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