Rethinking Representation in Clinical Research

Who’s in the Study?

Introduction

The underlying principle of a clinical study is to assess the safety and efficacy of an investigational product on a sample of the intended patient population. Therefore, to get the most accurate sampling and real-world data, a heterogeneous sample representative of the population should be included in the study. However, there can be a disparity in representation of patients of different ages, racial, ethnic, and other demographic and baseline characteristics (e.g., BMI, nicotine and alcohol use). Depending on the condition to be treated, the treatment effect and risk of adverse events may vary among these subpopulations. In a guidance for medical device studies, the FDA highlights the importance of collecting and assessing age, race, and ethnicity data. For FDA new drug applications, data must be presented by gender, age, race, and other (e.g., disease severity) subgroups (21 CFR 314.50(d)(5)(v) and (vi)). Similarly, the Australian Therapeutic Goods Administration requires age and sex information at a minimum when reporting adverse events.

The ethical principle of justice described in the Belmont Report applies to the fair selection of subjects. Equity is critical, and patients should have the resources and opportunities to participate. Lowering barriers can increase the patient pool to improve enrollment.

Although a study may not be powered to perform statistical analysis of subgroups, collecting detailed demographic information and looking at trends can inform what is to be expected in real-world usage of the investigational product. This can help focus post-market efforts if more information is needed about a particular subpopulation.

Removing barriers

Considering equity in the design of the study will help lower the barriers for participation to increase diversity. Each inclusion or exclusion criterion should be evaluated for clinical relevance. Often, criteria are chosen for short-term convenience such as only including fluent English speakers, so translations won’t be needed. However, this can unnecessarily exclude people and thus extend enrollment time.

Recruitment efforts should also be developed and distributed with equity in mind. Social media campaigns can be popular, effective, and inexpensive, but usually target a younger audience and those with internet access. To capture a broader participant pool, recruitment materials can be distributed in places that do not have a financial barrier such as community centers and libraries. Another example to increase visibility of recruitment materials to a diverse group of people is to create patient materials in different languages with infographics to attract patients with lower English literacy.

Decentralized studies have been used successfully to boost enrollment of diverse populations. Patients may not have access to reliable transportation or have physical limitations that makes traveling to a site difficult. Local facilities, at-home visits, and telehealth calls reduce patient travel burden.

Mistrust is a pervasive barrier to enrollment. Having a diverse research team and access to patient advocates can be encouraging for patients. Patients should feel that their voices are heard. Community engagement and involvement with patient advocates show that study participation is not a one-sided transaction.

Conclusion

Ensuring diverse patient populations in clinical studies is essential for collecting robust data to advance real-world evidence for patient health and a great approach to increase enrollment.

MED offers a variety of clinical trial services and is a full-service CRO. We have over 40 years of experience designing and executing clinical trials, ranging from early feasibility studies to multinational, controlled pivotal trials to post-market registries.

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