Leapcure’s Response to FDA’s Diversity Action Plan Guidance

Submitted Response to FDA

Subject: Comment on Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies
Commenter: Zachary Gobst, CEO of Leapcure
Submission Date: October 11, 2024
Regulatory Docket: FDA-2021-D-0789

Response:

At Leapcure, we believe in elevating the patient voice and empowering advocacy groups with technology to speed research progress. In our mission to accelerate research progress and in support of the objectives of the guidance to drive increased participation in clinical research for underrepresented populations, we propose that the FDA consider the role of incentives and education in supporting the objectives outlined in the law that FDA’s guidance is ultimately designed to address. Specifically, we propose that FDA’s framework consider product-market fit and adoption stage to further refine how diversity requirements are applied in clinical trials. Additionally, we recommend that demonstrating diversity in trial outcomes be considered a criterion for fast-tracking application reviews and approval timelines, similar to the principles applied in rare disease and medical device research where priority review of marketing applications is available. Lastly, we want to emphasize the importance of cultural competency and empathy in working with underrepresented communities, as this is vital for generating diverse enrollment and truly inclusive and valid clinical data. We discuss each of these in greater detail below.

Proposed Additional Framework Considerations:

1. Categorization of Trials Based on Product-Market Fit and Adoption Stage:
   • Clinical trials should be categorized based on their product-market fit with patients and physicians, taking into account how aware and accepting these groups are of the new therapy when compared to standard care.
   • Early-stage trials may still be working to build awareness and market traction, and as such, may require more flexibility in meeting diversity requirements. We agree that later-stage trials—where the treatment is gaining traction and has a clearer path to adoption—should face stricter diversity requirements, ensuring that treatments likely to be adopted by broader populations are designed to be inclusive from the outset.
2. Stricter Diversity Methodologies for Trials with High Product-Market Fit:
   • For trials that demonstrate high product-market fit, meaning the treatment is well-positioned for broad adoption, stricter diversity requirements should be enforced. These trials have the infrastructure, resources, and awareness necessary to meet inclusive enrollment goals.
   • By ensuring diversity-focused design at this stage, the FDA can help improve the real-world applicability of the treatment and generate data that reflects a broad cross-section of the population.
3. Leveraging Fast-Track Mechanisms for Diversity Data:
   • We further propose that the FDA apply fast-track principles used in rare disease and device research to encourage and reward trials that achieve diverse representation goals in their participant populations.
   • Just as rare disease and breakthrough device research fast-tracks treatments that demonstrate clear benefit potential in highly specialized populations, we believe diverse data from trials should be considered in the same light. If a trial demonstrates significant success in enrolling and generating outcomes for underrepresented populations, this should serve as a key factor for fast-tracking the review and approval timelines for applicants, akin to priority review.
   • This approach would incentivize sponsors to design their trials from the outset with diversity in mind, knowing that it could lead to an expedited path for FDA review and approval. The same like-for-like analysis used in rare disease research—where limited but targeted data leads to fast-tracking—can be applied here for diversity data.
4. The Role of Cultural Competency and Empathy in Inclusive Trials:
   • From a practitioner perspective, one of the most critical aspects of engaging underrepresented populations in clinical trials is the role of cultural competency and empathy in trial design and implementation. FDA’s final guidance should help drive cultural competency through education of stakeholders and provision of tools to better inform developers how cultural competency, empathy and sensitivity can help achieve the goals of the law and final guidance. 
   • While this may fall outside the FDA’s direct regulatory role, education of developers alongside issuance of the final guidance is paramount to ensuring that sponsors and researchers work with underrepresented communities in a manner that is culturally sensitive, respectful, and empathetic to the specific needs, values, and concerns of those populations. Without these practices, efforts to improve diversity in clinical trials may simply fail or may fail to generate valid, reliable data, as trust and engagement with these communities are compromised which could lead to trial discontinuations and lost data.
   • This cultural competency is not only about recruitment but also about sustaining engagement throughout the trial process, ensuring participants feel heard and supported, and that their concerns are addressed. Inclusive trials that employ culturally competent strategies are more likely to retain participants to completion and yield the data the law and guidance seek to address that is reflective of the true health outcomes across diverse groups.
5. Gradual Implementation of Restrictions Based on Adoption Stage:
   • As noted earlier, for earlier-stage trials, where awareness of the treatment is still developing, we support continued flexibility in diversity requirements by the FDA to ensure these trials can gather initial safety and efficacy data. 

Rationale:

• Building on Successful Fast-Track Models: By applying the same fast-track principles used for rare diseases and breakthrough devices to diversity data, the FDA can leverage an existing and successful framework. Just as fast-tracked treatments for rare diseases prove their value in small, targeted populations, trials that achieve diversity goals should also be fast-tracked based on their ability to deliver generalizable and equitable outcomes.
• The Power of Cultural Competency: Cultural competency and empathy in working with underrepresented populations are key to generating the inclusive, high-quality data the FDA is seeking. While this may not be a formal regulatory requirement, it is vital from a practitioner standpoint. Trials designed with these principles will be more likely to achieve the intended diversity outcomes, producing more reliable, valid data and fostering greater trust in the regulatory process.
• Tailored Diversity Enforcement: By categorizing trials based on their adoption stage, the FDA can ensure that early-stage innovations are not unduly burdened by diversity requirements, while ensuring that later-stage treatments meet the goal of inclusivity. This gradual approach strikes a balance between fostering innovation and promoting equity.

Conclusion

By incorporating product-market fit, leveraging fast-track mechanisms for diversity data, and emphasizing the importance of cultural competency in trial design, the FDA can create a more effective and equitable approach to improving diversity in clinical studies and research outcomes. We believe this holistic framework will ensure that clinical trials not only meet diversity goals but also generate inclusive data that is valid, reliable, and reflective of the real-world patient population.

Thank you for considering this comment. We believe these recommendations will strengthen the FDA’s efforts and promote both innovation and health equity in clinical research.

Sincerely,
Zachary Gobst
CEO, Leapcure