An unwelcome holiday gift

Should the federal government have the power to deny women with advanced breast cancer access to a drug they now have access to?

That is the principle at the center of the decision by the Food and Drug Administration (FDA), due on December 17th, over whether to revoke its 2008 approval of Avastin to treat late-stage breast cancer, which kills some 40,000 women in the United States each year.

Medicare covers cancer drugs when they have been approved by the FDA or are included in the National Comprehensive Cancer Network compendia of effective uses. Withdrawing approval for Avastin, which works to cut off blood flow to tumors, could hand down a sentence of earlier death to thousands of women.

Back in 2008, the FDA’s Oncologic Drugs Advisory Committee (ODAC) approved the use of Avastin in combination with chemotherapy because it stopped tumors from growing (progression-free survival) in the majority of patients. At the time the FDA wanted follow-up studies in more patients to confirm the benefits and check for side effects. The follow-up studies confirmed that Avastin stopped tumor growth and that some patients lived longer. Yet ODAC decided to recommend recalling Avastin for advanced breast cancer treatment because it didn’t increase overall median survival, a standard it imposed after the fact.

The shifting of standards is problematic. Many drugs in use today, such as Tykerb and Xeloda, were approved based on an increase in progression-free survival, even when they did not initially extend overall median survival. There’s a scientific reason for this: overall median survival only tells you what the median survival of all patients was with or without the drug. It doesn’t account for the fact that a drug may significantly extend the lives of some patients. A drug like Avastin may have toxic effects on many breast cancer patients, but for many others it can truly extend and improve life.

If Avastin is yanked because of a late-inning change in rules, it will set a deadly precedent for allowing a handful of advisors to dictate medical practice to millions of cancer patients. First, it will discourage the sort of real-world use that can inform and enrich larger clinical trials. Second, it will discourage companies from conducting and patients from enrolling in clinical trials. Finally, to the extent that ODAC shifts standards, it becomes an unaccountable panel that can control access to and reimbursement, not just for Avastin but for many other cancer drugs either on the market or in the pipeline.

Then there is the perception that ODAC changed the rules to comply with the cost containment mindset that shaped Obamacare. If the FDA accepts ODAC’s recommendation, then next month breast cancer patients with Avastin prescriptions will face the no-win scenario of either abandoning the treatment altogether or paying the drug’s $7,300 monthly price tag. Not much of a holiday gift.

For most ordinary American women, there is no extra $88,000 tucked away in their purse each year to pay for new medical expenses. This means that only the wealthy will have a choice of using Avastin. The rest will have to resign themselves to an earlier death. That holiday gift might be the last one they receive.