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Orphan drug development in the United States.
CPJ : Canadian Pharmaceutical Journal = RPC : La Revue Pharmaceutique Canadienne
|April 10, 1985
Summary
The Orphan Drug Act of 1983 incentivizes pharmaceutical companies to develop treatments for rare diseases. This legislation has successfully increased the number of orphan drug designations since its enactment.
Area of Science:
- Pharmaceutical Science
- Health Policy
- Regulatory Affairs
Background:
- Drug development traditionally prioritizes common diseases due to higher return on investment.
- Rare disease drug development faced significant financial and regulatory hurdles.
- The Orphan Drug Act was enacted to address this imbalance.
Purpose of the Study:
- To summarize the key provisions and impact of the Orphan Drug Act.
- To highlight the legislative mechanisms designed to encourage orphan drug development.
Main Methods:
- Review of the Orphan Drug Act's legislative provisions.
- Analysis of the impact of the Act on orphan drug designations.
Main Results:
- The Act established FDA recommendations for marketing approval.
- Incentives include exclusive marketing privileges and tax credits.
- Facilitated investigational availability and established the Orphan Product Board.
Conclusions:
- The Orphan Drug Act has demonstrably stimulated pharmaceutical interest in rare disease treatments.
- The legislation has led to a continuous rise in orphan drug designations.
- The Act serves as a successful model for incentivizing niche pharmaceutical development.