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GAO-23-105947 1 (2023-01-31)

handle is hein.gao/gaooex0001 and id is 1 raw text is: U.S. GOVERNMENT ACCOUNTABILITY OFFICE 441 G St. N.W. Washington, DC 20548 January 31, 2023 Chair Ranking Member Committee on Health, Education, Labor, and Pensions United States Senate The Honorable Cathy McMorris Rodgers Chair The Honorable Frank Pallone, Jr. Ranking Member Committee on Energy and Commerce House of Representatives Pediatric Cancer Studies: Early Results of the Research to Accelerate Cures and Equity for Children Act Pediatric cancer is the leading cause of death by disease past infancy among children from birth to age 14 in the United States, according to the National Cancer Institute. The National Cancer Institute estimated that in 2022 alone, 10,470 new cases of cancer would be diagnosed in children-of those, around 10 percent are expected to ultimately die from the disease.1 While survival rates for pediatric cancers more broadly have improved over the last few decades, survival rates for some pediatric cancers remain low. For example, the National Cancer Institute reports that only 10 percent of children with diffuse intrinsic pontine glioma, a rare brain tumor, survive 2 years after being diagnosed. There have been relatively few drugs approved to treat pediatric cancer. As of December 2022, there were 54 drugs approved to treat pediatric cancer.2 As a result, clinicians treating pediatric patients have limited options. Clinicians may opt to use a cancer drug approved for adults; however, in these instances, there may be limited available information specific to the safety and effectiveness of the drug in the pediatric population, according to Food and Drug Administration (FDA) officials.3 1National Cancer Institute, Childhood Cancers (Aug. 19, 2022), accessed Nov. 2, 2022, https://www.cancer.gov/types/childhood-cancers. 2Food and Drug Administration, Pediatric Oncology Drug Approvals (Dec. 2022), accessed Jan. 18, 2023, https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology-drug-approvals. 3Unapproved use of an FDA-approved drug is often called off-label use. Food and Drug Administration, Understanding Unapproved Use of Approved Drugs 'Off Label', (Feb. 5, 2018), accessed Dec. 6, 2022, https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-u napproved- use-approved-drugs-label. FDA officials noted that much of the pediatric cancer standard of care management includes use of cancer drugs not specifically approved for use in children. GAO-23-105947 Pediatric Cancer Studies Page 1

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