Physician perspectives on compassionate use in pediatric oncology

Pediatr Blood Cancer. 2019 Mar;66(3):e27545. doi: 10.1002/pbc.27545. Epub 2018 Nov 8.


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.

Methods: 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.

Results: 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.

Conclusion: 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.

Keywords: compassionate use; expanded access; investigational drugs; pediatric oncology.

MeSH terms

  • Attitude of Health Personnel*
  • Child
  • Clinical Competence*
  • Drugs, Investigational / therapeutic use*
  • Health Knowledge, Attitudes, Practice*
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / psychology
  • Oncologists / ethics*
  • Oncologists / psychology
  • Practice Patterns, Physicians' / ethics*
  • Surveys and Questionnaires


  • Drugs, Investigational