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Evolution and current status of the Orphan Drug Act
International Journal of Technology Assessment in Health Care
|January 1, 1992
Summary
The Orphan Drug Act incentivizes rare disease drug development, leading to new treatments. However, its effectiveness and costs require ongoing evaluation due to profitable orphan products.
Area of Science:
- Pharmacoeconomics
- Regulatory Science
- Drug Development
Background:
- The 1983 Orphan Drug Act addressed market and regulatory barriers to developing treatments for rare diseases.
- Changes in the pharmaceutical industry and regulatory testing influenced industrial interest in orphan drugs.
Purpose of the Study:
- To assess the impact of the Orphan Drug Act on the development and approval of orphan drugs and biologics.
- To evaluate the necessity and appropriateness of the Act's incentives for industrial development of orphan medical products.
- To examine controversies surrounding profitable orphan products and their benefit from the Act's provisions.
Main Methods:
- Analysis of drug and biologic development and approval data since the Orphan Drug Act's inception.
- Review of mandated explorations into the Act's incentive structure.
- Examination of controversies related to profitable orphan products.
Main Results:
- The Act has been associated with the development and approval of 40 drugs and 12 biologics for rare diseases.
- An additional 281 drugs and 141 biologics are in development and designated as orphans.
- Controversies have emerged regarding the financial benefits of the Act for certain profitable orphan products.
Conclusions:
- The Orphan Drug Act has demonstrably stimulated the development of orphan drugs and biologics.
- Continued assessment of the Act's benefits, costs, and incentive structure is necessary.
- The law's impact on market dynamics and accessibility of rare disease treatments warrants ongoing scrutiny.