Deriving Drug Discovery Value from Large-Scale Genetic Bioresources Proceedings of a Workshop (2016) / Chapter Skim
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1 Introduction and Themes of the Workshop
1 Introduction and Themes of the Workshop
Pages 1-8
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One way to increase the odds of success for compounds in the drug development pipeline is to adopt genetically guided strategies for drug discovery. A recent analysis of approved medicines indicated that using human genetic data to support the selection of drug targets and indications can roughly double the chances that a given drug will be clinically successful compared with drugs lacking genetic support (Nelson et al., 2015)
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However, many questions remain about the design of large cohort studies, the types of data that should be collected, and which business models could engage stakeholders most effectively. To examine how genetic bioresources could be used to improve drug discovery and target validation, the Roundtable on Genomics and Precision Health (previously called the Roundtable on Translating Genomic-Based Research for Health)
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To examine enabling partnerships and business models that can facilitate the use of genetic data for drug discovery. Participants at the workshop explored the current landscape of genomics-enabled drug discovery activities in industry, academia, and government; examined enabling partnerships and business models; and considered gaps and best practices for collecting population data for the purpose of improving the drug discovery process.
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Cohort studies are especially valuable when they include data collected over long periods of time, have extensive population participation, and allow for genotype- or phenotype-based recall studies, according to Mark Daly, the co-director of the Program in Medical and Population Genetics at the Broad Institute of the Massachusetts Institute of Technology and Harvard University. In addition, integrated approaches that include general population studies, family-based studies, research on founder populations, and studies of phenotypic-specific cohorts could potentially maximize opportunities for drug discovery, said Aris Baras, the vice president and co-head of the Regeneron Genetics Center.
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. The CTTV brings together expertise in drug discovery, chemistry, functional genomics, and electronic health records to study potential drug targets in the precompetitive realm, said Lon Cardon, the senior vice president of alternative discovery and development and the head of target sciences at GSK.
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Business Models That Support Drug Discovery Across Stakeholder Groups Progress of drug discovery and development efforts that begin within a single stakeholder organization can be greatly accelerated when such efforts are taken advantage of by larger collaborations involving multiple stakeholder groups. For example, patient and disease advocacy groups can gather genotypic and phenotypic data for investigators at academic institutions or pharmaceutical companies, help recruit individuals into clinical trials, provide treatment recommendations, and educate patient populations (see Chapter 4)
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. The SGC brings together government agencies, academic scientists, philanthropic organizations, pharmaceutical companies, and others to provide resources for discovering and validating drug targets.
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Potential challenges associated with drug discovery using data collected from cohort studies include incomplete annotation of intergenic sequences and a lack of knowledge about the effects of rare variants. These benefits and challenges are discussed within the context of specific genomic cohort efforts in Finland, Iceland, an integrated translational medicine institute, a pharmaceutical company, and a direct-to-consumer genetics company.
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