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Updated: May 10, 2026
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Summary
The Food and Drug Administration (FDA) finalized Orphan Drug Regulations from 1992. These updates clarify rules and improve the Orphan Drug Act implementation for rare disease treatments.
Area of Science:
- Regulatory Affairs
- Pharmaceutical Policy
- Orphan Drug Development
Background:
- The Orphan Drug Act of 1983 incentivizes rare disease drug development.
- The 1992 Orphan Drug Regulations provided initial implementation guidance.
- Experience since 1992 has identified areas for regulatory clarification.
Purpose of the Study:
- To issue final regulations amending the 1992 Orphan Drug Regulations.
- To clarify existing regulatory provisions.
- To address practical issues that have emerged since the initial regulations were established.
Main Methods:
- Review of existing 1992 Orphan Drug Regulations.
- Analysis of issues arising from regulation implementation.
- Development and finalization of amended regulations by the FDA.
Main Results:
- Final regulations amending the 1992 Orphan Drug Regulations have been issued.
- The amendments provide clearer regulatory provisions.
- Minor improvements have been incorporated to address identified issues.
Conclusions:
- The updated regulations aim to enhance the implementation of the Orphan Drug Act.
- These changes are expected to improve the process for developing drugs for rare diseases.
- The FDA continues to refine regulatory frameworks to support therapeutic innovation.
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