title: Access to Children's Oncology Group and Pediatric Brain Tumor Consortium phase 1 clinical trials: Racial/ethnic dissimilarities in participation.
abstract: BACKGROUND
  Phase 1 clinical trials introduce new therapies to humans with the goal of establishing their safety. A prior Children's Oncology Group (COG) study analyzed the proportional enrollment of patients by race, ethnicity, sex, and age for all trial phases. The current study evaluated the representation of patients by race, ethnicity, sex, and age in phase 1 clinical trials.
  
  
  METHODS
  This study evaluated 1348 children with 128 diagnoses enrolled in COG and Pediatric Brain Tumor Consortium phase 1 clinical trials in the United States from February 28, 2000 to December 29, 2008. Observed and expected proportions were calculated according to an established methodology with a representative population from Surveillance, Epidemiology, and End Results data, which included 27,766 children with the same International Classification of Diseases for Oncology (third edition) diagnostic codes.
  
  
  RESULTS
  Underrepresentation in phase 1 trials was seen for lymphohematopoietic (LH) tumors (9.3% observed vs 37% expected) versus solid tumors (90.6% observed vs 63% expected). Although representation was fairly proportional, Hispanics (12.6% observed vs 27% expected), particularly Hispanic females (6% observed vs 18% expected), were significantly underrepresented. The 0- to 4-year age group was underrepresented (11.7% observed vs 36.5% expected). By tumor type, the most significantly underrepresented groups were 0- to 4-year-old children and Hispanics for both solid cancers (11% observed vs 34.4% expected for 0- to 4-year-old children and 12% observed vs 24% expected for Hispanics) and LH cancers (16% observed vs 40% expected for 0- to 4-year-old children and 19.4% observed vs 33% expected for Hispanics).
  
  
  CONCLUSIONS
  Although sex and racial/ethnic groups are mostly proportionally represented in phase 1 trials, some specific subgroups such as Hispanic children are underrepresented and may benefit from focused accrual. Cancer 2016;122:3207-14. © 2016 American Cancer Society.
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia.
authors.forename: Ajay K
authors.initials: AK
authors.lastname: Nooka
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia.
authors.forename: Madhusmita
authors.initials: M
authors.lastname: Behera
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia.
authors.forename: Sagar
authors.initials: S
authors.lastname: Lonial
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia.
authors.forename: Margie D
authors.initials: MD
authors.lastname: Dixon
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia.
authors.forename: Suresh S
authors.initials: SS
authors.lastname: Ramalingam
authors.affiliation: Winship Cancer Institute, Atlanta, Georgia. rpentz@emory.edu.
authors.forename: Rebecca D
authors.initials: RD
authors.lastname: Pentz
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000379:methods*; D005006:Ethnicity / Q000706:statistics & numerical data*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010358:Patient Participation; D018579:Patient Selection; D011379:Prognosis; D044469:Racial Groups; D018426:SEER Program; D012737:Sex Factors; D014481:United States; D055815:Young Adult
keywords: Children's Oncology Group (COG); Pediatric Brain Tumor Consortium (PBTC); disparities; ethics; phase 1 trials