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. 2008 Dec 16:3:33.
doi: 10.1186/1750-1172-3-33.

Incentives for orphan drug research and development in the United States

Enrique Seoane-Vazquez  1 , Rosa Rodriguez-Monguio, Sheryl L Szeinbach, Jay Visaria
Affiliations

Incentives for orphan drug research and development in the United States

Enrique Seoane-Vazquez et al. Orphanet J Rare Dis. .

Abstract

Background: The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007.

Methods: Primary data sources were the FDA Orange Book, the FDA Office of Orphan Drugs Development, and the US Patent and Trademark Office. Data included all orphan designations and approvals listed by the FDA and all NMEs approved by the FDA during the study period.

Results: The FDA listed 1,793 orphan designations and 322 approvals between 1983 and 2007. Cancer was the main group of diseases targeted for orphan approvals. Eighty-three companies concentrated 67.7% of the total orphan NMEs approvals. The average time from orphan designation to FDA approval was 4.0 +/- 3.3 years (mean +/- standard deviation). The average maximum effective patent and market exclusivity life was 11.7 +/- 5.0 years for orphan NME. OD market exclusivity increased the average maximum effective patent and market exclusivity life of ODs by 0.8 years.

Conclusion: Public programs, federal regulations, and policies support orphan drugs R&D. Grants, research design support, FDA fee waivers, tax incentives, and orphan drug market exclusivity are the main incentives for orphan drug R&D. Although the 7-year orphan drug market exclusivity provision had a positive yet relatively modest overall effect on effective patent and market exclusivity life, economic incentives and public support mechanisms provide a platform for continued orphan drug development for a highly specialized market.

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References

    1. United States Food and Drug Administration The Orphan Drug Regulations. Final rule, 57 FR 62076 21 CFR Part 316. 1992.
    1. Asbury CH. The Orphan Drug Act. The first 7-years. JAMA. 1991;265:893–7. doi: 10.1001/jama.265.7.893. - DOI - PubMed
    1. National Institutes of Health Office of Rare Diseases. 2008. http://rarediseases.info.nih.gov (Accessed January 30, 2008).
    1. United States Congress Rare diseases act of 2002. Public Law 107–280 http://www.history.nih.gov/01docs/historical/documents/PL107-280.pdf (Accessed January 3, 2008).
    1. Office of Inspector General. United States Department of Health and Human Services The Orphan Drug Act implementation and impact. 2001. http://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf (Accessed January 3, 2008).

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