Improving Access to Accelerated Approval for Rare Disease Treatments
Author Information
Author(s): Miyamoto Brigitta E, Kakkis Emil D
Primary Institution: Kakkis EveryLife Foundation For Rare Diseases
Hypothesis
Can improved access to accelerated approval reduce development costs and increase the number of treatments for rare diseases?
Conclusion
Better access to accelerated approval could significantly reduce development costs and increase the number of rare disease drugs developed.
Supporting Evidence
- Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models.
- Better AA access could reduce development costs by approximately 60%.
- Improved access to AA could foster development of three times as many rare disease drugs for the same investment.
Takeaway
This study shows that making it easier to get drugs approved for rare diseases can save money and help more people get the treatments they need.
Methodology
The study devised clinical development programs using proposed clinical or surrogate endpoints for fifteen rare disease treatments.
Potential Biases
There may be risks of bias in the selection of surrogate endpoints.
Limitations
The study does not verify the validity of the proposed surrogate endpoints.
Statistical Information
P-Value
0.05
Statistical Significance
p<0.05
Digital Object Identifier (DOI)
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