FDA reviewers need a new transparent and predictable approach that advances innovation while maintaining scientific rigor. These are not in conflict, but the lack of a clear framework makes decisions seem like haphazard, discretionary, or worse, appear to compromise safety. CLL Society joined a broad swath of rare disease patients in support of a petition to the FDA calling for a legally binding regulation that would rely on a context based approach to identifying clinically appropriate endpoints, study methodology and a statistical analysis plan. This approach would not lower current evidentiary standards and would be durable with scientific advances while recognizing the particular circumstances of rare disease drug development. Read the petition here. Patients, clinicians, and researchers interested in supporting the petition can email [email protected]
Read the full comment letter here.
