Published on February 5, 2014
Setting the Scene: A Health Economics Perspective Adrian Towse Generating a NICE Assessment for Stratified Medicines Event Sponsored by BIVDA1 and ABPI2 London • 20 January 2014 1British In Vitro Diagnostics Association 2Association of the British Pharmaceutical Industry
Agenda • What are the elements of value in a diagnostic test? • • What institutional arrangements are needed for the assessment of value? • • A summary of nine case studies Options for appraising stratified medicines and diagnostics Generating the evidence, linking it to value, getting the technologies used • Where does the evidence come from? • The need to align incentives • Flexible pricing and coverage with evidence development • Challenge of diagnostic uptake 2
Framework to assess value of diagnostic test technologies in the context of treatment 1. Reducing drug adverse effects 5.Reducing uncertainty about value Value 4. Enabling Tx effective in a small fraction to be made available 2. Reducing time delays in selecting optimal Tx 3.Increasing adherence or willingness to start Tx Source: Garau et al. (2013) 3
Pathways for value of molecular diagnostics and key examples Source: Garau et al. (2013) 4
Agenda • What are the elements of value in a diagnostic test? • • What institutional arrangements are needed for the assessment of value? • • A summary of nine case studies Options for appraising stratified medicines and diagnostics Generating the evidence, linking it to value, getting the technologies used • Where does the evidence come from? • The need to align incentives • Flexible pricing and coverage with evidence development 5
Institutional processes for the value assessment of new diagnostics Dx-Tx pair launched simultaneously New Dx Dx not linked to a Tx Single Dx launched separately Dx assessed via Diagnostic-dedicated process Multiple Dx with same clinical use Dx linked to a Tx (companion Dx, personalised medicine) Dx-Tx joint assessment via Drug process Dx assessed via Diagnostic-dedicated process Dx assessed via Diagnostic-dedicated process Source: Garau et al. (2013) 6
Agenda • What are the elements of value in a diagnostic test? • • What institutional arrangements are needed for the assessment of value? • • A summary of nine case studies Options for appraising stratified medicines and diagnostics Generating the evidence, linking it to value, getting the technologies used • Where does the evidence come from? • The need to align incentives • Flexible pricing and coverage with evidence development • Challenge of diagnostic uptake 7
Molecular diagnostic tests: the evidence hurdle Marker Main study design Study size (patient numbers) Sponsor KRAS mutations (Anti-EGFR monoclonal antibodies in CRC) Retrospective cohort analysis of an RCT 1198 Drug developer & public research body (a) Oncotype DX® & (b) MammaPrint® (Prognostic/predictive in BrCa) Retrospective RCT cohorts (a) 688, 651, 895 (b) Prognostic: 117, 295, 307, 123 Predictive: 241 Diagnostic manufacturer RCTs (a) (b) Public research bodies Retrospective RCT cohort+ Healthy volunteers 1477, 162 Public research body Prospective cohort study 4471 (Terminated early) Payer Proof-of concept RCT 187 Diagnostic manufacturer CYP2C19 (Clopidogrel in ACS) 11248 6600 Public research body Source: Adapted from Towse et al. (2013) 8
Aligning incentives • Greater willingness on the part of payers to accept prices that reflect value • Will involve a need for price flexibility for drugs as evidence of their value for different groups of patients emerges over time • Consideration of some form of intellectual property protection (e.g. data exclusivity) for diagnostics to cover evidence of clinical utility • Realistic expectations about standards for evidence. This involves the use of CED and real-world evidence collection for both drugs and diagnostics • Public investment to complement the efforts of payers and manufacturers, recognising the limitations on the incentives for both to invest in evidence collection on all the questions that matter. Source: Towse and Garrison (2013) 9
Need for flexible pricing and more outcomes-based PAS Garrison et al (2013) Academy of Medical Sciences (2013) 10
France (INCa) – An approach to clinical implementation • 28 regional platforms • Partnerships between several laboratories located in university hospitals and cancer centres • Cooperation between pathologists and biologists • Compensate local pathologists for sample shipment • Free of charge to patients and hospitals • Public/private partnerships for molecular testing • Early phase network of 16 early-phase clinical trial centers (CLIP2) Source: Buzyn (2013) 11
France – estimates of economic impact of molecular testing PFS = progression free survival • Focus on cost-offset arising from not treating nonresponder patient subgroups identified through testing. • May explain willingness to fund the INCa initiative Source: Calvo (2011) 12
Sources Academy of Medical Sciences. (2013) Realising the potential of stratified medicines. London: Academy of Medical Sciences. Buzyn, A. (2013) How INCa is supporting the development of personalized medicine. Presentation at the 2013 WIN symposium. Paris. 10-11 July 2013. Available at: http://www.winsymposium.org/wpcontent/uploads/2013/07/WIN2013_Agnes-Buzyn-REVISED.190713.pdf Calvo, F. (2011) Personalized medicine: A nationwide initiative for an equal access to cancer treatment in France. 20 May. Available at: http://ec.europa.eu/research/health/pdf/event06/13052011/fabiencalvo_en.pdf Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should value-based pricing be applied to molecular diagnostics? Personalized Medicine. 10(1), 61-72. Garrison, L.P., Towse, A.T., Briggs, A., de Pouvourville, G., Grueger, J., Mohr, P.E., Severens, J.L., Siviero, P. and Sleeper, M. (2013) Performance-based risk-sharing--Good practices for design, implementation, and evaluation: Report of the ISPOR Good Practices for Performance-based Risksharing Task Force. Value in Health. 16(5), 703-719. Towse, A., Ossa, D., Veenstra, D., Carlson, J. and Garrison, L. (2013) Understanding the economic value of molecular diagnostic tests: Case studies and lessons learned. Journal of Personalized Medicine. 3(4), 288-305. doi: 10.339/jpm3040288. Towse, A. and Garrison, L.P. (2013) Economic incentives for evidence generation: promoting an efficient path to personalized medicine. Value in Health. 16(6Suppl), S39-S43. doi: 10.1016/j.jval.2013.06.003. 13
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