‘Groundbreaking’ Benefit/Risk Assessment Guidance Outlined
Four Sidley attorneys say an FDA draft guidance on benefit/risk assessment for new drugs and biological products “is [so] groundbreaking in its articulation of a vision of drug development that many entities involved in developing innovative drugs and biological products should review it carefully.” In an online post, the attorneys say the draft is of special interest to pre-commercial companies whose approach emphasizes efficiency and permits some residual uncertainty for clinical benefits and safety issues.
As reflected in the guidance, the attorneys write, FDA expects sponsors to focus their development activities in such a way that the data and information generated by those activities will minimize uncertainty in the benefit/risk analysis conducted by the agency during its review of NDAs and BLAs.
“Because FDA anticipates that clinical development will be structured in this manner,” the post says, “the agency also signals a relatively low tolerance for uncertainty, even in the area of rare disease drug development where patients express a desire for access and willingness to accept risk. Notably, FDA also puts industry on notice that active-control trials will be required in some cases, representing a departure from the established principle that FDA cannot require comparative efficacy data as a condition of approval.”
The attorneys’ analysis of the guidance covers:
- expectations of sponsors: structured benefit/risk planning;
- FDA’s approach to benefit/risk assessment;
- risk mitigation;
- uncertainty in the assessment of benefit and risk and rare diseases; and
- patient perspectives.