Exaggerated hopes for new cancer treatment can sometimes prove painful and costly to patients. Take the use of bone-marrow transplants, coupled with high-dose chemotherapy, for advanced breast cancer. Beginning in the late 1980s, some oncologists began prescribing the controversial treatment, which had worked with some other cancers, even though only one small study seemed to show an effect with breast cancer, while other evidence suggested the treatments might make patients sicker. But with few other therapies available, hope soon displaced caution.
The expensive, painful treatment quickly became the rage among oncologists, the media, litigators and lawmakers, all of whom pushed insurers to cover the procedure as women's last, best hope. "Hope trumped science" and "politics trumped policy," wrote George C. Halvorson, CEO of Kaiser Permanente, the big managed-care organization, and George J. Isham, medical director of HealthPartners, an HMO based in Bloomington, Minn. "[G]ood-hearted lawmakers" and courts around the country required insurers to cover the procedure, they explained, despite warnings that clinical trials were needed to determine its effectiveness.
As often happens, media coverage fueled enthusiasm, according to health writer Shannon Brownlee, a senior fellow at the nonpartisan New America Foundation think tank. "I looked at 1,000 stories," she says, and 90 percent portrayed the transplants as "women's last chance," framing the treatment as a Homeric epic with villains — the cancer and, eventually, insurance companies who refused to pay — a physician hero and a patient, suffering victim.
Entrepreneurship also played a role. Physicians and others set up local clinics to perform the therapy, further pressuring insurers to pay, says Richard A. Rettig, a co-author of the 2007 book False Hope: Bone Marrow Transplantation for Breast Cancer.
It was not the first time financial incentives trumped clinical judgment, said Guy B. Faguet, a professor emeritus of medicine at the Medical College of Georgia. For example, he said, when doctors own radiation facilities, radiotherapy use jumps 53 percent, charges rise 42 percent and consultation time between doctors and patients drops 18 percent, compared to the national average.
Ironically, when clinical trials were launched to test the bone-marrow transplant idea, the existence of the trials themselves signaled clinicians that the treatment was worthwhile, says Rettig, encouraging even more doctors to offer it. And the rush to treat made it harder to recruit trial subjects: Women who knew they could get the treatment outside of the trials were reluctant to participate because they feared they might be placed in the no-treatment "control" section of the experiments, Rettig says.
By the mid-1990s the transplant frenzy led at least eight states to legally require insurers to cover the procedure. In 1993, a California jury ordered the insurer HealthNet to pay cancer patient Nelene Fox $89 million for refusing coverage of the treatment. But in 1999, a large, definitive clinical trial found that the transplant/chemo combination treatment did not improve survival rates for breast cancer. And many women had suffered serious side effects, including heart failure, bone-marrow disease and even death.
By then at least 42,000 women had received the treatment, not including those in the clinical trials, at a total cost — at $80,000 per patient — of more than $3 billion.
Despite the costly bone-marrow transplant debacle, some cancer researchers and oncologists are optimistic that 21st-century cancer science — including understanding of genetic changes and molecular mechanisms — has advanced to the point where optimistic predictions about successful cancer treatments are no longer hype but reality. Food and Drug Administration chief Andrew von Eschenbach, who served as director of the National Cancer Institute (NCI) for four years, said in 2005 the institute had "committed itself [to] eliminate the suffering and death" caused by cancer by 2015.
But other analysts caution that such grand visions can be more of a danger than a help in cancer medicine. "Some people with visions are hallucinating," says Paul Goldberg, editor of The Cancer Letter, a medical newsletter. When it comes to cancer science, "sometimes the worst thing you can have is somebody with a vision."
In fact, points out Goldberg's wife Kirsten — publisher of the newsletter — von Eschenbach's "grandiose vision" actually crippled the NCI by shifting the focus to "far-out things like nanotechnology" while shortchanging approaches that might have borne more fruit.
"A visit to the annual [American Society of Clinical Oncology] meeting will convince one how desperate we are for any sign, no matter how minor, that a treatment might work," said Joseph V. Simone, a professor emeritus of pediatrics and medicine at the University of Utah, referring to how oncologists grasp at even the slimmest hint that a new treatment might work. "We share our patients' desperation, but we do them no favor by adopting unproven therapies as the standard of care."
Rettig favors a new partnership between the NCI, patient-advocacy groups and insurers that would issue formal public statements about which experimental treatments need clinical-trial evaluation. Insurers would then fund the clinical trials, promising to cover whatever procedures are proved safe and effective. Such a move, Rettig says, would protect insurers from the kinds of lawsuits they faced over bone-marrow transplants.