Diversity action plans are critical for clinical trial sponsors to consistently implement actions to improve racial and ethnic diversity in DCTs. Enrollment of a representative number of participants from underrepresented racial and ethnic populations helps ensure that the data generated in clinical trials and studies reflect the racial and ethnic diversity of the population expected to use drugs and medical products and may assist in identifying effects on safety or efficacy outcomes that may be associated with, or occur more frequently within these populations. While the FDA has published draft guidance for diversity action plans for the clinical trials generally, RDDC strongly recommends the FDA include the development of diversity action plans in the roles and responsibilities of DCT sponsors and develop specific guidance, or expand current guidance, for diversity action plans that account for the unique structure of DCTs.
As discussed above, DCTs offer unique benefits for increasing participation in clinical trials. The remote or hybrid trial design and the unique and innovative technological solutions employed by DCTs may necessitate additional consideration when developing diversity action plans. Specifically, in the formulation of the diversity action plan clinical trial sponsors should consider how decentralized features of the trial may be employed to increase participant diversity, and analyze which decentralized features will best meet diversity enrollment goals. Further, the action plan should describe specific trial enrollment and retention strategies that may be specific DCTs, including: digital health technology proficiency, language assistance, access to broadband required for use of decentralized technologies, and modification of technologies for persons with disabilities.
Patients may have more responsibilities in a DCT, and understanding their ability and willingness to meet these responsibilities is critical for the success and diversification of the trial. RDDC strongly urges FDA to include recommendations for clinical trial sponsors to engage patients and patient experiences when developing DCTs. Patient perspectives are essential in understanding the diseases or conditions at focus of the DCT, conceptualizing clinical benefits and risks, and ensuring the DCT is designed in a way that supports successful patient engagement. This is especially true for patients with rare diseases, who may face unique challenges accessing certain health technologies, in-person trial locations, and quality healthcare. Incorporating patient experiences in clinical trial design will assist the trial sponsor in understanding the clinical environment in which patients with the condition seek care, the patient and/or caregiver perspectives on the disease, its impacts, and therapeutic needs and priorities. By considering which aspect(s) of the patient experience are most effected by the disease or condition, clinical trial design, technology employed, and trial objectives and endpoints can be most effectively developed to ensure the most optimal applicability for patients and encourage full participation.
We are aware that the FDA is in the current process of developing guidance for the incorporation of patients' voices in medical product development and regulatory decision making. However, developing guidance for enhancing and incorporating patient voices cannot be successful if it is not encouraged consistently throughout other FDA guidance, including the development of DCTs.
A core aspect of DCTs is the deployment of digital health technologies (DHTs) that broaden the range of participation in the clinical trial and in the data. These technologies span a wide range of uses, from applications in general wellness to applications of medical devices. However, without addressing participant barriers in relation to the introduction of new technologies, DCTs cannot ensure participation from the lowest socioeconomic groups. Many patients from underserved populations, especially those with rare diseases, do not have access to broadband or lack the sufficient technology or training to use digital healthcare technologies.
In addition to recommending clinical trial sponsors to ensure the availability of DHTs for all DCT participants, FDA should encourage sponsors to provide training and resources as necessary for participants who may lack the technological proficiency, broadband, or other technological infrastructure required for participation and establish specific protocol in the case of technological unavailability or failure. FDA should also encourage sponsors to analyze any additional technological barriers that may prevent certain socioeconomic groups and rural individuals from trial participation.
Comprehensive recruitment strategies can enable clinical trial sponsors to recruit more diverse participants. While the Draft Guidance recommends some actions sponsors may take to partner with organizations, the FDA should expand the guidance to recommend sponsors partner with community, faith-based, nonprofit, and patient advocacy organizations for participant recruitment. These types of organizations often have membership groups and connections with local communities and are on the front line communicating within a diverse communities. These partnerships can sustain long-term DCT viability because these organizations can serve as hubs for clinical trial recruitment and other training that may be necessary for DCT participation.
In 2022 and 2023, RDDC partnered with Upequity to conduct an online survey titled Diversity, Equity, and Inclusion Efforts in Rare Disease Organizations Survey (DEI Survey). The DEI survey was developed to address rare disease patient organization's related to outreach, education, and support of patients of color with rare diseases. Based on the results of the survey, RDDC developed a set of recommendations to increase DEI across different organizations, including pursuing diversity in clinical trial recommendations. Specifically, the report recommends a series of actions clinical trial sponsors and other organizations can employ to increase the outreach to patients of color with rare diseases. The FDA should incorporate these recommendations into the Draft Guidance to ensure there are effective recruitment strategies. The recommendations include:
- Post clinical trial information on patient advocacy organization’s websites, social media, and other public-facing communication methods.
- Create a lay guide that provides an overview of what a clinical trial is and how to participate.
- Provide opportunities for the public to participate in forums that serve as an educational tool for families, including those living with rare disease, to address clinical trial information.
- Provide information about clinical trials through all modes of communication, even those outside of social media.
- Engage in targeted recruitment efforts of underserved/minority populations.
