When Oncologic Treatment Options Outpace the Existing Evidence: Contributing Factors and a Path Forward

Oncology is one of the most multidisciplinary areas of medicine, with most patients encountering multiple treatment modalities during the course of their disease. Rapidly occurring innovations in cancer care are continuously expanding the number of treatment options available. However, substantial variation in the amount and quality of evidence supporting new drugs, devices, and surgical approaches exists, compromising evidence-based treatment decisions. To address this important issue, the professional societies representing cancer care providers appointed a multidisciplinary working group: American College of Surgeons Commission on Cancer, American Society for Radiation Oncology (ASTRO), American Society of Clinical Oncology (ASCO), Society of Interventional Radiology (SIR), and the Society of Surgical Oncology. In addition, the working group included a patient and National Cancer Institute (NCI) representative. This manuscript identifies five factors contributing to differences in evidence development for cancer treatment modalities: (1) research funding, (2) methodological challenges to conducting randomized controlled trials in many therapeutic options, (3) regulatory agency oversight, (4) payment policies, and (5) hierarchy and sociological factors in medicine. It makes a series of consensus recommendations that address the need for more cross-disciplinary research and wider adoption of observational research, pragmatic trials, and reimbursement strategies.