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Trends In Orphan New Molecular Entities, 1983-2014: Half Were First In Class, And Rare Cancers Were The Most Frequent
Kathleen L Miller1, Michael Lanthier2
1Kathleen L. Miller (kathleen.miller@fda.hhs.gov) is an economist in the Office of Planning, Food and Drug Administration, in Silver Spring, Maryland, and an adjunct assistant professor in the Department of Health Policy and Management at the University of North Carolina at Chapel Hill.
Health Affairs (Project Hope)
|March 9, 2016
Summary
The Orphan Drug Act has successfully spurred innovation, with most approved orphan drugs being first-in-class treatments that significantly address unmet medical needs for rare diseases, particularly in oncology.
Area of Science:
- Pharmacology
- Public Health Policy
- Drug Development
Background:
- The Orphan Drug Act (1983) aimed to incentivize the development of treatments for rare diseases.
- Evaluating the effectiveness of this legislation is crucial for public health.
- Understanding the landscape of orphan drug approvals provides insights into rare disease treatment progress.
Purpose of the Study:
- To analyze the characteristics of orphan drugs approved as new molecular entities between 1983 and 2014.
- To assess the innovativeness and impact of these orphan drugs on unmet medical needs.
- To identify trends in orphan drug development, including therapeutic areas and sponsoring companies.
Main Methods:
- Retrospective analysis of 209 orphan drugs approved as new molecular entities from 1983-2014.
- Examination of drug characteristics, including novelty (first-in-class status) and regulatory review (priority review).
- Categorization of drugs by therapeutic area, with a focus on oncology, and analysis of company involvement over time.
Main Results:
- The majority of orphan drugs were highly innovative, with over 50% being first-in-class.
- A significant proportion (78%) received priority review, indicating substantial therapeutic potential.
- Rare cancer therapies constituted the largest segment (35%), with increased involvement from large pharmaceutical companies in recent years (2010-2014).
Conclusions:
- Orphan drugs approved under the Act have been highly innovative.
- These novel therapies have substantially reduced unmet medical needs for patients with rare diseases.
- The development of orphan drugs, especially for oncology, shows a positive trend with significant contributions to patient care.
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