Increasing Diversity, Equity, Inclusion, and Accessibility in Rare Disease Clinical Trials.
Pharmaceut Med
; 38(4): 261-276, 2024 Jul.
Article
in En
| MEDLINE
| ID: mdl-38977611
ABSTRACT
Diversity, equity, inclusion, and accessibility (DEIA) are foundational principles for clinical trials and medical research. In rare diseases clinical research, where numbers of participants are already challenged by rarity itself, maximizing inclusion is of particular importance to clinical trial success, as well as ensuring the generalizability and relevance of the trial results to the people affected by these diseases. In this article, we review the medical and gray literature and cite case examples to provide insights into how DEIA can be proactively integrated into rare diseases clinical research. Here, we particularly focus on genetic diversity. While the rare diseases DEIA literature is nascent, it is accelerating as many patient advocacy groups, professional societies, training and educational organizations, researcher groups, and funders are setting intentional strategies to attain DEIA goals moving forward, and to establish metrics to ensure continued improvement. Successful examples in underserved and underrepresented populations are available that can serve as case studies upon which rare diseases clinical research programs can be built. Rare diseases have historically been innovation drivers in basic, translational, and clinical research, and ultimately, all populations benefit from data diversity in rare diseases populations that deliver novel insights and approaches to how clinical research can be performed.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Clinical Trials as Topic
/
Rare Diseases
/
Health Services Accessibility
Limits:
Humans
Language:
En
Journal:
Pharmaceut Med
Year:
2024
Document type:
Article
Affiliation country:
Country of publication: