Overview
NINDS is a member of the Muscular Dystrophy Coordinating Committee (MDCC), a Federal Advisory Committee including NIH institutes, other Federal agencies, and patient advocates. The MDCC identifies the many challenges confronting people with muscular dystrophy and their families and coordinates efforts to address them. (View the MDCC Charter(pdf, 160 KB).) In 2005, the MDCC collected and integrated the recommendations of muscular dystrophy researchers, physicians, patients, family members, and other stakeholders into the Action Plan for the Muscular Dystrophies. The Action Plan(pdf, 690 KB) was revised and updated in 2015, and outlines priority needs to improve treatments and reduce disease burden for all forms of muscular dystrophy. This plan is meant to serve as a blueprint for research across the entire muscular dystrophy community.
MDCC Action Plan
Additional Action Plan Resources
- 2015 MDCC Action Plan Highlights(pdf, 164 KB)
- NIH Press Resease on MDCC Action Plan: Publication highlights release of muscular dystrophy action plan
- PubMed: The muscular dystrophy coordinating committee action plan for the muscular dystrophies
Previous MDCC Planning Efforts
The MD-CARE Act of 2001 directed the MDCC to develop a plan for conducting and supporting research and education on muscular dystrophy through the national research institutes, and to submit this plan to Congress within the first year of the establishment of the MDCC. This first planning stage led to the Muscular Dystrophy Research and Education Plan for NIH, which was submitted to Congress in August 2004 which formed the basis for a subsequent, more intensive planning process that produced the 2005 MDCC Action Plan for the Muscular Dystrophies(pdf, 625 KB), which was approved by the MDCC in December 2005. The next stage in planning is described in the 2015 MDCC Action Plan for the Muscular Dystrophies, above.
Strategies to Promote Diversity in Muscular Dystrophy Research Participation
While the prevalence of muscular dystrophies may vary by gender, race and ethnicity, researchers should design studies to ensure that research findings are applicable to all people affected by the condition being studied. Here we provide guidance on strategies and resources to reduce obstacles to research participation and encourage outreach to underrepresented people living with muscular dystrophies.
Strategies to overcome obstacles
General
- Broaden the eligibility criteria as appropriate so as not to exclude potential participants for reasons that are unlikely to affect the outcomes of the study.
- Minimize the burden of participating in the study by reducing the frequency and/or duration of clinic visits and overall time required.
- Allow for clinic visits in evenings or during weekends.
- Conduct periodic evaluation of recruitment and retention strategies.
Geographic
- Select study sites with ample numbers and diversity of potential study participants.
- Select study sites that minimize the travel of study participants.
- Provide support for study participant transportation, accommodations and parking as needed.
- Integrate remote data collection such as smartphone apps or wearables into the study design while also taking into consideration the need for access to broadband communication networks in rural areas.
Socioeconomic
- Have validated translations of consent forms and other relevant study documents available in languages that help ensure achievement of the planned enrollment.
- Include study personnel who are bilingual and culturally sensitive to the planned enrollment population. Consider enlisting the help of community ambassadors to build trust in the communities of potential study participants.
- Provide daycare for family members during study visits.
- Establish recruitment, enrollment and/or data collection sites in the community at locations that are convenient, familiar and trusted by potential study participants.
- Work with patient advocacy groups in outreach to underrepresented people to increase awareness of the study and to incorporate patient perspectives in the design of the study.
Additional Guidance
- Food and Drug Administration Guidance for Industry
- Publication: “Integrating Research into Community Practice— Toward Increased Diversity in Clinical Trials”, Woodcock et al., NEJM 2021
- Food and Drug Administration website on Clinical Trial Diversity
- NIH website on Inclusion of Women and Minorities as Participants in Research Involving Human Subjects
- NIH video guidance for applicants “Including Diverse Populations in NIH funded Clinical Research”
Funding Opportunities
Active
Inactive
Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01) - PAR-18-534
Field Initiated Projects Program (Development) - HHS-2020-ACL-NIDILRR-IFDV-0374
Advanced Rehabilitation Research Training (ARRT) grant - HHS-2018-ACL-NIDILRR-ARCP-0240
Advanced Rehabilitation Research Training (ARRT) Program - Community Living and Participation Department of Health and Human Services Administration for Community Living - HHS-2020-ACL-NIDILRR-ARCP-0368
Advanced Rehabilitation Research Training (ARRT) Program - Health and Function Department of Health and Human Services Administration for Community Living - HHS-2020-ACL-NIDILRR-ARHF-0369
2020 Request for Applications (RFA): International Research Grants in Congenital Muscular Dystrophy
Meetings
Membership
Lindsey A. Criswell, M.D., M.P.H., D.Sc.
Chair, MDCC; Federal Agency Representative
Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institutes of Health
Bethesda, Maryland