Drucken:Dieses+Poster+ist+121,92hoch+und+für+den+Druck+auf+einem+Großformatdrucker+ausgelegt.
Anpassen+des+Inhalts:Die+Platzhalter+im+Poster+wurden+bereits+für+Sie+formatiert.+Geben+Sie+Ihren+Text+in+den+Platzhaltern+ein,+oder+klicken+Sie+auf+ein+Symbol,+um+eine+Tabelle,+ein+Diagramm,+eine+SmartArtGrafik,+ein+Bild+oder+eine+Multimediadatei+hinzuzufügen.
Wenn+Sie+Aufzählungspunkte+hinzufügen+oder+entfernen+möchten,+klicken+Sie+auf+der+Registerkarte+"Start"+auf+die+Schaltfläche+"Aufzählungszeichen".
Wenn+Sie+weitere+Platzhalter+für+Titel,+Inhalt+oder+Textkörper+benötigen,+erstellen+Sie+eine+Kopie+des+gewünschten+Elements,+und+ziehen+Sie+es+an+die+gewünschte+Position.+Die+Funktion+"Intelligente+Führungslinien"+von+PowerPoint+hilft+Ihnen,+die+Elemente+am+restlichen+Inhalt+auszurichten.
Möchten+Sie+eigene+Bilder+anstelle+der+vorhandenen+Bilder+verwenden?+Kein+Problem!+Klicken+Sie+mit+der+rechten+Maustaste+auf+ein+Bild,+und+wählen+Sie+"Bild+ändern"+aus.+Behalten+Sie+die+Proportionen+von+Bildern+beim+Ändern+der+Größe+bei,+indem+Sie+eine+Ecke+ziehen.
Efficacy+of+an+orphan+drug:+The+patient+perspectiveCornelia)Dechant1,)MD;)Francesca)Granata2,)PhD;)Jasmin)Barman:Aksözen,)PhD3
1"[email protected]"[email protected]"[email protected]
THE"DISEASE:"ERYTROPIOETIC PROTOPORPHYRIA
Afamelanotide is"a"hormon analogon,"which"induces"skin"pigmentation"and"scavenges"radicals,"was"approved"”under"exceptional"circumstances”"by"EMA."In"2014"to"reduce"the"excruciating"pain"caused"by"severe"phototoxic"reactions"in"patients"suffering"from"erythropoietic protoporphyria (EPP).
THE"MEDICAMENT
Two"phase"II"and"three"phase"III"clinical"trials"with"347"participants"and"a"longTterm"observational"study"with"115"patients"from"the"Swiss"and"Italian"compassionate"use"and"early"access"schemes"showed"a"significant"extension"of"sun"exposure"time,"a"reduction"of"phototoxic"reactions"and"an"improvement"of"quality"of"life"under"the"therapy"with"afamelanotide.
THE+STUDIES
EPP"patients"under"therapy"describe"the"effect"of"the"treatment"as"transformative"and"life"changing."During"the"approval"process,"EPP"patients"for"the"first"time"in"the"history"of"EMA"were"allowed"to"participate"at"a"full"committee"meeting"and"their"description"of"the"clinical"benefit"contributed"to"the"positive"opinion"for"approval."However,"in"their"final"assessment"report,"EMA"claims"that"afamelanotide was"able"to)extent)sun)exposure)for)8)minutes)per)day)only For"standardization,"EMA"in"a"first"step"recalculated"the"median"and"in"a"second"step"divided"the"resulting"24h"(median)"additional"pain"free"sunlight"exposure"through"the"whole"period"of"the"study"(6"Month),"not"regarding"that"they"included"rainy"weather"periods"or"working"days."In"addition,"based"on"their"own"recalculation,"the"benefit"is"questioned"in"the"report"by"EMA,"although"such"a"standardization"obviously"cannot"capture"the"entire"benefit"of"the"treatment
RESULTS++BASED+ON+QOL+AND+SUN+EXPOSURE+TIME
EPP"is"an"ultraTrare"congenital"disease,"which"results"from"a"reduced"activity"of"ferrochelatase,"an"enzyme"of"the"heme biosynthesis."The"enzyme"deficiency"leads"to"an"accumulation"of"the"heme precursor"protoporphyrin IX"in"the"whole"body,"but"especially"in"blood,"bone"marrow"and"in"the"liver."Protoporphyrin IX"reacts"with"the"blue"part"of"the"visible"light"(400T410"nm)"and"causes"acute"photototoxic reactions"due"to"formation"of"oxygen"radicals"in"vessels"and"subcutaneous"tissue,"leading"to"unbearable"pain"and"a"severely"reduced"quality"of"life."Patients"with"EPP"have"to"wear protective clothing toprevent phototoxic reactions,"or remain indoors,which"leads"to"social"isolation"as"well.
