23 Recommendations in Modelling Precision Medicine: Learning from Case Studies in Tumour Agnostic Treatments, DPYD Genotyping and Maturity Onset Diabetes of the Young


Discussion Leader: Matthijs Versteegh, PhD, iMTA, Erasmus University Rotterdam, Rotterdam, Netherlands
Discussants: Sarah Wordsworth, PhD, Health Economics Research Centre, University of Oxford, OXFORD, OXF, UK; Balázs Nagy, PhD, Economic Modelling Division, Syreon Research Institute, Budapest, Hungary; Maureen Rutten-van Mölken, PhD, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, ZH, Netherlands

PURPOSE: 1) Demonstrate solutions to challenges in health economic modelling of precision medicine (PM) using three cases: tumour-agnostic treatments for NTRK fusion-positive (NTRK+) cancers, DPYD genotyping prior to fluorpyrimidine-based chemotherapy (ToxNav) and maturity onset diabetes of the young (MODY).

2) Discuss the appropriateness of these solutions based on published recommendations for modelling PM of the HEcoPerMed consortium and handling data limitations upon EMA approval.

DESCRIPTION: PM aims to better stratify patients, enabling more targeted healthcare. While holding the promise of generating more value for patients, the assessment of PM is complicated by data limitations and complex test-treatment pathways. To address these challenges, 23 modelling recommendations were published in 2021 by the EU-funded HEcoPerMed consortium, addressing the modelling of test-treatment combinations, non-randomized controlled data, additional elements of value, premature survival data and other issues.

This workshop demonstrates the practical application of the recommendations in three case studies. The health economic model on NTRK+ cancers shows how comparative effectiveness can be estimated for single-arm basket trials and how risk of death while waiting for gene test results can be incorporated. The MODY case demonstrates the importance of patient stratification, test positioning and predictive accuracy, and the impact of detecting of inheritable mutations on relatives. The ToxNav case evaluates routine DPYD testing for personalised dosing of fluorpyrimidine chemotherapy and toxicity management. Physicians’ compliance, and clinical decisions in real-world settings are incorporated. This workshop invites responses to the modelling solutions applied in the case studies and seeks input to improve the guidance.

The workshop benefits: health economists working in PM, manufacturers of PM, payers, regulatory authorities, and organisations like ICPerMed, EUnetHTA, EMA.


We apply real-time polling to invite comments on the appropriateness of the recommendations for modelling PM, and conclude with an audience discussion on the discrepancy between EMA and payer data needs.

Conference/Value in Health Info

2021-11, ISPOR Europe 2021, Copenhagen, Denmark




Economic Evaluation

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