August 16, 2010
First it was the mammography dust-up. Now it's high-priced drugs for breast cancer.
Late last week, the Food and Drug Administration received a letter from Susan B. Komen for the Cure, the pink ribbon-bedecked juggernaut among breast cancer treatment advocacy groups, demanding the agency maintain its approval of Genentech's Avastin for women with life-threatening metastatic breast cancer. "We recognize the benefits of Avastin overall are modest for women with metastatic breast cancer," Komen chief Nancy G. Brinker said in the letter. "However, we do know that for some women, Avastin offers a greater than modest benefit."
Lawmakers have also sent warning letters to the FDA. Sen. David Vitter, R-La., told The Washington Post over the weekend that he feared "this is the beginning of a slippery slope leading to more and more rationing under the government takeover of health care."
Women's access to the drug isn't at stake — at least for those with money. Avastin, designed to restrict the formation of new blood vessels that feed nutrients to fast-growing cancer tumors, has clearly shown its benefit for colorectal cancer, so it will stay on the market. Any physician can prescribe an approved drug for any condition if he or she thinks it will benefit the patient.
But an FDA decision to pull back its specific approval for the breast cancer indication would allow (not require) insurers — including Medicare and Medicaid — to reject paying for the Avastin, whose annual costs can reach $100,000 a year for those who live that long.
The Drawback to "Accelerated Approval"
Why would a government agency like the FDA incur the wrath of the breast cancer advocacy community so soon after the U.S. Preventive Services Task Force got rebuked by advocates and Congress for daring to suggest that annual mammograms for women under 50 probably weren't necessary?
Last month, an FDA advisory committee made up of academic experts without ties to the drug industry voted 12-1 to withdraw the agency's previous approval of Avastin for breast cancer. That nod, given in 2008, had been based on a single clinical trial that showed the drug, when added to chemotherapy in women with far-advanced disease, delayed tumor progression for an additional 5 1/2 months compared to women who got chemotherapy alone.
However, there was a catch. That single trial didn't last long enough to show whether the women on Avastin lived any longer. So the FDA gave the drug so-called "accelerated approval," which is a process created during the early days of the AIDS epidemic for rushing potentially life-saving drugs to market. Companies can begin marketing a drug given accelerated approval, but they must also conduct new trials to confirm that the surrogate marker of clinical benefit — in this case, postponement of tumor growth – actually postponed death.
The new evidence contained in the two confirmatory clinical trials submitted to the FDA earlier this year disappointed fans of the drug. In one, Avastin delayed tumor progression for three months, but the average length of time to death stayed the same. In the other, delay of tumor progression dropped to less than a month, and the patients on Avastin actually fared worse in terms of time until death than those on chemotherapy alone.
"We now have a number of well done studies that show no benefit to extension of life," the chairman of the committee, Wyndham Wilson of the National Cancer Institute, said near the conclusion of the meeting. "It hasn't been shown that it has a clinical benefit."
"When we approved this drug, there was considerable controversy over the magnitude of the improvement in progression free survival," said Richard Pazdur, director of the office of oncology drug products at the FDA. "We're... somewhat disappointed that the initial enthusiasm" for the drug was not borne out by the new trials.
The Political Hurdles
But as so often happens in health care, the science of well controlled clinical trials (sometimes called the gold standard of medical research) must also clear political hurdles before it can be incorporated into standard medical practice. Patient advocacy groups are in the business of providing hope to their donors, which for Komen's ubiquitous "Race for the Cure" fundraisers includes millions of women across the U.S. The group, which generates over $100 million a year for research, also spends $1 million a year lobbying, according to its latest Internal Revenue Service filing.
Drug companies are in the business of selling drugs, and, as Genentech's Sandra Horning argued to the advisory committee, they are also in the business of selling hope. Allowing patients with metastatic breast cancer to hope for a few more weeks that their tumors weren't progressing provided substantial solace to those women, she said. That comment provoked heated rebuttal from many advisors, including several of the practicing oncologists on the committee, about whether that was worth the vomiting, stomach pains and raised blood pressure that many women experience on the drug.
And then there's the bipartisan consensus on Capitol Hill, which backs health care cost control except when it means that it might cost votes. Vitter may be a Republican, but it was Sen. Barbara Mikulski, D-Md., who led the charge to include in the Patient Protection and Affordable Care Act (health care reform) a mandate that the government pay for all mammograms, no matter what the age of the patient.
If the FDA caves to the pressure and allows Genentech to keep advanced metastatic breast cancer on the Avastin label, it will be one more indication that the nation still isn't serious about controlling health care costs by complying with science-based medicine.
is the author of The $800 Million Pill: The Truth behind the Cost of New Drugs.
Panel Tells FDA to Withdraw Approval of Avastin for Breast Cancer(The Los Angeles Times)
Study: 'Accelerated' FDA Approval Too Slow for Cancer Drugs (USA Today)
Think Tank Calls for FDA to Forgo Approval of Drugs Cleared by European Regulator (The Hill)
Once More, Health Care Cost Control (The New York Times)