Blog April 30, 2018

Update on FDA’s Patient Benefit-Risk Assessment Program

patient making decisionOver the past several years, the U.S. Food and Drug Administration has begun efforts to further incorporate patient perspective on benefits and risks of potential new treatments in their regulatory reviews and to bring additional transparency and communication to the deliberation that goes into regulatory decisions. The agency has been responding to an increased awareness and understanding that a patient’s perception of the benefits and risks of different treatment options can vary based on the stage of disease, age of onset, available therapeutic choices, and whether a given treatment can improve their ability to function normally, slow the rate of disease progression, or impact other aspects of their quality of life. In other words, a 30-year-old man’s assessment of benefit and risk with respect to the treatment choices for his prostate cancer will likely differ from that of an 80-year-old man with the same disease. The belief is growing – both within the agency and externally — that those patient perceptions ought to be given weight in any regulatory decisions about such treatments.


On March 30, the FDA issued a statement about its activities to date and its plans to continue working closely with patients to incorporate their experiences into its Benefit-Risk assessments, to update the tools used to make those assessments, and to further improve the consistency and clarity of their communications about the reasoning behind specific regulatory decisions.


The agency first began incorporating a Benefit-Risk Framework into its regulatory reviews in 2013. An independent third-party evaluation of the FDA’s activities to date in this area has been generally positive: “Review staff, industry, health care providers, and patients and caregivers find the framework to be useful and effective in communicating the reasoning behind FDA’s decisions,” commented FDA Commissioner Scott Gottlieb, M.D.


The FDA’s new goal is to provide formal guidance by 2020 that further articulates the context in which the agency will make decisions and how it will incorporate its Benefit-Risk framework and templates into its review process. The agency also expects to issue further guidance and recommendations around:


  • Incorporating yet more patient experience and input into their decision making;
  • Organizing patient-focused public meetings on drug development to advance more systematic ways of gathering patient perspectives on specific conditions and treatments;
  • Encouraging patient stakeholders and others to conduct their own externally led, patient-focused drug development meetings;
  • Providing patients, caregivers, and advocates with more channels to provide meaningful input into drug development and regulatory decision-making, as well as easier ways to access information from others; and
  • Launching pilot programs and advancing policies in collaboration with the medical community, fostering trial design that places less burden on patients.


How much patient input will ultimately go into the FDA Advisory Committee recommendations and the agency’s regulatory decisions? What sort of tools might be needed to measure and report objectively on the benefit of new drugs to support their approval? And what will be required of industry to incorporate such varied evidence of patient-perceived benefit into their drug clinical trials? All this remains to be seen.