Pediatric Oncologists' Experiences With Compassionate Use.

e18627 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 (CU) programs, children who are not eligible for a clinical trial and for whom there are no known effective therapies may obtain investigational agents. Patients can access CU drugs only if a physician initiates an application. However, there is a lack of data on pediatric oncologists’ knowledge of and experiences with applying for investigational drugs on a CU basis, and the factors affecting their ability to obtain CU drugs. Methods: An electronic questionnaire was used to survey 132 pediatric oncologists to assess their awareness and utilization of CU programs, to identify barriers to its use, and to evaluate available institutional support and resources for CU applications. Results: We found that the process of applying for access to drugs in development is poorly understood, presenting a potential barrier to obtaining investigational drugs. Forty eight percent of the pediatric oncologists surveyed completed an application for CU. Of those who did not complete applications, 18% were unfamiliar with CU programs and 29% could not identify a potentially effective drug. More experienced providers and those at larger centers were more likely to obtain the desired drug. Fifty eight percent of those who completed an application needed assistance, and 78% of all respondents’ institutions lack support resources for CU applications. Finally, 83% of respondents believe that a service facilitating the application process would be helpful. Conclusions: We identified the lack of knowledge and paucity of support in the application process as immediate actionable barriers to wider utilization of CU in pediatric oncology. Our study suggests a possible healthcare disparity based on the experience of the provider and size of institution, with seasoned oncologists and major academic centers being more likely to successfully access investigational agents. In order to provide pediatric oncology patients equal opportunity to access investigational agents, pediatric oncologists may benefit from educational resources and assistance to support them through the CU request process.