Drugs

Clarification Wanted in Drug Benefit-Risk Guidance

David McFarland

— 2 min read
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Pharmaceutical Research and Manufacturers of America (PhRMA) says it shares FDA’s efforts to help advance the benefit-risk related initiatives under PDUFA 5, but believes that additional guidance and clarity are needed on implementation of the benefit-risk framework to help ensure timely patient access to innovative biopharmaceuticals that treat serious and life-threatening diseases, as well as new therapies for unmet needs that are safe and effective. The PhRMA comment letter calls on the agency to further develop and advance the regulatory science of the benefit-risk assessment and framework, enhancing communications with sponsors and patients, developing and promoting the use of science-based and transparent standards and methods, and advancing consistent implementation across the agency. The letter also contains specific recommendations and request for FDA clarification.

The International Society for Pharmacoepidemiology discusses a 2013 FDA proposed structured framework to evaluate evidence of the benefits and risks of drug and biologic products and recommends that in the guidance on framework content, FDA: 

  •           provide guidance on the content, format, and level of detail to be included in the framework;
  •          encourage sponsors to identify uncertainty in the evidence and provide guidance on how different types and levels of uncertainty should be considered when making benefit-risk determinations;
  •          work with external stakeholders to provide case studies to show how decision-analytic and patient preference methods could inform benefit-risk assessments, to evaluate the strength and weaknesses of the methods, and to identify current barriers to including these methods in benefit-risk assessments;
  •          provide guidance on assessing the effectiveness of risk mitigation activities for identified risks and how this information should be incorporated into post-marketing benefit-risk assessments; and
  •          develop a plan for providing training on the use of the framework for FDA reviewers, industry, patient organizations, and other stakeholders.

The Biotechnology Industry Organization (BIO) says it believes the benefit-risk framework “represents an important advancement in FDA product regulation because it reflects and reinforces the underlying reality that the FDA review process is grounded in the assessment of both benefits and risks.” BIO makes recommendations for increased transparency and flexibility of benefit-risk assessment in complete response letters, consistent use of the benefit-risk assessment across review divisions; and benefit-risk and the patient perspectives.

The Cancer Support Community says that incorporating the patient voice into benefit-risk assessments is essential to understanding the way a drug or biologic affects the whole patient, and allows doctors to identify patient needs and challenges early, allowing them to intervene as appropriate. It says that incorporation of the patient experience should continue past the approval of the medication and into the post-approval review setting.

Finally, the National Multiple Sclerosis Society says it is important that FDA acknowledge that the benefit-risk paradigm among a patient population varies greatly by disease state.

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