From Phase 1 design to market access, clinical trials that reflect the real world deliver better data, faster enrollment, and elevated regulatory confidence.
“No data on us without us.” This patient advocacy maxim, once a rallying cry, is now a regulatory and scientific imperative. When clinical trials fail to reflect the range of real-world patients, the consequences ripple across science, equity, and access. Therapies may be less effective or safe for the very people they aim to serve.
Nowhere is this gap more consequential than in oncology and rare disease research, where genetic and phenotypic variability drive treatment decisions. Underrepresentation is an issue of fairness, a threat to data integrity, regulatory confidence, and commercial viability. Slower enrollment, higher costs, and missed signals are just the beginning.
Across the globe, regulators and health authorities are responding. From the FDA’s proposed Diversity Action Plans to the EU’s Clinical Trials Regulation, the concept is clear: inclusive trials are essential. And clinical sponsors that act early see measurable returns.
Representation Is the Foundation of Precision Medicine
The effects of underrepresentation begin to influence trial outcomes as early as Phase 1. Decisions made in early clinical development, such as biomarker selection, pharmacokinetic sampling windows, and dose-ranging cohorts, shape which populations are studied and who benefits. In oncology and rare disease research, where genetic and phenotypic factors shape the development of targeted therapies, underrepresentation compromises scientific validity.
Examples of population-specific implications of underrepresentation include:
- Black women in the U.S. are 40% more likely to die from breast cancer, often due to differences in tumor subtypes and care access.[i]
- Asian patients have higher rates of EGFR mutations in non-small cell lung cancer, directly impacting treatment selection.[ii]
- Over 80% of rare diseases have a genetic origin, yet genomic datasets are disproportionately European, limiting the generalizability of findings.[iii]
Missing these or other populations increases the risk of missed safety or efficacy signals and undermines the power to detect population-specific insights that inform personalized treatment decisions.
Inclusion Drives Faster, More Efficient Trials
Patient variety in clinical trials also delivers clear operational and financial benefits. Recruitment delays are among the costliest inefficiencies in clinical development. According to industry data, 80–85 percent of clinical trials fail to meet initial enrollment goals, and each month of delay can cost sponsors between $600,000 and $8 million.[iv],[v] Inclusive strategies like multilingual materials, community engagement, and culturally informed site selection accelerate enrollment and reduce attrition.
In a 2023 AI-based site selection analysis, embedding diversity goals alongside enrollment targets resulted in significant improvements in representation, with increases in Hispanic, Black, and Asian enrollment of 14, 27, and 60 percent, respectively, without compromising overall recruitment speed.[vi] Culturally tailored, community-driven recruitment enhances retention, reduces screen failure rates, and minimizes costly protocol amendments.[vii]
Regulatory and Payer Confidence Depends on Representative Data
An equitable approach also supports regulatory alignment and market access. The FDA’s Drug Trials Snapshots summary from 2022 found that 75 percent of participants in pivotal trials were white, and there was an underrepresentation of Black, Hispanic, and Asian populations.[viii] As regulators explore the most effective mechanisms to improve representation, sponsors can address these gaps now and position themselves to meet evolving expectations.
Payers and health technology assessment bodies are also evaluating trial populations with greater scrutiny. When data do not reflect real-world demographics, confidence in a product’s broad applicability is compromised, making payer reimbursement more challenging to achieve.
In high-cost areas such as oncology and rare disease, this scrutiny influences both pricing and patient access. Sponsors who embed inclusion early are gaining a competitive edge in data quality, enrollment timelines, and payer engagement. Conversely, failure to address these gaps can result in regulatory delays, restricted market access, and costly post-approval requirements, outcomes far more expensive than getting it right from the start.
Real-World Impact, Measurable Results
Sponsors who make the investment often see measurable results. Studies demonstrate that trials employing culturally tailored and community-engaged recruitment strategies, such as partnerships with patient advocacy organizations, translation of materials, and trusted clinical outreach, consistently show higher retention and lower screen failure rates compared to standard approaches.[ix]
The Akari Foundation, a Texas-based nonprofit focused on supporting Hispanic patients with rare diseases, offers a compelling example of culturally tailored outreach. By providing education in Spanish and addressing barriers such as transportation, Akari connects underserved patients with trials they would not otherwise have access to.
Your Health Equity Strategy Starts Here
At Ergomed, we view diversity as a scientific imperative and a strategic advantage. From Phase 1 through pivotal trials, we collaborate with sponsors to integrate inclusion at every stage, identifying underserved populations, establishing trusted partnerships with patient advocacy organizations, and implementing culturally informed recruitment strategies that foster trust and accelerate enrollment. In our next blog on this topic (insert link), we will discuss how to operationalize inclusiveness in clinical research using practical, proven approaches. Ready to accelerate equitable, high-impact trials? Contact Ergomed to get started.
[i] DeSantis, C. E., Ma, J., Goding Sauer, A., Newman, L. A., & Jemal, A. (2017). Breast cancer statistics, 2017, racial disparity in mortality by state. CA: A Cancer Journal for Clinicians, 67(6), 439–448.
[ii] Shigematsu, H., & Gazdar, A. F. (2006). Somatic mutations of epidermal growth factor receptor signaling pathway in lung cancers. International Journal of Cancer, 118(2), 257–262.
[iii] Landry, L. G., Ali, N., Williams, D. R., Rehm, H. L., & Bonham, V. L. (2018). Lack of diversity in genomic databases is a barrier to translating precision medicine research into practice. Health Affairs, 37(5), 780–785.
[iv] Idnay B, Fang Y, Butler A, Moran J, Li Z, Lee J, Ta C, Liu C, Yuan C, Chen H, Stanley E, Hripcsak G, Larson E, Marder K, Chung W, Ruotolo B, Weng C. Uncovering key clinical trial features influencing recruitment. J Clin Transl Sci. 2023 Sep 4;7(1):e199.
[v] Lawrence CE, Bruce VNM, Salberg LD, Edwards T, Morales C, Palm M, Bernard GR. Quantitative assessment of the impact of standard agreement templates on multisite clinical trial start up time. J Clin Transl Sci. 2023 Sep 6;7(1):e204.
[vi] Theodorou, B., Glass, L., Xiao, C., & Sun, J. (2024). FRAMM: Fair ranking with missing modalities for clinical trial site selection. Patterns, 5(3), 100944.
[vii] National Academies of Sciences, Engineering, and Medicine. (2022). Improving representation in clinical trials and research: Building research equity for women and underrepresented groups.
[viii] U.S. Food and Drug Administration. (2023). Drug trials snapshots summary report: 2022.
[ix] Igwe, J.-K., & Wangdak Yuthok, T. Y. (2023). Opportunities to increase the science of diversity and inclusion in clinical trials: A strategic research network approach. Journal of the American Heart Association, 12(e030042).