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4 Business Models That Support Bioresource Discovery and Collaboration
Pages 39-48

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From page 39...
... (Ehm) Public–private partnerships with well-defined deliverables and timelines can facilitate the discovery and validation of new tar gets that companies can incorporate into their therapeutic devel opment programs; provide new insights into known, existing targets; enable a significant increase in the knowledge of tracta ble disease biology and disease pathways; and create a rich, comprehensive, integrated knowledge base that is easy to use and available to the entire global research community.
From page 40...
... Speakers in the third session were asked to examine potential precompetitive business models and investments that can support discovery efforts and opportunities across stakeholder groups. ENGAGING PATIENTS FOR DRUG DISCOVERY Pancreatic cancer is the only major type of cancer with a 5-year survival rate in the single digits and is a health threat in the United States and around the world, said Lynn Matrisian, the vice president of science and medical affairs at the Pancreatic Cancer Action Network (PanCan)
From page 41...
... Percentages correspond to the percent of total pancreatic cancers with mutations and/or copy number alterations in the indicated gene or pathway, and shaded ovals indicate possible therapies based on the molecular aberrations. SOURCE: Lynn Matrisian, National Academies of Sciences, Engineering, and Medicine workshop presentation, March 22, 2016.
From page 42...
... This system allows researchers to access another layer of information that supplements clinical records and thus creates opportunities for novel discoveries, Matrisian said. COLLABORATIVE APPROACHES TO GENOMIC DRUG DISCOVERY Utilizing EHR-Linked Biobanks for Drug Discovery Previous efforts to assess the impact of genetic variation on clinical phenotypes have relied on population studies performed through genetic analysis consortiums, said Meg Ehm, the director of external strategic alliances for genetics at GlaxoSmithKline (GSK)
From page 43...
... This analysis revealed that the variant was associated not only with a reduced risk of type 2 diabetes, but also with a reduced risk of coronary heart disease, thus providing supportive evidence that GLP1R agonists are not likely to be associated with an unacceptable increase in cardiovascular risk. The Genomic Resources for Drug Discovery Consortium grew out of enthusiasm about associating genetic variants with clinical outcomes, but participating companies also intend to work toward developing evidence that links potential drug targets to disease progression, predicting out
From page 44...
... , which brings high-level government, industry, and nonprofit foundation partners together to identify and validate the most promising biological targets for therapeutics. The specific goals of AMP are to discover and validate new drug targets that companies can incorporate into their therapeutic development programs; to provide new insights into known, existing targets; to enable a significant increase in knowledge of tractable disease biology and disease pathways; and to create a rich, comprehensive, integrated knowledge base that is easy to use and available to the entire global research community, said David Wholley, the director of research partnerships for FNIH.
From page 45...
... Though funding procedures and workflows have been complicated at times, AMP has succeeded in building a knowledge portal that provides aggregated human genetic data from more than 150,000 individuals across a wide range of ages and ethnicities, and has created tools within the portal to allow easy, integrated interrogation across multiple datasets while maintaining individual-level data privacy, he said. The challenges for this type of collaboration include both incentivizing investigators to share data and managing data restrictions, such as consent and regulations on exporting data out of countries, Wholley said.
From page 46...
... Finally, the consortium has used the expertise, chemical compound collections, and other resources made available through commercial partnerships to work on proteins or protein families that have been previously viewed as intractable or unusable in a drug development program. As an example of this approach, Bountra cited work on a family of proteins with a special motif called bromodomains, which were thought to be undruggable due to their complex biology and protein structures.
From page 47...
... It's the right thing to do for industry." There is hope that the creation of a new collaborative research ecosystem that involves the pharmaceutical industry, academic researchers, patients, government, and private funders, will generate many more novel medicines, Bountra continued. THE SUSTAINABILITY OF BUSINESS MODELS How can collaborative business models for drug discovery be sustainable over time?
From page 48...
... 48 USING GENETIC BIORESOURCES FOR DRUG DISCOVERY One shortcoming of public–private partnerships, Wholley said, is that few of them have clearly defined metrics for when enough work has been done on a topic or outline plans for how the partnership will prioritize and advance projects. Looking forward to future steps to ensure consortium relevance for the long term is also important.


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