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Expediting drug development--the FDA's new "breakthrough therapy" designation
Rachel E Sherman1, Jun Li, Stephanie Shapley
1From the Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD.
The New England Journal of Medicine
|November 15, 2013
Summary
The U.S. Food and Drug Administration (FDA) introduced a new "breakthrough therapy" designation. This expedited pathway is for drugs showing substantial improvement over current treatments for serious conditions.
Area of Science:
- Regulatory Science
- Drug Development
- Clinical Trials
Background:
- The U.S. Food and Drug Administration (FDA) offers expedited programs for serious conditions.
- These programs aim to accelerate the availability of novel therapeutics.
Purpose of the Study:
- To introduce and explain the FDA's new "breakthrough therapy" designation.
- To outline the criteria for this expedited drug review pathway.
Main Methods:
- The designation is based on preliminary clinical evidence.
- Evidence must demonstrate substantial improvement over existing therapies.
Main Results:
- The "breakthrough therapy" designation is now part of the FDA's expedited program portfolio.
- This designation signifies a potentially significant advancement in treating serious conditions.
Conclusions:
- The new designation provides a faster route for promising drugs.
- It requires robust early clinical data showing clear advantages over current standards of care.
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