Patients with metastatic breast cancer who benefited from taking Genentech/Roche's Avastin are running a grassroots social media campaign, including a Facebook page and an online petition, to stop the FDA from withdrawing approval for that indication.

The agency's Oncology Drugs Advisory Committee voted 13-0 against full approval of Avastin (bevacizumab) for first-line treatment of metastatic breast cancer in July ([A#0072030001]).

A discussion on a breast cancer website led a 48-year-old woman with triple negative breast cancer named Christi Turnage to create the online petition, with the technical help of the spouse of another patient, on the "Care2" social networking website.

The petition argues that Avastin has miraculous effects in some cases, such as in some women with triple negative breast cancer and that FDA withdrawal would amount to a "death sentence" for those who are responding well.

The petition had over 2,600 signatures as of Aug. 2 and organizers are hoping to get at least 10,000. If an indication is withdrawn for metastatic breast cancer, insurers will stop covering the medication and patients will have to foot a big bill for the drug's cost, the petition warns.

"This medication is not being pulled for safety reasons. Current clinical studies show that it didn't extend lives as long as the FDA thought it should," the petition states.

Avastin had received accelerated approval in 2008 with paclitaxel chemotherapy for metastatic breast cancer. But the ODAC committee decided that the progression free survival was not clinically meaningful and voted that the indication should be removed from the label of the drug, which is approved for a number of other cancers: metastatic colorectal cancer, non-small cell lung cancer, glioblastoma, and metastatic kidney cancer. FDA will make a decision by Sept. 17 about whether to follow the committee's recommendations on metastatic breast cancer.

In an interview, Turnage said the petition really started to gain steam after her 19-year-old son Josh on his own accord posted the petition as an event on a Facebook page and word started to spread. The issue then drew the support of Sen. David Vitter (R-LA), who expressed concerns that the recommendation for Avastin withdrawal was "potential early signs of Obamacare rationing" in July 30 post on his website. The senator noted that he had sent a letter expressing his concern to Richard Pazdur, director of FDA's Office of Oncology Drug Products.

"I was even more alarmed that the FDA's Advisory Committee may soon pull this drug not because of safety concerns but because of cost-effectiveness," Vitter wrote. "The decision about whether patient should use life-extending drug is a decision that should be made solely between a doctor and a patient, not a government panel."

Withdrawal May Not Deter Insurance Coverage

Vitter asserts that a withdrawal of an indication could mean no insurance coverage from commercial carriers as well as Medicare. However, some patients have said that their insurers have indicated they will continue to cover the drug regardless of its approval status, as long as it is included in practice guidelines from the National Comprehensive Cancer Network. Furthermore, the Centers for Medicare and Medicaid Services recognizes off-label uses of cancer drugs that are included in NCCN guidelines.

The NCCN is due to hold a webinar on treatment of metastatic breast cancer on Aug. 16, although it's unclear whether the group is now reviewing its guidelines.

Grassroots petition aside, some official breast cancer advocacy groups strongly supported the withdrawal of an indication for metastatic breast cancer. San Francisco-based Breast Cancer Action, for example, agreed with the ODAC decision. A drug should meet one of three conditions, explained the organization's program manager Kim Irish. It should improve overall survival, and/or improve quality of life, and/or be less costly than available treatments. And based on the research, in Breast Cancer Action's view Avastin does not meet these criteria.

Turnage argues that subset data showing positive effects for some patients might not be available but that the drug is clearly benefiting some patients. After a diagnosis with Stage IV triple negative cancer, she herself is now completely cancer-free on Avastin and said there are no treatment other options if she has to stop taking it. Poly ADP ribose polymerase inhibitors are looking very promising for triple negative disease but are not yet commercially available.

An expanded access program is up-and-running for BSI-101, the PARP developed by BiPar and now owned by Sanofi Aventis, but it is only open to patients with no measurable disease. So ironically, Turnage said she is doing so well on Avastin that she does not qualify. Meanwhile, two of her friends who do have measurable disease are in a lottery and hoping they can make it into the expanded access program.

Similarly, Lori Baur, another patient with triple negative disease believes her survival is dependent on access to Avastin. Baur recalls that when she was diagnosed with cancer with widespread metastases to the brain in early 2009, her doctor initially gave her about six to eight weeks to live. Baur said she is now doing well on Avastin. Doctors don't take a cookie cutter approach to cancer treatment, rather they personalize their practices depending on a patient's tumor type, Baur argues.

"Why is Avastin being singled out? It doesn't work for everyone but none of the drugs do," Baur said in an interview.

What A New Label Might Look Like

Genentech declined to comment on its plans for patients should FDA decide to go along with the committee's recommendation in September but said it will be prepared in the event of a withdrawal of the indication.

One possibility is that FDA will not close the door completely on the metastatic breast cancer indication, and instead Avastin will be labeled for patients that are benefiting from or have previously benefited from the drug.

That was the relabeling approach that FDA used when AstraZeneca's non-small cell lung cancer drug Iressa (gefitinib) failed to satisfy the requirements for full approval in a post-marketing study. In contrast, Pfizer is completely withdrawing its acute myeloid leukemia therapy Mylotarg (gemtuzumab) -the first product to be pulled from the market after gaining accelerated approval - and it will be available only through a treatment IND (Also see "Pfizer Pulls Mylotarg For Safety 10 Years After Accelerated Approval" - Pink Sheet, 28 June, 2010.). Mylotarg's problem centered around liver toxicity, however, not the weaker-than-expected efficacy seen with Iressa and Avastin.

-Emily Hayes (e.hayes@elsevier.com)

Avastin Reimbursement Worry Drives Patient Petition To FDA

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