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Related Experiment Videos

The US orphan drug programme 1983-1995.

S R Shulman1, M Manocchia

  • 1Tufts Center for the Study of Drug Development, Tufts University, Boston, Massachusetts, USA. sshulman@infonet.tufts.edu

Pharmacoeconomics
|August 5, 1997
PubMed
Summary
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The US Orphan Drug Act incentivizes rare disease drug development. Analysis from 1983-1995 shows 631 designations and 121 approvals, with cancer and genetic diseases most common. Biotechnology firms increasingly participate.

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.

Related Experiment Videos

  • 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.