FDA on further Benefit-Risk framework – an outlook

PDUFA VI Draft Goals Letter just posted online

The Draft Goals Letter was just posted online here. There are lots of interesting things in this. Here is a quote from the Regulatory Decision Tools Subgroup that is especially interesting for Benefit-Risk:

“FDA discussed its proposal to explore the appropriateness of incorporating additional decision-analytic approaches that could inform FDA’s regulatory decision-making through the B-R Framework. As part of this proposal, the agency proposed convening at least one workshop to review potential approaches, such as the use of trial endpoints that capture ranked benefit and harm outcomes at the patient-level, and their applicability within FDA’s drug regulatory context. Industry indicated that the value in this proposed area of exploration would need to be weighed against other biostatistics-related proposals under discussion.”

FDA publishes draft guidance on BR assessment including patient perspective

The FDA published a draft guidance on “Factors to Consider Regarding Benefit- 11 Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions”. Notably it contains a section called “How FDA Considers Benefit-Risk in Patient Focused Medical Device Product Availability, Compliance, and Enforcement Decisions”. It also discusses patient preferences on the benefit, patient tolerances of risk, patient impact, and patient preferences for availability.