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ASustainable,Multi-stakeholderApproachforMAPPS- MedicinesAdaptivePathwaystoPatients
AcceleratedDevelopmentofAppropriatePatientTherapies
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
MeetJane.JanewasDiagnosedwithAdvancedCanceraYearAgo
Atfirst,hertumour respondedtotreatmentbutithasnowcomeback,andJaneknowstheoutlookisbleak.
Thereisanewexperimentaltreatmentbeingtested.Resultsfromstudiesandearlyclinicaltrialslookpromisingbutitisstillearlydays.
Jane’sreactionisswift:“Ineedtogivethisatry”
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
MeetJohn.JohnHasaHighRiskforCancer,GivenhisFamilyHistory
Johnbelievesthat,ifhegetscancer,itisstilladecadeortwoaway.
Oneday,Johnhears“thereisanewexperimentalcancertreatment,itisanovelconcept.Results fromstudiesandearlyclinicaltrialslookverypromisingbutitisstillearlydays.”
John’sreactionisswift:“Ihopethisproductisavailableafterhavingbeenthoroughlytriedandtested– Ihavenoappetiteforuncertainty.” 3
©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
Whoisright?WebelievethatbothJaneandJohnare“right”,giventheirrespectivesituations,andthatHealthcareDecision-MakershaveanobligationtobothJaneandJohn.
MedicinesAdaptivePathwaystoPatients(MAPPs)seekstofosteraccesstobeneficialtreatmentsfortherightpatientgroupsattheearliestappropriatetimeintheproductlife-spaninasuitablefashion
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
• AstraZenecaisdeputyprojectleader;Lygatureisprojectcoordinator
• EstablishcollaborativesolutionsandaddressbarrierstotheimplementationofMAPPs
• Bringtogether patients,regulators,industry,HealthTechnologyAssessment(HTA)bodies,payers,andacademicsundertheumbrellaoftheIMI
• Providepatientswithmoreappropriateaccesstoinnovativemedicines
• Initiateworkpackagestoaddressexistingbarriersandtestconceptstodevelopa‘tool-kit’tofacilitateMAPPs
AMulti-StakeholderIMIProjectledbytheEuropeanMedicinesAgencyInvestigatingMAPPs
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
• FundedbyIMI-JU• 01June2015– 1Dec.2017• 23EFPIAmembers• 2patients’organisations• 2HAs,2HTAs• Payersasobservers• Totalcost € 2260000
APublicPrivatePartnership
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
AddressingStakeholderConcerns…
• WhichmedicinesshouldMAPPsfocuson?
• ShouldMAPPsbesomethingforspecialcases,concentratingonthecomplexitiesofdemonstratingthereal-lifeadded-valueofuniquetreatments?
• Ifwedevelopnewapproachestodatageneration,howcanwepreventadeclineinthequalityofevidence?
• Doesanadaptiveapprovalmeanalsoanadaptivepricing&reimbursement?
• Howcanweensurethattheproductwillnotbeusedoff-labelandthecommitmentsaremet?
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
… &FacilitatingMAPPs
• ProduceactionablerecommendationstoassistwiththeimplementationandadoptionofMAPPs
• FindsolutionstothechallengesoftheimplementationofMAPPsandfosterthealignedunderstandingofconsortiummembersandtheirconstituents
• Distributefindings,keydiscoveriesandcasestudiesfromongoingorcompletedMAPPspilotprojects,creatingaMAPPsrepositoryofknowledgeandopportunities
• SupportIMI2 byfacilitatingtheinclusionofMAPPsenablers,toolsandmethodologies toaddressitschallengesandopportunities
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
WorkPackage1:Evidencegenerationovertheentireproductlifecycle
SarahGarnerNICE
SolangeRohouAstraZeneca
WP1LeadsGOAL: Determinehowtheactualperformanceofthenewtherapywilldictateitsusebasedontheevidenceofitseffectivenessandsafetyovertime
WorkPackage1hastwocoreobjectives:
• AnalyseandmonitoractivitiesthatoccurbothwithinandoutsideofIMIprogrammesthatprovideandgenerateevidenceaboutMAPPs
• DevelopagapanalysisofthecurrentandfutureneedsofevidencegenerationtosupportMAPPs.
