Clinical trial enrollment, and why it matters to all of us.
Every time a doctor has to make a decision, a risk/benefit analysis must be done to ensure that the course of care offers the greatest positive outcome for the patient. The basic tenet of evidence-based medicine is that this analysis be carried out with a body of high-quality information, ideally derived from randomized clinical trials and for which there is consensus agreement. This is especially true in the case of cancer, where intuitive notions are often contradicted by actual clinical trial data, and the trend toward personalized medicine recognizes that there is a clear variation in the behavior and treatment response among individual cancers.
The advancement of tailored and evidence-based medicine is directly related to the number of informative clinical trials and subjects enrolled in these studies. Despite increased attention and funding for trials, the proportion of patients with cancer enrolled remains dismally low.
A recent Institute of Medicine report concluded that the clinical trial machinery is broken, with an increasing amount of bureaucracy, inefficiencies, mismanagement, disincentives, and logistical hurdles resulting in low accrual, high costs, and studies that never reach completion. This report primarily addressed the system of cooperative groups that are funded by the National Cancer Institute. Along with research by pharmaceutical companies usually focused on getting drugs approved or expanding market share, these groups represent the main source of data available for making decisions and adopting new diagnostic and therapeutic approaches.
This issue’s cover story, “The Treatment Option You May Be Missing” by Laura Beil, presents the many dimensions of clinical research—the challenges and opportunities. There are many explanations for the state of affairs in clinical research. The pendulum of regulatory rules seems to swing in response to every crisis, ranging from safety issues to investigator misconduct or concerns about patient privacy.
The medical field and research within it still remain fragmented and seemingly in a “Tower of Babel” mode, with a lack of communication and large-scale analytic capacity because few data standards exist. Rising health care costs and diminishing reimbursements have cut staffing and limited the flexibility needed to take on research. Even noble-hearted investigators fall prey to petty scientific snobbery, insular behavior, competing priorities, and conflicts of interest (not necessarily financial in all cases).
There is no doubt that a balance must be struck between protecting patient safety and advancing innovative ideas. There is also a conflict between the need for oversight and for streamlining the efficiency of developing and running a study.
However, the news is not all bad. With health care reform, there may be an opportunity to revolutionize clinical research through the use of medical information technology and broadening the number of clinical centers where research takes place. This will require clinical trials to be simplified so that they can be integrated into routine care. For example, every time a medical record is updated with a new diagnosis, the information system might trigger a notification that the patient be informed of a specific trial and automatically set up the steps needed for further screening and, if applicable, enrollment, treatment, and follow-up through the study. The data entry would be through the established electronic medical record system, and much of the monitoring and oversight could be conducted remotely by a clinical research organization.
The revolution in biological discovery is fueling interesting new diagnostic assays and drugs, but our limited ability to test them threatens their prioritization and integration into routine care. The collective response of the research community, the government, and the public will play out over the next few years, and we will continue to bring our readers the facts and perspectives on this process.
Debu Tripathy, MD
Professor of Medicine, University of Southern California
Co-leader, Women’s Cancer Program at the USC/Norris Comprehensive Cancer Center