Drug Evaluation Committee Estimation of Therapeutic Effects for Rare Diseases
December 2022
Currently, technological innovations—particularly in the field of genetic technology—are advancing, making it possible to develop innovative drugs for diseases with high unmet medical needs, such as rare diseases. The development of drugs for rare diseases faces numerous challenges, including the difficulty of conducting clinical trials with sufficient statistical power due to the small number of patients,and the use of a placebo as a control raises ethical concerns due to the severity of the disease, among other factors. However, in recent years, several statistical methods for estimating therapeutic efficacy have been proposed, and regulatory authorities have published numerous documents related to the development of orphan drugs.In light of these circumstances, we investigated statistical methods for inferring the therapeutic efficacy of orphan drugs, as well as recent application examples, and compiled our findings into this report.The report includes mathematical explanations of the statistical methods, primarily intended for statisticians, while the guidance and case studies related to the development of drugs for rare diseases are intended for a broader audience of professionals involved in drug development. We hope this report will be of assistance to all those involved in the development of drugs for rare diseases.
Japan Pharmaceutical Manufacturers Association (JPMA) Drug Evaluation Committee
Data Science Subcommittee FY2022 Continuing Task Force 3