Understand The R&D Process

FDA Review and Approval

After completion of the first 3 phases of clinical trials, the Sponsor analyzes the data. If the data shows that the therapy is effective and safe, the company submits a New Drug Application (NDA) to the FDA. Your group may wish to become a member of the FDA Advisory Committee if one is needed during the review process.

An NDA will contain:
- Data from all the preclinical and Phase 1, 2 and 3 studies, plus analyses of that data.
- Information about how the therapy is produced.
Review of an NDA can take up to 1 year. The FDA:
- Establishes that the data demonstrates that the therapy is both safe and effective.
- Weighs the benefits and potential risks of the therapy.
- Decides what information should be included in the package insert to instruct doctors about how to use the therapy and for which patients.
- Ensures that the therapy will be manufactured to the highest quality.
If the data cannot be clearly interpreted to grant approval or disapproval, the FDA may decide to have an FDA Advisory Committee review the evidence in a public meeting to gain additional perspectives to include in their decision making process.
- NCATS Toolkit provides more information on FDA Advisory Committees in Clinical Trials and FDA Review: Participate in the FDA Advisory Committee Meetings.
If the therapy is approved, then manufacturing on a large scale begins and the drug can be sold to patients outside of clinical and research studies.

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)