Root Causes Of Unavailability And Delay EFPIA - CRA Report 2024

Therootcausesofunavailabilityofinnovativemedicinesanddelayinaccess:Shorteningthewait ExecutiveSummary1 Theunprecedentedspeedofinnovationexhibitedoverthelastfiveyearsandthepromiseoftheindustrypipeline2provideanimportantopportunitytoimproveoutcomesforpatients.Thereiscommonagreementthatthevalueofinnovationisrealisedonlywhenpatientsbenefitfromadvancesintreatment.However,asignificantnumberofmedicinesarenotavailableacrossallEuropeanUnion(EU)markets.3 EFPIAhasformanyyearslookedatthelengthoftimeittakesformedicinestobemadeavailable.AsillustratedbythemostrecentdatainthePatientW.A.I.T.IndicatorSurveyin2023,theaveragetimetoreimbursementforinnovativetreatmentsacrossEUandEuropeanEconomicAreacountrieshasreached531days,rangingfrom126daysinGermanyto990daysinTurkey.4TherearepatientaccessinequitieswithinEurope,withsignificantdifferencesacrosscountriesinthenumberofproductsavailableatanygiventimeandthatthetimetakenpriortonationalreimbursementalsovariessignificantlyfromonecountrytoanother.Theindustrysharesconcernaboutthesedelaysandrecognisethatdelaysandtheunavailabilityofmedicinesharmpatients.TheseconcernsareimportantcontextforthedebateregardingtheimpactoftheEU’sGeneralPharmaceuticalLegislationandwhetheritwillimproveaccesstomedicinesforpatientsintheEU. Overthepastfouryears,EFPIAhasdocumentedtherootcausesofaccessinequalityandfound10interrelatedfactorsthatexplainunavailabilityanddelay(definedaslengthoftimefromEuropeanmarketingauthorisationtoavailabilityatthememberstatelevel)withregardtoinnovativemedicines,buildingontheW.A.I.T.analysis.5ThesefactorsarerootedinmedicinesaccesssystemsandprocessesintheEUmemberstatesandthecorrespondingimpactoncommercialdecision-making.Theyincludeaslowregulatoryprocess,lateinitiationofmarketaccessassessment,duplicativeevidencerequirements,reimbursementdelays,andlocalformularydecisions.Becausetherootcausesaremultifactorial,theycanbesolvedonlybystakeholdersworkingtogether. 1Terminology:ThispaperhasadoptedtheterminologyusedintheupdatedW.A.I.T.analysis.Definitionsareintheglossary. 2IQVIA(2024)GlobalTrendsinR&D2024.Availableat:https://www.iqvia.com/insights/the-iqvia-institute/reports-and-publications/reports/global-trends-in-r-and-d-2024-activity-productivity-and-enablers[accessedApril2024] 3ThispaperfocusesprimarilyonrootcausesofdelaywithrespecttoproductsapprovedcentrallybytheEMA.Therearenon-centrallyapprovedmedicinestowhichmanyoftheserootcauseswouldalsoapply. 4ThePatientW.A.I.T.Indicator2023Survey.Note:NodatesweresubmittedintotalforMalta,soitisexcludedfromtherangeandaveragecalculation. 5https://www.efpia.eu/publications/downloads/efpia/the-root-causes-of-unavailability-and-delay-to-innovative-medicines/ Table1:Therootcausesofdelaysandunavailability Category Potentialrootcauses Thetimebeforemarketingauthorisation 1.Thespeedoftheregulatoryprocess 2.Accessibilityofmedicinesbeforemarketingauthorisation Thepriceandreimbursementprocess 3.Initiationoftheprocess4.Thespeedofnationaltimelinesandadherence Thevalueassessmentprocess 5.Misalignmentonevidencerequirements6.Misalignmentonvalueandprice 7.Thevalueassignedtoproductdifferentiationandchoice Healthsystemconstraintsandresources 8.Insufficientbudgettoimplementdecisions 9.Diagnosis,supportinginfrastructure,andrelevancetopatients Thesubnationalapprovalprocess 10.Multilayerdecision-makingprocess Theindustryconsidersthattherootcausesofunavailabilityanddelaycouldbeaddressedthroughcollaborativeworkwithmemberstates,theEuropeanCommission,andotherstakeholdersonproposalstoimproveavailabilityandreducedelays.Thisworkmustoccurfromthebeginningoftheprocess,includingproposalstospeeduptheregulatoryprocesstodeliversafe,high-qualitydiagnostics,vaccines,andtreatmentstopatientsasfastaspossible.TheindustrywelcomestheEuropeanParliament’scommitmenttodevelopingafuture-proofregulatoryframeworkthatisreflectedinitsrecentlyadoptedpositiononproposedreformstoEUpharmaceuticallegislation.TheindustryalsosupportsthepragmaticdecisionbyMembersoftheEuropeanParliamenttonotlinkregulatorydataprotection(RDP)tomedicineavailability. Findingworkablesolutionstoimprovepatientaccesstomedicineswillrequiremulti-stakeholdercollaborationandconsiderationofthemultifactorialnatureofrootcausesofunavailabilityanddelay.EFPIAanditsmembershaveworkedonaseriesofspecificproposalstoimprovepatientaccesstoinnovativemedicinesandreduceinequalitiesacrossEurope.Thefollowingaresomeoftheseproposals: Acommitmentfromtheindustrytofilepricingandreimbursement(P&R)applicationsinallEUcountriesnolaterthantwoyearsafterEUmarketauthorisation,providedthatlocalsystemsallowit.ThiscommitmentreflectsthejointambitionofindustryandsocietytomakeinnovationforunmethealthneedsavailabletopatientsandhealthsystemsacrossEuropeassoonaspossible. Thecreationofaportalwheremarketingauthorisationholders(MAHs)canprovidetimelyinformationregardingthetimingandprocessingofP&Rapplicationsinthe30Europeancountries.SuchinformationincludesthereasonsfordelayinaP&RdecisionortheMAHhavingnotfiledinaparticular market.Theportalisnowinitssecondyearofoperation,andEFPIAhaspubl