CEI Comments to the Food and Drug Administration Regarding PDUFA Reauthorization

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The Competitive Enterprise Institute appreciates the opportunity to submit these comments regarding the FDA’s proposed recommendations for reauthorization of the Prescription Drug User Fee Act. PDUFA has helped patients by ensuring that the FDA has greater resources available to review New Drug Applications and Biologics License Applications, and in turn ensuring that the agency is capable of providing more timely access to new medicines. 

Still, there remain numerous shortcomings in agency oversight of drug and biologics development and marketing. CEI is therefore pleased to see that the FDA’s proposed recommendations for PDUFA reauthorization include several items that could begin to address these concerns. Among these are the agency’s proposals to explore more formalized and transparent tools for weighing the risks and benefits of new medicines and a commitment to use patient reported outcome measures in assessing risks and benefits.

No medicines are perfectly “safe” in the sense that they have no potentially negative side effects. For many products, including drugs and biologics that treat serious life-threatening or disabling diseases, medicines may be considered safe enough, even in the presence of substantial known risks. Unfortunately, the FDA’s current process for assessing and balancing benefits and risks is largely ad hoc, informal, and qualitative, relying primarily on the intuitive judgment of the agency’s medical review staff and expert advisory committees. Implementing a more formalized and comprehensive benefit-risk framework would better advance the agency’s important goals. 

More formalized, quantitative benefit-risk analysis is not a panacea. But implementing improved processes would help manufacturers better understand the kinds of data that must be generated during pre-clinical and clinical testing, help FDA product reviewers better systematize their decision-making, and help patients and medical clinicians better assess the utility of new products when prescribing treatment regimens. Although the quantitative assessment of benefits and risks will always suffer from inadequacies, making greater use of quantitative assessments will nevertheless lead to an evaluation process that is more transparent, predictable, and rational. 

Denying approval for a drug or biologic that is known to be risky, but which nevertheless has a positive benefit-risk balance could harm more patients than it would help – particularly when the product is intended to treat serious life-threatening or disabling conditions that have few other available treatment options. Indeed, while benefit-risk analysis and benefit-cost analysis have been criticized for inappropriately justifying decisions to place potentially risky products on the market, a more appropriate name for the process would be “risk-risk assessment.” That framing best describes why the analysis is useful:  Its ultimate goal is to identify which of many possible choices would lead to the best, or safest, overall outcome.

In the end, benefit-risk assessment is not a dispositive tool that can make choices for agencies. Rather, it merely provides a framework for agency decision makers to “organize available information” by forcing them “to state their assumptions clearly, exposing possible biases to criticism and correction.” Therein rests another important feature of benefit-risk assessment in the drug and biologics approval process. Ultimately, the purpose of benefit-risk assessment is not to replace the judgment of medical experts in the decision-making process, but to put the FDA’s expert judgments on record, explain the agency’s reasons for approving or denying approval for new products, and hold those decisions up to public scrutiny.