U.S. FDA has produced a new guidance document, titled “E2C (R2) Periodic Benefit Risk Evaluation Report (PBRER) - Guidance for Industry.”
When a new medicinal product is approved for marketing, demonstration of safety and efficacy are generally based on data from a limited number of patients, many studied under the controlled conditions of randomized trials. Often, higher risk subgroups and patients with concomitant illnesses that require use of other drugs are excluded from clinical trials, and long-term treatment data are limited. Moreover, patients in trials are closely monitored for evidence of adverse events. In clinical practice, monitoring is less intensive, a broader range of patients are treated (age, comorbidities, drugs, genetic abnormalities), and events too rare to occur in clinical trials might be observed (e.g., severe liver injury). These factors underlie the significance of continuing analysis of relevant safety, efficacy, and effectiveness information throughout the life cycle of a medicinal product — promptly (as important findings occur) and periodically — to allow an overall assessment of the accumulating data. Although the majority of new information will be safety-related, new information about effectiveness, limitations of use, alternative treatments, and many other aspects of the drug’s place in therapy may be pertinent to its benefit-risk assessment.
Posted by Dr. Tim Sandle