FDA Gives Update on Benefit-Risk Assessment Program

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The agency has release a report providing an overview of steps taken to enhance benefit-risk assessment in the review of drugs.

On March 30, 2018, FDA released an update on its implementation of efforts to enhance the agency’s benefit-risk assessment and communication in the drug review process. The document, “Benefit-Risk Assessment in Drug Regulatory Decision-Making,” provides an overview of the agency’s progress since the program began in 2013 as part of the Prescription Drug User Fee Act (PDUFA) V.

The document also discusses the enhancement of the Benefit-Risk Framework, and FDA plans on publishing guidance by June 2020 on the agency’s decision-making context and framework for benefit-risk assessment. This framework will provide clarity in communicating the agency’s reasoning behind drug regulatory decisions. Part of the goal of this effort is to understand patient care and evaluate benefits and risks from the patient’s perspective.

FDA has applied this framework during the past few years, and an independent third-party has conducted an evaluation of FDA’s work. According to FDA, the evaluation found that “review staff, industry, healthcare providers, and patients and caregivers find the framework to be useful and effective in communicating the reasoning behind the FDA’s decisions.”



After reviewing patient-focused drugs developed in more than 20 disease areas, FDA concluded that patient input informs the clinical context and provides insight in which to frame benefits and risk and also provides a direct source of evidence on benefits and risks. The agency is developing guidance for the use of more widespread development of patient experience data. Efforts to include patient experiences include hosting patient-focused drug development public meetings, launching pilot programs, and providing more channels for communication.

“The benefit-risk implementation plan issued today is part of the FDA’s ongoing commitment to advancing our mission of protecting and promoting public health. It marks another important step forward in increasing transparency of FDA decisions as well as streamlining the process by which we obtain input from the patient and stakeholder communities. We know that, in the battle against disease, engaged and informed patients are our best allies and our greatest resource,” said FDA Commissioner Scott Gottlieb, MD, in a statement.

Source: FDA