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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:

• Clinical trial design and interpretation challenges resulting from the small number of patients with the individual disorders 
• The lack of drug development precedent for most rare diseases
• The lack of established endpoints, including biomarkers and clinical outcome measures, for some rare disease clinical trials

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 involved 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:

• How to collect high quality and fit-for-purpose data for rare disease clinical trials
• Use of data sources to inform rare disease drug development
• Design and analysis methodologies for use in rare disease clinical trials

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. 

For the workshop agenda, participant biographies and to register for this workshop, please visit: CDER and JHU CERSI Rare Disease Workshop. The workshop agenda, presentation materials, and recording/video will be added to the noted webpage when available.

For questions, please email  CDER-JHU CERSI Rare Disease Workshop