ new way “ is not defined – new for the tissue establishment or for the world . Literature based or retrospective safety evaluation could be accepted in some cases . CAs should have an option to allow innovative products to their own member state market. This way the local CA can set the initial quality , safety and efficacy standards to the new products until harmonised EU-wide guidance on the new product is issued . A quicker adoption of common EU requirements for the new products is also one of the targets of the revision of BTC legislation . 0 This really depends on the area and activity in question . Also the „ new way “ is not defined – new for the tissue establishment or for the world . Literature based or retrospective safety evaluation could be accepted in some cases . CAs should have an option to allow innovative products to their own member state market. This way the local CA can set the initial quality , safety and efficacy standards to the new products until harmonised EU-wide guidance on the new product is issued . A quicker adoption of common EU requirements for the new products is also one of the targets of the revision of BTC legislation . * Q16 Are you aware of cases where blood , tissues and cells are used to treat patients , without proven clinical benefit ? Yes -X No Please describe the case (s) you are aware of briefly 357620 282575 For example we are not sure how the clinical benefit of ovarian tissue freezing is proven or how it should be done . With the current organisation of CAs , it is not likely that all of them would be able to to advise TEs on the clinical studies needed to prove the clinical efficacy and in some cases it would not be ethical to run a controlled trial . For example we are not sure how the clinical benefit of ovarian tissue freezing is proven or how it should be done . With the current organisation of CAs , it is not likely that all of them would be able to to advise TEs on the clinical studies needed to prove the clinical efficacy and in some cases it would not be ethical to run a controlled trial . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted Member States are responsible for deciding the regulatory status of products / substances . They might classify as blood , tissues and cells ( Substances of Human Origin) or under another legal framework such as the pharmaceutical or medical device frameworks . EU level regulatory advice can be sought on whether the legislation on Advanced Therapy Medicinal Products would apply ( from the Committee for Advanced Therapies ) and on whether the medical device legislation would apply ( from an expert group of medical device authorities ). * Q17 Are you aware of cases where the regulatory classification of a substance of human origin is unclear ? Yes - X No 2362200 573577 Serum eye drops – T&C/ blood / medicinal products / medical devices . Platelet rich plasma - T&C/ blood / medicinal products / medical devices . Fecal matter transplant - T&C/ medicinal products . Adipose tissue injections into knee joint – T&C/ medicinal products / single medical procedure ( depending on the exact site of injetion and treatment effect sought ). Impact is that it is much more difficult to get a medical device or medicinal product authorized and therefore it could be less accessible to patients . On the other hand , application of medicinal products regulation to a procuct would reduce some safety and efficacy concerns . 0 0 Serum eye drops – T&C/ blood / medicinal products / medical devices . Platelet rich plasma - T&C/ blood / medicinal products / medical devices . Fecal matter transplant - T&C/ medicinal products . Adipose tissue injections into knee joint – T&C/ medicinal products / single medical procedure ( depending on the exact site of injetion and treatment effect sought ). Impact is that it is much more difficult to get a medical device or medicinal product authorized and therefore it could be less accessible to patients . On the other hand , application of medicinal products regulation to a procuct would reduce some safety and efficacy concerns . Please provide information on case (s) you are aware of Description of the product / substance The regulatory framework it borders The impact of this for patients 1 2 3 Please provide information on case (s) you are aware of Description of the product / substance The regulatory framework it borders The impact of this for patients 1 2 3 * Q18 Do you consider that there are substances / products being regulated under one legal framework but would be better regulated under another ? Yes - X No No answer -137160 2339340 It is relatively difficult to apply medicinal product regulation to FMT and platelet rich plasma and therefore it would be easier to regulate them under tissues and cells . Also serum eye drops would be easier to handle , if they belong under BTC and not as medicinal products . But the current BTC regulation raises one important problem – the product cannot be given to te patients to use at home . Distribution is only between establishments or establishmets and hospitals . 0 It is relatively difficult to apply medicinal product regulation to FMT and platelet rich plasma and therefore it would be easier to regulate them under tissues and cells . Also serum eye drops would be easier to handle , if they belong under BTC and not as medicinal products . But the current BTC regulation raises one important problem – the product cannot be given to te patients to use at home . Distribution is only between establishments or establishmets and hospitals . Please provide information on substances / products that you consider are not regulated under the most suitable framework Description of the product / substance Why you consider it is not regulated under the most suitable framework 1 2 3 Please provide information on substances / products that you consider are not regulated under the most suitable framework Description of the product / substance Why you consider it is not regulated under the most suitable framework 1 2 3 Q19 How would you assess the impact of a new EU level structure or committee to advise Member States on whether a substance falls under the BTC legislation or not , equivalent to those for ATMPs and medical devices ? The EU level structure or committee should advise Member States on under which legislation one or the other substance falls ( blood or tissues or medical device or ATMP or medicinal product (non-ATMP) ) . This committe e should also advise the member states on the authorisation of the products that fall under more than one legislative frameworks . Assessments concluding that a substance or product does not fall under one or the other specific legislation are helpful , but not as helpful as refe r ring where the product should belong . Ideally t here should be one committe e to make the decision , but i f one committe e cannot be formed , the decision should be made in c ooperation of different committ ees ( medicinal product , BTC, ATMP , medical device ) . 895350 10036556 If you have further comments on your answer please enter them here 2000 character (s) maximum 2000 character (s) maximum * Q20 If an EU level structure or committee as described in Q19 were established , do you consider that it should co-ordinate decisions with the equivalent committees in the medicinal product and medical device frameworks ? Yes X – ideally there would be one com m ittee No No answer * Q21 Are the donation , procurement and testing provisions for blood , tissues and cells that are used to manufacture medicinal products or medical devices adequate ? Very inadequate Somewhat inadequate Adequate - X Somewhat too stringent Much too stringent I don't know Please describe the specific provisions you consider should be changed and why . 2000 character (s) maximum 2000 character (s) maximum Q22 If you have further comments on the subject of innovation in blood , tissues and cells please enter them here . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted Sufficiency of supply of blood , tissues and cells Although an objective of the BTC legislation was to ensure a sustainable supply of critical blood , tissues and cells , the evaluation showed that there are dependencies on certain Member States and on third countries for certain substances , in particular plasma for the manufacture of medicinal products . In addition , it was highlighted that there is a lack of legal provisions to ensure appropriate emergency measures in the event of sudden supply interruptions . Q23 What effect would mandatory EU monitoring and routine reporting of sufficiency data ( mandatory reporting of donations , distribution , import, export and use by BTC establishments to national authorities and to the Commission ) have? Additional costs and administrative burden for establishments and authorities Yes . Transparency for citizens Could be . 