NEWS

Costs Studies Show Clinical Trials, Standard Therapy May Be Equal

Tom Reynolds

It is a common lament that insurers are crippling progress in cancer treatment by refusing to pay treatment costs for patients in clinical trials.

But evidence is emerging that routine care costs for trial participants may be no greater than for patients treated outside trials. And while a major federally funded study gears up to answer that question definitively, industry sources say that insurers are moving to cover routine care costs in trials, that some highly reputable insurers have long done so, and that the true barriers to improved clinical trial participation lie elsewhere.

Findings presented in May at the American Society for Clinical Oncology’s annual meeting in New Orleans suggest that routine care costs for trial participants are comparable to those for other cancer patients. One study done by the American Association of Cancer Institutes at five large cancer centers found that 6-month direct medical charges for 35 patients in phase II trials averaged $57,542, compared with $63,721 for 35 matched patients not in trials.

Two studies at Memorial Sloan-Kettering Cancer Center in New York had similar findings. The first compared hospital costs and physician charges for 152 Medicare-eligible patients treated with chemotherapy and found that costs for patients in trials averaged $30,775, or 20% lower than the $37,055 for patients who got standard care. The second study used patients in two phase I trials as their own controls and reported that trial costs were similar to the costs of standard chemotherapy.

"We now have clear evidence that cancer clinical trials . . . cost no more than standard therapy," Derek Raghavan, M.D., chief of medical oncology at the University of Southern California-Norris Comprehensive Cancer Center, said at the ASCO meeting.



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Dr. Derek Raghavan

 
Other experts said, however, that the studies publicized at ASCO are less than convincing. "These studies were small, they weren’t well controlled, costs weren’t reliably collected," said Martin Brown, Ph.D., chief of the National Cancer Institute’s Health Services and Economics Section.



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Dr. Martin Brown

 
A pair of reports published in the Journal of the National Cancer Institute provide more solid evidence but are still limited to single institutions, Brown added. A study from Kaiser Permanente in Oakland, Calif. (J Natl Cancer Inst, Jan. 19, 2000) found costs 10% higher in trials than out, but if expensive bone marrow transplants were excluded, the difference disappeared. In the other, an analysis of trials at the Mayo Clinic in Rochester, Minn. (J Natl Cancer Inst, May 19, 1999), the authors concluded that "clinical protocols may add relatively little" to the already high cost of cancer treatment.

New Study

The limited evidence on trial costs has prompted a new study, funded by NCI, NIH, and the National Science Foundation, that is designed to answer the question more definitively. The Costs of Cancer Treatment study, conducted by RAND Corp. of Santa Monica, Calif., will analyze costs for 1,500 patients, half of them in phase II or III trials and half receiving standard treatment.

To ensure that it is representative of trials nationwide, the RAND study will be conducted in a broad range of settings, from teaching hospitals to community-based clinics, randomly selected from among all U.S. institutions, clinics, and practices participating in NCI-sponsored cooperative group studies. And a thorough analysis of costs, derived from patient interviews along with medical and financial records, is designed to uncover costs that may have been missed in earlier studies, said principal investigator Dana Goldman, Ph.D., a RAND economist.

Because studies typically have examined records only at the institution performing the trial, "it’s possible that some people, especially off-protocol, may be getting care from providers outside the institution," incurring costs that are missed in the analysis, he said. "So we are actually going to the patients and asking them, ‘where are you getting care?’ " Results of the study are expected in 2002.

‘Moving in the Right Direction’

Joseph Newhouse, Ph.D., a Harvard economist who heads the Sloan Center for the Managed Care Industry, Cambridge, Mass., agreed that the industry is moving in the right direction—to pay for the experimental arm as it would pay for the control arm, though he added the move may be partly inspired by "threat of litigation."

States are also taking action. Rhode Island passed a law in 1991 requiring coverage for cancer patients in trials. Maryland, Ohio, and Georgia followed suit in 1998, Virginia in 1999, and Arizona, Illinois, and Louisiana in 2000. (Georgia’s law is limited to children and Ohio’s to state employees.)

