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

Part 1: Foundations and Pre-Clinical Research
Categories: Drug Development, RDCA-DAP
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About Course

Drug development begins long before the first patient enrolls in a clinical trial. This course, which is Part 1 in our series ‘Rare Disease Drug Development: What Patients and Advocates Need to Know’, will cover:


  • the steps in drug development
  • the roles of those involved
  • how to lay a strong foundation during the pre-clinical period and
  • the transition from pre-clinical to clinical studies.


The focus of this series is on rare diseases and how patients and patient advocates can play a significant role in rare disease drug development.


The course is available free online and can be accessed on mobile. It has been created by the National Organization for Rare Disorders (NORD) in collaboration with FDA’s Center for Drug Evaluation and Research (CDER) and the Critical Path Institute. Funding 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 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

Transitioning from Pre-Clinical to Clinical Research

Course Resources

Course Resources

Course Resources

Course Resources

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