Natural History Studies and Registries in the Development of Rare Disease Treatments
Public Workshop: In-Person and Virtual
May 13, 2024 | 10am-4pm (eastern)
The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Diseases Team within the Office of Rare Diseases, Pediatrics, Urologic and Reproductive
Medicine; Office of New Drugs; Center for Drug Evaluation and Research and the National Institutes of Health’s Division of Rare Diseases Research Innovation within
the National Center for Advancing Translational Sciences, is hosting a public
workshop. The workshop will bring together rare disease patient advocates,
academic researchers, regulated industry, and other key stakeholders to discuss considerations for the use of natural history study and registry data in rare disease
drug development programs.
Rare diseases impact more than 400 million people worldwide, including 25-30 million people in the United States. Yet, the vast majority of rare diseases have no FDA-approved treatment. All drug product development programs benefit from a firm scientific foundation, including an understanding of disease natural history; however,
the natural history of rare diseases is often poorly understood. Natural history studies allow researchers to track the progression of illness in patients over time, and information from these studies can play an important role throughout drug product development, from discovery to clinical trial design to the post-marketing period. A registry can be defined as an organized system that collects clinical and other data in a standardized format for a population defined by a particular disease, condition, or drug exposure. When considering whether to use an existing registry—or develop a new registry— for regulatory purposes, the data collected should be assessed for its ability to address scientific objectives. Both registries and natural history studies can play an important role in informing the careful planning of clinical studies for use in rare disease drug development.
Workshop Goals:
This workshop will address the role and design of registries and natural history studies to inform the development of rare disease treatments, including aspects of clinical trial design. It will also present considerations for:
- collecting registry and natural history data that are fit for regulatory purposes
- the use of registries and natural history studies to inform regulatory decision-making
This is a hybrid event with options to participate in-person or remotely.
For questions or to request special accommodations, please email admin@reaganudall.org.
Agenda
10am |
Welcome & Opening RemarksPatrizia Cavazzoni, MD, Center for Drug Evaluation and Research, FDA |
---|---|
10:15am |
"What Are Registries and Natural History Studies?"Dominique Pichard, MD, MS, National Center for Advancing Translational Science, NIH "Why Registries and Natural History Studies are Critical to Rare Disease Treatment Development"Kerry Jo Lee, MD, Center for Drug Evaluation and Research, FDA |
10:30am |
Getting Started: Developing Registries and Designing Natural History StudiesLeslie Gordon, MD, PhD, The Progeria Research Foundation Q&A Session |
11:25am |
Addressing Challenges in Registry and Natural History Data CollectionBenjamin Forred, MBA, ACRP-CP, Sanford Research Reactor Panel |
12:25pm |
Funding OpportunitiesPhilip J. Brooks, PhD, National Center for Advancing Translational Sciences, NIH |
12:40pm |
LUNCH |
1:35pm |
Collecting Fit for Purpose Data to Inform Regulatory Decision MakingJennifer Farmer, MS, Friedreich's Ataxia Research Alliance Reactor Panel |
2:35pm |
Natural History Studies and Registries that Informed Regulatory Decision MakingExample: Nulibry for molybdenum cofactor deficiency Example: Lumasiran and Nedosiran for Primary Hyperoxaluria Reactor Panel |
3:45pm |
Closing Remarks |