895350 10054209 Q24 What effect would sharing of reported donation and supply monitoring data on an EU platform have? Additional costs and administrative burden for establishments and authorities Yes . Information for policy makers ( for vigilance and sufficiency measures ) Yes Q25 What would be the impact of mandatory rapid notification to the national authority , and by them to other Member State authorities , in the case of a sudden significant drop in supply due to an incident or other crisis ? Imrove d information exchange could be beneficial to handle supply issues in a crisis situation * Q26 What other measures could be introduced in legislation to address a sudden drop in supply due to a crisis ? Co- operative actions between blood and tissue establishments - X Notification to the national authority with a response at Member State level Notification to the EU level with collective response co-ordination Other No answer Please describe 1000 character (s) maximum Please describe 1000 character (s) maximum Some blood and tissue establishments and competent authorities have in place preparedness / contingency plans for emergencies such as infectious disease outbreaks , natural disasters or military conflicts . * Q27 What would be the effect of making such preparedness / contingency plans mandatory ? It would raise many concerns It would raise some concerns It would have no impact It would bring some improvements - X It would bring many improvements No answer 320675 493494 There could be general preparedness / contingency plans , but specific problems will always have to be solved depending on the actual situation , as the emergency cases can be very different . . There could be general preparedness / contingency plans , but specific problems will always have to be solved depending on the actual situation , as the emergency cases can be very different . . Please describe the key concerns 1500 character (s) maximum Please describe the key concerns 1500 character (s) maximum Q28 If you have further comments on the topic of ensuring a sustainable supply of essential blood , tissues and cells . Please list any other measure you consider would support this objective . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted General comments and supporting documents Q29 If you have general comments on other topics related to the revision of the EU legislation on blood , tissues and cells , please enter them here . Text of 1 to 2000 characters will be accepted Text of 1 to 2000 characters will be accepted S hortage s reporting or mandatory contingency plan s could be considered as possible measures to tackle the shortages of plasma but would need to be further analysed . The reporting mechanism could be useful to have a better overview of the situation and to be able to plan necessary measures . The actual usefulness would depend on the subsequent use of this data and the mechanisms foreseen , for example for mutual assistance among Member States . EU should focus on avoiding wastages and ensuring that available plasma is used to the maximum extent possible . EU should also have a policy to increase the accessibility to the recombinant coagulation factors . Common EU approach on appreciation of ap heresis donors could be helpful , but general blood donation should continue according to VUD principles . You may upload one supporting document to your submission here . Only files of the type pdf,txt,doc,docx,odt,rtf are allowed THANK YOU FOR YOUR CONTRIBUTION! Targeted Consultation on the Revision of the EU Legislation on Blood, Tissues and Cells Fields marked with * are mandatory . Fields marked with * are mandatory . Introduction The Commission has launched an initiative to revise the EU legislation on blood, tissues and cells ( BTC ), addressing a number of shortcomings identified in an evaluation of the legislation published in 2019 . The i n i t i a t i v e a i m s t o : update the legislation to provide a more flexible alignment with scientific and technological developments tackle the (re-)emergence of communicable diseases, including lessons learnt from the COVID-19 p a n d e m i c • focus on the increasing commercialisation and globalisation of the sector. This Targeted Consultation supplements a Public Consultation that is open in parallel on the European Commission Have your Say port a l . It is targeted at organisations (not individuals) that are directly involved in or impacted by the fields concerned and are familiar with the current legislation and its implementation. It will feed into the Impact Assessment process that will lead to the revision of the EU legislation on blood, tissues and cells. The scope of the impact assessment, and of this consultation, is limited to the EU legislation on blood, tissues and cells. Thus, it does not address possible changes to other EU legal frameworks, such as those for advanced therapy medicinal products, other medicinal products or medical devices, but it does explore issues at the borderlines between the blood, tissues and cells frameworks and those other regulated frameworks. If your organisation is among those targeted in this consultation, you are advised to complete both surveys, as questions in the Public Consultation are not repeated here or, in some cases, the topics are addressed again but explored in more depth in this survey. An external contracted study will also gather evidence and views to support the Impact Assessment. Apart from the first section entitled 'About you', you are not obliged to answer all survey questions. You are advised to answer only those questions for which you have experience or expertise . Please note also that not all the shortcomings identified in the evaluation of the BTC legislation are addressed in this consultation. Some shortcomings are considered more appropriate for exploration in participatory workshops organised in the context of the external study. About you * Language of my contribution Bulgarian Croatian Czech Danish Dutch English Estonian - X Finnish French German Greek Hungarian Irish Italian Latvian Lithuanian Maltese Polish Portuguese Romanian Slovak Slovenian Spanish Swedish Organisation name 255 character(s) maximum 506730 90805 Ministry of Social Affairs Ministry of Social Affairs Organisation scope International Local National - X Regional Organisation size Micro (1 to 9 employees) Small (10 to 49 employees) Medium (50 to 249 employees) - X Large (250 or more) Transparency register number (if applicable) 255 character(s) maximum Check if your organisation is on the transparency register . It's a voluntary database for organisations seeking to influence EU decision-making. 659130 98425 - 0 - Which of the following best describes the work of your organisation ? Blood collection and/or blood banking Plasma collection for manufacture of medicinal products Tissue or cell donation or banking for transplantation Tissue or cell donation or banking for assisted reproduction Transfusion of blood and blood components Clinical application of tissues or cells - transplantation Clinical application of tissues or cells - assisted reproduction Government oversight of blood or tissue establishments (inspection, authorisation , vigilance) - X Medical ethics Standards setting Pharmaceutical industry – plasma derived medicinal products Pharmaceutical industry – other BTC derived medicinal products Non-industrial developers of blood, tissue or cell based medicinal products Representation of donors of blood, tissues or cells Representation of patients treated with blood tissues or cells or products manufactured from them Government oversight of medicinal products - X Government oversight of medical devices X Research using blood, tissues or cells Other field relevant to this consultation X You selected 'Other'. Please describe the relevant work of your organisation to this consultation Policy development and regulation at the national level Text of 1 to 1000 characters will be accepted Country where the organisation is based or where it has its main office Please add your country of origin, or that of your organisation . Afghanistan Djibouti Libya Saint Martin Åland Islands Dominica Liechtenstein Saint Pierre and Miquelon Albania Dominican Republic Lithuania Saint Vincent and the Grenadines Algeria Ecuador Luxembourg Samoa American Samoa Egypt Macau San Marino Andorra El Salvador Madagascar São Tomé and Príncipe Angola Equatorial Guinea Malawi Saudi Arabia Anguilla Eritrea Malaysia Senegal Antarctica Estonia - X Maldives Serbia Antigua and Barbuda Eswatini Mali Seychelles Argentina Ethiopia Malta Sierra Leone Armenia Falkland Islands Marshall Islands Singapore Aruba Faroe Islands Martinique Sint Maarten Australia Fiji Mauritania Slovakia Austria Finland Mauritius Slovenia Azerbaijan France Mayotte Solomon Islands Bahamas French Guiana Mexico Somalia Bahrain French Polynesia Micronesia South Africa Bangladesh French Southern and Antarctic Lands Moldova South Georgia and the South Sandwich Islands Barbados Gabon Monaco South Korea Belarus Georgia Mongolia South Sudan Belgium Germany Montenegro Spain Belize Ghana Montserrat Sri Lanka Benin Gibraltar Morocco Sudan Bermuda Greece Mozambique Suriname Bhutan Greenland Myanmar /Burma Svalbard and Jan Mayen Bolivia Grenada Namibia Sweden Bonaire Saint Eustatius and Saba Guadeloupe Nauru Switzerland Bosnia and Herzegovina Guam Nepal Syria Botswana Guatemala Netherlands Taiwan Bouvet Island Guernsey New Caledonia Tajikistan Brazil Guinea New Zealand Tanzania British Indian Ocean Territory Guinea-Bissau Nicaragua Thailand