getting to access : context for innovative approaches for access to innovative therapies
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D U R H A N E W O N G - R I E G E R
ContextforInnova-veApproachesforAccesstoInnova-veTherapies
Innova&veMedicinesOfferOpportuni&esandChallenges
� Whythisforumma9ers:Innova-vedrugtherapiesaremedicinesthatcan:¡ Offerasubstan-alimprovementoverexis-ngtherapies;forexample,biologicscanmodifytheunderlyingcauseofacondi-onratherthanjusttreatthesymptoms;
¡ Addressanunmetneed;manydrugsforrarediseasearethefirsttobedevelopedforthatcondi-on
¡ Targetthe(unique)characteris-csofasubgroupofpa-entsbasedongene-cmakeupand/orotherindividualcharacteris-cs
¡ Curethedisease;forexample,byelimina-ngthecause(hepa--sCvirus),replacingabnormalcells(stemcellreplacement),ormodifyingthefaultygene
Innova&veMedicinesOfferOpportuni&esandChallenges
� Butinnova-vetherapiescomeatapricethatisoKenconsidered“unaffordable”undercurrentassessmentprocesses¡ Individually,therapiestendtobemuchhigherthanthe“old”therapies
theyarereplacing¡ Thenumberofinnova-vetherapiesisincreasing,asistheimpactondrug
budgets¡ Ourassessmentprocessesdonotaddressinnova-on;theyweredeveloped
todeterminehowmuchincrementalcostshouldbepaidforincrementalbenefitstoexis-ngtherapiesandhowtoallocatedrugbudgetstoachievemostoverallhealthofapopula-on,regardlessofthecondi-onsandneed
¡ Individualhigh-costtherapiescanhaveaniden-fiableimpactonoveralldrugplanbudgets,whetherprivateorpublic
AccesstoMedicines:Vic-mofAging
� Agingeconomieswithlimited,zero,ornega-vegrowthpuSngpressureongovernmentstomeetincreasedneedswithfewerresources
� Aginghealthinfrastructuresorganizedaroundout-datedmodelsofcareandinefficienttechnology¡ Resourcesallocatedaroundhospital-basedcri-calcare¡ Lackofinvestmentinchronicdiseasesandpreven-on
� Agingpopula-onsrequiringmorehealthcare¡ Be9erhealthcare,nutri-on,safetyresultinmorepeoplelivinglongerandgeSngdiseasesofoldage
� Agingdrugbudgets¡ Stableordecreasingbudgetsbuiltoninfluxofgenericsubs-tutes¡ Innova-vetherapiesofferingunprecedentedbenefitstobroaderaswellasnichepopula-onsbutat
unprecedentedprices� AgingR&Dapproaches
¡ Research“quagmire”indiscretephasesandRCTgoldstandard¡ Earlyregulatoryapprovals(forunmetcri-calneeds)leadingtorejec-onbypayersforlackof
evidence¡ Lackofacceptedscien/ficmethodologytoevaluate“real-world”data(andlackoftechnologyto
collect)
Innova/onsElsewhere
� ADAPTSMART(EMA/EUNEHTA/IMI)� NEWDIGS-MIT� UKHST� Germany,FranceManagedAccess/CED
EURORDIS
� NewBlueprinttoCutcostsandfasttrackR&D� EarlydialogueandEuropeancollabora/onondetermina/onofvalue
� Europeancoopera/onframeworkforfairpricesandsustainablehealthcarebudgets
� Con/nuumofevidencegenera/on
Innova/veMedicinesOfferOpportuni/esandChallenges
� Identify options for access that work for all ¡ Supportmanagedaccess(criteria-based,monitored)¡ Addresseskeyconcernsofallstakeholder(/meliness,placeintherapy,value,
budgetimpact)¡ Forwardlookingtobestintegratefuturemedicines(rare,personalized/
targeted,stemcellandgenemodifica/on,repurposed,biosimilars)� Iden/fywaysforallstakeholderstoworkcollabora/velytowardconsensusapproach¡ Iden/fywhatisworkingincurrent(Canadian)approachesandshouldbe
enhanced¡ Iden/fywhatisgeXnginthewayandstrategiesforaddressing¡ Iden/fyareasofagreement(keep),disagreement(resolve),andambiguity
(clarify)¡ Iden/fypathwaystoopencommunica/on,transparency,andengagement
(ofallstakeholders)