The U.S. Food and Drug Administration (FDA) has demonstrated a commitment to expanding diversity in clinical trials. Decentralized Clinical Trials ( DCTs) contribute to this effort by enhancing convenience for trial participants, reducing burdens on caregivers, expanding geographic access, and facilitating research on rare diseases and diseases affecting populations with limited mobility.

This is the fourth edition of our blog series on decentralized clinical trials DCTs. Click here to explore the series.

Decentralized Clinical Trial Draft Guidance

In May 2023, the FDA released Draft Guidance regarding the implementation of DCTs for drugs, biological products, and devices. DCTs are clinical trials where some or all trial-related activities occur at locations other than traditional clinical trial sites. For example, all trial activities may occur via telehealth, or the trial may be hybrid and the administration of the investigational product may occur at a traditional site while follow-up visits are conducted at-home or via telehealth.

As we discussed previously, access to telehealth providers and digital health technologies have greatly expanded the types of trial-related activities that can be conducted remotely and the scope of data that can be collected. The use of decentralized elements, such as obtaining laboratory tests at a local facility (rather than requiring a participant to travel to an academic medical center) or conducting follow-up visits in a participant’s home or via telemedicine, increases the convenience of such activities for research participants and can assist in diversity efforts. Such increased access and convenience offer a more patient-centric approach to clinical trials.

A key element in the Draft Guidance centers around the Sponsor’s responsibilities for striving towards diversity and inclusiveness in their trial populations. The Draft Guidance specifies that Sponsors engaging in outreach through local health care institutions (for example, pharmacies and clinics) may facilitate recruitment of participants in areas where there are limited or no traditional clinical trial sites. In addition, Sponsors using local health care providers located close to the participant and/or facilitating the conduct of trial-related activities in the participant’s home (such as via telemedicine or other digital health technologies) reduces the need for travel which may improve engagement, recruitment, and retention for participants with challenges accessing traditional sites. Furthermore, the Draft Guidance notes that using local health care providers may also help reduce cultural or linguistic barriers to participation by using providers that understand the needs of the local community.

Why Prioritizing the Patient Matters

In a press release announcing the FDA’s new Draft Guidance, FDA Commissioner Robert M. Califf, M.D. specifically highlights the value of DCTs in regards to enhancing diversity and states:

“As we seek to improve our evidence generation system, decentralized clinical trials may enhance convenience for trial participants, reduce the burden on caregivers, expand access to more diverse populations, improve trial efficiencies, and facilitate research on rare diseases and diseases affecting populations with limited mobility.”

Ultimately, DCTs provide a more patient-centric approach to clinical trials by increasing access to trials to participants that previously may have not had the time or geographic proximity to participate. This patient-centric approach may facilitate research on rare diseases and diseases affecting populations with limited mobility and may expand access to more diverse populations that better reflect the populations that will use the approved products.

FDA’s commitment to DCTs builds on the agency’s previous initiatives to increase diversity in clinical trials via the creation of “Race and Ethnicity Diversity Plans” and the prioritization of data collection of racial and ethnic minorities. DCTs appear to be the next step in FDA’s goal to diversify enrollment and improve data collection and analysis to assist FDA in its mission of ensuring the safety and efficacy of approved products.

FDA’s Commitment to Diversity

The DCT Draft Guidance builds on FDA’s previous efforts at addressing diversity. In October 2016, FDA issued guidance on the Collection of Race and Ethnicity Data in Clinical Trials which focused on methods to the collection of data related to racial and ethnic populations. This guidance was followed by draft guidance on establishing “Race and Ethnicity Diversity Plans” issued in April 2022 which asserts the need for improved enrollment of racial and ethnic minorities to improve data collection and analysis for Sponsors. The guidance primarily arms Sponsors with new recommendations in developing a “Race and Ethnicity Diversity Plan” to bolster the enrollment of certain populations, such as Black or African American, Hispanic/Latino, Indigenous and Native American, Asian, Native Hawaiian and Other Pacific Islanders, and other persons of color, in clinical trials. Many of these groups, although often disproportionately impacted by disease burden, are often underrepresented in clinical studies.

As part of the Race and Ethnicity Diversity Plan, Sponsors are recommended to: lay out enrollment goals for underrepresented racial and ethnic participants, describe available data of a disease or condition as it relates to underrepresented populations, discuss the current understanding of evidence of disparities, specify study design features that will support the analysis of the effectiveness of the medical product in light of race and ethnicity data, summarize differential findings, specify plans to collect data to explore the differences in safety and/or effectiveness associated with race and ethnicity throughout the developmental life cycle of a medical product, and include clinical pediatric studies as part of the pediatric development of the product.

The Race and Ethnicity Diversity Plan guidance centers on the enrollment of participants themselves. The guidance aims to increase the accuracy of the data Sponsors collect while tackling the issue of disproportionately low enrollment of certain participants. The goal of the guidance is to bolster the public’s faith in clinical data and provide Sponsors with more weight behind their conclusions of the efficacy of their products.

DCTs may help achieve such goals by providing an additional avenue to increase enrollment and facilitate high quality data collection and analysis. The DCT Draft Guidance builds on FDA’s previous initiatives by providing additional opportunities to diversify enrollment, maintain engagement and retention, and, therefore, assist FDA in its mission of ensuring the safety and efficacy of approved products.

We will continue to monitor FDA for any rule changes or guidance that affect or improve DCT opportunities.

Daniel S. Costandy contributed to this article.