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Single-Study Approvals: Quantum of Evidence Required.
Frank J Sasinowski1, Michelle L Butler1
1Hyman, Phelps & McNamara, PC, Washington, DC, USA.
Therapeutic Innovation & Regulatory Science
|September 28, 2019
Summary
Demonstrating a new drug
Area of Science:
- Pharmaceutical Sciences
- Regulatory Affairs
Background:
- The legal requirement for demonstrating new drug effectiveness has been debated since 1962.
- Establishing substantial evidence for new molecular entities presents unique challenges.
Purpose of the Study:
- To clarify the pathways for new drug approval based on a single adequate and well-controlled study.
- To explain the Food and Drug Administration's (FDA) interpretation and application of these pathways.
Main Methods:
- Analysis of statutory revisions and FDA guidance documents.
- Review of illustrative examples of FDA drug approvals.
Main Results:
- Two distinct pathways exist for single-study drug approvals.
- A 1997 statutory revision allows approval with confirmatory evidence.
- A 1998 FDA guidance outlines the 'statistically very persuasive study' pathway.
Conclusions:
- Both pathways offer viable routes for FDA drug approval.
- Understanding these pathways is crucial for regulators, industry, patients, and investors.
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