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Updated: Jun 1, 2026
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Drug safety sciences and the bottleneck in drug development
1Hamner-University of North Carolina, Institute for Drug Safety Sciences, Schools of Medicine and Pharmacy, University of North Carolina, Chapel Hill, North Carolina, USA. pbwatkins@med.unc.edu
Clinical Pharmacology and Therapeutics
|May 20, 2011
Summary
US Food and Drug Administration (FDA) regulators required a large new clinical trial for a chronic disease drug due to potential liver safety concerns. This decision imposes significant costs and delays on the drug sponsor.
Area of Science:
- Pharmacology and Drug Development
- Clinical Trial Design
- Regulatory Affairs
Background:
- A new drug for chronic disease treatment demonstrated efficacy.
- Potential liver safety concerns were identified in two subjects during review.
Purpose of the Study:
- To assess the necessity of a large-scale clinical trial for drug approval.
- To evaluate the impact of regulatory requirements on drug development timelines and costs.
Main Methods:
- Review of a new drug application by regulatory agencies.
- Identification of potential safety signals (abnormal liver chemistries).
- Imposition of a prerequisite for a large clinical trial (20,000 patients, 1 year).
Main Results:
- Efficacy of the drug was agreed upon by regulators.
- Uncertain liver safety liability identified in a small number of subjects.
- A large, costly clinical trial mandated as a condition for approval.
Conclusions:
- Regulatory review identified a potential safety issue requiring further investigation.
- The required clinical trial presents substantial financial and market risks to the sponsor.
- The drug development pathway can be significantly impacted by safety signal evaluation.
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