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

11| SMARegistriesWorkshop2017

WholeGroupDiscussion

• Othercountryspecificchallenges• Coreefficacyassessments

• HowtoensurethatPMSdatacapturedisaccurateandcomplete- whattrainingandresourcesarerequiredforsites

(includinglessexperienced)?