Clinical Trial Design and Drug Approval in Oncology: A Primer for the Advanced Practitioner in Oncology

Evidenced-based practice requires timely and accurate integration of scientific advances. This presents a challenge for the oncology clinician given the robust pace of scientific discovery and the increasing number of new drug approvals and expanded indications for previously approved drugs. All currently available antineoplastic therapies have been developed through the clinical trials process. Advanced practitioners (APs) in oncology are often involved in the conduct of clinical trials as primary investigators, sub-investigators, study coordinators, or in the delivery and monitoring of care to patients enrolled in these trials. A prerequisite to evidenced-based practice is understanding how clinical trials are conducted and how to critically analyze published results of studies leading to U.S. Food & Drug Administration approval. Any AP involved in the clinical management and supportive care of patients receiving antineoplastic therapies should be able to critically review published data to glean findings that warrant a change in practice. The goals of this manuscript are to summarize key elements of the clinical trial process for oncology drug development and approval in the United States and to provide a primer for the interpretation of clinical data.