Drug Evaluation Committee Symposium on "Current Status and Issues of Estimating Treatment Effectiveness in Clinical Development of Rare Diseases
Feb 16, 2023
We are pleased to post the presentation materials of the symposium "Current Status and Issues of Treatment Effect Estimation in Clinical Development of Rare Diseases" (February 20, 2023) hosted by the Data Science Subcommittee Continuing Task Force 3 in FY2022.
This symposium will organize new study designs and statistical approaches that could be applied to clinical development for rare diseases, deepen our understanding of them based on actual cases, and discuss issues related to the estimation of treatment effects from the standpoint of industry, government, and academia, in order to provide an opportunity to actively utilize scientific designs and approaches. The objective is to increase momentum for the active use of scientific design approaches. In addition to mathematical explanations of statistical methods for statisticians, the program also includes guidance and application examples related to the development of drugs for rare diseases.
- 0_Program (409KB)
- 1-1_Deliverables of the Task Force (341KB)
- 1-2_Current status and guidance related to Rare disease development (544KB)
- 1-3_Use of historical control and examples (2.25MB)
- 2-1_Study design using master protocols and examples (1.59MB)
- 2-2_CID Pilot Program and Examples (829KB)
- 2-3_Summary of N-of-1 design and Complete n-of-1 design and analysis methods (1.59MB)
- 3-1_A Study on Quantification of Therapeutic Effects for Rare Diseases (Introduction of guidelines developed by AMED, etc.) (1.98MB)
- 3-1_Reference 1: Bayesian guidelines (final version) (585KB)
- 3-1_Reference 2: Bayesian Guideline Q&A (final version) (366KB)
-
As of December 2023Reminder of Dr. Hirakawa's pdf document in 3-1: Please limit the use of this document to personal use.