EuroBioForum 2013 - Day 1 | Katherine Payne


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EuroBioForum 2013 2nd Annual Conference
27-28 May 2013 - Hilton Munich City, Munich, Germany



Towards market access for personalised medicines: opportunities and recommendations

Katherine Payne
Professor of Health Economics, Health Sciences - Economics, University of Manchester
Member EuroBioForum Strategic Advisory Board


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EuroBioForum 2013 - Day 1 | Katherine Payne

  1. 1. 27-28 May 2013, Munich, GermanyEuroBioForum 2013 2nd Annual ConferenceKatherine PayneThe University of Manchester
  2. 2. Personalised Medicine(s)?• From a hyped theoretical concept to clinical application:now the perfect solution to offer good value for money?• Targeting of medicines using a biomarker or genetic-baseddiagnostic to identify the eligible patient population• The most common current applications involve stratifyingcancer patient populations using companion diagnostics• But other personalised applications in medicine areemerging eg. stratified (risk-based) breast screeningprogrammes
  3. 3. The Context• Poor global economic climate: greater emphasis on theneed to effectively use finite healthcare budgets• There is substantial diversity in how healthcare systems areprovided and funded across European countries• There is a commonality in the need for decision-makersworking within these healthcare systems at local, regionalor national levels to think about how best to spend theavailable healthcare budget effectively• Decision-makers need/want a sufficiently robust evidencebase to reassure them they are spending the resources inthe best way possible
  4. 4. Whose perspective?Suppliers Suppliers and User UserAcademic community(science & appliedresearch)Government (health policymakers)SocietyManufacturer(pharmaceutical &diagnostic)Third party healthcarepayersGeneral publicPrivate laboratories(molecular & pathology)Health servicecommissioners (national,regional, local)PatientsHospital basedlaboratories(molecular & pathology)Reimbursement & drugformulary committeesHospital based pharmacyservicesClinicians(primary care, general &specialist hospital)
  5. 5. From market access to patient benefit• Medicines: regulatory and reimbursement hurdles• Same (general) hurdles are relevant to a companiondiagnostic but the specifics (processes and evidentiaryrequirements) completely different• Different levels of decision making:– national (centralised)– provider (hospital or primary care)– patient-clinician level• Different evidence requirements likely to be considered tobe useful and sufficient to inform introduction of apersonalised medicine
  6. 6. Health Technology Assessment• There is substantial variation across Europe in:– the process of funding and producing HTAs– the technical details used in the evaluative methods– the intended use of the HTA reports• HTAs can potentially be used to inform clinical guidelines orreimbursement decisions for local, regional or national use• In some jurisdictions, HTAs have a more formal legal statusand are used by national decision makers working forhealthcare third party payer organisations
  7. 7. Economic evaluation:A framework for measuring ‘added’ valueINPUTSProcess ofhealth careOUTPUTSResources:MedicinesManaging sideeffectsTests and labStaffMonitoring etcOutcomes:% cases cancercuredLife years gainedQuality adjusted lifeyears1. Pharmacogenetic test totarget a treatment forbreast cancer2. Current practice (no test)Perspective: the healthcare systemTime horizon: lifetime
  8. 8. A Model-Based Economic EvaluationQALYs CostsFor 100 women, costs £148,000 more for new drug A but gain 6 QALYsIncremental cost per QALY = £24,700 per QALYCosts for new drug A = (70 x 3,000) + (30 x 3,200) = £306,000Costs for standard care = (60 x 1,500) + (40 x 1,700) = £158,000£3,000£3,200£1,500£1,70070 women have a response to new drug A60 women have a response to standard careQALYs for new drug A = (70 x 0.80) + (30 x 0.20) = 62QALYs for standard care = (60 x 0.80) + (40 x 0.20) = 56
  9. 9. CostsFor 100 women, costs £28,250 less for gene test and gain 2 QALYsCosts for gene test = £2,037,50Costs for no gene test = £2,320,00QALYs for gene test = 61QALYs for no gene test = 59Gene test = 68.75 women have a responseNo Gene test = 65 women have a responseQALYs£3,100£3,300£1,600£1,800£3,000£3,200£1,500£1,700Testcosts£100
  10. 10. NICE and Personalised MedicineCompanion diagnostic‘with’new medicineEstablished treatmentpathway & > 1 testTechnology AppraisalIs the medicine cost effective?Diagnostics AssessmentProgrammeIs the diagnostic cost effective?
  11. 11. Poor (unfocussed) clinicalevidence base34 studies: 25 patient cohortsNo-meta analysis due toheterogeneity: alleles included,patient cohorts, outcomesNot possible to populate aneconomic modelNot currently possible torecommend CYP2D6 testing inwomen receiving treatmentwith tamoxifen
  12. 12. Example recommendations:short termRecommendation Possible ApproachTo understand the current use of HTA to informreimbursement and payer decisions for companiondiagnostic medicines across EuropeStructured document reviewsupported by a survey of keystakeholdersTo identify and create a database of existingreimbursement and payer systems for diagnosticsand medicines across EuropeStructured document reviewsupported by a survey of keystakeholdersTo identify how companion diagnostics are pricedand/or charged for at the provider level acrossEuropeSurvey of providers in hospitalsand service commissionersTo understand the extent of variation intechnologies and processes for the conduct ofcompanion diagnostic testing within and acrossEuropean countriesSurvey of research and clinicallaboratories
  13. 13. Example recommendations:medium termRecommendation Possible ApproachTo understand how existing reimbursement andpayer systems for diagnostics and medicines acrossEurope must be re-aligned to facilitate market entrySemi-structured interviews andsurvey of key stakeholdersTo produce guidance on the technicalities of thedesign and conduct of the HTA process specific tocompanion diagnostic medicinesEstablish a working group of keyHTA bodiesTo understand the implications of moving from singlecompanion diagnostics to multiple/profiles forevidence requirements and service provisionSemi-structured interviews andsurvey of key stakeholdersTo describe and quantify gaps in the evidence baseand the added value of future research to reducecurrent uncertainties to support the introduction ofcompanion diagnosticsEconomic modelling with formalvalue of information methods
  14. 14. Example recommendations:medium/long termRecommendation Possible ApproachTo identify incentives to encourage manufacturers to investin the production of a robust evidence base to support theclinical & cost effectiveness of companion diagnosticmedicinesEstablish a mixed workinggroup of key HTA bodiesand manufacturersTo identify and target national funding for research tounderstand the added value of technologiesEstablish working group ofHTA/ research fundingbodiesTo align the use of HTA for the reimbursement of companiondiagnostics in line with existing practice for medicinesEstablish a working groupof key HTA bodiesTo produce clear and explicit guidance on the evidencedecision makers working at national and local providerslevels would like to support the reimbursement ofcompanion medicine diagnosticsEstablish a mixed workinggroup of key stakeholdersincluding HTA bodies,manufacturers andhospital providers
  15. 15. AcknowledgmentsReflections on market access for personalized medicine:Recommendations for EuropeKatherine Payne, The University of ManchesterLieven Annemans, Ghent University