1| SMARegistriesWorkshop2017
SMARegistriesWorkshop,Amsterdam,May2017
TREAT-NMD,AnneOyewole
PostMarketingSurveillance(PMS)
2| SMARegistriesWorkshop2017
PostMarketingSurveillance(PMS)• PMS(alsoknownas Phase IV)isthepracticeofmonitoringthesafetyofa
pharmaceuticaldrugandisessentialtoassureproductsafetyandlong-termefficacy
• PMScommitment:imposedorvoluntary• DifferenttypesofPMSapproaches:spontaneousreportingdatabases,
prescriptioneventmonitoring,electronichealthrecords,patientregistries• PMShelpstofurtherrefine,orconfirmordeny,thesafetyofadrug• PMSdataiscriticalinhelpingpharmaceuticalcompaniesandthe
regulatorsreliablymeasurethehealthandqualityoflifeofpatients
3| SMARegistriesWorkshop2017
WhyusePMSPatientRegistry?Allowsthecollectionoflarge-scaleandlong-termdataandalsoallowstheopportunitytocollectinformationrelatingtothefollowing:
• Monitoringofsafety/efficacyoftherapeutictreatmentincludingadverseeffectsanddrug-druginteractions
• Assessmentofaccesstotreatmentandreimbursementissues(includingimpactofreimbursementpolicy)
• Health-caresystemaccessandutilisation• Physicianexperience• Patient-reportedoutcomes• Compliance• Burdenofillness• Collectionofhealtheconomicendpoints• Collectionofproductusagestatisticsandcostassessment
4| SMARegistriesWorkshop2017
WhatistheincentiveforpatientstobeinaPMSRegistry• PatientparticipationinPMScontributestodrugdevelopmentandtheimprovementof
healthcare• Patientscontributerealworldexperienceandhealthdatathatcannotbeobtainedin
phases1-3ofclinicaltrials• Theevidencecollectedfrompatientshelpstheregulatorsandpharmaceutical
companiesdeterminetheeffectiveness/safetyoftreatment
5| SMARegistriesWorkshop2017
PTCObservational‘Non-Interventional’Study
• Recruitmentof200patientsacrossEurope(consentrequired)
• Adefinedgroupofpatient(aged5>withnon-sensemutation)arefollowedfor5years
• Patientarenotrandomlyassignedandthereisnoblinding
• Datacapturedonthesafetyandeffectivenessisstoredinacertified,regulatorycompliantPMSregistry(e.g.database)
• DatahelpsPTCandEMAreliablymeasurethehealthandqualityoflifeofpatients
6| SMARegistriesWorkshop2017
LessonsLearntfromPMSinDMD- Regulators
Termsandconditionsofapprovalmayberestrictive:
• Companymaybeunabletoselectsites• Numberofpatientsandfollowupperiodispre-determined
• Outcomemeasuresareimposed• Differentregulatorsmaystipulatedifferenttermsandconditions
7| SMARegistriesWorkshop2017
LessonsLearntfromPMSinDMD- CountryDifferences
• Delaysinprescribingproductoccurreddueto§ Nationalapprovalprocess- variationfromcountrytocountry
despiteEMA(conditionalapproval)§ Negotiationsonpriceandethicalconsentonacountrylevel
• GermanyhaveafasterroutefordrugapprovalcomparedtootherEuropeancountries
• The‘payers’ofthedrugcanstipulatefurtherconditionsandeveninsistthatdataiscollectedinadifferentway
Europe
8| SMARegistriesWorkshop2017
LessonsLearntfromPMSinDMD– PatientRecruitment• NotalltrialpatientsrollintoaPMSphase
§ Dependsoncountry§ Siteandethics
• Oncethedrugisprescribedpatientshavetosignacontracttoensuretheyattendfollowupappointments
9| SMARegistriesWorkshop2017
LessonsLearntfromPMSinDMD- PMSSystem
• Clinicianreportedentriesonly• RegulatorycompliantITsystemis
required• Standarddataentrymaybedifficultto
achieveduetonationaldifferences• Notallcliniciansindifferentcountries
willbeexperiencedwithfunctionaltests.Trainingmayberequired(oragreementthatdatacanbeomitted)
10| SMARegistriesWorkshop2017
LessonsLearntfromPMSinDMD– OutcomeMeasures
Best Outcome Measures
Industry
Academics
PatientsRegulators
Clinicians