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The Orphan Drug Act. The first 7 years
1Robert Wood Johnson Foundation, Princeton, NJ 08543-2316.
JAMA
|February 20, 1991
Summary
The Orphan Drug Act has boosted the development of rare disease treatments. Post-Act data shows increased industry sponsorship of orphan drugs, indicating greater focus on these vital therapies.
Area of Science:
- Pharmacoeconomics
- Regulatory Science
- Drug Development
Background:
- The 1983 Orphan Drug Act aimed to incentivize rare disease drug development by reducing regulatory hurdles.
- Key provisions included market exclusivity, tax credits, and streamlined regulatory processes.
Purpose of the Study:
- To analyze the impact of the Orphan Drug Act on orphan drug development and market dynamics.
- To compare industry and government data before and after the Act's implementation.
Main Methods:
- Comparative analysis of industry and government data from pre- and post-Orphan Drug Act periods.
- Examination of trends in marketed and experimental orphan drug sponsorship and designation.
Main Results:
- Industry sponsorship of marketed orphan drugs increased from 34 (pre-Act) to 39 (post-Act).
- A significant rise in experimental orphan drugs with 301 gaining designation post-Act.
- While many orphan products have low sales, some generate over $100 million annually, prompting legislative review.
Conclusions:
- The Orphan Drug Act is associated with a notable increase in orphan product development.
- Further examination of the law's costs and benefits is recommended for future policy considerations.