Research Funding Opportunity from CDMRP/PRMRP for Fiscal Year 24

In our ongoing effort to advance scientific understanding and advancing treatment research in all Fragile X-associated conditions and disorders, we are sharing information on an available funding opportunity.

Peer Reviewed Medical Research Program (PRMRP)

Over $32M has been awarded for Fragile X-associated conditions and disorders research since 2010, when Fragile X was first included as an eligible condition for funding from the Congressionally Directed Medical Research Program (CDMPR) Peer Reviewed Medical Research Program (PRMRP), including the recent award of up to $11.4 M for a clinical trial for individuals living with Fragile X-associated Tremor/Ataxia syndrome (FXTAS). Thanks to the efforts of the NFXF advocates, Fragile X, which includes all FMR1-associated conditions and disorders, is once again included as an eligible topic area for Fiscal Year 24 (FY24). The PRMRP is within the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP) – a global funding organization that fosters novel approaches to congressionally targeted biomedical research areas. CDMRP receives annual appropriations that are disease or condition-specific. Congress appropriated $370M in funding For the FY24 budget within the PRMRP.

Since 2010, the NFXF and the Fragile X community have continued to advocate in Congress for policies and research funding for Fragile X-associated conditions and disorders. We have nominated numerous consumer reviewers from the Fragile X community to participate in the peer and programmatic PRMRP review process. The consumer reviewer provides a perspective that is complimentary to the scientific expertise and helps the scientist understand the human side of how the research will impact the Fragile X community.

Important and New Opportunities for Fiscal Year 24

• Rare Diseases and Conditions Portfolio – Fragile X syndrome and Fragile X-associated conditions and disorders are included in this new category.*
  *While not new, hereditary ataxia is also listed as an eligible condition within the PRMRP, which may be an additional option for FXTAS projects. Learn more here.
  
• Providing Feedback about Research Needs – The NFXF was invited to and participated in a conversation to identify research gaps, the most urgent needs, and an explanation as to why these unmet needs are important to the Fragile X community.
• Impact Award
• Webinar with PRMRP Program Manager – April 10, 2024. Registration required.

FY24 PRMRP Funding Opportunities/Mechanisms for Fragile X-associated Conditions and Disorders Research:

• Clinical Trial Award: Supports the rapid implementation of clinical trials of novel interventions. The Preproposal deadline is May 13, 2024, 5:00 p.m. ET
• Discovery Award: Supports the exploration of a highly innovative new concept or untested theory – Letter of Intent deadline is May 6, 2024, 5:00 p.m. ET
• Investigator-Initiated Research Award: Supports research that will make an original and important contribution to the field of research or patient care in the Topic Area(s) of interest – Letter of Intent deadline is May 6, 2024, 5:00 p.m. ET
• Impact Award: Supports applied research, defined as use-inspired and practice-oriented science that brings together outcomes from basic research, and insights from the real-world environment, to foster clinical applicability – Letter of Intent deadline May 13, 2024, 5:00 p.m. ET
• Lifestyle and Behavioral Health Interventions Research Award (LBIRA): Supports the development, evaluation, and implementation of therapies, technologies, and innovations that will enhance patient experience and improve outcomes. Letter of Intent deadline is May 13, 2023, 5:00 p.m. ET
• Technology/Therapeutic Development Award: Supports the translation of promising preclinical findings into clinical applications for prevention, detection, diagnosis, treatment, and/or quality of life – Letter of Intent deadline is May 13, 2024, 5:00 p.m. ET

Learn more about the FY24 funding mechanisms:

The NFXF can help! The PRMRP program appreciates partnerships between patient advocacy groups and researchers.

The NFXF Research Facilitation portfolio leverages our skills to create meaningful partnerships with all researchers, from initial concept through the dissemination of results. We support our research facilitation mission through the NFXF Research Readiness Program, our services menu, focused projects, and advocacy. We recommend all researchers engage in the program early for support from concept through the conclusion of the research project.

PRMRP Strategic Goals for Fiscal Year 24

Foundational Studies

• Identify biological mechanisms underlying disease onset, disease progression, or phenotype/symptomatic heterogeneity, including studies to address sex, gender, ethnic and/or racial differences.
• Elucidate how biomarkers (including genotype) are linked to disease phenotype or subtype.
• Develop novel preclinical models that recapitulate the phenotype of human disease

Diagnosis

• Identify and validate objective biomarkers to predict onset, response to therapy, disease complications and/or disease progression.
• Develop and validate improved diagnostic criteria and screening tools for early detection or to track disease progression.
• Determine the physiological impact related to diagnosis and/or timing of a diagnosis.

Treatment

• Develop and test pharmacological or nonpharmacological treatments, or improve upon existing treatments, especially those that will minimize side effects.
• Develop and test curative strategies to include tissue engineering, genetic approaches, or protein replacement.
• Develop and test interventions to improve neuropsychological outcomes and cognitive symptoms and other comorbidities as defined by those with lived experience.
• Develop and test strategies to support ongoing treatments during life transitions (i.e., pediatric to adult care).

Epidemiology

• Conduct population-based studies to identify risk (i.e., carrier status), lifestyle determinates of health or protective factors that influence onset, progression and/or outcomes.
• Conduct natural history/longitudinal studies to understand incidence, prevalence, and progression of the disease/condition and carrier and modifier gene status.
• Develop and validate research tools to collect, mine, and integrate real-world data (patient-reported data, longitudinal data, etc.) with electronic medical records to guide precision medicine approaches.
• Develop clinically relevant endpoints for clinical trials.