Department of Defense Grant: Innovative Duchenne Muscular Dystrophy Idea Development Award

The FY24 DMDRP IDA promotes new ideas that are still in the early stages of development and have the potential to yield impactful data and new avenues of investigation. This award supports conceptually innovative, high-risk/high-reward research that could lead to critical discoveries or major advancements that will accelerate progress in improving outcomes for individuals with DMD. Applications should include a well-formulated, testable hypothesis based on strong scientific rationale.

New Investigators: The FY24 DMDRP IDA mechanism encourages applications from independent investigators in the early stages of their careers (i.e., within 10 years of their first faculty appointment or equivalent) or applications from established investigators new to DMD research.

• The New Investigator – Early Stage category is designed to allow applicants early in their faculty appointments to compete for funding separately from established investigators.

• The New Investigator – Transitioning category is designed to allow investigators in an area other than muscular dystrophy, at or above the level of Assistant Professor, seeking to transition to a career in DMD, thereby bringing their expertise to the field.

Applications from New Investigators and Established Investigators will be peer and programmatically reviewed separately. Principal Investigators (PIs) using the New Investigator – Early Stage category or New Investigator – Transitioning category are strongly encouraged to strengthen their applications by collaborating with investigators experienced in DMD research and/or possessing other relevant expertise. It is the responsibility of the applicant to describe how the included collaboration will augment the PI’s expertise to best address the research question. All applicants for the New Investigator categories must meet the specific eligibility criteria described in Section II.C, Eligibility Information.

Preliminary data relevant to DMD that supports the feasibility of the research hypotheses and research approaches are required. Preliminary data may include unpublished results from the laboratory of the PI, research team or collaborators named on the application.

Key elements of this award are as follows:

• Innovation: Research deemed innovative may introduce a new paradigm, challenge current paradigms, look at existing problems from new perspectives, or exhibit other uniquely creative qualities.

• Impact: Research that has high potential impact may lead to major advancements and significantly accelerate progress toward improving outcomes for individuals with DMD.

It is the responsibility of the PI to clearly and explicitly articulate the project’s innovation and its potential impact on DMD. The project’s impact to both DMD research and to individuals with DMD should be articulated, even if clinical impact is not an immediate outcome. Applications that demonstrate exceptional scientific merit but lack innovation and high potential impact do not meet the intent of the IDA.

Clinical trials are not allowed under this funding opportunity.

A clinical trial is defined in 45 CFR 46.102 as a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include a placebo or another control) to evaluate the effects of the interventions on biomedical or behavioral health-related outcomes.

Studies that do not seek to measure safety, effectiveness, and/or efficacy outcome(s) of an intervention are not considered clinical trials.

For the purposes of this funding opportunity, research that meets the definition of a clinical trial is distinct from clinical research. Clinical research encompasses research with human data, human specimens, and/or interaction with human subjects. Clinical research is observational in nature and includes:

(1) Research conducted with human subjects and/or material of human origin such as data, specimens, and cognitive phenomena for which an investigator (or co-investigator) does not seek to assess the safety, effectiveness, and/or efficacy outcomes of an intervention. Research meeting this definition may include but is not limited to: (a) mechanisms of human disease, (b) diagnostic or detection studies (e.g., biomarker or imaging), (c) health disparity studies, and (d) development of new technologies.

(2) Epidemiologic and behavioral studies that do not seek to assess the safety, effectiveness, and/or efficacy outcomes of an intervention.

(3) Outcomes research and health services research that do not fit under the definition of clinical trial.

Excluded from the definition of clinical research are in vitro studies that utilize human data or specimens that cannot be linked to a living individual and meet the requirements for exemption under §46.104(d)(4) of the Common Rule.