Ensuring the safety and efficacy of human drugs and biological products is critical, and clinical trials are an essential part of evidence-based review and approvals. However today, more and more drugs are coming to market with evidence from a single controlled clinical investigation. This can lead to the introduction of more biased results and a lack of independent substantiation to verify the drugs’ benefits.
In September 2023 the agency issued draft guidance – Demonstrating Substantial Evidence of Effectiveness with One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence – which expanded on the types of confirmatory evidence accepted. This expansion can lead to additional therapies on the market lacking appropriate evidence of safety and efficacy.
In comments provided to the FDA, Arnold Ventures made the following recommendations for change to the most recent guidance:
- Clarify that pre-clinical information collected before the controlled clinical investigation begins cannot demonstrate confirmatory evidence of patient benefit and harm after the intervention is studied.
- Require clinical trial sponsors to publicly post sufficient details of design, conduct, and analysis of the controlled clinical investigation to allow evaluation by the scientific community.
- Require a product’s label to state when direct evidence of patient benefit is not available.
