What Does “Safe” and “Effective” Mean at the FDA?

Today, the Niskanen Center submitted comments to the Food and Drug Administration (FDA), arguing that its structured benefit-risk assessment framework fails to address the true causes of new drug approval delays. In order to meet the Agency’s statutory obligations under the Food and Drug Administration Safety and Innovation Act, the FDA needs to clarify its standards for safe and effective drugs. Until it does so, the structured benefit-risk assessment framework will simply serve as a band-aid to a far more systemic regulatory problem.

From the executive summary:

The Food and Drug Administration’s Draft PDUFA V Implementation Plan provides a welcome set of recommendations for modernizing the Agency’s IT infrastructure and accommodating the need for electronic submissions of new drug applications. However, the plan fails to effectively communicate the benefits and risks associated with new drug approvals by ignoring the fundamental lack of clarity regarding the standards by which the Agency determines a drug’s “safety” and “effectiveness.”

In order to meet its statutory obligations under the Food and Drug Administration Safety and Innovation Act, we offer a number of recommendations that can improve the Agency’s Draft PDUFA V Implementation Plan: (1) narrow pre-market approval considerations to safety and effectiveness, not hypothetical off-label uses; (2) clarify the specific standards for what constitutes a “safe” and “effective” drug; (3) limit the use of the structured benefit-risk assessment laid out in the Draft PDUFA V Implementation Plan to the post-market approval process; and (4) move to embrace a tiered order of effectiveness model for new drug approvals.

Read the full comments here.