Understand The R&D Process

Tips to Learn More

FDA has several resources that can help you learn more about the therapy development process. Although not specific to patient groups, the information is provided in an easy to understand manner:
- How Does FDA Approve a Drug? provides a quick overview of the drug approval process in an animated 2 minute video.
- The Drug Development Process provides a basic overview of the approval process for drugs from discovery to postmarketing studies. The process is very similar for biologics. Each phase of the process has links to subsequent pages of more information.
- The Device Development Process provides a similar overview for medical devices.
- The Center for Drug Evaluation and Research (CDER) Learn Training and Education offers educational and information modules about human drug regulation and activities through podcasts, education courses, and videos.
  - Drug Approval: Bringing a New Drug to the Market , is a fictional case study navigating the drug approval process from conducting nonclinical testing and clinical trials to submitting a new drug application to FDA. It is in the form of an easy-to-read Fact Sheet.
Global Genes , a nonprofit with the mission of connecting, empowering, and inspiring the rare disease community, provides several resources focused on the therapy development process.
- Drug Development Roadmap (2015) includes a glossary of terms and a brief description of each of the stages of the therapy development process from the patient group perspective.
- From Molecules To Medicine: How Are New Drugs & Therapies Developed? (2018) goes into greater detail about each of the stages as well as provides insight on how your patient group can become involved in the process.
- The 10 Things You Need to Pursue Research For Your Disease: What Are the Options for Research and Why is it so Hard? (2015) is a 1-hour video presentation given by Brad Margus, who is both a parent of two children with a rare disease and a founder of a nonprofit patient group for that disease. Brad did not start out with a strong background in science and presents his advice for navigating the process of research for rare diseases in an empowering and practical manner.
European Patients’ Academy is a website focused on education and training to increase the ability of patients to understand and contribute to medical research and development. This site includes online courses, webinars, and a searchable toolbox with over 3,000 resources.

Resources

Preparation for Clinical Trials

FDA Guidance for Industry: Good Laboratory Practices Questions and Answers U. S. Food and Drug Administration (FDA) (link)

Developing Products for Rare Diseases & Conditions U. S. Food and Drug Administration (FDA) (link)

Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review U. S. Food and Drug Administration (FDA) (link)

Tips to Learn More

How Does FDA Approve a Drug U. S. Food and Drug Administration (FDA) (link)

The Drug Development Process U. S. Food and Drug Administration (FDA) (link)

The Device Development Process U. S. Food and Drug Administration (FDA) (link)

CDER Learn Training and Education U. S. Food and Drug Administration (FDA) (link)

Drug Approval: Bringing a New Drug to the Market U. S. Food and Drug Administration (FDA) (link)

Drug Development Road Map Global Genes (link)

From Molecules To Medicine: How Are New Drugs & Therapies Developed? Global Genes (link)

The 10 Things You Need to Pursue Research For Your Disease: What Are the Options for Research and Why is it so Hard? Global Genes (link)

European Patients' Academy European Patients' Academy on Therapeutic Innovation (link)