Incentives for Orphan Drug Research in the US
Author Information
Author(s): Seoane-Vazquez Enrique, Rodriguez-Monguio Rosa, Szeinbach Sheryl L, Visaria Jay
Primary Institution: Ohio State University
Hypothesis
What are the characteristics and impacts of orphan drug designations and approvals in the US from 1983 to 2007?
Conclusion
Public programs and policies support orphan drug research and development, with various incentives leading to a modest increase in orphan drug approvals.
Supporting Evidence
- 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 years.
- The orphan drug market exclusivity provision increased the average maximum effective patent life by 0.8 years.
Takeaway
This study looks at how the government helps companies make drugs for rare diseases, showing that while there are some benefits, the impact isn't huge.
Methodology
The study analyzed FDA data on orphan drug designations and approvals from 1983 to 2007, using statistical methods to assess differences.
Potential Biases
Potential bias in data collection and analysis due to reliance on FDA records.
Limitations
The study does not estimate how many orphan drugs would have been approved without the ODA incentives.
Participant Demographics
The study included data on orphan drug designations and approvals across various sponsors, including universities and companies.
Statistical Information
P-Value
p<0.001
Statistical Significance
p<0.001
Digital Object Identifier (DOI)
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