British Virgin Islands Guyana Niger The Gambia Brunei Haiti Nigeria Timor-Leste Bulgaria Heard Island and McDonald Islands Niue Togo Burkina Faso Honduras Norfolk Island Tokelau Burundi Hong Kong Northern Tonga Mariana Islands 895350 746379 Cameroon Iceland North Tunisia Macedonia Canada India Norway Turkey Cape Verde Indonesia Oman Turkmenistan Cayman Islands Iran Pakistan Turks and Caicos Islands Central African Republic Iraq Palau Tuvalu Chad Ireland Palestine Uganda Chile Isle of Man Panama Ukraine Cambodia Hungary North Korea Trinidad and Tobago China Israel Papua New United Arab Guinea Emirates Christmas Italy Island Paraguay United Kingdom Clipperton Jamaica Peru United States Cocos (Keeling) Japan Philippines United States Minor Outlying Islands Islands Colombia Jersey Pitcairn Islands Uruguay Comoros Jordan Poland US Virgin Islands Congo Kazakhstan Portugal Uzbekistan Cook Islands Kenya Puerto Rico Vanuatu Costa Rica Kiribati Qatar Vatican City Côte d’Ivoire Kosovo Réunion Venezuela Croatia Kuwait Romania Vietnam Cuba Kyrgyzstan Russia Wallis and Futuna Curaçao Laos Rwanda Western Sahara Cyprus Latvia Saint Yemen Barthélemy Czechia Lebanon Saint Helena Ascension and Tristan da Cunha Zambia Democratic Republic of the Congo Lesotho Saint Kitts and Nevis Zimbabwe Denmark Liberia Saint Lucia Your first name 339725 22225 Elen Elen Your family name 333375 37465 Ohov Ohov Email 339725 25400 [email protected] [email protected] Do you wish to be informed regarding further Commission events or publications related to this topic? Please keep me informed regarding the BTC revision process - X Do not use this email address to contact me except for confirmation of my submission to this consultation The Commission will publish all contributions to this targeted consultation. You can choose whether you would prefer to have your details published or to remain anonymous when your contribution is published. Fo r the purpose of transparency, the country of origin, organisation name and size, and its transparency register number, are always published. Your e-mail address will never be published. Opt in to select the privacy option that best suits you. * Contribution publication privacy settings The Commission will publish the responses to this public consultation. You can choose whether you would like your details to be made public or to remain anonymous. Anonymous The name of your organisation , the field(s) that your organisation works in, the country where your organisation is based and your contribution will be published as received. Your personal name will not be published. Please do not include any personal data in the contribution itself. Public Your name, the name of your organisation , the field(s) that your organisation works in, the country where your organisation is based and your contribution will be published as received. Please do not include any personal data in the contribution itself. - X I agree with the personal data protection provisions SECTION A Keeping EU technical requirements up to date with scientific and medical knowledge and practice The BTC evaluation showed that, over time, many new substances of human origin being used in patients do not fall within the scope of the BTC legislation. Some fall wholly or partially under other frameworks nationally and some are unregulated at the EU level. These substances do not meet the defined scope and definitions of the basic acts for blood and for tissues and cells. Please note that this section does not address those substances that might border or fall under other frameworks (medicinal products or medical devices). Such borderline substances are addressed below in the innovation section. Q1 Should the scope and/or definitions of the revised legislation be drafted to include any of the following? 2460656 2277110 0 0 5000624 1515110 0 0 No - exclude from the scope of BTC legislation Include donation , procurement / collection and testing only in the BTC scope Include all steps up to clinical use and vigilance in the BTC scope No answer Blood used for clinical purposes other than transfusion ( e.g . platelet rich plasma or serum eye drops) Blood , tissues or cells used for non- clinical research or teaching Other No - exclude from the scope of BTC legislation Include donation , procurement / collection and testing only in the BTC scope Include all steps up to clinical use and vigilance in the BTC scope No answer Blood used for clinical purposes other than transfusion ( e.g . platelet rich plasma or serum eye drops) Blood , tissues or cells used for non- clinical research or teaching Other You selected 'Other'. Please describe 1000 character(s) maximum 104775 713740 0 0 Q2 Should the legislation include in its scope substances of human origin that do not meet the definitions of blood, tissues or cells (e.g. breast milk or intestinal microbiota) but are applied to patients? Yes No No answer Q3 If you have further comments on the extension of the BTC scope to substances not currently included (apart from substances that border other frameworks such as advanced therapy medicinal products or medical devices), please enter them here. 312208 242359 There should be a legal mechanism that allows member states to include novel products in the scope of BTC legislation. This would apply to products that do not fit under other legislations and to which the application of BTC legislation is considered optimal by the competent authorities and expert bodies. This would prevent novel approaches to be stuck in the “grey area” with no common approach. Distribution of some SoHo products to patient’s home in a member state could be acceptable, if approved by that countries’ national CA (e.g. for serum eye drops). even if they do not meet the definition of BTC 0 There should be a legal mechanism that allows member states to include novel products in the scope of BTC legislation. This would apply to products that do not fit under other legislations and to which the application of BTC legislation is considered optimal by the competent authorities and expert bodies. This would prevent novel approaches to be stuck in the “grey area” with no common approach. Distribution of some SoHo products to patient’s home in a member state could be acceptable, if approved by that countries’ national CA (e.g. for serum eye drops). even if they do not meet the definition of BTC 1000 character (s) maximum 1000 character (s) maximum Q4 The European Commission has proposed reinforcing the mandate of ECDC, including a role in routine surveillance of communicable disease test results among BTC donors in the EU. Do you have comments on this proposal? 1000 character(s) maximum 269875 26882 We are not sure what is meant by surveillance of test results among BTC donors. The test for communicable diseases of BTC donors have to be negative in order to proceed with the donation. Does ECDC mean to follow donors, who test positive on some communicable diseases after more than one donation or if communicable disease is discovered after transfusion/transplant ? This is currently done by national CAs. Otherwise, c ommunicable disease test results of BTC donors and donor candidates are not routinely collected by competent authority, so it would be helpful to know, how ECDC is planning to carry out this surveillance and what benefit is expected in order to give more detailed response. ECDC’s scientific advice for complex situations is of course always appreciated. 0 We are not sure what is meant by surveillance of test results among BTC donors. The test for communicable diseases of BTC donors have to be negative in order to proceed with the donation. Does ECDC mean to follow donors, who test positive on some communicable diseases after more than one donation or if communicable disease is discovered after transfusion/transplant ? This is currently done by national CAs. Otherwise, c ommunicable disease test results of BTC donors and donor candidates are not routinely collected by competent authority, so it would be helpful to know, how ECDC is planning to carry out this surveillance and what benefit is expected in order to give more detailed response. ECDC’s scientific advice for complex situations is of course always appreciated. Q5 Should scope and technical quality and safety rules differ for different types of donation settings ? 3324224 5006340 0 0 3267074 4110990 0 0 4781550 3549015 0 0 4743450 3006090 0 0 3257549 2110740 0 0 2343150 1424940 0 0 3241675 2678430 You selected 'Other'. Please descibe the donation setting you are referring to. The first option “not processed” should be more clearly explained and illustrated with examples. We consider retransplantation of a piece of skin, blood vessel or bone during the same surgical procedure something that should not be included in the scope of the legislation. On the other hand all of these activities require flushing, cleaning, cutting and sometimes shaping of the tissue, that under other conditions could be considered processing. We are in position that purely mechanical processing in the operating room should not be included in the scope of BTC legislation , if the material is used on the same patient during the same surgical procedure. The cut-off point within the same surgical procedure would be more complex processing, that involves special chemical agents or enzymes and that should already be within the scope of BTC legislation. This should be determined by CAs or expert bodies using case by case approach, if needed. 