Rare Disease Drug Development Series: What Patients and Advocates Need to Know

Categories: Drug Development, RDCA-DAP
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About Course

Patients are necessary contributors in the research and development process of treatments for all diseases and disorders. This is especially true for rare diseases: when patient populations are smaller than 200,000 people—sometimes much smaller—the need for individuals to join research studies is greater. But clinical trial participation is not the only role patients can play in drug development. From beginning to end, patients can play an important role in laying the foundation for research and continue contributing feedback until a safe and effective treatment receives FDA approval.


Rare disease patients are often the experts on their own conditions. Patient organizations can help set standards for what needs to be treated in the first place, help gather data on patients’ health, and act as proactive partners to the research community over the time it takes to develop and approve potential treatments. To do that effectively, patients and patient advocacy organizations need to understand the drug development process inside and out.


The National Organization for Rare Disorders (NORD®) has created this free online course in collaboration with the FDA’s Center for Drug Evaluation and Research (CDER) and the Critical Path Institute, designed to help patients and patient advocacy groups understand the drug development process. Each module is valuable on its own, and together they act as a comprehensive guide to the role of patient advocacy in rare disease drug development.


Start learning today, and receive updates as new modules are added!


Funding for this course was made available through the Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP®), a collaborative agreement with the Critical Path Institute funded by the FDA.

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Course Content

Drug Development Overview

  • Module Introduction
  • Overview of the Drug Development Process
  • The Five Steps in Drug Development
  • Rare Disease Challenges in the Drug Development Process
  • How Can Patients and Advocates Help Accelerate Drug Development?

Roles in Drug Development

Natural History Studies

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