Improving equity in cancer care: How RWE is transforming regulatory decisions for underrepresented populations

Regulatory evaluation of cancer medications has traditionally relied on randomized clinical trials designed with specified patient populations demonstrating a clear unmet need and aligned to the targeted mechanism of action to demonstrate a favorable benefit-risk profile. However, certain patient populations – racial and ethnic groups, pediatric or elderly populations, and those with comorbidities – have been historically underrepresented in these studies, resulting in a limited understanding of how medicines work across diverse groups of eligible patients. For example, a review of clinical trials used for US Food and Drug Administration (FDA) approvals of new molecules and biologics in 2020 found that trial populations included 8% Black and 11% Hispanic individuals, while these individuals represent 13% and 16%, respectively, of the overall US population.¹

Recognizing that treatment response can vary by patient characteristics, including race, age, and ethnicity, FDA and other global health authorities have emphasized the importance of inclusivity in clinical research to gain a more comprehensive understanding of the safety and effectiveness of new therapies across all relevant patient populations. Real-world evidence (RWE) has emerged as a critical tool for addressing persistent gaps in representative patient data by helping to inform recruitment strategies for more representative trial populations and to improve understanding of how innovative treatments perform in underrepresented populations in the post-market setting.

This is particularly vital in cancer research, where disease heterogeneity, the rapid pace of treatment innovation, and the focus on long-term outcomes amplify the demand for robust RWE. In response, important advancements in real-world data (RWD) curation and RWE methodology are making it increasingly feasible to evaluate the safety and effectiveness of cancer medications in historically underrepresented populations, helping enable more informed regulatory decisions and clinical practice and improved patient care.

Recent RWE advancements to support regulatory decisions in less-studied populations

Regulators have become more explicit in their expectations for representative trial populations, opening the door for significant improvements in the availability of more data across diverse demographic and phenotypic subgroups to inform regulatory and clinical decisions.

Oncology-specific RWD sources are increasing steadily due to collaborations with oncology healthcare providers, electronic healthcare record systems, and public-private partnerships. As these new data sources emerge, ensuring the data are fit-for-use and meet quality standards to generate scientifically robust and reliable RWE is critical. For example, in studying underrepresented patient populations in RWD, it is important to consider geographic representation, quality and completeness in the capture of race, ethnicity, age, and sex, and the availability of other genetic, environmental, and sociocultural aspects that are associated with prognosis and/or access to quality healthcare.

In 2022, FDA issued specific guidance on the importance of diversity action plans to improve enrollment of underrepresented populations in clinical studies.² That development has prompted the consistent and systematic use of RWE to inform diversity plans in both clinical trials and postmarketing studies. For example, Johnson & Johnson, through a collaboration between its Global Epidemiology team in the Office of the Chief Medical Officer and J&J Innovative Medicine R&D teams, is using RWD to estimate race and ethnicity distributions in the target disease populations in order to set evidence-based recruitment strategies and inform diversity action plans submitted to regulators. In postmarketing studies, fit-for-use RWD is also being used to ensure that safety monitoring and benefit-risk assessments reflect all patients eligible for treatment.

Additionally, RWE is being applied to evaluate product use in patients with comorbid conditions who are often excluded from traditional trials, providing a more comprehensive understanding of treatment outcomes in real-world settings. This is particularly important for therapies that are used in elderly cancer patients who have undergone extensive prior treatment, such as rare hematologic malignancies. For example, RWD is being used at Johnson & Johnson to characterize baseline risk and outcomes, such as cardiovascular events, that are commonly observed in elderly cancer patients in order to better understand how these medicines work in patient groups who were not initially studied in clinical trials due to frailty.

The important role of RWE in fostering greater inclusivity in cancer research

As FDA and other health authorities continue to expand the use of RWE in regulatory decisions, it is essential to recognize the critical role of RWE in enabling more representative safety and effectiveness assessments for cutting-edge treatments for cancer where significant unmet patient needs persist.

Recent applications of RWE illustrate its ability to enable a more comprehensive assessment of how treatments impact diverse patient populations to inform interventions designed to increase access to effective and safe cancer care for all patients. Embracing RWE as a complementary tool alongside clinical trials can enable regulators and health technology assessment bodies to meet the evolving needs of public health and foster greater inclusivity in cancer treatment evaluations.

Dina Gifkins is Executive Director, Global Epidemiology, Office of the Chief Medical Officer, Johnson & Johnson Innovative Medicine; Kourtney Davis is Vice President and Head, Global Epidemiology, Office of the Chief Medical Officer, Johnson & Johnson Innovative Medicine

References

Food and Drug Administration. 2020 Drug Trials Snapshots Summary Report. February 2021. Accessed March 18, 2024. https://www.fda.gov/media/145718/download
Food and Drug Administration. Diversity action plans to improve enrollment of participants from underrepresented populations in clinical studies [draft guidance]. Issued 14 April 2022. Accessed 17 January 2025. https://www.fda.gov/media/179593/download

Improving equity in cancer care: How RWE is transforming regulatory decisions for underrepresented populations

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