Value-Based and Affordable Precision Medicine: Recommendations for Health Economic Modelling, Innovative R&D Funding and Provider-Payment


Discussion Leaders: Maureen Rutten-Van Mölken, PhD, Erasmus School of Health Policy & Management (ESHPM), Erasmus University Rotterdam, Rotterdam, Netherlands Balázs Nagy, PhD, Economic Modelling Division, Syreon Research Institute, Budapest, PE, Hungary;Matthijs Versteegh, PhD, Institute for Medical Technology Assessment, Rotterdam, Netherlands; Sarah Wordsworth, PhD, Health Economics Research Centre, University of Oxford, Oxford , OXF, UK

Presentation Documents

PURPOSE: 1) To discuss recommendations for health economic modelling of precision medicine (PM) and demonstrate their application using 3 case-studies – maturity-onset diabetes of the young, tumor-agnostic NTRK inhibitors, and DPYD genotyping (ToxNav) prior to fluorpyrimidine-based chemotherapy. 2) To provide an overview of innovations in R&D funding and provider-payments for PM and discuss the way forward in risk-sharing agreements. 

DESCRIPTION: There is an increase in the development of healthcare interventions described as PM, with the EU investing €3.2 billion in PM research in 2017 alone. PM holds great promises for improved health outcomes and reduced costs because it enables targeting of therapies. Despite this, PM has been slow to enter routine clinical care, partly due to a lack of effectiveness evidence and because therapies are often priced at the margin, based on ‘what the market can bear’. Although health economic modelling methods are suited to assess the value of PM, the myriad of challenges that may converge in modelling PM calls for additional guidance. This workshop invites responses to the preliminary recommendations of the EU-funded HEcoPerMed project for modelling of PM, such as “If a genetic test is not part of routine care, include the downstream costs and benefits of testing for both individuals who test positive and individuals who test negative in the model” and “When effectiveness is estimated relative to a historical cohort, obtain data on the distribution and prognostic value of the gene mutation in that cohort”. It demonstrates the application of these recommendations in three case studies. The workshop concludes with an overview of financing and payment models for PM and discusses their suitability to increase the development and uptake of PM. The workshop benefits: a) health economists working in PM, b) manufacturers of PM, c) payers, d) regulatory authorities, e) international organizations such as ICPerMed, EUnetHTA, EMA.

Conference/Value in Health Info

2020-11, ISPOR Europe 2020, Milan, Italy




Health Policy & Regulatory

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