FDA issues draft Guidance on Confirmatory Evidence of Clinical Trials
![GMP meets Development](files/eca/userImages/training.img/Z-ECA-GMP-meets-Development.jpg)
Recommendation
5-7 November 2024
Hamburg, Germany
GMP and FDA Compliance in Pharmaceutical Development and IMP Manufacturing
The Confirmatory Evidence guidance supplements and expands the recommendations in the 2019 Substantial Evidence of Effectiveness draft guidance by providing further detail on the use of data drawn from one or more sources (e.g., clinical data, mechanistic data, real-world data). It also provides examples of data types that could be considered confirmatory evidence and additional information on the flexibility in the amount and type of evidence needed to establish effectiveness.
Types of Confirmatory Evidence
Following is a list of examples that can be used to substantiate one adequate and well-controlled clinical investigation to demonstrate substantial evidence of effectiveness:
- Clinical evidence from a related indication (e.g., submission of a new drug or biologics license application for a new indication for an already approved therapy)
- Mechanistic or pharmacodynamic evidence (e.g., obtained from in vitro testing)
- Evidence from relevant animal data
- Evidence from other members of the same pharmacological class
- Natural history evidence
- Real-World Data/Evidence (RWD / RWE)
- Evidence from expanded access use of an IMP
Early engagement with the agency to discuss any plans to use RWE as confirmatory evidence is recommended. Sponsors should continue to meet with the agency throughout product development, particularly if changes to the clinical investigation or confirmatory evidence are contemplated.
More information is available in FDA's draft guidance Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence.
Related GMP News
23.07.2024Pilot Results on Regulatory-Led RWE Generation
23.07.2024ICH M12 Guideline on Drug Interaction Studies
20.06.2024Draft ICH M14 Guideline on Real-World Data for Safety Assessment
20.06.2024Glossary of Terms and Definitions for Innovative Clinical Trials
21.05.2024Real-World Evidence: FDA´s Considerations for Non-Interventional Studies
14.05.2024How to Handle and Reserve Samples from BA / BE Studies