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The Strategic Plan for Translational Research is composed of two parts: I. overcoming obstacles to pre-clinical therapy development; and II., overcoming obstacles to early phase clinical research for new drugs or new applications for pre-existing drugs.
I. Preclinical Therapy Development.
A. Conduct market analyses for diseases in MDA’s program
B. Develop a business plan to promote potential collaborations between industry and academia in the field of neuromuscular disease research
C. Provide access to regulatory consultants and educational services for all levels of the process of product development
D. Provision of funding mechanisms to further goal-oriented projects, including funding for contracts and infrastructure
E. Provision of resources for support and dissemination of critical animal models
F. Provision of resources for critical GMP manufacturing needs
G. Support for biomarker identification for small-molecule therapeutics, as early indicators of dosing, toxicity, and potential efficacy
II. Clinical Research Support.
A. Establishment of an MDA Data Safety Monitoring Board (DSMB) that can be utilized for MDA-funded trials
B. Development of nationally accepted and implemented study design and outcomes measurements for clinical trials, with FDA involvement, through:
- National databases
- Common protocols identifying a preferred patient class for each MD disease
- Natural history studies
- Clinical-trial working groups or consortia with statistical input
- Training for clinicians to learn specific efficacy tests (e.g., MUNE for measuring motor neuron function or QMT for strength testing)
C. Facilitation of nationally based clinical trials by supporting patient identification, enrollment, and participation, including a database of eligible patients willing to consider research protocols, and support for enrollment and participation costs
D. Establishment of a resource on ethics
- Create committee addressing ethical issues in clinical trials and produce a consensus paper
- Utilize committee members to review clinical trial protocols on a case-by-case basis
E. Development of physicians trained in clinical research through Clinical Research Training Grants.
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