The article outlines the key points of the approach to be used in order to improve participation in clinical studies conducted in the US.
Table of content
The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a draft guidance document dedicated to Diversity Action Plans to improve the enrollment of participants from underrepresented populations in clinical studies. Once finalized, the document will provide information about the applicable regulatory requirements, as well as additional clarifications and recommendations to be taken into consideration by the parties responsible for clinical trials.
At the same time, provisions of the guidance are non-binding in their legal nature, nor are they intended to introduce new rules or impose new obligations. Moreover, the authority explicitly states that an alternative approach could be applied, provided such an approach is in line with the existing legal framework and has been agreed with the authority in advance.
In particular, the present guidance is intended to assist sponsors conducting specific clinical studies involving drugs, biological products, and devices in fulfilling the requirements for Diversity Action Plans under sections 505(z) and 520(g)(9) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by section 3601 of the Food and Drug Omnibus Reform Act of 2022 (FDORA). Sections 505(z)(3) and 520(g)(9)(A) of the FD&C Act mandate sponsors to submit Diversity Action Plans in the form and manner specified by FDA guidance.
The primary goal of these plans is to enhance the enrollment of historically underrepresented populations in clinical studies, thereby improving the robustness and applicability of evidence for the intended use population. These plans must be focused on the sponsor’s enrollment goals, the rationale behind these goals, and the strategies to achieve them.
Additionally, the FDA is required to update or issue guidance to sponsors on the format and content of their Diversity Action Plans, which includes demographic characteristics such as age, sex, and racial and ethnic backgrounds.
Purpose and Scope of the Guidance
The FDA is issuing this guidance to comply with section 3602 of FDORA, which requires updates or issuance of guidance on Diversity Action Plans as stipulated by sections 505(z) and 520(g)(9) of the FD&C Act. This document outlines the format and content requirements for Diversity Action Plans, including the timing and submission processes.
It also details the criteria and processes for the FDA to evaluate sponsors’ waiver requests under sections 505(z) or 520(g)(9). Additionally, the guidance offers recommendations for sponsors wishing to publicly disclose key information about their Diversity Action Plans. This guidance replaces the previous draft guidance issued in April 2022, focusing on diversity plans to improve enrollment from underrepresented racial and ethnic populations in clinical trials.
At the same time, it is important to mention that it does not cover all issues related to the clinical development of medical products, such as clinical study design, endpoints, or necessary data for marketing submissions, for which sponsors should refer to other FDA guidance documents.
Regulatory Background
First of all, the authority acknowledges that clinical studies play a crucial role in determining the safety and effectiveness of medical products intended for various conditions or diseases. However, certain populations in the United States are often underrepresented in biomedical research, including clinical studies, even though they may bear a higher burden for specific conditions or diseases compared to their representation in the general population.
This underrepresentation can be due to several factors, including assumptions about the feasibility of enrolling a representative population and the impact on study timelines, as well as a lack of strategies to ensure the inclusion and retention of a diverse study population. Ensuring diverse enrollment in clinical studies is essential for generating data that reflect a broader population, thus improving the generalizability of results and supporting equitable and timely access to medical innovations.
The FDA has issued several guidance documents to promote the inclusion of diverse populations in clinical studies. These documents provide recommendations on enrolling representative populations considering demographic factors such as race, ethnicity, sex, and age group and include general measures to enhance diversity in clinical studies.
These measures are scientifically appropriate and may extend to the post-approval setting. Scientific experts and stakeholders have also suggested strategies to ensure diverse clinical study participation, emphasizing the need for intentional and deliberate efforts to achieve representativeness in clinical study populations.
Importance of Diversity in Clinical Studies
Diverse clinical study populations ensure that medical products are tested in a representative sample of the intended use population. Factors to consider when setting enrollment goals include demographic characteristics, clinical characteristics such as comorbidities and disease etiology, and access to standard preventive and diagnostic care.
FDA regulations require that sponsors include demographic information in their clinical study reports and marketing submissions, ensuring that effectiveness and safety data are presented by race, gender, and age.
Requirements and Recommendations for Diversity Action Plans
Consistent with section 3602(a) of FDORA, this guidance focuses on Diversity Action Plans aimed at achieving adequate representation of different age groups, sexes, and racial and ethnic demographics in clinical studies. Sponsors are encouraged to consider broader health disparities and differential access to healthcare that may impact clinical study participation, including geographic location, gender identity, sexual orientation, socioeconomic status, physical and mental disabilities, pregnancy, lactation, and comorbidities.
These factors should be taken into account when developing enrollment goals for Diversity Action Plans, supporting subgroup analyses where relevant.
Submission and Evaluation of Diversity Action Plans
The requirements for Diversity Action Plans apply to clinical studies commencing enrollment after 180 days from the publication of the final guidance. Sponsors should not submit Diversity Action Plans for clinical studies where protocols or Investigational Device Exemption (IDE) applications are submitted within 180 days of the final guidance publication, or for studies that do not require an IDE and are approved by an institutional review board within the same period.
The guidance describes the format and timing for submitting Diversity Action Plans and the criteria for evaluating waiver requests from sponsors.
Conclusion
In summary, by virtue of the present guidance, the authority intends to assist the parties responsible for clinical studies in ensuring the accuracy and reliability of the results by ensuring the proper representation of various groups of patients. The authority mentions that certain patient groups could be insufficiently represented, creating additional risks for such groups due to the lack of information deriving from the studies.
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