Improving the Efficiency and Effectiveness of Genomic Science Translation Workshop Summary (2014) / Chapter Skim
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5 Role of Advocacy in Facilitating Translation of Basic Scientific Research
5 Role of Advocacy in Facilitating Translation of Basic Scientific Research
Pages 31-38
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From page 31... ...
The ability to translate research results into outcomes for patients is urgently needed, and patient advocacy groups play a significant role in translating discoveries into patient treatments. Pat Furlong, founding president and chief executive officer of Parent Project Muscular Dystrophy (PPMD)
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From page 32... ...
Academic institutions have actually turned down funding because PPMD's board-directed cost policy could not be adjusted to provide additional funding for overhead expenses. As a result, the foundation has established its own lab to work on validating drug candidates so that it can reduce the risk on some of its investments.
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From page 33... ...
The TACT model is a comprehensive and integrated one that brings together experts from academia, regulatory agencies, industry, clinical practice, and basic science with parent advocates to discuss proposals in a confidential review setting and inform industry and academia about patient needs. The review process is more than that of a typical academic advisory committee, resulting in greatly increased coordination and credibility among nonprofit organizations, industry, and funders.
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From page 34... ...
The foundation will also identify and support drug candidates with the greatest likelihood of success, more quickly recruit subjects for trials by identifying potential subjects and supporting their participation, develop biomarkers and validate new functional endpoints, and encourage the use of centralized institutional review boards. The identification of markers in early disease would have the most impact for patients if the progression of the illness could be slowed by targeting these markers, Furlong said, but because the disease has so much clinical variability, there likely will not be one single drug to treat DMD.
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From page 35... ...
Partnerships for Drug Development Foundations have influenced pharmaceutical companies by encouraging them to embrace novel models for risk sharing, said Sherer. However, development of these models takes time, flexibility, and perseverance.
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From page 36... ...
The funding allowed target validation, animal testing, and a drug discovery effort that has yielded a series of molecules targeting a neurotransmitter receptor for glutamate. In 2012, Vanderbilt University announced a major collaboration with Bristol-Myers Squibb to move the therapy forward into the clinic.
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From page 37... ...
Advocacy groups are changing, said Sharon Terry, Roundtable co-chair and president and chief executive officer of Genetic Alliance. Young parents do not necessarily sign up for a foundation and send in $50 per year.
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From page 38... ...
He added that "we have highly motivated academic researchers that volunteer their time, take lower indirect costs, share their tools, share their data, and are happy and excited in their careers to be a part of this effort. It's [about]
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