500 character(s) maximum Q6 Should the processing of BTC that are not stored be regulated regardless of the donation setting? 4606925 3093720 3063875 2484120 4638675 1785620 3089275 1055370 You selected 'Other'. Please describe the processing setting that you consider should be regulated. 276225 214729 We presume that first and second option mean that the tissue is reapplied during the same surgery when it was removed. Please also see our comment on the definition of processing from the previous answer. We presume that first and second option mean that the tissue is reapplied during the same surgery when it was removed. Please also see our comment on the definition of processing from the previous answer. 1000 character (s) maximum 1000 character (s) maximum Q7 The following terms are currently defined in the basic act for blood (Directive 2002/98/EC). Do you consider that any of these should be revised? blood blood component blood product autologous transfusion blood establishment hospital blood bank serious adverse event serious adverse reaction blood component release deferral distribution haemovigilance 171450 206375 0 0 inspection none Please give details of the definition(s) you think should be revised and why. 285750 257810 We think that none of these blood directive definitions need to be changed. 0 0 We think that none of these blood directive definitions need to be changed. 2000 character (s) maximum 2000 character (s) maximum Q8 Are there additional terms related to blood that should be defined in a basic act ? Yes 133350 9525 0 0 No No answer 0 -635 Please list the additional terms Please list the additional terms Q9 The following terms are defined in the basic act for tissues and cells (Directive 2004/23/EC). Do you consider that any of these should be revised? cells tissue donor donation organ 130810 203835 0 0 procureme nt P rocessing Preservation Q uarantine storage distribution human application serious adverse even t serious adverse reaction tissue establishment allogeneic use autologous use N one Please give details of the definition(s) you think should be revised and why 276225 232410 As the new and complex processing methods that could be used within the same surgical procedure, could require authorisation within the scope of BTC legislation, the „simpler“ processing methods should still be out of the scope. It should be added to the processing definition that simple mechanical processing (cutting to correct size or shape, flushing with saline etc.) in the operating room should not be included in the scope of BTC legislation, if the material is used on the same patient during the same surgical procedure. The current definition „all operations involved in the preparation, manipulation, preservation and packaging“ could be confusing, as preparation and manipulation also happens during the same surgical procedure. The new definition could be: Processing - means all operations involved in the preparation, manipulation, preservation and packaging of tissues or cells intended for human applications. Manipulation should exclude substantial manipulation in terms of ATMP regulation. Mechanical processing of the patient material that is to be transplanted back to the same patient during the same surgical procedure is excluded from the scope of this directive. It would also be necessary to further specify what actions are considered to be covered under the procurement and processing under BTC legislation to better distinguish them from the actions in the context of manufacturing of advanced therapy medicinal products. 0 0 As the new and complex processing methods that could be used within the same surgical procedure, could require authorisation within the scope of BTC legislation, the „ simpler“ processing methods should still be out of the scope. It should be added to the processing definition that simple mechanical processing (cutting to correct size or shape, flushing with saline etc.) in the operating room should not be included in the scope of BTC legislation, if the material is used on the same patient during the same surgical procedure. The current definition „all operations involved in the preparation, manipulation, preservation and packaging“ could be confusing, as preparation and manipulation also happens during the same surgical procedure. The new definition could be: Processing - means all operations involved in the preparation, manipulation, preservation and packaging of tissues or cells intended for human applications. Manipulation should exclude substantial manipulation in terms of ATMP regulation. Mechanical processing of the patient material that is to be transplanted back to the same patient during the same surgical procedure is excluded from the scope of this directive. It would also be necessary to further specify what actions are considered to be covered under the procurement and processing under BTC legislation to better distinguish them from the actions in the context of manufacturing of advanced therapy medicinal products. 2000 character (s) maximum 2000 character (s) maximum Q10 Are there additional terms related to tissues and cells that should be defined in a basic act? Yes 114300 7620 0 0 No No answer Please list the additional terms 2000 character(s) maximum Q11 Does the description and role of the Responsible Person in a blood or tissue establishment need to be improved? 85725 233680 0 0 Yes No No answer Please explain how it should be improved 1000 character (s) maximum Please explain how it should be improved 1000 character (s) maximum Q12 Do you consider that a role for physicians in blood or tissue establishments should be defined in a basic act? 114300 233680 0 0 Yes No No answer Q13 Which of the following responsibilities should be specified in legislation for a physician? Donor selection policies 133350 191135 0 0 BTC allocation policies Adverse reaction investigation and follow-up 190500 177800 0 0 Clinical outcome (efficacy) monitoring Other 307975 531495 The need to specify any of these responsibilities in the legislation depends on the extent of the specification as some degree of flexibility should be considered. The need to specify any of these responsibilities in the legislation depends on the extent of the specification as some degree of flexibility should be considered. You selected ' Other '. Please describe 1000 character (s) maximum You selected ' Other '. Please describe 1000 character (s) maximum Q14 If you consider that there are other key personnel roles in blood and tissue establishments that should be defined in a basic act, please give details here. 365916 244475 - - 1000 character (s) maximum 1000 character (s) maximum The EU legislation includes many technical rules to be followed by blood and tissue establishments. According to the evaluation, many of these rules are currently out of date. The evaluation also concluded that the rules should be extended to include donor protection and the protection of children born from medically assisted reproduction. The Commission is considering three possible options for setting and updating these technical rules: By professionals : the blood and tissue establishments would conduct their own risk assessments and establish rules based on the conclusions, together with professional society guidance. This process would be reviewed for approval by inspectors from the competent authority. EU law would require that professionals follow the rules and guidance of named expert bodies such as ECDC and EDQM , in consultation with professional associations. All detailed technical requirements would be described in EU legislation and kept up-to-date with regular amendments. Q15 Which of the proposed policy options is most appropriate to define and update each of the following technical rules? You may choose different options for different aspects. 