INTERPRETATION+OF+THE+EMA
SINGLE"PERSON"OBSERVATION"FOR"2"MONTH"EACH WITH"&"WITHOUT"AFAMELANOTIDE
CONCLUSION
The"pivotal"study"describes"a"significant"prolongation"of"sun)exposure)time)of)55h)(mean/)patient;)28.6)h)median))over)6)months).The"quality of life (QoL)"scores,"measured by an"EPPTspecific questionnaire,"were 31 ± 24%"of maximumprior to afamelanotide treatment,"rose to 74%"after"starting afamelanotide andremained at"this level during the entire observation period."Only minor"adverseevents attributable to afamelanotide,"predominantly nausea,"were recorded.
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Schmerzen in Relation zur Wetterlage vor und nach Therapiebeginn
Wetterlage: 0- Regen, 1- bewölkt/tlw. Regen, 2- tlw. bewölkt, 3- Sonne
Schmerzen: 0- keine, 1- gering, 2- belastend, 3- sehr belastend, 4- nicht mehr arbeitsfähig, 5- verrücktmachend
Therapiebeginn
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Vermummung in Relation zur Wetterlage vor und nach Therapiebeginn
Wetterlage: 0- Regen, 1- bewölkt/tlw. Regen, 2- tlw. bewölkt, 3- Sonne
Schutzbekleidung: 0- keine Vermummung, 1- nur Hut und Schal, 2- volle Montur
Therapiebeginn
Pain irelation to theweather conditions before and after)treatmentwith afamelanotideBlue:"Weather condition 0:"Rain,"1:"cloudy/"rainy;"2:cloudy"/sunny;"3:"SunRed:"Pain: 0:"no pain;"1:"mild"pain;"2:"moderate"pain;"3:"very painful;"4:"not"possible to work because of pain;"5:"to be out"of onesmind with pain
Based on"the PainTDiary of Cornelia"Dechant"2017
Protection clothes in)relation to theweather conditions before and after)treatmentwith afamelanotideBlue:"Weather condition 0:"Rain,"1:"cloudy/"rainy;"2:cloudy"/sunny;"3:"Sun;"Yellow:"protection/clothes : 0:"none ,"1:"hat"and scarf,"2:"full protection(hat","scarf,"gloves,"socks...)Based on"the PainT Diary of Cornelia"Dechant"2017
Although"EPPTPatients"report"about"a"significant"longer"sun"exposition"time"and"an"eminent"improvement"of"quality"of"life"under"therapy"with"afamelanotid,"the"postThoc"recalculations""of"the"EMA"and"the"associated"seemingly"short"prolongation"of"sun"exposure"time"led"to"a"severe"impairment"of"the"reimbursement"process"of"afamelanotideTtherapy"in"most"of"the"countries"of"the"EU.We"think"that"a"more"patientTcentered"approach"to"describe"the"benefit"is"necessary"to"avoid"such"situations,"especially"in"rare"diseases."
SINGLE"PERSON"OBSERVATION"FOR"2"MONTH"EACH WITH"&"WITHOUT"AFAMELANOTIDE
Literature:"Biolcati,"G.,"Marchesini,"E.,"Sorge,"F.,"Barbieri,"L.,"SchneiderTYin,"X.,"&"Minder,"E."I."(2015)."LongTterm"observational"study"of"afamelanotide in"115"patients"with"erythropoietic protoporphyria."British'Journal'of'Dermatology,"172(6),"1601T1612.Langendonk,"Janneke G.,"et"al.""Afamelanotide for"erythropoietic protoporphyria.""New'England'Journal'of'Medicine 373.1"(2015):"48T59.«Patients"involved"in"discussions"on"benefits"and"risks"of"a"medicine"at"CHMP"for"the"first"time»"http://www..ema.Europe.eu/docs/en_GB/document_library/Press_release//2014/10/WC500176355.pdf
European"Public"Assessment"report"(EPAR)"afamelanotide:""23"October"2014,EMA/CHMP/709396/2014"Rev.1Committee"for"Medicinal"Products"for"Human"Use"(CHMP)http://www.ema.Europe.eu/docs/en_GB/document_library/EPARTPublic_assessment_report/human/002548/WC500182309.pd
Quality"of life (QoL)"scores vs."treatment duration."The"treatment period is indicated on"the abscissa and the QoL scores on"the ordinateLongTtermobservational study of afamelanotide in"115"patients with erythropoietic protoporphyria
This"private"„painTdiary“"written by an"EPP"Patient,"used as documetation for theinsurance ,shows an"impressive effect on"the daily life of this patient("with regard tothe pain and the protection clothes)"""""
