Our survey, Physician perspectives on compassionate use in pediatric oncology, was published on the same day as FDA Commissioner Scott Gottlieb announced the FDA’s commitment to improving its expanded access (compassionate use) program.
Gottlieb’s statement addressed the key findings of an FDA-sponsored independent Expanded Access Program Report that found that “confusion with program navigation, multi-stakeholder coordination, and administrative burden were the most frequently-cited challenges.”
While the overall impression of the program was positive among physicians, Gottlieb said “Stakeholders also reported some challenges across the physician and patient journey through the program, that, if properly addressed, could meaningfully enhance the program.”
Our study, which we conducted together with the Albert Einstein College of Medicine, for the first time inquired about the experiences of pediatric oncologists with compassionate use application process. Only 37% of pediatric oncologists we surveyed rated themselves as competent to navigate the application process. Those who had never completed an application incorrectly believed that pediatric patients had a lower likelihood of receiving FDA authorization for obtaining expanded access.
Over three quarters of oncologists in our study reported that their institutions do not provide support, resources, or training regarding the compassionate use application process, or that they are unsure of whether this support is available.
FDA plans to update the Expanded Access portion of the website, and to establish an agency-wide Patient Affairs Staff and Health Care Provider Affairs Program. “The Patient Affairs Staff is already in place and charged with serving as the “home base” and primary point of entry for patients and physicians starting the EA process and navigating them through the steps,” Gottlieb said.
In our study, as in the FDA report, the “pain points” for physicians varied significantly based on the level of experience with compassionate use. And our study, as well as the FDA’s assessment report, concludes that the program needs to be made easy for patients, physicians, and other stakeholders to understand and navigate. Read the study below:
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.
This study surveyed 132 pediatric oncologists to assess awareness and utilization of compassionate use programs, to identify barriers to their use, and to evaluate available institutional support and resources.
We found that the process of applying for access to drugs in development is poorly understood, which presents a barrier to obtaining investigational drugs. Fifty‐seven percent of the pediatric oncologists applied for compassionate use. Providers from larger institutions or with more than 15 years of clinical experience were more likely to complete an application and obtain investigational agents for their patients.
Identified perceived and actual barriers to compassionate use application submission suggest pediatric oncologists may benefit from educational resources and support to ensure children with cancer equal access to investigational agents and care.