5305425 3618865 5305425 3166110 5324475 2785110 0 5334000 2213610 5334000 1213485 5330190 908685 3891915 5196840 0 3922395 4701540 3914775 4038600 0 3975735 1645920 18 5299710 118110 3920218 1268186 4730115 5775960 3907155 5059680 3968115 4503420 3947160 4106545 3960495 3413760 0 4684395 2743200 4760595 2049780 0 3937635 609600 3952875 137160 19 You chose 'other' for one or more of the rules. Please describe the alternative option you propose, specifying the rule/requirement you are referring to. Regarding rules on donor protection and foll o w up, the basic principles and requirements should be foreseen in the EU and national legislation. For example, when setting the age limits the need to protect minors needs to be considered. More detailed rules could in addition be foreseen in the expert level guidance documents (for example age considerations related to clinical quality and safety). Main principles of donor compensation should be set in the EU legislation, while Member States should also have some flexibility in applying the donor reimbursement/compensation rules, which in case of tissues and cells could also depend on the specific type of donation. For blood donation the principle of VUD should be maintained. Regarding quality and safety requirements ( f.ex mandatory testing for communicable diseases and genetic testing of gamete, traceability rules), the main principles should be covered in the EU legislation and more detailed guidance could be agreed by the European level expert bodies. Regarding rules on donor protection and foll o w up, the basic principles and requirements should be foreseen in the EU and national legislation. For example, when setting the age limits the need to protect minors needs to be considered. More detailed rules could in addition be foreseen in the expert level guidance documents (for example age considerations related to clinical quality and safety). Main principles of donor compensation should be set in the EU legislation, while Member States should also have some flexibility in applying the donor reimbursement/compensation rules, which in case of tissues and cells could also depend on the specific type of donation. For blood donation the principle of VUD should be maintained. Regarding quality and safety requirements ( f.ex mandatory testing for communicable diseases and genetic testing of gamete, traceability rules), the main principles should be covered in the EU legislation and more detailed guidance could be agreed by the European level expert bodies. Q16 If option 2, or a combination including option 2 is implemented, which rules should be defined by ECDC ? Rules for donor deferral/exclusion to prevent transmission of communicable diseases - X Requirements for donor selection questionnaires in relation to communicable disease transmission risk - X Communicable diseases to be screened in donors routinely and in specific circumstances - X Communicable disease testing methods to be applied (e.g. serology, NAT etc.) - X Rules for test kit selection and validation - X Rules on confirmatory testing of initially reactive tests - X Rules for testing laboratory good practice - X Rules on reporting of positive donor testing results to competent authorities or ECDC, if required by legislation – X Rules on donor sample archiving, if required by legislation - X Requirements for validation of existing or new microbial inactivation technologies - X Rules on combining measures (donor questionnaires, testing, microbial inactivation) to achieve required safety levels of BTC - X Other - X You selected 'Other'. Please describe other rules/requirements that you consider should be defined by reference to ECDC 287655 249555 As there could be significant differences in testing and microbiological inactivation protocols, ECDC should be available for consulting on specific cases not directly described in the laws or guidelines. As there could be significant differences in testing and microbiological inactivation protocols, ECDC should be available for consulting on specific cases not directly described in the laws or guidelines. 1000 character (s) maximum 1000 character (s) maximum Q17 If option 2, or a combination including option 2, is implemented, which parts of EDQM guidance should be referenced in EU legislation? -114300 25400 Good Practice Guidelines (GPG) for blood (as currently) The entire EDQM tissue and cell guide -104775 11430 Good Practice Guidelines (GPG) for tissues and cells The EDQM tissue and cell guide excluding Section C -95250 -13335 Blood component monographs Other specific sections in the EDQM guides -114300 4445 Tissue and cells component monographs - The entire EDQM blood guide No answer You selected 'Other'. Please list the sections of the guides that you consider should be referenced 1000 character(s) maximum Q18 What do you consider to be the appropriate role(s) of professional and scientific associations in the setting of technical rules for BTC? 0 -635 They should define their standards independently and those standards should be taken into account by those setting the rules for the EU They should be formally consulted on all rule changes by those setting the rules for the EU left 9525 They should be represented in expert committees established to support those setting the rules for the EU Their standards should be considered for direct referencing in EU legislation Other - You selected ' Other '. Please describe 1000 character (s) maximum You selected ' Other '. Please describe 1000 character (s) maximum Q19 Can you propose an expert body that sets standards for genetic testing of gamete or embryo donors? 180975 221615 0 0 Yes No Please provide details 500 character(s) maximum Q20 Please provide details of any other expert bodies that could be considered to define technical safety and quality rules for reference in EU legislation if option 2 is implemented, describing the technical quality and safety criteria in which they are expert 295275 266700 1000 character (s) maximum 1000 character (s) maximum Q21 Do you have comments regarding the process (e.g. participation, transparency, consultation, evidence basis) that should be followed for updating guidance by ECDC, EDQM or other expert bodies if option 2 is adopted? Yes 0 -635 No Please provide your comments here 1000 character (s) maximum Please provide your comments here 1000 character (s) maximum Q22 If policy option 3 is implemented, how can EU legislation be kept up to date most efficiently? 0 0 Revised legislation is proposed by the European Commission following guidance published by expert bodies 0 -635 The European Commission establishes a series of expert scientific committees to continuously review evidence and propose changes The European Commission incorporates technical experts in its relevant 238125 252730 policy team to review evidence and update legislation Other 269875 534247 We consider it important that EU legislation is regularly reviewed and updated in view of technological and scientific scientific developments and a mechanism should be foreseen in the basic acts providing for a review based on expert input from scientific committees and involving Member State competent authorities . We consider it important that EU legislation is regularly reviewed and updated in view of technological and scientific scientific developments and a mechanism should be foreseen in the basic acts providing for a review based on expert input from scientific committees and involving Member State competent authorities . You selected ' Other '. Please describe 1000 character (s) maximum You selected ' Other '. Please describe 1000 character (s) maximum Q23 Please enter here any further comments you may have on how technical safety and quality rules can be kept up to date with science, technology and epidemiology 2000 character (s) maximum 2000 character (s) maximum SECTION B Improving oversight of blood, tissue and cell activities The evaluation indicated that variable national approaches to oversight of blood, tissue and cell activities in Member States results in a lack of trust and create barriers to the exchange of blood, tissues and cells between Member States. 3505200 392430 0 0 Q24 Would adding any of the following general principles in EU legislation increase confidence in oversight practice? Independence from the regulated sector Adequate administrative capacity 19050 -54610 0 0 Lack of personal conflicts of interest of inspectors at each inspection Legal mandate of inspectors (to issue orders to cease activity, to seize documentation and/or samples, etc.) -104775 -58420 0 0 -113665 201295 0 0 Transparency to citizens Skill and competence of inspectors and other authority officials Other You selected 'Other'. Please describe what other regulatory principles should be defined 1500 character (s) maximum 1500 character (s) maximum Q24.1 You selected 'Transparency to citizens'. Which of the following aspects of transparency would you consider appropriate for inclusion in EU legislation? Publication of national aggregated annual reports of inspection, authorisation and vigilance activities - yes Publication of individual results of blood and tissue establishment inspections / authorisations - no Publication of the details of serious non-compliances, cessation orders or the detection of illegal practice of significance to public health - no 123824 1196975 0 0 Q24.2 You selected 'Competence of authority officials'. How can competence of authority officials be promoted/ensured by EU legislation? 3095624 735965 0 0 Specific qualification requirements -30480 2540 0 0 Regular participation in international training events (e.g. PIC/S) Certification in an EU training programme Assessment of competence by EU auditors 3057525 10160 0 0 Certification in a national training programme Participation in multi-country inspections or mutual audits -66675 -5715 0 0 Requirements for regular participation in EU organised training events Regular participation in national training events Other You selected 'Other'. Please describe 1000 character(s) maximum 295275 102235 Shortage of training events could be a problem , if regular participation is sought . Shortage of training events could be a problem , if regular participation is sought . Q24.3 How can the administrative capacity (resources) of an authority be promoted /strengthened by EU legislation? Requirements for specific staff numbers per population size Assessment of the adequacy by EU auditors 133350 12065 0 0 Participation in multi-country inspections or mutual audits Sharing of inspector resources between Member States Other You selected ' Other '. Please describe 1000 character (s) maximum You selected ' Other '. Please describe 1000 character (s) maximum The current legislation describes the key requirements for authorisation of blood and tissue establishments. The following questions explore how these might be improved in revised legislation Q25 Which of the following should be considered in revised legislation? 