Risk/Benefit Analysis: What, How, and Why?

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With the fifth reauthorization of the Prescription Drug User Fee Agreement (PDUFA V) one and half years underway, we welcome Dr. Rob Metcalf to the CMM platform to explore how PDUFA V accelerates patient access to medicines. Dr. Metcalf is Vice President of Global Regulatory Affairs at Eli Lilly and Company and a member of the FasterCures Benefit Risk Advisory Council, which provides recommendations to expand patient input in shaping new medicine development and influencing regulatory decisions.

Approving safe and effective new medicines has always been the main charge of the Food and Drug Administration (FDA). The FDA's decision about a potential new treatment for use hinges on the evaluation of that medicine's benefit/risk profile and requires the determination that the benefits of the drug outweigh the risks associated with it. In the U.S., the FDA has the responsibility to make these decisions, and they have been called upon to do so in a manner that protects and promotes public health.

The environment in which benefit-risk assessments are made is extremely complex. Regulators are required to make decisions in a timely fashion, and they must take into consideration the perspectives of more and more stakeholders. Many of these stakeholders' value systems differ as to which potential benefits and risks are relevant and to what extent each matters.

Lack of structure and transparency in the current system has led to decisions that are inconsistent and unpredictable, difficulty for regulators in defending decisions and articulating their underlying logic, and perceptions that decisions do not optimize patient perspectives. Structured benefit/risk assessment is a tool that can help the agency manage the complexities associated with regulatory decision making and communication.

While the FDA's decision to approve or reject a new medicine relies mainly on scientific data about safety and how well a treatment worked in clinical trials, patients also bring an important voice in assessing the benefits and risks of a potential medicine. For many patients, the potential risks of a new medicine may be surpassed by the potential improvements that a new medicine could make to their health or quality of life. This patient perspective can help enrich the FDA's knowledge of a disease state and bolster its decisions.

Ultimately, patients have the most to lose if there is a lack of balance between drug benefits and risks. The most recent iteration of the Prescription Drug User Fee Agreement (PDUFA) aims to improve this process by gathering input from patients, who know the day-to-day realities of their condition better than anyone, into a benefit/risk framework that will be eventually be utilized in all regulatory approval decisions. Tune in next month when I come back to discuss how benefit/risk has become more than just a regulatory process--it has become an opportunity for patients to have a voice in shaping their access to future medicines.