The FY21 SRP TRPA mechanism supports partnerships between clinicians, research scientists and other disciplines that will accelerate the movement of promising ideas in scleroderma into clinical applications. The ultimate goal of translational research is to move a concept or observation forward into clinical application that is relevant to active-duty Service Members, Veterans, other military beneficiaries, and the American public. This award supports the development of translational research collaborations between two or more investigators to address a central problem or question in scleroderma in a manner that would be less readily achievable through separate efforts.
Observations that drive a research idea may be derived from a laboratory discovery, populationbased studies, or clinician’s firsthand knowledge of patients and anecdotal data. However, members of the partnership should not view translational research as a one-way continuum from bench to bedside. The research plan must involve a reciprocal flow of ideas and information between basic and clinical science, reflecting an intellectual synergistic partnership between clinicians, research scientists and/or other disciplines.
Applicants are encouraged to leverage resources and expertise at the National Center for Advancing Translational Sciences (NCATS) to improve efficiency and accelerate the translational process. A list of NCATS programs and resources supporting pre-clinical and clinical innovation can be found at https://ncats.nih.gov/research.
The FY21 SRP TRPA requires multiple PIs. At least two, and up to three, PIs must partner in one overarching study. One partner in the collaboration must be a clinician. Multi-institutional partnerships are encouraged. At least one member of the partnership must have experience in either scleroderma research or scleroderma patient care as demonstrated by active funding and/or recent publications. A proposed project in which the partner merely supplies tissues or access to patients will not meet the intent of this award mechanism. One PI will be identified as the Initiating PI and will be responsible for the majority of the administrative tasks associated with application submission. The other PI(s) will be identified as a Partnering PI(s). All PIs should contribute significantly to the development of the proposed research project, including the Project Narrative, Statement of Work (SOW), and other required components. If recommended for funding, each PI will be named to an individual award within the recipient organization. For individual submission requirements for the Initiating and Partnering PIs, refer to Section II.D.2, Content and Form of the Application Submission. It is the responsibility of the PIs to describe how their combined expertise will better address the research question and explain why the work should be done together rather than through separate efforts.
Deadlines:
To be considered for funding, applications must address at least one of the following FY21 SRP Translational Research Partnership Award (TRPA) Focus Areas. Selection of the appropriate Focus Area(s) is the responsibility of the applicant.
• Define biomarkers (‘omics and/or molecular markers, cell subsets, imaging, patient-reported outcomes) that help inform therapeutic choices (immunosuppressive/anti-fibrotic) or predict course (morbidity) and quality of life.
• Utilize systems biology, multi-omics, and/or preclinical screening approaches (including but not limited to high-throughput screens, the development of animal models, three-dimensional tissue culture and/or organoids) with the intent to develop drug testing models in order to understand the heterogeneity of disease as well as to develop prevention and therapeutic interventions.
• Conduct studies of diverse populations to include the development of cohorts and identification of potential measures of patient outcomes to better understand the burden of the disease.
• Define functional role of epigenetic changes, multiple cell types, and molecules that mediate pathogenesis and/or initiate or propagate organ-specific disease activity using preclinical models and clinical samples.
• Conduct population-based or cohort studies to understand the prevalence, heterogeneity, and course of this disease, its manifestations, and its impact on health outcomes and activities for daily living. Examples include but are not limited to the following areas of emphasis. ○ Understand the unique burden of disease in diverse populations. ○ Understand disease heterogeneity (course of disease, prevalence, and associated factors). ○ Utilize disease registries linked to biological samples and high-quality clinical data and patient-reported outcomes. ○ Conduct fine phenotyping of clinical subsets to address heterogeneity.
• Understand and improve the impact of disease and its treatment on the patient’s experience and quality of life. Examples include but are not limited to the following areas of emphasis. ○ Develop interventions to improve coping with disease. ○ Identify main concerns of patients to inform development and validation of patientreported outcomes. ○ Understand the link between molecular laboratory, and clinical measures and the patient’s quality of life.
• Develop and validate short- and long-term organ-specific and composite clinical outcomes measures to determine treatment efficacy. Examples include but are not limited to the following areas of emphasis. ○ Develop better quantifiable and reproducible measures to assess clinical manifestations including skin, heart, Raynaud phenomenon, calcinosis cutis, or gastrointestinal tract morbidity in scleroderma. ○ Validate patient-reported outcome measurements to aid in the approval of drug and therapies. ○ Develop and validate intermediate biological/surrogate endpoints to support larger clinical proof-of-concept/proof-of-mechanism trials.
Additionally, an exploration of disease and/or population heterogeneity within the context of the proposed research is expected.
Independent investigators at all academic levels (or equivalent) are eligible to be named as a PI on the application. One partner in the collaboration must be a clinician (M.D., D.O., or equivalent) with clinical duties and/or responsibilities. At least one member of the partnership must have experience in either scleroderma research or scleroderma patient care as demonstrated by active funding and/or recent publications. An eligible PI, regardless of ethnicity, nationality, or citizenship status, must be employed by, or affiliated with, an eligible organization.
The anticipated direct costs budgeted for the entire period of performance for an FY21 SRP TRPA will not exceed $750,000. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.
