The US orphan drug programme 1983-1995

Area of Science:

• Food and Drug Law
• Pharmacoeconomics
• Drug Development Policy

Background:

• The Orphan Drug Act (ODA) is a key US legislation promoting rare disease treatments.
• Global interest in orphan drug incentives necessitates understanding US program experiences.
• The ODA aims to encourage development of drugs for conditions affecting small patient populations.

Purpose of the Study:

• To review the US Orphan Drug Act legislation and regulations.
• To analyze 13 years of US orphan drug activity (1983-1995).
• To examine the impact of regulatory challenges and other FDA programs on orphan drug development.

Main Methods:

• Review of the Orphan Drug Act and associated regulations.
• Descriptive data analysis of orphan drug designations and approvals from 1983-1995.
• Examination of court decisions impacting FDA's authority under the ODA.
• Analysis of FDA's early access, fast-track, and user fee waiver programs.

Main Results:

• Over 13 years, 631 orphan drug designations and 121 marketing approvals were granted.
• Cancer and genetic diseases accounted for 40% of all orphan drug indications.
• Drugs with investigational new drug designation and fast-track status showed shorter development times.
• Biotechnology firms' share in orphan drug activity increased over time.
• Orphan drugs benefited from FDA user fee waivers, accounting for 32% of foregone revenue.

Conclusions:

• The US Orphan Drug Act has facilitated development, with notable activity in cancer and genetic diseases.
• Regulatory interpretations and court decisions may impact the predictability of orphan drug exclusivity.
• FDA's expedited programs and user fee waivers appear beneficial for orphan drug sponsors.
• The increasing role of biotechnology signifies evolving landscape of rare disease drug development.