Avastin Breast Cancer Decision Leaves Questions

As CBS News notes, breast cancer patients are "dismayed" at the FDA's decision to pull Roche's Avastin drug for breast cancer treatment in the U.S.

The blockbuster drug will no longer be available for treatment of advanced breast cancer, a blow to women with the disease who have been lobbying to keep the drug available as an option when all others have failed. The drug remains on the market to treat colon, lung, kidney, and brain cancers.

"I did not come to this decision lightly," stated FDA commissioner Dr. Margaret Hamburg. "Sometimes despite the hopes of investigators, patients, industry and even the FDA itself, the results of rigorous testing can be disappointing."

“We are disappointed with the outcome. We remain committed to the many women with this incurable disease and will continue to provide help through our patient support programs to those who may be facing obstacles to receiving their treatment in the United States,” commented Hal Barron, M.D., chief medical officer and head, Global Product Development at Roche, which makes the cancer drug through its Genentech division. “Despite today’s action, we will start a new Phase III study of Avastin in combination with paclitaxel in previously untreated metastatic breast cancer and will evaluate a potential biomarker that may help identify which people might derive a more substantial benefit from Avastin.”

"We know that this decision was a difficult one for the FDA and respect the length of time they spent deliberating the many issues that impact patients," stated the Susan G. Komen breast cancer advocacy organization in a statement. "We want to ensure that women who are successfully using Avastin today continue to have access to the drug, and that their treatment be covered by third-party payers."

The FDA's controversial decisions stems from research showing that there’s no conclusive proof that it extends lives of breast cancer patients, while it presents dangerous side effects including severe high blood pressure, massive bleeding, heart attack or heart failure, and perforations in the stomach and intestines.

Hamburg justified the FDA decision based on the rationale that “it is clear that women who take Avastin for metastatic breast cancer risk potentially life-threatening side effects without proof that the use of Avastin will provide a benefit, in terms of delay in tumor growth, that would justify those risks.”

The FDA action isn't an outright ban, as it leaves an opening for doctors to still prescribe the drug — although insurers may not pay for it, and a year’s treatment for breast cancer can run upwards of $100,000. Medicare’s response to the FDA move is they “will monitor the issue and evaluate coverage options as a result of action by the FDA but has no immediate plans to change coverage policies,” said spokesman Don McLeod to the Washington Post.

The decision didn't come out of the blue. In 2008, the FDA’s fast-track approval of Avastin for breast cancer was contingent upon continued studies of the drug’s usefulness, but research did not indicate that users’ tumor growth was slowed or their lives lengthened. 

Given that the drug is under fire beyond breast cancer treatment, it begs the question why the FDA singled out breast cancer applications of the drug. In September, the New York Times reports, five patients being treated with Avastin for eye disease at the Veterans Affairs medical center in Los Angeles became blind.

As a treatment for "wet," age-related macular degeneration and other eye diseases, Avastin is comparatively cheap at $50 an injection, which saves Medicare and patients hundreds of millions of dollars annually as compared to another Genentech drug for eye disease, Lucentis, which costs $2,000 an injection. 

However, earlier this year, four other patients at the Veterans Affairs hospital in Nashville suffered infection from bacterially contaminated Avastin, leaving one man blind and brain damaged, and his family filing a $4 million suit. 

Proponents of Avastin claim two million plus injections of the drug have been administered in eye treatment since 2005 with few problems. Oncologists and advocacy groups are split on the issue with some saying the FDA action against Avastin is due to cost — a claim disputed by the agency which says its due to science and lack of clinical proof.

This latest FDA decision is one more in a series of ongoing debate about cancer drugs and public expectations of effective treatment of life-threatening disease.

It’s a hot topic on Twitter, while the full text of the FDA’s decision and a Q&A is available here.