Drug Evaluation Committee Points to Consider in Cost-Effectiveness Evaluation Learned from Case Studies

Data Science Subcommittee

March 2023

In Japan, the cost-effectiveness evaluation system will be fully introduced in 2019 for the purpose of drug price adjustment, and the Center for Health Economics and Evaluation has begun to release evaluation results after the full introduction (9 products as of January 2023). Looking overseas, the National Institute for Health and Care Excellence (NICE) in the U.K. has been conducting similar cost-effectiveness evaluations for about 20 years, albeit with a different framework from that in Japan, and many evaluation examples have already been accumulated and published. Many examples of cost-effectiveness evaluations have already been accumulated and published.

In cost-effectiveness evaluation, transparency and validity of the evaluation are ensured by a public analysis organization reviewing the analysis results of a company, and a wide range of points have been raised by public analysis organizations regarding the setting of analysis conditions, construction of disease transition models, estimation of model parameters, and methods for dealing with data uncertainty. We believe that there are many lessons to be learned from NICE and Japanese precedents of cost-effectiveness evaluation, and this report, although not exhaustive, summarizes typical issues on each topic and describes points to be considered and their countermeasures.

In cost-effectiveness evaluation, it is necessary to select a method that is considered reasonable and appropriate while making maximum use of available information to provide information that contributes to decision-making for efficient allocation of medical resources. Therefore, although cost-effectiveness evaluation does not determine the only correct value, we believe that it is beneficial for practitioners to understand the points to keep in mind from the discussion of prior cases in order to conduct an appropriate cost-effectiveness evaluation. We hope that this report will assist in the selection of appropriate methods and implementation of analyses.

Committee on Drug Evaluation, Japan Pharmaceutical Manufacturers Association
Data Science Subcommittee 2022 Ongoing Task Force 7

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