5457825 2223135 0 0 4990284 1900374 5400675 3202305 5454015 2699385 4973955 1510665 5438775 1061085 5446395 603885 You selected 'Other'. Please describe here other approaches for improving BE/TE authorisation requirements 334241 275705 Authoris ation for import is important, but does not need revised or specifically considered during the revision of the directives. Authoris ation for import is important, but does not need revised or specifically considered during the revision of the directives. 1000 character (s) maximum 1000 character (s) maximum Q26 There is a Commission hosted public platform with a compendium of authorised tissue establishments, indicating the activities for which they are authorised . Should there be one for Blood establishments too? 122975 0 0 0 Yes No No answer Q27 The current legislation does not require inspection or authorisation of the following entities by competent authorities. Should this be added in revised legislation? 5021286 2489835 5454015 4128135 5423535 3526155 5446395 3137535 5466245 2794635 5012055 2063115 5027295 1293495 5027295 1689735 5446395 851535 5431155 523875 Blood and tissue establishments in third countries supplying the EU Other Blood and tissue establishments in third countries supplying the EU Other You selected 'Other'. Please describe the other entities that you consider should be authorised 348615 259715 TEs that are getting their material from third countries to supply EU are currently authorised and that includes an audit of third country TE by the importing TE in EU, which should be enough . TEs that are getting their material from third countries to supply EU are currently authorised and that includes an audit of third country TE by the importing TE in EU, which should be enough . 1500 character (s) maximum 1500 character (s) maximum Q28 How should the requirements for national authorities be defined and updated? 5372100 3909060 0 0 5334000 3070860 0 0 5353050 2499360 0 0 5353050 1985010 0 0 5381625 1489710 0 0 5372100 1108710 0 0 You selected 'Other' for one or more of the requirements for national competent authorities. Please describe, specifying who you consider should define the requirements. right 289560 In all the mentioned areas, basic principles and requirements should be included in the EU legislation. However, for uniform application it is very important to have detailed guidance that could be set by the relevant EU expert bodies and expert groups. For example, for annual vigilance reporting and annual donation and use reporting it is important that the detailed guidance foresees the methodology to ensure that comparable data is collected. 0 0 In all the mentioned areas, basic principles and requirements should be included in the EU legislation. However, for uniform application it is very important to have detailed guidance that could be set by the relevant EU expert bodies and expert groups. For example, for annual vigilance reporting and annual donation and use reporting it is important that the detailed guidance foresees the methodology to ensure that comparable data is collected. Q29 Should the possibility for donors or patients to report adverse outcomes or complaints directly to the competent authority be required in legislation? 177165 9525 Yes No No answer Q30 Please describe here any further comments you may have on improving oversight of blood, tissue and cell activities right 263526 Donors and patients should always be allowed to ad d ress their complaints to competent authorities , which we believe is the case in EU. 0 0 Donors and patients should always be allowed to ad d ress their complaints to competent authorities , which we believe is the case in EU. 2000 character (s) maximum 2000 character (s) maximum SECTION C Supporting innovation for patient benefit The BTC evaluation found that innovation was not facilitated optimally. In particular, while the tissue and cell legislation includes some requirements for preparation process authorisation , the blood legislation only specifies the required characteristics of blood components for transfusion and does not require preparation process authorisation . Strengthening the authorisation of preparation processes of BTC (non-ATMP) Q31 Do you consider that new preparation processes or clinical uses for blood, tissues or cells (non-ATMP) should require a specific authorisation ? Yes 112395 271780 0 0 No No answer Q32 If authorisation of preparation processes is introduced across blood, tissues and cells (non-ATMP), which of the following should apply? 4514850 3496311 0 0 5255895 2994660 3891915 2545080 5309235 1981200 4554855 1272540 3884295 594360 4572000 544195 0 0 Authorised preparation processes should be shared and recognised between Member States Authorised preparation processes should be listed in a public register/ compendium Authorised preparation processes should be shared and recognised between Member States Authorised preparation processes should be listed in a public register/ compendium Q33 If you consider that there are other key principles relating to preparation process authorisation that should be addressed in legislation please describe them. right 248285 We would not tie the preparation process authorisation to the clinical use, as the same process could have more than one clinical application and once authorised the professionals would have some freedom in clinical application. This would lead to discussions about the degree of novelty of the clinical application, which is difficult to define, while the quality and safety of the tissue or cell product remains unchanged. Novelty of the clinical application for blood is easier to define. For Q31: Need for specific authorisation for a new preparation processes depends on the definition of new preparation processes and needs further analysis. Probably there is need for some kind of risk-benefit assessment to ensure safety of donors and recipients. A mechanism for scientific advice could be considered at the EU level. The scope and exact procedure would need to be defined taking into account safety and proportionality considerations. Also, the borderline with ATMP would need to be considered and in view of the hospital exemptions authorization procedure. 0 0 We would not tie the preparation process authorisation to the clinical use, as the same process could have more than one clinical application and once authorised the professionals would have some freedom in clinical application. This would lead to discussions about the degree of novelty of the clinical application, which is difficult to define, while the quality and safety of the tissue or cell product remains unchanged. Novelty of the clinical application for blood is easier to define. For Q31: Need for specific authorisation for a new preparation processes depends on the definition of new preparation processes and needs further analysis. Probably there is need for some kind of risk-benefit assessment to ensure safety of donors and recipients. A mechanism for scientific advice could be considered at the EU level. The scope and exact procedure would need to be defined taking into account safety and proportionality considerations. Also, the borderline with ATMP would need to be considered and in view of the hospital exemptions authorization procedure. 