Landmark Cancer Clinical Trials and Real-World Patient Populations: Examining Race and Age Reporting.

Thejus Jayakrishnan,Rami Manochakian,Kianna Nguyen,Mizba Baksh,Ricardo Parrondo,Ayesha Samreen,Meghna Ailawadhi,Aneel Paulus,Asher Chanan-Khan,Taimur Sher,Vivek Roy,Sikander Ailawadhi,Sonikpreet Aulakh

doi:10.3390/cancers13225770

Thejus Jayakrishnan, Rami Manochakian + Show 11 more

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https://doi.org/10.3390/cancers13225770

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Abstract

Simple SummaryFood and Drug Administration (FDA) drug approvals from July 2007 to June 2019 were reviewed to identify oncology approvals, and trials with age details were reviewed for the study. We hypothesized that the clinical trials that do not report race are likely to suffer from a higher degree of age disparity. The study demonstrated that a significant number of clinical trials leading to cancer drug approvals suffer from racial and age disparity when compared to real-world populations and that the two factors may be interrelated. Age discrepancy between the clinical trial population and the real-world population was higher for studies that did not report race (mean difference −8.8 years (95% CI −12.6 to −5.0 years)) vs. studies that did report it. We recommend continued efforts to recruit diverse populations in clinical trials and make concerted efforts to implement national strategies in order to realize healthcare equity. In the meantime, detailed reporting of patient demographic characteristics in publications should be considered standard.Background: Concern exists that the clinical trial populations differ from respective cancer populations in terms of their age distribution affecting the generalizability of the results, especially in underrepresented minorities. We hypothesized that the clinical trials that do not report race are likely to suffer from a higher degree of age disparity. Methods: Food and Drug Administration (FDA) drug approvals from July 2007 to June 2019 were reviewed to identify oncology approvals, and trials with age details were selected. The outcomes studied were the weighted mean difference in age between the clinical trial population and real-world population for various cancers, the prevalence of race reporting and association of age and race reporting with each other. Results: Of the 261 trials, race was reported in 223 (85.4%) of the trials, while 38 trials (14.6%) had no mention of race. Race reporting improved minimally over time: 29 (85.3%) in 2007–2010 vs. 49 (80.3%) in 2011–2014 vs. 145 (85.4%) during the period 2015–2019 (p-value = 0.41). Age discrepancy between the clinical trial population and the real-world population was higher for studies that did not report race (mean difference −8.8 years (95% CI −12.6 to −5.0 years)) vs. studies that did report it (mean difference −5.1 years, (95% CI −6.4 to −3.7 years), p-value = 0.04). Conclusion: The study demonstrates that a significant number of clinical trials leading to cancer drug approvals suffer from racial and age disparity when compared to real-world populations, and that the two factors may be interrelated. We recommend continued efforts to recruit diverse populations.

Highlights

The unfortunate reality of the lack of representation of racial and ethnic minorities in clinical trials is a major barrier to the achievement of health equity [1–5]
We reviewed Food and Drug Administration (FDA) drug approvals from July 2007 through June 2019 and identified drug approvals pertaining to malignant hematology and oncology
Clinical the cutting cuttingedge edgeofofmedicine medicineleading leading novel therapies. They lead to the improvement in overall survival or disease-free survival, the context of cancer, they lead to the improvement in overall survival or disease-free surideally at an acceptable qualityquality of life of and cost tocost the patient and society

Summary

Introduction

The unfortunate reality of the lack of representation of racial and ethnic minorities in clinical trials is a major barrier to the achievement of health equity [1–5] While this is of importance to all aspects of medicine, the relevance in the context of cancer stems from the fact that the cancer treatment is life-altering, and differences based on the race and ethnic characteristics of the patient have implications on survival and quality of life. Certain cancer diagnoses are seen more frequently in some racial/ethnic subgroups, making this under-representation even more challenging [6] Another factor that represents an important baseline characteristic of the trial population, with implications on the applicability of the trial results in the real-world setting, is patient age [2]. Race reporting improved minimally over time: 29 (85.3%) in 2007–2010 vs. 49 (80.3%) in 2011–2014 vs. 145 (85.4%) during the period

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