Project Title: Economic Impact of Clinical Trials Among Children Diagnosed with Cancer 

Principal Investigator: Marilyn Schapira

Co-Investigators: Lamia Barakat, Jeffrey Silber, Charles Bailey

Project Dates: 2015-2017

More than 60% of children with cancer under age 15 receive treatment through a therapeutic clinical trial compared to about 5% of those who are older than 20 years. Understanding the costs of treatment associated with clinical trial participation is critical in determining if the effects of treatment justify the costs from a societal perspective, including costs borne by the patient and family. This study compared the economic impact and health-related quality of life outcomes among children with cancer who were treated on Phase III clinical trials to those who receive non-trial standard treatment. Outcomes evaluated include survival, direct and indirect costs, and health-related quality of life. Findings will inform decision-making for children with cancer and their families who are considering taking part in therapeutic clinical trials.


Schapira MM, Barakat LP, Silber JH. Reply to Assessing clinical trial effects on outcomes among pediatric and adolescent and young adult (AYA) patients with cancer. Cancer 2021 Feb 15;127(4):649-650. doi: 10.1002/cncr.33251. epub Oct 29, 2020.


Schapira MM, Stevens EM, Sharpe JE, Hochman L, Reiter JG, Calhoun SR, Shah SA, Bailey LC, Bagatell R, Silber JH, Tai E, Barakat LP. Outcomes among pediatric patients with cancer who are treated on trial versus off trial: A matched cohort study. Cancer. 2020 Aug 1;126(15):3471-3482. doi: 10.1002/cncr.32947. Epub 2020 May 26. PMID: 32453441.