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Center of Excellence in Regulatory Science and Innovation


Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials:  Considerations and Tools


Two-day virtual event

Day 1:   Tuesday, May 2, 2023  Recording  Slides

Day 2:   Wednesday, May 3, 2023  Recording  Slides



Background and Meeting Objectives

On May 2 and 3, 2023, the FDA’s Center for Drug Evaluation and Research (CDER) and the Johns Hopkins University’s Center of Excellence in Regulatory Science and Innovation (JHU CERSI) will host a jointly sponsored virtual workshop on addressing challenges in the design and analysis of rare disease clinical trials.

Advances in rare diseases, particularly in the field of genetics, result in an active, highly innovative, and rapidly evolving area of science and drug development. Nonetheless, rare disease drug development represents an area of high unmet medical need. Rare diseases affect a large number of individuals in the United States. Approximately 25 to 30 million Americans have a rare disease (about 1 in 10 Americans).

There are many challenges in rare disease drug development that are broadly recognized by the rare disease drug development community, such as:

The design and conduct of rare disease drug development trials is informed by multiple data sources, including natural history studies or registries. It is important for data to be high quality and fit-for-purpose. The use of innovative trial designs and analysis methods – including adaptive designs and Bayesian methods – are potential strategies to address the challenges of rare disease drug development.  The use of these strategies is increasing among drug development sponsors, and there is a need for more education regarding the use of complex innovative trial designs and analyses for rare disease clinical trials. 

The primary audiences for the workshop are academic investigators, small or emerging pharmaceutical and biotechnology companies, patient advocacy groups and other stakeholders in the design of registries, natural history studies, or clinical trials and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of rare disease clinical trial design and analysis.

This workshop will discuss:

Participation in this workshop will allow attendees to improve their familiarity and understanding of guidelines and approaches for high quality rare disease clinical trials. While specific questions about applications will not be addressed, topics in general will be discussed and case studies will be presented as learning examples.



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Click here for speaker biographies for Day 1

Click here for speaker biographies for Day 2