Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: a comprehensive review of approaches and challenges.



This study aimed to perform a comprehensive review of modelling approaches, methodological and policy challenges in the economic evaluation (EE) of precision medicine (PM) across clinical stages.


First, a systematic review was performed to assess the approaches of EEs in the past 10 years. Next, a targeted review of methodological papers was conducted for methodological and policy challenges in performing EEs of PM. All findings were synthesized into a structured framework that focused on Patient population, Intervention, Comparator, Outcome, Time, Equity and ethics, Adaptability and Modelling aspects, named the "PICOTEAM" framework. Finally, a stakeholder consultation was conducted to understand the major determinants of decision making in PM investment.


In 39 methodological papers, we identified major challenges to the EE of PM, including that PM applications involve complex and evolving clinical decision space, clinical evidence is sparse due to small subgroups and complex pathways in PM settings, a one-time PM application may have lifetime or intergenerational impacts but long-term evidence is often unavailable, equity and ethics concerns are exceptional. In 275 EEs of PM, current approaches did not sufficiently capture the value of PM but that of targeted therapies, nor differentiate Early EEs from Conventional EEs. Finally, policy makers perceived the budget impact, cost-savings and cost-effectiveness of PM as the most important determinants in decision-making.


There is an urgent need to modify existing guidelines or develop new reference case that fits into the new healthcare paradigm of PM to guide decision making in R&D and market access.






Published Version (Please cite this version)


Publication Info

Chen, Wenjia, Yi Wang, Yaroslava Zemlyanska, Dimple Butani, Nigel Chong Boon Wong, Suchin Virabhak, David Bruce Matchar, Yot Teerawattananon, et al. (2023). Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: a comprehensive review of approaches and challenges. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research. p. S1098-3015(23)02596-2. 10.1016/j.jval.2023.05.001 Retrieved from https://hdl.handle.net/10161/27530.

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David Bruce Matchar

Professor of Medicine

My research relates to clinical practice improvement - from the development of clinical policies to their implementation in real world clinical settings. Most recently my major content focus has been cerebrovascular disease. Other major clinical areas in which I work include the range of disabling neurological conditions, cardiovascular disease, and cancer prevention.
Notable features of my work are: (1) reliance on analytic strategies such as meta-analysis, simulation, decision analysis and cost-effectiveness analysis; (2) a balancing of methodological rigor the needs of medical professionals; and (3) dependence on interdisciplinary groups of experts.
This approach is best illustrated by the Stroke Prevention Patient Outcome Research Team (PORT), for which I served as principal investigator. Funded by the AHCPR, the PORT involved 35 investigators at 13 institutions. The Stroke PORT has been highly productive and has led to a stroke prevention project funded as a public/private partnership by the AHCPR and DuPont Pharma, the Managing Anticoagulation Services Trial (MAST). MAST is a practice improvement trial in 6 managed care organizations, focussing on optimizing anticoagulation for individuals with atrial fibrillation.
I serve as consultant in the general area of analytic strategies for clinical policy development, as well as for specific projects related to stroke (e.g., acute stroke treatment, management of atrial fibrillation, and use of carotid endarterectomy.) I have worked with AHCPR (now AHRQ), ACP, AHA, AAN, Robert Wood Johnson Foundation, NSA, WHO, and several pharmaceutical companies.
Key Words: clinical policy, disease management, stroke, decision analysis, clinical guidelines

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