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

Abstract

ObjectivesThis 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.MethodsFirst, 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.ResultsIn 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.ConclusionsThere 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.