Review of Racial and Ethnic Representation of Participants Enrolled in Pediatric Clinical Trials of Oncology Drugs Conducted Through FDA Written Requests.

Importance: The Best Pharmaceuticals for Children Act states that in issuing a written request (WR), the US Food and Drug Administration (FDA) shall consider the adequate representation (eg, proportionate to the disease population) of children from racial and ethnic minority populations. If the terms of the WR are fulfilled, the FDA may grant an additional 6 months of exclusivity for any unexpired patents and exclusivities attached to approved indications. Objective: To report on the race and ethnicity of participants enrolled in pediatric studies conducted in response to WRs for which pediatric exclusivity was granted between 2001 and 2021. Design, Setting, and Participants: This retrospective review examines pediatric exclusivity request submissions for oncologic drugs that received pediatric exclusivity between December 2001 and January 2021 based on fulfillment of the requirements of a WR that were identified using the FDA's Document Archiving Reporting and Regulatory Tracking System. Demographic data were manually abstracted from supporting study reports, and data were pooled across submissions for the analysis. Data were analyzed throughout 2022 and 2023. Main Outcomes and Measures: Representation by race, sex, and ethnicity in pediatric studies conducted in response to WRs. Results: A total of 22 pediatric exclusivity requests were identified, comprising 40 studies and 2025 patients. Most trials (26 [65%]) in the analysis were cooperative group studies. Representation by race was as follows: American Indian/Alaska Native (13 [0.6%]), African American/Black (228 [11.3%]), Asian (92 [4.6%]), Native Hawaiian/other Pacific Islander (33 [1.6%]), White (1303 [64.3%]), other (194 [9.6%]), and unknown/not reported (162 [8.0%]). Representation by sex was female individuals (41.2%) and male individuals (58.8%). Ethnicity was as follows: Hispanic (226 [5.7%]), non-Hispanic (910 [22.5%]), unknown/not reported ethnicity (2800 [69.1%]), and other ethnicity (114 [2.8%]). Conclusions and Relevance: The study results suggest that overall, representation of participants of racial and ethnic minority groups in studies supporting pediatric exclusivity requests appear comparable with the racial distribution of childhood cancers in the US based on data from the National Childhood Cancer Registry Explorer. However, fewer Hispanic participants were enrolled in the trials we reviewed (8%) compared with the representation of Hispanic patients in the National Childhood Cancer Registry (28%). This discrepancy may be partially explained by the large proportion of participants with unknown information regarding ethnicity. Further research into the reasons for the large proportion of participants with missing ethnicity information is needed.

Equitable Access to Clinical Trials: How Do We Achieve It?

The mismatch between the study populations participating in oncology clinical trials and the composition of the targeted cancer population requires urgent amelioration. Regulatory requirements can mandate that trial sponsors enroll diverse study populations and ensure that regulatory revue prioritizes equity and inclusivity. A variety of projects directed at increasing accrual of underserved populations to oncology clinical trials emphasize best practices: broadened eligibility requirements for trials, simplification of trial procedures, community outreach through patient navigators, decentralization of clinical trial procedures and institution of telehealth, and funding to offset costs of travel and lodging. Substantial improvement will require major changes in culture in the educational and professional practice, research, and regulatory communities and will require major increases in public, corporate, and philanthropic funding.

Highlights of FDA Oncology Approvals in 2022: Tissue-Agnostic Indications, Dosage Optimization, and Diversity in Drug Development.

In 2022, cancer drug development continued to progress rapidly despite the lingering COVID-19 pandemic. Highlights of U.S. drug approvals for oncology indications this year include ongoing development in rare diseases and molecular subgroups, improved dosage optimization, and updated data for drugs granted accelerated approval, with confirmatory studies demonstrating verification of clinical benefit in some instances, as well as indication withdrawal when clinical benefit was not verified.

Oncology Expanded Access and FDA's Project Facilitate.

The Oncology Center of Excellence at the U.S. Food and Drug Administration launched Project Facilitate on May 31, 2019, to assist oncology health care providers with Expanded Access requests for investigational drugs. Expanded Access, sometimes called "compassionate use," is a regulatory pathway for physicians caring for patients who have a life-threatening condition or a serious disease to gain access to an investigational drug for treatment when no comparable or satisfactory alternative treatment options are available. Herein we describe the Project Facilitate program and the process for requesting Expanded Access to an investigational drug.