The new article pays special attention to the content of Diversity Action Plans, emphasizing the aspects related to the relevant goals.
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Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a draft guidance document dedicated to Diversity Action Plans to improve the enrolment of participants from underrepresented populations in clinical studies.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.
Content of the Diversity Action Plan
Under sections 505(z) and 520(g)(9) of the FD&C Act, a Diversity Action Plan must include:
- The sponsor’s goals for enrollment in the clinical study, disaggregated by race, ethnicity, sex, and age group of clinically relevant study populations.
- The sponsor’s rationale for such goals.
- The sponsor’s explanation of how the sponsor intends to meet such goals.
The document provides guidance on the form and content of a Diversity Action Plan and offers recommendations to ensure the plan meets FDA requirements. It is also important to mention that while the requirements and recommendations for Diversity Action Plans are generally aligned across drugs and devices, the types of submissions and their timing differ due to unique statutory and regulatory requirements and administrative considerations.
Enrollment Goals: Basics
According to the guidance, a Diversity Action Plan must include specific enrollment goals for a clinical study, disaggregated by the race, ethnicity, sex, and age group of the clinically relevant study population. These demographic characteristics should be detailed for subsets of the population (e.g., Asian, Black/African American).
Enrollment goals should be informed by the estimated prevalence or incidence of the disease or condition in the U.S. intended use population for the medical product being studied. In some cases, increased enrollment of certain populations may be necessary to identify clinically important differences in responses to the drug or medical device among different subsets of the study population.
A rationale must be provided for the proposed enrollment goals, especially if they deviate from the estimated prevalence or incidence of the disease or condition in the intended use population. The Diversity Action Plan should include a description of the general approach and rationale, including the methodology used to derive target enrollment goals.
Considerations for Multiple Clinical Studies
For sponsors conducting several clinical studies to support marketing authorization, the enrollment goals for each study should be consistent with an overall strategy to achieve proportionate representation of the clinically relevant population. This ensures that even if individual studies do not have proportionate representation, the overall clinical development program does.
Special Considerations for Rare Diseases
In the case of rare diseases where the clinical development program may consist of a single, small pivotal study, it might be challenging to detect differences in safety and effectiveness across the study population due to the small number of participants. However, consistent representative enrollment can provide opportunities for hypothesis generation and further study.
Sources for Enrollment Data
As it is stated in the document, sponsors should use appropriate sources, such as demographically representative registries, publicly available epidemiological surveys, and published literature, to obtain information about the estimated prevalence or incidence of the disease or condition across the affected population by race, ethnicity, sex, and age group. When using non-public sources like electronic health records or privately held information, sponsors should provide the rationale for their approach, a summary of the analysis, and citations for the data sources.
Setting Enrollment Goals
In cases where data is limited or unavailable to characterize the incidence or prevalence of the disease or condition, sponsors should consider the following approaches:
- Use prevalence and incidence information for the broader disease population when data for a specific subset is unavailable.
- For a general-use population (e.g., preventive vaccine), use US census data for setting enrollment goals.
- For populations with limited or no demographic data, general US population demographics may be used to set enrollment goals.
Global Considerations
According to the document, the FDA supports well-designed and conducted multi-regional clinical studies to provide evidence of safety and effectiveness for FDA-regulated medical products. A Diversity Action Plan for a multi-national clinical study must describe participant enrollment goals for the entire study, considering differences in disease characteristics, medical practices, and available therapies.
The overall study design should account for the need to enroll a population representative of the US intended use population as part of the overall medical product development program.
Geographic and Socioeconomic Factors
It is further stated in the present guidance, sponsors should consider additional characteristics such as geographic location and socioeconomic status (SES) if data suggests these factors impact study outcomes. The Diversity Action Plan should describe if and how these factors informed the enrollment goals and identify strategies to mitigate barriers to enrollment and retention.
Enrollment goals must be disaggregated by:
- Race: List goals for each category according to FDA guidance.
- Ethnicity: List goals for each category according to FDA guidance.
- Sex: List goals for each category according to FDA guidance.
- Age Group: List goals for clinically relevant age subsets according to FDA guidance.
The FDA explains that a well-structured Diversity Action Plan ensures that clinical studies reflect the demographic characteristics of the intended use population, thereby enhancing the generalizability and applicability of study findings. Furthermore, study sponsors are encouraged to engage with FDA review divisions early in the planning process to address any factors that may affect the enrollment goals and strategies described in their Diversity Action Plans.
Conclusion
In summary, the present FDA guidance outlines the basics of the content requirements for a Diversity Action Plan to be followed by the parties involved in order to ensure its completeness. The document pays special attention to enrollment goals and the way they should be determined.
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