Throughout history, decision-makers have relied on clinical trials to make evidence-based improvements in the delivery of health care. From the first recorded study of whether beans or meat better prepared warriors in biblical times, to the discovery that lemons treated scurvy among ailing British sailors better than vinegar or cider, carefully controlled scientific studies have inspired medical advances for centuries. They are just as influential today, especially in the evolving world of cancer treatment.
What does a clinical trial do?
A clinical trial may seek answers to any number of questions: whether a drug works, how it compares to other treatments, or whether it causes side effects, for example. Without such data, winning government approval for next-generation drugs and other treatment advances would be impossible. Moving a new drug from idea to pharmacy shelf is an often-lengthy process, one that frequently depends on how long it takes to enroll enough patient-volunteers. It typically takes eight to 12 years for a drug candidate to make the journey from lab to clinic. At a time when some standout new cancer treatments are showing promise over traditional therapies, that may seem like a long time to wait.
Clinical trial volunteers accept certain risks when participating—that the drug may not work for them, or that the treatment may lead to harmful side effects or other complications. In return, they gain early access to potential new therapies—under the close scrutiny of a panel of experts and may get a pay-it-forward satisfaction from helping to advance scientific knowledge. Multiple layers of review help protect the patient’s safety and interests throughout the process, says Dr. Ricardo Alvarez, Director of Cancer Research & Breast Medical Oncologist at Cancer Treatment Centers of America® (CTCA).
But clinical trials are not for everyone. Some patients are not good candidates; others choose not to enroll. Overcoming both hurdles remains a critical challenge for 21st century researchers. “The ultimate goal is to learn,” Dr. Alvarez says. “I think the only way we are going to make a change in the natural history of the disease is to make clinical trials a first-line recommendation.”
A drawback: Low enrollment
Unfortunately, only 3 or 4 percent of adult cancer patients in the United States enroll in clinical trials. The low participation rate often carries consequences—to the trial and the body of research—further delaying drug approval and presenting challenges to the trial’s success, says Dr. Glen Weiss, Director of Clinical Research at CTCA Phoenix. “Clinical trials are critical to all aspects of medicine, but especially cancer care, because the stakes are so high,” Dr. Weiss says.
Dozens of targeted anti-cancer drugs are in the research pipeline. The challenge for oncologists is deciphering the complexity the genomic era has brought. The light illuminating that path is clinical trial data. “I have been in oncology 20 years, and I can tell you, in the past three years, there has been a revolution in how we think about clinical trials and how we treat patients,” Dr. Alvarez says. “We are selecting more of these patients based on their tumor’s molecular profile, the genomic analysis, the proteomics. This is called the personalization of medicine.”
