The new article is dedicated to enrollment goals, highlighting the aspects related to the rationale behind them and the way they should be measured.
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Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a draft guidance document dedicated to Diversity Action Plans to improve the enrolment of participants from underrepresented populations in clinical studies.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.
Rationale for Enrollment Goals
According to the guidance, the Diversity Action Plan must include a detailed rationale for the enrollment goals. This rationale should provide sufficient information and analysis to explain how the sponsor determined the enrollment goals.
In particular, the sponsor’s rationale should include:
- Background information on the disease or condition being investigated, including an overview of its natural history, risk factors, prevalence, and incidence estimates, if available.
- Any additional background information that justifies the enrollment goals.
If the sponsor plans to conduct multiple clinical studies to support a single marketing submission, they may specify enrollment goals across these studies. The rationale must explain the differing enrollment goals across studies and how each study contributes to the overall enrollment goals for the clinical development program of the medical product.
Specific Data and Information
The document further outlines a specific scope of information to be included depending on the product in question. For instance, the rationale should describe data and information suggesting potential differences in the safety and effectiveness of the investigational drug across the clinically relevant population, such as differences in pharmacokinetics (PK) or pharmacodynamics (PD).
Sponsors should include available data on differences in PK, PD, safety, or effectiveness by sex, age, or genetic variations prevalent in certain racial and ethnic populations. The rationale should also consider other population-level or individual characteristics impacting clinical outcomes, such as socioeconomic status (SES), geographic location, and comorbidities. Sponsors must provide citations for the data sources used.
For medical devices, the rationale should describe data and information about the potential for differential safety and effectiveness of the device across clinically relevant populations. This includes data on expected differences in safety or effectiveness by sex, age, or genetic variations.
The rationale should also consider other population-level or individual characteristics that may impact clinical outcomes, such as SES, geographic location, and comorbidities. Sponsors must evaluate relevant factors affecting device performance across a diverse population and provide citations for the data sources used.
For example, variations in skin pigmentation affecting device performance should be considered when setting enrollment goals. The rationale should describe how available information was considered in setting these goals.
Measures to Meet Enrollment Goals
The Diversity Action Plan must explain how the sponsor plans to achieve the specified enrollment goals. The relevant section should include a description of the enrollment and retention strategies for the study population.
As explained by the FDA, the Diversity Action Plan should focus on specific measures addressing the enrollment and retention of participants in the clinical study.
Examples of strategies include:
- Sustained Community Engagement: Engaging with community advisory boards, navigators, community health workers, patient advocacy groups, local healthcare providers, and community organizations.
- Cultural Competency Training: Providing training for clinical investigators and research staff to build trust, engage with participants from diverse backgrounds, and avoid biased communication and behavior.
- Participant Awareness: Improving awareness and knowledge of the clinical study, including language assistance for individuals with limited English proficiency.
- Reducing Participant Burden: Minimizing unnecessary procedures, offering convenient site locations, providing transportation and dependent care assistance, allowing flexible study visit hours, and reimbursing incurred costs.
- Improving Access: Limiting exclusion criteria, selecting site locations that facilitate enrollment of a representative population, and considering the accessibility needs of individuals with disabilities.
- Study Decentralization: Utilizing decentralized study methods when appropriate.
Monitoring Enrollment Goals
The Diversity Action Plan should also include a plan to monitor enrollment goals during the study to ensure they are met. This involves specifying how and when study enrollment will be monitored and any measures to be undertaken if the study is not on track to meet its goals.
This information should be included in Diversity Action Plan modifications and briefing packages for meetings related to the clinical study.
Conclusion
In summary, the present guidance outlines special considerations intended to ensure the Diversity Action Plans are comprehensive and effectively promote the enrollment of a diverse and representative study population, enhancing the validity and applicability of clinical study results.
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