Venture Philanthropy Strategies to Support Translational Research Workshop Summary (2009) / Chapter Skim
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1 Getting Started in Translational Research
1 Getting Started in Translational Research
Pages 9-20
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From page 9... ...
Creating novel mechanisms to support translational research is a large step, and for many voluntary health organizations it is a leap into the unknown. Several voluntary health organizations have spent decades in the core business of raising money and supporting basic research, providing direct patient care, supplying capital for facilities, or making small-scale individual academic grants.
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From page 10... ...
Richard Insel, executive vice president of research for the Juvenile Diabetes Research Foundation, offered four key points to consider: • Know your disease • Know yourself • Know your partners • Identify your goal KNOW YOUR DISEASE To know where to begin and what needs to be funded, voluntary health organizations need a strong understanding of their target diseases and conditions -- a map of their diseases. What is really known about the disease?
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From page 11... ...
Over time, developing ways to share this analysis and assessment with the broader field, constantly communicating new developments as they occur, becomes an important contribution in the grant funding that voluntary health organizations provide.
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From page 12... ...
Hood agreed and expressed that Parkinson's disease drug development is slowed not just because markers do not exist to definitively diagnose Parkinson's disease or measure its progression, but because biomarkers of a drug's effectiveness are also inadequate. For a voluntary health organization involved in translational research, investment in biomarkers is as important as investment in promising new therapeutics.
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From page 13... ...
Developing a translational research program requires many things from a voluntary health organization: commitment, expectation management, the readiness to constantly examine and reexamine progress and risk tolerance, and the willingness to change when things are not working. If an organization's culture is resistant to this process, it may not be the right time to move into funding translational research.
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From page 14... ...
"There is a huge expectation built there that needs to be managed because the vast majority of such studies will be failures," he explained. Expectations One of the biggest challenges that many voluntary health organizations face is managing expectations, too much hype, and too much overpromise, explained Insel.
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From page 15... ...
Voluntary health organizations are in a unique role in that they can provide an overview of the state of the research, in addition to providing insight into and education for the patient community. An organization's leadership and culture determine where the organization will tread, so it is important that everyone is on board.
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From page 16... ...
As Dennis Choi of Emory University highlighted, forming partnerships with academia, industry, government, and other voluntary health organizations is necessary in order to take a particular therapy all the way from idea to cure. Partnership management was a common theme throughout the workshop.
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From page 17... ...
Voluntary health organizations using venture philanthropy can help engage researchers and guide their efforts toward a "sweet spot," Insel said, where proof-of-concept trials begin as quickly as possible in order to remove as much risk as possible from the broad gap between basic research and clinical development. Ultimately, voluntary health organizations need to approach the process of translational research the same way pharmaceutical companies do.
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From page 18... ...
In his experience, Insel said that the Juvenile Diabetes Research Foundation learned that many of the assays that had been developed in the academic community had to be reformatted for compatibility with high-throughput screens. Thus, a voluntary health organization may choose to focus on validation.
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From page 19... ...
• Make a list of knowns and unknowns, and focus funding on address ing and overcoming the unknowns as efficiently as possible. Once there is sufficient basic research in place, it becomes a more attrac tive opportunity for the private sector to step in, continue research and development, and drive therapies into the clinic.
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