Related Experiment Video
Updated: Aug 6, 2025
A Novel Method for Involving Women of Color at High Risk for Preterm Birth in Research Priority Setting
Published on: January 12, 2018
Metrics, baseline scores, and a tool to improve sponsor performance on clinical trial diversity: retrospective cross
Tanvee Varma1, Michelle Mello2,3,4, Joseph S Ross5,6,7
1Yale School of Medicine, New Haven, CT, USA.
Objective:
To develop a measure for fair inclusion in pivotal trials by assessing transparency and representation of enrolled women, older adults (aged 65 years and older), and racially and ethnically minoritized patients.
Design:
Retrospective cross sectional study.
Population:
Sponsors of novel oncology therapeutics that were approved by the US Food and Drug Administration over 1 January 2012 to 31 December 2017.
Data Sources:
Trial data from Drugs@FDA, ClinicalTrials.gov, and corresponding publications; cancer incidence demographics from US Cancer Statistics and the American Cancer Society.
Main Outcome Measures:
Transparency measures assess whether trials publicly report participant sex, age, and racial and ethnic identity. Representation measures assess whether trial participant demographics represent more than 80% of the US patient population for studied conditions, calculated by dividing the percentage of study participants in each demographic subgroup by the percentage of the US cancer population with the studied condition per group. Composite fair inclusion measures assess average transparency and representation scores, overall and for each demographic group. Results are reported at the trial, product, and sponsor levels.
Results:
Between 1 January 2012 and 31 December 2017, the FDA approved 59 novel cancer therapeutics, submitted by 25 sponsors (all industry companies) on the basis of 64 pivotal trials. All 25 sponsors (100%) reported participant sex, 10 (40%) reported age, and six (24%) reported race and ethnicity. Although 14 (56%) sponsors had adequate representation of women in trials, only six (24%) adequately represented older adults, and four (16%) adequately represented racially and ethnically minoritized patients (black, Asian, Hispanic or Latinx). On overall fair inclusion, one sponsor scored 100% and the median sponsor score was 81% (interquartile range 75-87%). More than half of sponsors (13 (56%) of 25) fairly included women, 20% (n=5) fairly included older adults, and 4% (n=1) fairly included racially and ethnically minoritized patients in trials. 80% of product had pivotal trials that fairly included women, 24% fairly included older adults, and 5% fairly included racially and ethnically minoritized patients.
Conclusions:
This novel approach evaluates trials, products, and sponsors on their fair inclusion of demographic groups in research. For oncology trials, substantial room was noted for improved inclusion of older adults and patients who identify as black or Latinx and transparency around the number of participants identifying as Native Hawaiian, Pacific Islander, American Indian, and Alaska Native. These measures can be used by sponsors, ethics committees, among others, to set and evaluate trial diversity goals to help spur progress toward greater research equity in the US.
Insights
Fair inclusion in cancer trials remains a challenge, with significant underrepresentation of older adults and racial/ethnic minorities. Improved transparency and representation measures are crucial for advancing research equity.
Area of Science:
- Oncology Clinical Trials
- Health Equity Research
- Regulatory Science
Background:
- Pivotal oncology trials often lack diverse representation, potentially limiting treatment generalizability.
- Assessing fair inclusion requires evaluating both transparency in reporting and demographic representation against patient populations.
Purpose of the Study:
- To develop and apply a novel measure for assessing fair inclusion in pivotal oncology trials.
- To evaluate the transparency and representation of women, older adults, and racial/ethnic minorities in US FDA-approved cancer drug trials.
Main Methods:
- Retrospective cross-sectional study of 64 pivotal trials for novel oncology therapeutics approved between 2012-2017.
- Data sources included Drugs@FDA, ClinicalTrials.gov, US Cancer Statistics, and American Cancer Society.
- Fair inclusion was measured by trial transparency (reporting demographics) and representation (comparing trial demographics to US cancer patient demographics).
Main Results:
- While all sponsors reported participant sex, only 40% reported age and 24% reported race/ethnicity.
- Adequate representation was achieved for women in 56% of trials, older adults in 24%, and racial/ethnic minorities in 16%.
- Overall fair inclusion scores varied, with only one sponsor achieving 100%; 56% fairly included women, 20% older adults, and 4% racial/ethnic minorities.
Conclusions:
- Substantial improvements are needed in the inclusion and reporting of older adults and racial/ethnic minorities in oncology trials.
- The developed fair inclusion measures can guide sponsors and ethics committees in setting and evaluating diversity goals.
- Enhancing research equity requires a concerted effort to ensure clinical trial populations reflect the diversity of patients with cancer.
More Related Videos
04:53A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
Published on: September 20, 2019
06:55Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index
Published on: January 8, 2020
Related Concept Videos
01:11Clinical Trials: Overview
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
01:21Factors Affecting Drug Response: Overview
01:22Diversity in Cell Signaling Responses
Graded and Abrupt Responses
Some signaling systems generate...
01:20Sputum Studies II: Culture and Sensitivity
Sputum culture and sensitivity is a medical procedure used to diagnose bacterial infections in the respiratory tract and select the most appropriate antibiotics for treatment. This process involves analyzing sputum samples of thick and opaque secretions produced in the lungs and airways. These samples are collected from patients and then sent to the laboratory for analysis.
The test can identify various pathogens responsible for respiratory infections, including Streptococcus,...