Comments on Postmarketing Studies Draft Guidance
Kaiser Permanente says that expanding the scope of circumstances when postmarketing requirements (PMRs) can apply could increase available scientific evidence about drugs, which will support evidence-based treatment decisions and improve the quality of care. The company strongly encourages FDA “to apply and enforce meaningful PMRs to assess serious risks and the safety and clinical benefit of drugs approved under expedited programs.” Specifically, it says the agency should (1) require manufacturers to evaluate clinical endpoints such as morbidity and mortality in all PMRs when a drug is approved using a surrogate endpoint as the primary endpoint; (2) include a conspicuous consumer- and provider-facing “gray box” warning for any drug approved on the condition of completing a PMR, indicating that the drug’s safety and clinical benefit are subject to further review; and (3) require manufacturers to make robust information about PMRs publicly available so the evidence can be used to support treatment decisions, pharmacy benefit design, and other important healthcare system functions that improve quality of care.
Pharmaceutical Research and Manufacturers of America recommends that the agency clarify how it intends to implement its new authorities under the Substance Use-Disorder Prevention that Promotes Opioid Recovery and Treatment (SUPPORT) act. It says FDA should provide more details on when it proposes to require a postmarketing study or postmarketing clinical trial to assess the serious risk of reduced effectiveness under the conditions of use prescribed in the drug’s labeling. It also calls on the agency to more clearly describe the process for applying the law’s stepwise approach for requiring postmarketing studies or clinical trials, and it recommends revisions to the draft’s “New Safety Information” and the FDA rationale for a postmarketing requirement.
The National Center for Health Research asks that the guidance clarify that post-market studies can supplement but not replace essential information about safety or efficacy. It also says that when FDA requires a clinical trial or another type of study and the sponsor fails to follow the regulatory requirements, the agency should use all available enforcement powers to ensure the needed information is delivered in a timely and complete manner.
Pfizer says the process for determining new postmarketing requirements both during the drug review period and post-market should be predictable, articulate a clear scientific rationale regarding the scientific questions addressed, and allow for sufficient time for FDA-sponsor dialog and review of study objectives, feasibility, and design.
Finally, the Biotechnology Innovation Organization says that ensuring that postmarket requirements are feasible, efficient, and effective and that information gained from them is effective in supporting benefit/risk assessments requires a practical and science-based evaluation of the feasibility and relevance of the research question that begins pre-approval and extends throughout a product’s lifecycle.