accelerated development of appropriate ... -...

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