FDA Benefit/Risk Guidance Analyzed
Three Faegre Drinker consultants say an FDA September draft guidance on benefit/risk assessments for drugs and biological products “is an important step in increasing transparency of the FDA decision-making process. It gives sponsors, patient advocacy organizations, and other stakeholders a view into how FDA is using the relatively new benefit/risk assessment process and raises helpful points for considering benefits and risks in a drug development program.”
In an online analysis, the consultants say that in cases where a specific treatment offers clear, meaningful benefits or has clear advantages over currently approved treatments, those benefits may outweigh the risks. In other cases, they say, the agency may consider subpopulations and determine that the benefits to them outweigh the risk even if the same is not true for the broader populations.
The guidance also addresses how FDA deals with uncertainty when conducting benefit/risk assessments. The consultants say the guidance indicates that FDA may tolerate greater uncertainty (such as limits on understanding the patient population, differences between clinical studies and the anticipated real-world use of a therapy, questions about how a drug may interact with other drugs, and lack of clarity on the meaningfulness of the potential benefits of the therapy) for a serious disease with high unmet medical needs, including those that may be going through accelerated approval.
Uncertainty is especially common, the draft says, in candidate therapies for rare diseases and FDA may use more regulatory flexibility when making decisions on products to treat such diseases.
The consultants say the draft guidance, especially the content on patient experience data and the critical role it plays in a drug development program, dovetails with the patient-focused drug development series the agency has been developing as required under PDUFA 6 and the 21st Century Cures Act.