NBCC’s Chief Programs Officer Michelle Tregear and Chief Policy Officer Melanie Wyne sent the following letter to Richard Pazdur, MD, director of the Oncology Center of Excellence at the US Food and Drug Administration (FDA).
Dear Dr. Pazdur,
We were pleased to represent the National Breast Cancer Coalition (NBCC) at the recent FDA/AACR/ASA workshop on the use of overall survival (OS) data in oncology clinical trials. We were encouraged to hear from each panel that regulators and industry agree OS data is critical to the FDA’s mission to protect public health by ensuring the safety, efficacy, and security of drugs and other medical products. As you well know, NBCC believes that drugs must be approved based on clinically meaningful outcomes rather than unvalidated surrogate endpoints and that we need more drugs that save or meaningfully improve patients’ lives, not simply more drugs.
We have long made our position clear on the need for high-level evidence of clinical benefit for drug approval. NBCC recently submitted comments on the draft FDA Guidance on Accelerated Approval Trial Design for Oncology Drugs. Our chief concern with the draft guidance is that it perpetuates the use of a surrogate endpoint, progression-free survival, as a measure of clinical benefit. It also fails to advance the inclusion of trained patient advocates in the design and conduct of clinical trials.
We appreciate the FDA’s history of working with patients. We are grateful for our history of interaction with the FDA and participation in your committees and panels. We are especially grateful that FDA representatives have participated in NBCC’s education and training programs for lay advocates.
We do believe, however, that a more robust patient voice must be included. While we heard repeatedly during the workshop that the patient’s voice was critical, only one patient advocate was included in the workshop. There are many voices in the patient advocate community. Moreover, while many patient groups promote getting more drugs to the market faster, often without an understanding of whether a clinical benefit is present, you should include those groups that take a different approach. While NBCC recognizes that we are all desperate to find interventions that will save or significantly prolong lives without reducing the quality of life, we should not accept anything less than real, high-level evidence that approved treatments do this.
Having worked on several pivotal trials as informed advocates, we appreciate the importance of a strong FDA with high drug and device development, testing, and approval standards. We are especially supportive of the FDA’s resolve in the face of pressure from interests that desire to speed up the approval process at the potential expense of rigor and safety. We will remain strong supporters of an efficient, well-run, and robust FDA that will benefit all Americans and preserve the highest level of public health while employing the latest and best technology and science.
We look forward to continuing to work with the FDA and other relevant policymakers on these critical issues.
Michelle Tregear, Chief Programs Officer
Melanie Wyne, Chief Policy Officer