ASCO has called for greater access to and education about phase I clinical trials, the first-in-human studies of new agents designed to fight cancer.
In a policy statement released Dec. 15, the Society stresses the critical importance of phase I clinical trials in cancer research and treatment, emphasizing that this research offers greater potential as a treatment option for many patients than was the case in the past, due to development of molecularly targeted agents, biomarker tests to identify patients likely to respond to treatments and innovative clinical trial designs.
“The Critical Role of Phase I Trials in Cancer Research and Treatment” ASCO policy statement, which updates a 1997 version, was published online in the Journal of Clinical Oncology.
“With new agents that target specific abnormalities in a patient’s tumor, better tests to identify those abnormalities and more sophisticated clinical trial designs, today’s phase I trials in cancer offer patients a greater likelihood of benefit than ever before,” said ASCO President Peter Paul Yu, MD, FACP, FASCO. “Patients shouldn’t shy away from phase I trials and doctors should present these trials as options for eligible patients throughout the period of active cancer treatment, not only when all other treatment options have failed.”
The ASCO policy statement reviews evidence that shows patients who participate in phase I trials may experience improved quality of life, psychological advantages, and direct medical benefits. Yet, according to the Society, significant barriers discourage patient enrollment in research.
One of the longstanding barriers—insurance coverage—has been addressed. The Patient Protection and Affordable Care Act (ACA) of 2010, for example, requires many private payers to cover routine patient costs in clinical trials; however, the coverage rules for Medicare are unclear and vary across the U.S. Furthermore, Medicaid and health insurance plans that are grandfathered under the ACA are not required to provide coverage.
“The uneven laws and regulations create disparities in patient access to phase I trials and deny many patients the opportunity to try a novel therapy and contribute to knowledge about their disease,” said the statement’s lead author, Jeffrey S. Weber, MD, PhD, a member of the ASCO Cancer Research Committee. “CMS should address these disparities and fix these gaps.”
Phase I clinical trials are an important step in translating basic research into clinical practice. Researchers use these studies to determine the recommended dose and schedule of an investigational agent, observe its therapeutic effect, and assess its safety profile. Additional phase I studies, called phase Ib, may also be used to evaluate new schedules of existing agents or combinations of new agents with established agents or radiation.
In addition to specifically focusing on the special issues related to conducting phase I trials in children, the ASCO policy statement recommends concrete steps be taken to creating a healthcare delivery and payment system and expanding educational efforts that support decisions by patients to participate in these trials.
1. (a) The Centers for Medicare & Medicaid Services should recognize that phase I cancer clinical trials meet the therapeutic intent requirement of the Medicare National Coverage Determination for routine patient care costs in clinical trials. (b) State Medicaid programs should reimburse for routine patient costs associated with clinical trials, including phase I trials.
2. (a) Professional societies’ educational efforts should target improving clinicians’ and researchers’ abilities to discuss the purposes and risk-benefit assessment of patients’ participation in phase I trials in cancer. (b) Professional societies and patient advocacy organizations should develop enhanced educational materials for patients to explain the goals of phase I trials in cancer.
3. Professional societies should enhance educational materials for clinicians and researchers to help overcome challenges to phase I trial enrollment, such as incomplete understanding of insurance coverage and attitudes that phase I trials should be considered only after other treatment options fail.
4. (a) Researchers and trial sponsors should include language on therapeutic intent in phase I trial protocols. (b) Researchers and trial sponsors should utilize phase I trial designs that minimize the number of patients exposed to potentially subtherapeutic doses of the agent being tested.
5. The biopharmaceutical industry should initiate pediatric phase I trials for agents directly relevant to childhood cancer(s) during the late phase I or early phase II adult drug development program timeframe.