1000 character (s) maximum 1000 character (s) maximum Q34 What would be your assessment of the cost and administrative burden of introducing a requirement for authorisation of new preparation processes or clinical uses for blood, tissues or cells (non-ATMP), including clinical studies proportionate to the assessed risk? For competent authorities - significant For blood and tissue establishments - significant For clinical users - significant Q35 Please enter here any futher comments you may have on preparation process authorisation 2000 character (s) maximum 2000 character (s) maximum Defining whether, and if so which, BTC requirements should be applied to a substance /product Member States are responsible for deciding the regulatory status of substances/products. They might classify them as blood, tissues and cells (Substances of Human Origin) or under another legal framework such as the pharmaceutical or medical device frameworks. The BTC evaluation identified that some substances/products are regulated under different frameworks (BTC, medicinal products, medical devices) in different Member States. EU level regulatory advice can be sought on whether the legislation on Advanced Therapy Medicinal Products would apply (from the Committee for Advanced Therapies) and on whether the medical device legislation would apply (from an expert group of medical device authorities). An equivalent advisory mechanism is not established in the current BTC legislative framework. Q36 If an EU mechanism were introduced to advise on whether, and if so which, BTC requirements should apply to a substance/product, what is your view on the following statements regarding its possible role? 4048125 2023110 0 0 4067175 803910 0 0 5438775 1261110 0 0 4044315 1706880 Fully agree Partially agree Do not agree No answer It should advise on whether a substance / product should be subject to all, or certain , provisions of the BTC legislation It should advise on the appropriate legislative framework not when the BTC framework is not considered relevant The criteria it would apply should be defined in BTC legislation It should publish its advice Fully agree Partially agree Do not agree No answer It should advise on whether a substance / product should be subject to all, or certain , provisions of the BTC legislation It should advise on the appropriate legislative framework not when the BTC framework is not considered relevant The criteria it would apply should be defined in BTC legislation It should publish its advice Q37 If such an advisory mechanism were introduced, which of the following should be included in its composition? 113914 9525 0 0 Member State BTC competent authorities Patient representatives Blood and tissue establishment representatives Donor associations 152400 -31115 0 0 152400 218440 0 0 Health Technology Assessment bodies 126785 271780 0 0 Scientific experts 152400 253365 0 0 Clinical experts Others You selected 'Other'. Please describe which other type of expert should be included 303742 282787 Generally , r egulators should determine the regulatory status of the product. Scientific experts and clinical experts participating in the regulatory advisory group, should have sufficient regulatory experience to help to solve the questions where it is too difficult to find consensus between the regulators themselves. 0 Generally , r egulators should determine the regulatory status of the product. Scientific experts and clinical experts participating in the regulatory advisory group, should have sufficient regulatory experience to help to solve the questions where it is too difficult to find consensus between the regulators themselves. 1000 character (s) maximum 1000 character (s) maximum 139065 685165 0 0 Q38 If such a mechanism were introduced, who should be eligible to request advice on whether a substance/product should be subject to the BTC legislation (in part or in its entirety)? 152400 262255 0 0 National BTC competent authorities Blood and tissue establishments 150495 -34925 0 0 152400 243205 0 0 Researchers 161925 262255 0 0 Industry Professional associations Others You selected 'Other'. Please describe who else you consider should be able to request advice 1000 character (s) maximum 1000 character (s) maximum Interaction between advisory mechanisms on regulatory status of substances/products 152400 488315 0 0 Q39 Does your organisation have experience of developing therapies that are at the borderlines with other EU regulated frameworks? Yes No From your experience, how easy have the following aspects been? 5219700 689610 0 0 5229225 1313815 0 0 5905500 2866390 0 0 5233035 2057400 You indicated that some aspect was complex or you selected 'Other'. Please describe 280035 316230 1500 character (s) maximum 1500 character (s) maximum 114300 1137285 0 0 Q40 If an EU mechanism is established to advise on whether, and if so which, requirements of the BTC legislation should apply to certain substances/products, should this mechanism interact with equivalent advisory structures in other frameworks (e.g. Committee on Advanced Therapy Medicinal Products and the Medical Device Classification and Borderlines Group)? Yes No No answer Which of the following topics should be the subject of that interaction? 123825 469265 0 0 123825 -48260 0 0 Co-ordination regarding advice/recommendations on which regulatory framework should apply in borderline cases 85725 450215 0 0 Coordination regarding the application of the regulatory status (scope) criteri a in the different legal frameworks to ensure coherent advice Exchange of information in circumstances where advice/recommendations from one mechanism has an impact on another framework Other You selected 'Other'. Please describe what other topic you consider should be included in the scope of that interaction 1000 character (s) maximum 1000 character (s) maximum 137160 676275 0 0 Q41 Do you or your organisation have experience of working with substances /products that are subject to provisions of more than one regulated frameworks (BTC, pharmaceutical products, medical devices)? Yes No Please indicate the borderline(s) with which you have experience 99060 224790 0 0 95250 11430 0 0 BTC and plasma derived medicinal products ( seerumtilgad ) 98210 252730 0 0 BTC and other classical medicinal products ( FMT ) BTC and advanced therapy medicinal products ( P õlve süstitavad rasvarakud – kas jõuavad liigeseõõnde või mitte ) 123825 9525 0 0 BTC and medical devices ( Luuimplantaadid ) 98210 13970 0 0 BTC and food ( Rinnapiimapank ) 98210 8255 0 0 Other ( ravimid ja meditsiiniseadmed . Munasarjakoe vitrifitseerimislahus ) 272415 509905 Medicinal products and medical devices. The use of non- CE marked, but industrially prepared vitrification solution for ovarium tissue has been complex, as there was initially no consensus whether this should be medical device or medicinal product. Medicinal products and medical devices. The use of non- CE marked, but industrially prepared vitrification solution for ovarium tissue has been complex, as there was initially no consensus whether this should be medical device or medicinal product. You selected ' Other '. Please describe 1500 character (s) maximum You selected ' Other '. Please describe 1500 character (s) maximum Given your experience, how easy do you consider it is to comply with requirements across the frameworks? 3990976 1797685 0 4886325 778510 0 0 4914899 1292860 0 0 Easy to comply Rather easy to comply Rather challenging Challenging Meeting all technical provisions when more than one framework applies Inspection and authorisation procedures when more than one framework applies Vigilance reporting when more than one framework applies Other Easy to comply Rather easy to comply Rather challenging Challenging Meeting all technical provisions when more than one framework applies Inspection and authorisation procedures when more than one framework applies Vigilance reporting when more than one framework applies Other You indicated that compliance with one or more type of requirement was challenging or that there were aspects other than those listed. Please describe 267566 219421 Different CAs could have different interpretations on legislative requirements, which can make it very difficult to proceed with the authorisation that covers more than one framework. Different CAs could have different interpretations on legislative requirements, which can make it very difficult to proceed with the authorisation that covers more than one framework. 2000 character (s) maximum 2000 character (s) maximum 130331 488315 0 0 Q42 Do you consider that blood competent authorities should be able to authorise storage of plasma that is collected for the manufacture of medicinal products? Yes No No answer 260639 503035 Blood competent authorities should have all the competence and experience to authorise such storage Blood competent authorities should have all the competence and experience to authorise such storage Please explain your reasons 1000 character (s) maximum Please explain your reasons 1000 character (s) maximum Q43 To what extent do you consider the current blood donor selection and testing requirements appropriate for plasma collected for manufacture of plasma-derived medicinal products? Inappropriate 152400 262890 0 0 Somewhat inappropriate Appropriate No answer Please explain what you consider is inappropriate 1000 character(s) maximum Q44 Have you experienced difficulties related to the BTC legislation when importing tissues or cells for the manufacture of ATMPs or importing manufactured ATMPs 152400 243205 0 0 Yes No No answer Please explain and suggest how this might be resolved in revised BTC legislation 302895 285750 We answered „no“ mainly because we have not had this kind of experience yet , but we do not expect to have any problems with applying the import legislation, should we need it . We answered „ no“ mainly because we have not had this kind of experience yet , but we do not expect to have any problems with applying the import legislation, should we need it . 