Clinical trial enrollments that reflect the demographic diversity of the impacted patient population generate insightful scientific knowledge on how therapies work across all those affected by the disease. Trials with diverse participants also may improve access to promising care for patients whose age, gender, race, and ethnicity might be underrepresented. Soon this approach will infuse most study designs, as the FDA is poised to require clinically relevant populations for most pivotal trials.

For decades, trial demographics have shown patterns of disparities that underrepresented some patient populations. For example, 13 of the 15 new cancer therapies approved by the U.S. Food and Drug Administration (FDA) in 2021 had enrollments overwhelmingly comprised of white male participants.¹ Other studies have documented the routine exclusion from trials of people with disabilities, chronic illnesses, hearing or vision disorders or psychiatric impairments, even when supportive options could be permitted or provided.²

The FDA has led global regulatory efforts to help industry transform its trial populations, establishing distinct diversity and inclusion (D&I) programs, like Project Equity and Project Silver.^3,4 The FDA also drafted guidance in April 2022 on developing a Race and Ethnicity Diversity Planto outline how sponsors should proactively assess the racial and ethnic makeup of their trials’ intended patient populations, set enrollment goals for patient subpopulations, and build enrollment strategies to reflect clinically relevant populations.⁵ In December 2022, the U.S. Congress took steps to compel sponsors to have Diversity Action Plans in place for most pivotal drug and device clinical trials as a requirement for new drug and device applications, following the finalization of revised FDA guidance.⁶ For more background on this long-standing issue, read IQVIA Institute's report Advancing Diversity in Clinical Development.

Many sponsors face challenges in expertise and resources in developing diversity plans. However, a diversity plan helps sponsors understand, identify, enroll, and retain clinically relevant participants from historically underrepresented populations. These individuals include those belonging to racial and ethnic minorities, identifying with sexual or gender minorities, or experiencing economic, geographic, linguistic, or cultural barriers to healthcare services. They also may be patients older than 75, or with comorbidities like HIV, organ dysfunctions, mobility issues, or brain metastases.

We believe that taking a proactive trial lifecycle approach is key to achieving diversity alongside overall clinical development goals and timelines. D&I should be part of trial DNA, as an influencer of every trial aspect, from design to site selection and activation to recruiting, monitoring, and retaining patients. When D&I considerations are woven into trial planning from the beginning, concerns about adding time and cost can be mitigated or eliminated.

We offer sponsors a holistic framework for both FDA Diversity Plan development and operationalizing their trials’ diversity tactics, with specific roles for staff of the sponsor and CRO, and anticipated outputs to drive the approach forward:

1. Characterize D&I demographics of the clinically relevant population
2. Develop baseline D&I enrollment goals
3. Reflect D&I insights in trial design and align site identification with D&I enrollment goals
4. Embed D&I in site activation plan, communications and training
5. Develop D&I recruitment support and planning
6. Deliver D&I in patient recruitment with tailored strategies
7. Leverage D&I for patient retention and continuous engagement

As a critical part of their D&I planning, sponsors should actively hear how patients feel about their disease, treatment options, and the potential burdens a trial might present. Surveys, social listening and focus groups, particularly with a patient advocacy partner, will gather patients’ preferences to inform the D&I plan. Advocacy partnerships may also provide paths to assistance with recruitment and retention, particularly from community-based patient or physician groups. Some sponsors formalize these alliances into trial or community advisory boards, whose input helps ensure a trial has culturally diverse staff and culturally appropriate and universally designed materials. These boards recognize how patients’ experiences, ethnicity, and culture can impact what patients know and perceive about a trial. For example, patients can differ in their cultural and belief sensitivities to many aspects of a trial, including the   characteristics of the investigational therapy; the accessibility, duration, or frequency of clinic visits; the testing and procedures; the chance of randomization to a placebo; and the collection of patient-reported outcomes. Patients’ input about all these points shape the formation of a robust D&I plan.

Translating the qualitative data of patients’ insights into actionable trial features can be daunting. We use an algorithm to yield quantitative outputs that sponsors can use to calculate and compare how different protocol modifications might impact patient burden. As part of our Data-Informed Protocol Assessment (DIPA), this approach to D&I helps validate study designs and leads to earlier identification of enrollment challenges, potentially reducing the need for later amendments.

A comprehensive approach to D&I for clinical development of your oncology assets can be a tremendous benefit to you as a sponsor and to the patients seeking innovative therapies. Contact us to discuss how we can help support your clinical research D&I goals and FDA Diversity Plan needs.