Medical Perspective: More Clinical Trials, Organization, Clinical Databases

John L. Marshall, an oncologist specializing in gastrointestinal tract cancers, reports in a Washington Post article “Fighting a Smarter War on Cancer” (2009) that “despite the many recent advances in detection and treatment, of [every] 50 patients, 40 of them are likely to lose their fight” (para. 2) with cancer. Marshall is concerned with the “enormous cost of cancer care” (para. 3) and how exactly it is helping treatment. He writes, “At the moment, there is a giant disconnect between patient, the cost of care and the clinical benefit of the treatment--a disconnect that has caused us to lose perspective” (para. 4).

Marshall suggests that research in cancer treatment has only made minor progress since President Nixon signed into law the National Cancer Act. Advancements in chemotherapy, particularly less invasive avenues or administering treatment, have succeeded “with several types of less-common cancers” (para. 7), but they have not worked for more commonly diagnosed cancers, which “represent an enormous public health problem, consuming the majority of our cancer-specific health-care costs and research dollars” (para. 7).

While clinical trials, in a majority of cases, represent the best possible chance of survival for some patients, Marshall suggests that “[f]ew cancer clinical trials are designed to [actually] ‘cure’ patients” (para. 8). Clinical trials, he states, “typically cost millions of dollars (often including taxpayer support), take years to complete and can involve thousands of patients” (para. 8) and normally only extend the average patient survival rate for one month. Marshall suggests that Americans are afraid that clinical trials for cancer treatment under a healthcare reform plan will be harder to fund and less available to patients—hindering innovation in the fight to cure cancer.

If the Obama administration were to call on oncologists to help choose which cancer treatments would be included in a public option plan and which would fail to meet some 'value' standard, we would have no guidelines to follow. The Food and Drug Administration uses safety and efficacy as standards for drug approval, but neither of those considers the magnitude of benefit or cost. I frequently ask my students and peers if there is a cancer drug today that they would pay for out of pocket if they had to. We all have patients who lack insurance but have some financial means, making this an exercise that is played out regularly in our clinics.

After a long pause, someone invariably will say ‘Gleevec,’ a pill that treats a terrible disease called chronic myeloid leukemia. Gleevec, a brand name for imatinib, costs up to several thousand dollars a month, but it makes this leukemia 'go away' for a long time with very few side effects -- a true magic bullet. Very few cancer drugs can be described as having this kind of value. "Thank God for my insurance" is a common line from patients. (para. 9-10)

Marshall explains that fewer than five percent of cancer patients in the U.S. participate in clinical trials. That means that “more than 95 percent are treated with the "standard of care" -- a legal term denoting a minimum level of care for an ill or injured person” (para. 12). Under “standard of care” options insurance companies will pay for a set amount of treatments and drugs. But if innovations are going to be made in the fight against cancer, data needs to be collected (and made available to physicians) about how patient’s react to treatments. “Standard of care” programs generally limit the the type and overall value of data that might be collected. If more people were allowed (by insurance programs) to enter into clinical trials, then representative data can be collected and analyzed to reveal a larger picture that is simply impossible with the restrictions of the standard programs of care. Marshalls states that the profession’s “understanding of cancer therapies comes from the 5 percent of patients who enroll in trials, a tiny database when we consider the highly variable nature of the disease. In fact, most of our evidence has not come from clinical trials performed in the United States but from nations where patients have little access to advanced care except through participation in such trials” (para. 14).

Marshall calls for a dedicated exploration and dissemination of data (a comprehensive clinical database”) “on the genetic makeup of patients and their cancers. We can no longer diagnose cancers using only a microscope. We must profile them at a molecular level to determine precise treatments, instead of using our current trial-and-error approach” (para. 16). Marshall believes that healthcare reform will jump start a system that is not nearly as effective as it should be (Marshall, 2009).