Drug Evaluation Committee Method for Estimating Therapeutic Effects of Rare Diseases
Data Science Subcommittee
December 2022
Currently, technological innovations such as gene technology are making it possible to create innovative drugs for diseases with high unmet medical needs, such as rare diseases. There are many challenges in the development of orphan drugs, including the difficulty of conducting clinical trials with sufficient power due to the small number of patients, and the ethical issues involved in using placebo as a control due to the severity of the disease, etc. Recently, several statistical methods have been proposed to estimate therapeutic effects, In addition, several documents related to the development of orphan drugs have been published by regulatory authorities. In light of this situation, we surveyed statistical methods for estimating therapeutic effects of orphan drugs and their recent applications, and summarized them in this report. The report includes mathematical explanations of statistical methods mainly for statisticians, and guidance and case studies related to the development of drugs for rare diseases are presented for a wide audience of persons involved in drug development. We hope that this report will be of help to those involved in the development of orphan drugs.
Japan Pharmaceutical Manufacturers Association, Committee on Drug Evaluation
Data Science Subcommittee FY2022 Ongoing Task Force 3
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