commiss regul ec april lai provis implement criteria design medicin product orphan medicin product definit concept quot similar medicin product quot quot clinic superior quot commiss european commun regard treati establish european commun regard regul ec european parliament council decemb orphan medicin product articl thereof regul ec call commiss adopt provis implement articl adopt definit quot similar medicin product quot quot clinic superior quot order implement articl regul ec addit detail factor consid establish preval return invest satisfactori natur altern method diagnosi prevent treatment help sponsor committe orphan medicin product inform present accord guidanc drawn commiss pursuant articl regul ec natur medicin product concern probabl condit treat rare lai overli prescript requir establish criteria met assess criteria refer articl basi inform object commun measur field rare diseas account order ensur respect market exclus provis laid articl regul ec lai definit concept quot similar medicin product quot quot clinic superior quot definit account work experi committe proprietari medicin product evalu exist medicin product relev opinion scientif committe medicin product medic devic definit support guidelin foreseen articl regul ec provis updat regularli light scientif technic knowledg experi design regul orphan medicin product measur provid regul conform opinion stand committe medicin product human adopt regul articl purpos regul lai factor consid implement articl regul ec orphan medicin product establish definit quot similar medicin product quot quot clinic superior quot purpos implement articl abovement regul intend assist potenti sponsor committe orphan medicin product compet author interpret regul ec articl criteria design preval condit commun purpos establish pursuant subparagraph articl regul ec medicin product intend diagnosi prevent treatment life threaten chronic debilit condit affect person commun specif rule appli document list provid accord guidanc drawn pursuant articl regul ec document includ append authorit refer demonstr diseas condit medicin product administ affect person commun time applic design submit data includ detail condit intend treat justif life threaten chronic debilit natur condit support scientif medic refer document submit sponsor includ refer review relev scientif literatur provid inform relev databas commun databas commun refer made databas countri provid extrapol made diseas condit consid framework commun activ rare diseas inform provid case diseas condit includ project financi support commun order improv inform rare diseas relev extract inform includ detail preval diseas condit question provid potenti return invest purpos establish pursuant subparagraph articl regul ec medicin product intend diagnosi prevent treatment life threaten debilit chronic condit commun incent market medicin product commun gener suffici return justifi necesari invest specif rule appli document provid accord guidanc drawn pursuant articl regul ec data includ detail condit intend treat justif life threaten debilit chronic natur condit support scientif medic refer document submit sponsor includ data cost sponsor incur develop medicin product document provid includ detail grant tax incent cost recoveri provis receiv commun countri case medicin product authoris indic medicin product investig indic clear explan justif method apport develop cost indic provid statement justif develop cost sponsor expect incur submiss applic design provid statement justif product market cost sponsor incur past expect incur year medicin product authoris provid estim justif expect revenu sale medicin product commun year authoris cost revenu data determin accord gener accept account practic certifi regist account commun document provid includ inform preval incid commun condit medicin product administ time applic design submit exist method diagnosi prevent treatment applic design medicin product orphan medicin product submit accord paragraph paragraph articl irrespect applic design submit accord paragraph sponsor addition establish exist satisfactori method diagnosi prevent treatment condit question method exist medicin product signific benefit affect condit purpos establish pursuant articl regul ec exist satisfactori method diagnosi prevent treatment condit question method exist medicin product signific benefit affect condit rule appli detail exist diagnosi prevent treatment method condit question authoris commun provid make refer scientif medic literatur relev inform includ authoris medicin product medic devic method diagnosi prevent treatment commun justif method refer paragraph consid satisfactori justif assumpt medicin product design sought signific benefit affect condit gener provis sponsor appli design medicin product orphan medicin product appli design stage develop medicin product applic market authoris made applic design submit therapeut indic authoris medicin product case market authoris holder appli separ market authoris cover orphan indic sponsor obtain design orphan medicin product medicin product intend prevent treat diagnos diseas condit provid complet applic design laid guidelin articl submit case medicin product design committe orphan medicin product refer criteria design made articl articl regul articl definit definit articl regul ec appli term regul quot substanc quot mean substanc manufactur medicin product human defin articl direct eec purpos implement articl regul ec orphan medicin product definit appli quot signific benefit quot mean clinic relev advantag major contribut patient care purpos implement articl regul ec orphan medicin product definit appli quot activ substanc quot mean substanc physiolog pharmacolog activ quot similar medicin product quot mean medicin product similar activ substanc substanc contain authoris orphan medicin product intend therapeut indic quot similar activ substanc quot mean identici activ substanc activ substanc princip molecular structur featur necessarili molecular structur featur act mechan includ isom mixtur isom complex ester salt coval deriv origin activ substanc activ substanc differ origin activ substanc respect minor molecular structur structur analogu macromolecul differ origin macromolecul respect molecular structur proteinac substanc differ due infidel transcript translat differ structur due post translat event glycosyl pattern tertiari structur differ amino acid sequenc major pharmacolog relat protein substanc group biolog compound intern proprietari inn stem consid similar monoclon antibodi bind target epitop consid similar polysaccharid substanc ident saccharid repeat unit number unit vari post polymeris modif includ conjug polynucleotid substanc includ gene transfer antisens substanc consist distinct nucleotid differ nucleotid sequenc purin pyrimidin base deriv major antisens substanc addit delet nucleotid significantli affect kinet hybridis target consid similar gene transfer substanc differ sequenc signific substanc consid similar differ structur relat modif ribos deoxyribos sugar backbon replac backbon synthet analogu differ vector transfer system close relat complex partli defin substanc relat viral vaccin relat cell therapi product radiopharmaceut activ substanc differ origin radionuclid ligand site label molecul radionuclid coupl mechan link molecul radionuclid provid act mechan quot clinic superior quot mean medicin product shown provid signific therapeut diagnost advantag provid authoris orphan medicin product wai greater efficaci authoris orphan medicin product assess effect clinic meaning endpoint adequ control clinic trial gener repres kind evid need support compar efficaci claim medicin product direct compar clinic trial gener comparison base endpoint includ surrog endpoint case methodolog approach justifi greater safeti substanti portion target popul case direct compar clinic trial except case greater safeti greater efficaci shown demonstr medicin product make major contribut diagnosi patient care articl entri forc regul enter forc dai adopt commiss appli dai regul bind entireti directli applic member state brussel april commiss erkki liikanen member commiss oj