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Combined-Indications Significance Level of Multiple Related Indications Developed Simultaneously
Jiacheng Yuan1, Ray Zhu2, Daniel Jia3
1Allergan Inc, 2525 Dupont Drive, MI2-2D, Irvine, CA, 92612, USA. jiacheng.yuan@allergan.com.
Therapeutic Innovation & Regulatory Science
|December 10, 2020
Summary
Developing multiple related indications simultaneously for drug approval can be more efficient. This approach allows for a less stringent combined-studies significance level compared to traditional sequential development.
Area of Science:
- Pharmaceutical Sciences
- Biostatistics
- Drug Development
Background:
- Regulatory approval typically requires two confirmatory studies per indication, each at a one-sided 0.025 significance level.
- Existing approvals for related indications can sometimes allow for one confirmatory study for a new indication.
Purpose of the Study:
- To explore strategies for simultaneous development of multiple related drug indications.
- To maintain a desired combined-studies significance level while streamlining the approval process.
Main Methods:
- The study proposes statistical frameworks for evaluating multiple indications concurrently.
- It does not involve human or animal subject studies.
Main Results:
- Strategies are presented for simultaneous indication development, allowing for a less stringent overall significance level.
- An example strategy involves a combined-studies significance level of one-sided 0.0000156 for two indications, with individual studies at 0.00395.
Conclusions:
- Simultaneous development of related indications is feasible for marketing registration.
- This approach can achieve regulatory approval with a more lenient combined-studies significance level than the standard two-study paradigm per indication.
Keywords:
combined-studies evidencecombined-studies significance levelconfirmatory studiesstrength borrowingMore Related Videos
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