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
INPROGRESS
• CompleteareportofMAPPsresearchgapsbasedonthereviewofmatureIMIprojectstoinformhowbesttoleveragepre-existingknowledge
• CreateahorizonscanningreportoffutureIMIandnonIMIprojectstoanticipatetheoutcomesoffutureprojectstoinformMAPPs
• CreatecollaborativeresearchproposalsbasedongapanalysisofwhatADAPTSMARTneedstoaccomplish
WorkPackage1:StatusofActions
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
WorkPackage2:DesigningaNewMAPPspathway
AndreBroekmansLygature
LukMaesBristol-MyersSquibb
WP2Leads GOAL:Createanewdevelopmentpathwaywherepatients,regulators,HTA/payers,practitioners,andindustryarealigned
WorkPackage2hasthefollowingobjectives:• CreateastandardisedglossaryoftermsforMAPPs• Identifyaseamlesspathwayfrompre-clinicalresearchto
marketaccess• DefineengagementcriteriaofproducttoentertheMAPPs
pathways• Developtoolstoensureappropriateuseofproductsby
patients• IntegratefindingsfromtheEMApilotprojectandother
relevantinitiativestoMAPPs 11
©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
COMPLETED• ADAPTSMART Glossary is availablefordownload
INPROGRESS• CreateproposalforpragmaticoperationalcriteriaforMAPPsdecision(s)
• Selectthemethods/toolset&dataforsamplescenarios
• CreatebriefingdocumentsabouttheEMApilotsforworkinggroupsintheproject
• Createareportwithrecommendationsforthetools/systemstobeusedtoguideappropriateuse
WorkPackage2:StatusofActions
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
INPROGRESS• Mappingofthedifferent
transition/engagementmomentswithstakeholders thatarepartoftheMAPPsprocess.
ADAPTSMART hasidentified23keystakeholderdecisionpointsthatMAPPsmustsuccessfullyaddress
WorkPackage2:StatusofActions
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
WorkPackage3:ImplicationsofDecision-makingandSustainability
RichardBarkerCASMI
AliciaGranadosSanofiGenzyme
WP3LeadsGOAL: Addresstheopportunitiesandobstaclestoasuccessfulandethicalimplementationofadaptivedecisionmakinginresearch,regulatory,andmedicalpractice
WorkPackage3includesthefollowinginitsscope:• R&Defficienciesandinefficiencies• Pricingarrangements,risk-sharing,effectivepatient
accessandbusinesscasesforsponsors• Ethicalimplicationsforpatientsandclinicians• Intellectualpropertyandregulatorydataprotection• Legalimplicationsforalldecisionmakers• Personaldataprotection 14
©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
INPROGRESS
• Createamatrix thatcontrastskeydecisionpointsofcurrentvs.futureprocessesè availableontheAdaptSmart.euwebsite
• Createaninventoryandpossiblepaperoncurrentlyavailablemanagedentryagreements & establishrecommendationsontheapplicabilityofmanagedentryagreementsè podcastavailableontheAdaptSmart.euwebsite
• Establishthepointstoconsideronethical&legalaspectsofadaptivedecisionmaking
WorkPackage3:StatusofActions
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
WorkPackage3:ApplicationofManagedEntryAgreements(MEAs)
• Anarrangement betweenamanufacturer andpayer/provider thatenablesaccessto(coveragereimbursementof)ahealthtechnology,subjecttospecifiedconditions.
• Canuseavarietyofmechanismsto
– addressuncertaintyabouttheperformanceoftechnologies
– managetheadoptionoftechnologiesinordertomaximizeeffectivelytheiruse,orlimittheirbudgetimpact
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
Financial-basedMEAs
• Agreementsbetweenmanufacturerandpayersbasedonobservablefinancialperformance– Priceagreementbasedonmanufacturer’smarketshare– Price-volumeagreements– Pricingbychannel(discountoncertainproducts/channels)– Capitation(discountsforspecificpatients)– Freeinitiation(Patient/dosedependentdiscount)– Portfolioagreement(discountsbasedonmanufacturer’sportfolio)
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
Outcomes-basedMEAs
• Agreementsbaseondefinedoutcomes(generallyclinical)oragreementsbasedonthedevelopmentofnewevidence.Thisinclude
– Patientrisksharingagreement(pricedecidedbasedonpatientsubgroupswithrespecttoprobabilityofbenefitsoftreatment)
– Performance-linkedreimbursement(e.g.outcomeguaranteed)
– Conditionalcoverage(reimbursementlinkedtothedevelopmentofadditionalevidence)
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
AdaptSmart.EU– AResourceforMAPPsandProjectStatus
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
Someusefuldocumentsreleasedonthewebsite
• Engagementcriteriadocument: http://adaptsmart.eu/wp-content/uploads/2016/02/ADAPT-SMART-Engagement-Criteria-Final1.pdf
• ShortreportoftheworkshoponManagedEntry (5July2016): http://adaptsmart.eu/wp-content/uploads/2016/02/ADAPT-SMART-Managed-entry-short-WS-report.pdf
• ShortreportonSeamlesspathway(6July2016): http://adaptsmart.eu/wp-content/uploads/2016/02/ADAPT-SMART-Seamless-pathways-short-WS-report.pdf
• Forinfo:theCharlesRiverAssociatesreportcommissionedbyEFPIA: http://adaptsmart.eu/report-by-charles-river-associates-use-of-managed-entry-agreements-for-products-with-a-conditional-or-exceptional-authorisations/
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©CopyrightIMIADAPTSMART|ThisprojecthasreceivedfundingfromtheInnovativeMedicinesInitiative2JointUndertakingundergrantagreementNo115890.ThisJointUndertakingreceivessupportfromtheEuropeanUnion’sHorizon2020researchandinnovationprogrammeandEFPIA.
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A Sustainable, Multi-stakeholder Approach for MAPPS - Medicines Adaptive Pathways to Patients
Accelerated Development of Appropriate Patient Therapies
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