Wrong Way in the War on Cancer

By Merrill Goozner, Gooznews. Posted May 25, 2009.

“Most of off-label use is done with good intentions,” observes University of Chicago oncologist Richard Schilsky, the current president of the American Society of Clinical Oncology. But he adds that “no data is collected on whether it’s an effective strategy or not. And most of it gets reimbursed.”

Meanwhile, only 3 percent to 15 percent of adult cancer patients (it depends on whom you ask) are enrolled in clinical trials that would show if those regimens are truly effective or refines their best use (when treatment should be started or stopped, for instance, or in what doses, in combination with what other treatment approaches, and for which patient sub-groups). “The extensive off-label use of drugs allows patients access without having to participate in clinical trials,” Schilsky said. “At some point the treatment crosses over from evidence-based to completely not evidence-based. And when you cross that line, the cost of care runs up and patients become diverted from participating in clinical trials.”

Pediatric oncology serves as the model for cancer researchers who believe enrolling more patients in clinical trials would speed up the process of learning which therapeutic interventions work best for which patients. The NCI-funded Pediatric Oncology Group, which is now part of the non-profit Children’s Oncology Group, over the past five decades enrolled about 90 percent of all children with cancer in clinical trials. This enabled pediatric oncologists to steadily refine the treatment regimens to the point where survival rates today are over 80 percent, up from 20 percent in the 1960s.

“We have made stunning progress” in treating childhood cancers, says Sharon Murphy, a visiting fellow at the Institute of Medicine and former head of the Pediatric Oncology Group. “The trials built over time on previous learning [by] incorporating better scientific understanding of the science of the disease into treatment. It led to risk stratification,” she explains, which is the holy grail of personalized medicine that researchers often talk about in adult oncology but, alas, has proven elusive except in a few situations such as HER2-positive breast cancers.

Yet not all pediatric oncologists believe repeating that approach in adult oncology will lead to similar progress. Adult tumors can take decades to develop and are resistant to treatment. Pediatric cancers, in contrast, usually “arise from embryonic development and develop into cancers that are much more susceptible to chemotherapy and radiation,” explains Joseph V. Simone, director of the University of Florida’s Shands Cancer Center.

In any case, getting more adults patients enrolled in clinical trials won’t be easy. Many cancer patients confronting their life-threatening illness are understandably reluctant to take a 50-50 chance of getting put in the arm of a clinical trial that doesn’t receive the experimental therapy. And that’s only half the problem. Most patients are treated by community oncologists in private offices, not at academic medical centers that get NCI financial support to enroll patients in trials.

“It takes a lot of time and support staff to carry out a clinical trial,” notes John Mendelsohn, director of the M.D. Anderson Cancer Center at the University of Texas in Houston and chairman of the IOM committee evaluating NCI’s clinical trial system. “That requires reimbursement.”

Medicare will reimburse physicians for the cost of patients’ usual care if they are enrolled in a clinical trial. But usual care doesn’t include the cost of setting up a system to solicit informed consent, collect complete pre-trial information, conduct rigorous follow-up, and perform all the other tasks crucial to generating scientifically valid data. The result, a recent study showed, is that just 30 percent of all cancer clinical trials listed in the government’s registry (www.clinicaltrials.gov) recruit all the patients required by the trial’s original protocols.[5]

Given these constraints on rapidly expanding the clinical trial system, some experts say a better approach to rapidly improving cancer care would focus on collecting extensive information about the 1.3 million Americans who are diagnosed and treated for cancer every year. As the nation’s physicians move toward adopting electronic medical records (spurred on by the incentives in the Obama administration’s $767 billion economic stimulus plan), it should enable oncologists to record their patients’ demographic and genetic information (properly protected for privacy), their diagnoses, their treatments, and, eventually, their outcomes. This information could then be analyzed retrospectively to see what works and translated into guidelines for better care. Such a “learning system” would be a major step on the road toward personalized medicine, the proponents say, because the analysis could also identify which subgroups responded best to which treatments and regimens.

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Merrill Goozner spent more than 25 years in the news business as a foreign correspondent, economics writer and investigative reporter for the Chicago Tribune and other publications. He is the author of The $800 Million Pill: The Truth Behind the Cost of New Drugs and blogs at Gooznews.com.

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