Advances in medicine often begin with an insight in the laboratory or at the bedside, when physicians and scientists believe they have discovered a better way to treat a disease or solve a clinical problem.
However, without rigorous evaluation, innovations cannot be assumed to be either effective or safe. Indeed, the annals of medicine are filled with seemingly great ideas that proved ineffective or even harmful to patients when they were carefully evaluated. Today, we require proof — usually a clinical trial before recognizing a new drug, biologic therapy or device as a standard of treatment. Of course, it is also important to recognize that today’s standards of therapy will almost invariably be replaced tomorrow. The cycle of innovation to assessment and reassessment is one of the foundations of medical science.
Yet this cycle is facing a crisis, in part because too few patients are enrolled in clinical trials. The gap between the knowledge advancement we could have and the nearly stagnant reality is especially evident in cancer research. Clinical research for childhood malignancies (which account for less that 2 percent of all cancer) stands as the unparalleled exemplar, with the vast majority of children participating in clinical trials as part of their treatment. For children with cancer, clinical trials are accepted as the norm by physicians, patients and their parents. In fact, data on children’s clinical trials suggest that simply participating improves outcomes, even if the therapy being studied is not fully effective. This reflects the fact that clinical trials reduce the variability of treatment modifications, such as physicians’ more subjective changes in dose or regimen. In all, U.S. childhood cancer death rates declined by more than 50 percent from 1975 to 2006, averting 38,000 deaths.
But meanwhile, less than 5 percent of adults diagnosed with cancer enter a clinical trial, in part because they are largely treated by oncologists in private practice — unlike children, who are primarily treated at academic medical centers. The low rate of participation in clinical trials hurts the development and regular renewal of national standards of cancer treatment, the collection of biological samples from large populations of patients and the ability to comprehensively monitor outcomes.
Given the cost and complexity of modern cancer care, it is incumbent on physicians to assess the diagnosis and management of oncology patients objectively, rigorously, efficiently, constantly and successfully. Innovations need to be complemented with the assessment and reassessment of presumed standards of care. From my perspective, this is best done through clinical trials and protocols, modeling what has been done over the past decades in childhood cancer. That standard should be incorporated into adult cancer care, although this will require oncologists and patients to transform their attitudes and recognize clinical trials as essential to improving the outcome of cancer.
Philip A. Pizzo, MD
Stanford University School of Medicine
Carl and Elizabeth Naumann Professor, Pediatrics, Microbiology and Immunology