Mohit Ghose, a spokesman for the American Association of Health Plans, said the industry opposes such mandates and that market forces will compel insurers to cover patients in trials.

In New Jersey, a voluntary coalition of insurers, providers, researchers, ethicists, legislators, drug makers, and consumer advocates forged an agreement to cover costs of patients in trials. Mary Todd, M.D., deputy director of the Cancer Institute of New Jersey in New Brunswick, said the state worked toward voluntary agreement to avoid the adversarial climate that can result when mandates are imposed.

At the federal level, the Patient’s Bill of Rights, which would extend the mandate nationally, is still under debate in Congress. But President Clinton’s recent directive that Medicare pay patient care costs in trials (see News, July 5) will have enormous direct and indirect impacts, said Diana Petitti, M.D., director of research and evaluation at Kaiser Permanente of Southern California in Pasadena and vice chair of the National Cancer Policy Board. Medicare beneficiaries make up about 65% of all cancer patients, she said, and Medicare is a bellwether for the entire health care industry.



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Dr. Diana Petitti

 
"Whether the RAND study shows trial costs are more expensive or not more expensive is probably a moot point now that the decision has been made at the public policy level," she added.

Mary McCabe, director of NCI’s Office of Clinical Research Promotion, said that in many cases, insurers have covered patients in trials only as demonstration projects. "We’ve been seeing a gradual change" in favor of coverage, she said, "but we’d like to see it become a national standard rather than the exception." Unless they have convincing evidence that trials are not hurting their bottom line, insurers may resist making permanent policy commitments to trial coverage, McCabe said.

One issue uniting all parties is the desire to avoid a scenario that up until now has been unfortunately common: lawsuits forcing insurers to cover trial costs. The most notorious have been bone marrow transplants for breast cancer. Despite a lack of positive data from large, randomized clinical trials in the United States, insurers were forced to pay for thousands of these procedures.

Opinions diverge on the extent to which insurers are to blame for poor trial enrollment, but everyone seems to agree it is not the only barrier. "Many fingers have been pointed at insurance coverage policy as the reason there aren’t more patients in clinical trials," Petitti said, but available evidence suggests the problem is more complicated. For example, when United HealthCare, a Minneapolis HMO, launched a pilot program in 1999 to encourage its members to join trials, only about a dozen patients responded. Researchers frequently cite patient’s lack of knowledge about trials, reluctance to be randomized, and belief that the treatment has not been sufficiently tested as reasons for nonparticipation.

Definitions Unclear

A 1999 report from the U.S. General Accounting Office found that nearly all plans surveyed, while formally excluding "experimental treatments," commonly made exceptions for patients in "legitimate" trials. However, GAO noted, "there is little agreement on which trial services constitute standard care, and, therefore, payment for services provided in a trial can vary widely."

Petitti asserted that "routine care costs have never been a big issue" for insurers, In fact, she said, costs vary so much from one patient to another that insurers typically have no way to know whether a patient is in a trial—a point also made in the GAO report. Infrastructure costs—salaries for trial coordinators, recruiters, and data managers; costs of maintaining institutional review boards; and costs of other administrative functions needed to run a trial—are the big expenses that make institutions reluctant to take part.

"The good thing about taking away this myth of noncoverage, and showing through empiric data that it really isn’t more costly to give treatment in trials, will be that people can start focusing on ways to pay these infrastructure costs," she said.

Goldman expects that the RAND study will not only provide better data on trial costs but also will help policy makers reconfigure the health care payment landscape.

"There’s the narrow question of, ‘Is there a problem right now?’ " he said. "But there’s also the broader question of, ‘Can we design better reimbursement mechanisms that would encourage people to participate in clinical trials?’ " One possibility, he said, might be a health care tax that would be used to reimburse insurers for additional trial costs in exchange for providing members with blanket coverage for approved trials.


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