FDA Bevacizumab Decision

FDA Bevacizumab Decision

Peter Paul Yu, MD, FASCO, FACP

Nov 20, 2011
This week the FDA decided to withdraw bevacizumab's indication for treatment of metastatic breast cancer. The earlier indication had been approved under FDA's accelerated approval process wherein final approval was conditional on the generation of confirmatory evidence based on clinical trials. Such trials did not show the expected benefit leading to the rendering of the controversial decision to withdraw the FDA indication.  Between the initial accelerated approval and this week's decision, practice patterns had been set using bevacizumab in the routine care of breast cancer patients with metastatic disease and a strong clinical impression was left on many patients and practitioners that at least a subgroup of this population did derive clinical benefit.

The FDA accelerated approval policy stems from a laudable desire to speed cancer patients' access to new therapies. It goes hand in hand with the CMS policy of "Coverage with Evidence Development," or CED, that allows provisional reimbursement for new drugs pending further supporting evidence development, but unlike the FDA policy, CED also provides for a mechanism to acquire that evidence through mandatory reporting of outcomes to a registry. This is a more rapid manner to fill the knowledge gap than depending on the completion of randomized clinical trials.

A third federal initiative that might speed up evidence generation and that holds great potential to better inform drug approval and reimbursement policy is the FDA mini-Sentinel Program. The mini-Sentinel Program now tracks data on 100 million patient lives through those patients' electronic health records (EHRs). These are patients who receive care at integrated health care delivery systems that use a variety of EHR vendors, but who have agreed to use a distributed data network system to answer queries about patient data and medication use to evaluate for adverse events and potentially confounding factors. Presently, the focus is on drugs that have already obtained FDA full approval, but eventually, such a system could be used for comparative effectiveness research and earlier in the drug approval process.

Together such initiatives as FDA's accelerated approval, CMS' coverage with evidence development, and FDA's mini-Sentinel Program may, if combined, lead to a more rapid and learning health care system that allows patients access to new treatments without exposing those same patients to excessive risk for adverse events or the health care system to costly, wasted expense. ESAs and bevacizumab had biologically plausible stories with enticing clinical benefits, but that is not sufficient.


The ideas and opinions expressed on the ASCO Connection Blogs do not necessarily reflect those of ASCO. None of the information posted on ASCOconnection.org is intended as medical, legal, or business advice, or advice about reimbursement for health care services. The mention of any product, service, company, therapy or physician practice on ASCOconnection.org does not constitute an endorsement of any kind by ASCO. ASCO assumes no responsibility for any injury or damage to persons or property arising out of or related to any use of the material contained in, posted on, or linked to this site, or any errors or omissions.

Back to Top