Before It's Too Late: Multistakeholder Perspectives on Compassionate Access to Investigational Drugs for Pediatric Patients With Cancer.

Patients and their families, physicians, drug companies, and regulatory agencies have common goals: to find effective therapies for life-threatening conditions. In oncology, the lines between clinical research and treatment are often blurred; parents and physicians of patients who have exhausted standard-of-care treatments and cannot participate in a clinical trial are likely to consider seeking compassionate use access to investigational drugs; however, knowledge and perspectives about compassionate use may differ among these groups. There are unique considerations associated with providing compassionate use to children diagnosed with cancer, including evaluation for potential developmental toxicities, the need for pediatric-specific dosing and formulations, informed consent, and, when appropriate, patient assent.

Physician perspectives on compassionate use in pediatric oncology.

Background: Targeted cancer treatments are almost always first studied in adults, even when there is a biologically plausible potential for efficacy in children. Through compassionate use programs, children who are not eligible for a clinical trial and for whom there are no known effective therapies may obtain access to investigational agents, including drugs under development for adults. However, little is known about pediatric oncologists' experiences with applying for and obtaining compassionate use agents.