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Published on: December 11, 2016
Precision Medicines' Impact on Orphan Drug Designation
Christine M Mueller1, Gayatri R Rao2, Katherine I Miller Needleman1
1Office of Orphan Products Development, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clinical and Translational Science
|July 13, 2019
Summary
The Orphan Drug Act (ODA) incentives boost rare disease drug approvals. However, precision medicine is not the main driver of increased orphan drug designations or the creation of orphan subsets from common diseases.
Area of Science:
- Pharmacoeconomics
- Drug Development
- Regulatory Science
Background:
- The Orphan Drug Act (ODA) provides incentives for developing drugs for rare diseases.
- Orphan drug designation has seen increased application in recent years.
- Precision medicine's role in creating 'orphan subsets' of common diseases is debated.
Purpose of the Study:
- To evaluate the basis of orphan drug designations.
- To assess the impact of precision medicine on orphan drug designations and subsets.
- To determine if precision medicine drives the increase in orphan drug designations.
Main Methods:
- Analysis of orphan drug designation data.
- Assessment of the proportion of designations based on precision medicine and orphan subsets.
- Comparison of trends in orphan drug designations over time.
Main Results:
- The increasing volume of orphan drug designations is not primarily driven by precision medicine creating orphan subsets.
- Orphan subsets of common diseases constitute a small fraction of overall orphan drug designations.
- The ODA continues to be a significant factor in rare disease drug approvals.
Conclusions:
- Precision medicine's impact on orphan drug designations is less significant than previously suggested.
- The ODA's incentives remain crucial for rare disease drug development.
- Further research is needed to fully understand the evolving landscape of orphan drug development.
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