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Single-Study Approvals: Quantum of Evidence Required.
Frank J Sasinowski1, Michelle L Butler2
1Hyman, Phelps & McNamara, PC, 700 13th St NW, Washington, DC, 20005, USA.
Therapeutic Innovation & Regulatory Science
|December 10, 2020
Summary
Demonstrating new drug effectiveness often requires more than one study. This paper clarifies two distinct FDA pathways for drug approval using a single adequate and well-controlled study, crucial for new molecular entities.
Area of Science:
- Pharmaceutical Sciences
- Regulatory Affairs
- Medical Law
Background:
- The statutory requirement for demonstrating new drug effectiveness has been debated since 1962.
- New molecular entities (NMEs) present unique challenges in proving substantial evidence of effectiveness.
- FDA regulations have evolved to provide pathways for single-study drug approvals.
Purpose of the Study:
- To elucidate the two distinct pathways for achieving FDA drug approval based on a single adequate and well-controlled study.
- To clarify the criteria and application of these pathways, particularly for new molecular entities.
- To provide illustrative examples of how the Food and Drug Administration (FDA) utilizes these approval pathways.
Main Methods:
- Analysis of statutory revisions and FDA guidance documents pertaining to drug effectiveness demonstration.
- Review of historical FDA approval decisions and case examples.
- Exegesis of regulatory pathways for single-study drug approvals.
Main Results:
- Identification of two primary pathways for single-study drug approval: the 1997 statutory revision pathway and the 1998 FDA guidance pathway (single statistically very persuasive study).
- Explanation of the distinct criteria and evidence requirements for each pathway.
- Illustrative examples demonstrating the practical application of these pathways by the FDA.
Conclusions:
- Two independent and viable pathways exist for demonstrating substantial evidence of effectiveness using a single adequate and well-controlled study.
- Understanding these pathways is crucial for regulators, industry stakeholders, patients, and investors.
- These pathways offer valuable routes to FDA approval for new drugs, especially NMEs.
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