By Elizabeth Danielson, MHA, Director, Payor Relations
The U.S. Food and Drug Administration’s (FDA) recent announcement that it is recommending removing approval for use of Avastin® (bevacizumab) for the treatment of metastatic breast cancer has generated considerable interest and strong opinions. Informed and concerned people view the decision differently: the FDA’s decision is endorsed by some oncologists and patient advocacy groups and condemned by others. If the FDA affirms its decision upon appeal, then organizations that pay claims – commercial health plans and Medicare intermediaries – don’t have the luxury of an opinion; they must act, and by taking action, affect the treatment decisions of oncologists and their patients. In deciding what to do, they must be able to defend their decision as both evidence-based and consistent with their overall drug coverage policies.
The FDA acknowledges that Avastin can still be prescribed for treatment of metastatic breast cancer even if it withdraws approval for that indication. But it would create the risk that insurance won’t cover it, making it unaffordable for most women. A great deal of cancer treatment involves off-label use of drugs – more than 50 percent by some estimates.1 Many, but by no means all, payors routinely cover off-label use of drugs in cancer care, especially if the drug’s use is supported by recommendations in NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines™) and NCCN Drugs & Biologics Compendium (NCCN Compendium™), which is derived from the NCCN Guidelines™. Thus, coverage policies often are not based on FDA recommendations. Some payors, especially “secondary” payors (stop loss carriers and reinsurers), won’t cover any drug that is used in the off-label setting, even for a cancer diagnosis. That policy, if applied to treatment that is considered standard of care and is recommended in the NCCN Compendium™, leads to many cancer patients being left without coverage for life-extending treatment.
A recent article in Bloomberg News2 observed that “NCCN Guidelines are recognized in more than 100 countries as standards in how to diagnose, treat and reduce the risk of cancer.” The article goes on to say that UnitedHealthcare, WellPoint, Aetna, and Humana rely on the NCCN Guidelines and NCCN Compendium as a basis for coverage. In 2008, CMS identified three new compendia – including the NCCN Compendium – that are approved in addition to the American Hospital Formulary Service (AHFS) Compendium for use by Medicare contractors to determine drug coverage for cancer treatment. Of the four compendia, the NCCN Compendium is widely considered to be the most up-to-date, comprehensive, and authoritative, with the most transparent process.
What resources and processes should be used as the basis for coverage determinations if the FDA, NCCN Guidelines, and NCCN Compendium are overlooked? Some payors rely on their own medical directors, or hire consultants to review evidence and make recommendations about which off-label uses should be covered. But are the conclusions of a small number of reviewers equivalent to the conclusions of a broad panel of sub-specialists that make up the NCCN Guidelines Panels?
The NCCN Guidelines, from which the NCCN Compendium is derived, are arrived at through the most comprehensive, transparent process of any that are currently available. As noted in the Bloomberg News article, a subset of NCCN Guidelines Breast Cancer Panel Members has been involved in clinical trials or consulted with Genentech; this information is updated at least annually and is documented in detail at NCCN.org.3 According to William T. McGivney, PhD, CEO of NCCN, NCCN Guidelines Panel Members are not allowed to receive more than $50,000 in total from outside companies or more than $20,000 from any one company. Every effort is made to prevent the substance or appearance of conflict of interest in the decisions of NCCN Guidelines Panels. If a panel member’s outside funding exceeds these limits, he or she can no longer serve on the panel. Where a conflict of interest exists or appears to exist, NCCN Guidelines Panel Members are excluded from discussion about that issue.
Dr. Robert Carlson, a breast cancer specialist at Stanford Cancer Institute and Chair of the NCCN Guidelines Panel for Breast Cancer, confirmed that the panel discussed Avastin three times during the past several months, anticipating that FDA might withdraw its approval. “The panel sees no new information in the current data sets that would alter [its] recommendations,” Dr. Carlson told Bloomberg News.
Also quoted in the Bloomberg News article, Dr. Alan Rosenberg, VP of medical policy at WellPoint, stated that their internal advisors “recognize that the NCCN’s [Compendium] is strongly supported.” In an October article in the ASCO Post,4 UnitedHealthcare’s senior vice president for oncology, Dr. Lee Newcomer, affirmed that UnitedHealthcare bases coverage decisions on the NCCN recommendations. “This takes the decision-making to the academic experts who we feel are best qualified to make the call, and second, the process is transparent…. So for us, the NCCN would also need to withdraw its recommendation…. If the NCCN still believes we should cover bevacizumab, we will.”
In the same article, Dr. James Cross, head of national medical policy and operations at Aetna, said that “If breast caSncer is removed by the FDA as an indication for bevacizumab, Aetna would consider removing coverage, but we would also consider the position of the NCCN on this issue, as we have covered drugs for indications in NCCN’s Compendium with a consensus rating of 2B or greater.”
The FDA’s December 15, 2010 statement on its website5, “Questions and Answers about Avastin,” states that “patients currently receiving Avastin for breast cancer should speak with their oncologists … about whether to continue their treatment or explore other treatment options. Oncologists should use their medical judgment when deciding whether their patients should continue using Avastin with the drug paclitaxel, take paclitaxel alone, or consider other treatment options.”
This statement by FDA spokesperson Erica Jefferson, quoted in the Bloomberg News article, is especially relevant. She states that the FDA views the [NCCN] Guidelines Panel as a “well-respected group of oncologists” whose recommendations sometimes differ from the [FDA’s] decision on approval. “With any cancer therapy, oncologists should use their medical judgment when deciding what treatment might be best for their patients when FDA-approved options have been exhausted.”
Of the options available – FDA guidance, independent review of evidence, or NCCN Guidelines recommendations – the latter represents the best available option.
1Ratner M and Gura T, Nature Biotechnology, 2008.
2Bloomberg News. “Insurers Back Roche’s Avastin Even as FDA Says It Doesn’t Work.” January 10, 2011.
3http://www.nccn.org/disclosures/panel_list.asp?ID=53 (Note: website registration at nccn.org is required to view content)