1000 character (s) maximum 1000 character (s) maximum Q45 Have you experienced difficulties related to the BTC legislation when exporting tissues or cells for the manufacture of ATMPs, or exporting manufactured 161925 253365 0 0 ATMPs? Yes No No answer Please explain and suggest how this might be resolved in revised BTC legislation 1000 character(s) maximum 250248 11597 The problems are related with the export of starting material, as it is not defined anywhere what conditions have to apply, so we had to come up with the conditions ourselves. BTC legislation should have guidance on the appropriate conditions on starting materials. The problems are related with the export of starting material, as it is not defined anywhere what conditions have to apply, so we had to come up with the conditions ourselves. BTC legislation should have guidance on the appropriate conditions on starting materials. Interplay between regulatory frameworks when more than one applies to a substance /product Q46 To what extent do you consider that interplay between regulated frameworks (BTC, medicinal products, medical devices) would be improved by increased cooperation between authorities in the different sectors at Member State level ? Significantly . Q47 To what extent do you consider that interplay between regulated frameworks (BTC, medicinal products, medical devices) would be improved by increased cooperation between authorities in the different sectors at EU level ? Significantly . Q48 If you have general comments on other topics related to innovation in the BTC sector, please enter them here SECTION D Sufficiency of supply of blood, tissues and cells Although an objective of the BTC legislation was to ensure a sustainable supply of critical blood, tissues and cells, the evaluation showed that there are dependencies on certain Member States and on third countries for certain substances, in particular plasma for the manufacture of medicinal products. In addition, it was highlighted that there is a lack of legal provisions to ensure appropriate emergency measures in the event of sudden supply interruptions. All 3 policy options under consideration include measures to monitor sufficiency of supply on a routine basis and an alert requirement in the case of sudden supply threats. Q49 How would you rate the cost and administrative burden of implementing requirements for reporting and monitoring of activity data (e.g. donations, supply, shortages) nationally and at an EU level? For blood and tissue establishments Depends on the way it will be organised . For competent authorities Depends on the way it will be organised . For hospitals/clinics that use blood, tissues and cells in patients Depends on the way it will be organised . A significant reliance of the EU on the US for its supply of plasma for medicinal product manufacture is well documented and the international exchange of haematopoietic stem cells is understood and essential for matching purposes. Significant imports of some other BTC are also reported, notably corneas and bone. Q50 How can the EU ensure sufficiency of BTC supply for EU patients without relying on imports from third countries? 4844415 2318385 4836795 1998345 4844415 1579245 4813935 1190625 4836795 862965 4813935 535305 4810125 596265 0 0 5821680 318135 5854065 1270 Supply planning at the regional , national or EU level Provisions to allow export bans Other Supply planning at the regional , national or EU level Provisions to allow export bans Other You selected 'Other'. Please explain what other measures could help the EU to achieve strategic independence of BTC supply 28575 241936 EU should focus on ensuring appropriate use of plasma and avoiding wastage (for example, by assessing the situations where member states or centres do not provide their plasma for manufacturing ) . EU should also have a policy to increase the accessibility to the recombinant coagulation factors. M echanisms for compensation for donors differ quite significantly between member states. Common EU approach on appreciation of donors could be helpful , but each member state should still have the option to introduce m ore stringent requirements in their legislation . G eneral blood donation should continue according to VUD principles . 0 0 EU should focus on ensuring appropriate use of plasma and avoiding wastage (for example, by assessing the situations where member states or centres do not provide their plasma for manufacturing ) . EU should also have a policy to increase the accessibility to the recombinant coagulation factors. M echanisms for compensation for donors differ quite significantly between member states. Common EU approach on appreciation of donors could be helpful , but each member state should still have the option to introduce m ore stringent requirements in their legislation . G eneral blood donation should continue according to VUD principles . 1000 character (s) maximum 1000 character (s) maximum Q51 How would you assess the burden (financial and administrative) of these measures for stakeholders and authorities? 4695825 1716405 0 0 4707255 1237615 4690110 854710 0 0 4733925 535305 0 4752975 3106420 4699635 2832100 4669155 2496820 4722495 2138680 Q52 If you have other comments on measures to support the achievement of BTC sufficiency, please enter them here 2000 character(s) maximum right 73025 Supply planning at regional, national and/or EU level could be among measures to be further discussed and analysed as well as the possibility to allow export bans. 0 0 Supply planning at regional, national and/or EU level could be among measures to be further discussed and analysed as well as the possibility to allow export bans. Q53 How can it be ensured that BTC are allocated according to clinical need? Requirements for priority allocation rules at establishment level - led by clinicians Requirements for priority allocation rules at national level - led by clinicians Requirements for priority allocation rules at EU level - led by clinical expert committees 209550 0 0 0 No requirements - leave establishments collect and supply according to demand Other 885825 10223754 You selected 'Other'. Please explain what other measures could help to ensure allocation according to clinical need 274493 245687 Setting allocation priorities through a separate administrative body would not improve the supply situation. When deciding about allocation priorities, the ethical and clinical aspects should be the main criteria to be considered. Setting allocation priorities through a separate administrative body would not improve the supply situation. When deciding about allocation priorities, the ethical and clinical aspects should be the main criteria to be considered. 1000 character (s) maximum 1000 character (s) maximum Q54 If you have general comments on other topics related to the sufficiency of the BTC supply, please enter them here 2000 character(s) maximum General comments and supporting documents Q55 If you have general comments on other topics related to the revision of the EU legislation on blood, tissues and cells, please enter them here. 85724 -5715 The requirements on storage and processing of infectious materials ( cells, tissues and organs ) should be revised based on the actual scientific evidence of disease transmission. This is something from the EDQM guidelines, but separation of materials with positive viral markers from other patients ’ materials may not be reasonable, as there may not be any risk of transmission . It is also necessary to review and harmonise implementation of the EU legislation from the point of view of data protection requirements. This concerns for example data required as part of adverse reaction reporting, access to personal data in the context of surveillance and cross-border collaboration between tissue/blood establishments (issues related to security, anonymization, pseudonymization, etc ). Consideration could be given at the EU level also to the issues of cross-border transportation of donor material (for example, security and customs procedures, qualification as an emergency medical flight). The COVID-19 pandemic has brought up these challenges as different Member States, airports and carriers are applying different conditions to the transportation of donor material. 0 0 The requirements on storage and processing of infectious materials ( cells, tissues and organs ) should be revised based on the actual scientific evidence of disease transmission. This is something from the EDQM guidelines, but separation of materials with positive viral markers from other patients ’ materials may not be reasonable, as there may not be any risk of transmission . It is also necessary to review and harmonise implementation of the EU legislation from the point of view of data protection requirements. This concerns for example data required as part of adverse reaction reporting, access to personal data in the context of surveillance and cross-border collaboration between tissue/blood establishments (issues related to security, anonymization, pseudonymization, etc ). Consideration could be given at the EU level also to the issues of cross-border transportation of donor material (for example, security and customs procedures, qualification as an emergency medical flight). The COVID-19 pandemic has brought up these challenges as different Member States, airports and carriers are applying different conditions to the transportation of donor material. 2000 character (s) maximum 2000 character (s) maximum You may upload one supporting document to your submission here. Only files of the type pdf,txt,doc,docx,odt,rtf are allowed THANK YOU FOR YOUR CONTRIBUTION!