Abstract LB-146: Compassionate use navigator addresses challenges and facilitates access to investigational drugs for pediatric oncologists

Abstract Background: Targeted cancer treatments are almost always studied first in adults, and even when there is a biologically plausible potential for efficacy in children, studies very rarely include pediatric patients. Through compassionate use (expanded access) programs, children who are not eligible for clinical trials and for whom there are no satisfactory therapies available may have access to experimental therapies, including drugs under development for adults. Even when potential benefits of a promising experimental treatment may outweigh the potential risks of a relatively untested drug, the process to obtain the drug is complex. A physician must obtain a patient's consent, agreement from a pharmaceutical company to provide an investigational drug, and approvals from the FDA and an IRB. These time-consuming and poorly understood steps often present barriers to obtaining access to investigational drugs. Some physicians and patients do not know that applying for investigational drugs is an option. Objectives: 1) To design a web portal to assist families and physicians with submission of compassionate use applications for promising, investigational drugs for children with cancer, and 2) To study pediatric oncologists’ awareness and utilization of the compassionate use program, and identify barriers to its use. Methods: The Compassionate Use Navigator is a new program of Kids v Cancer (www.kidsvcancer.org/compassionate-use) that provides: a) An option for physicians to request help from Compassionate Use Navigator staff in completing a compassionate use application; b) Information on rules and procedures for obtaining expanded access for physicians and patients, including FDA and pharmaceutical company contacts; c) Instructions on how to apply for compassionate use; and d) Develops a registry of outcomes of compassionate use applications for children. A questionnaire has been developed to assess pediatric oncologists’ experiences with and barriers affecting use of compassionate use programs to be distributed before and after go-live of the Navigator in February 2016. Results: The Compassionate Use Navigator was created as a web-based tool and concierge service for pediatric oncologists and families who want to gain access to investigational drugs outside of clinical trials when no satisfactory FDA-approved treatment options are available. Conclusion: The complex process to obtain compassionate use of an investigational drug may be a barrier to utilization of the program, particularly in childhood cancer. Studies are needed to explore the effects of the Compassionate Use Navigator on the number of applications over time, and evaluate pediatric oncologists’ perceptions about the compassionate use program before and after implementation of the Compassionate Use Navigator. Citation Format: Elena Gerasimov, Lindy Zhang, Scott Moerdler, Michael Roth, Nancy Goodman, Daniel Weiser. Compassionate use navigator addresses challenges and facilitates access to investigational drugs for pediatric oncologists. [abstract]. In: Proceedings of the 107th Annual Meeting of the American Association for Cancer Research; 2016 Apr 16-20; New Orleans, LA. Philadelphia (PA): AACR; Cancer Res 2016;76(14 Suppl):Abstract nr LB-146.