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Enabling Precision Medicine The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop (2017) / Chapter Skim
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6 Reflecting Back and Looking Forward: Key Themes and Potential Next Steps in Genetics-Enabled Drug Development
Pages 83-92

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From page 83...
... In the final workshop session, Laura Nisenbaum, Russ Altman, Bray Patrick-Lake, and John Carulli, director of precision medicine at Biogen (at the time of the workshop) , summarized the key points from each of the four workshop panel sessions.
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From page 84...
... Development of Patient-Friendly Resources and Educational Materials Establishing new and improved educational resources, such as a userfriendly clinical trials database, could empower providers to identify genetic diseases, discuss clinical trials as treatment options, when necessary, and
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From page 85...
... This and other issues could be addressed collaboratively between trial sponsors and patient advocacy groups; accordingly, advocacy organizations could develop patient-friendly, graphical representations of complex topics or issues, such as genetic variation or benefit–risk assessment, that could be adapted to different diseases. Solicitation and Incorporation of Patient Input Patient engagement, David Leventhal said, affects all the main drivers of drug valuation, including cost, revenue, timing, risk, and intangibles.
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From page 86...
... ENPV modeling and similar approaches can support trial sponsor decisions to increase patient engagement throughout the drug development process. Return of Individual Genetic and Summary Results from Clinical Trials Many clinical research participants are interested in receiving the results of genetic testing performed during the course of research, Jane Perlmutter said.
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From page 87...
... Accrual and Retention Strategies To address the complexity of genetics-enabled clinical trials and the historical difficulties faced in clinical trial accrual and retention, trial sponsors are developing innovative methods for enhancing trial recruitment,
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From page 88...
... Novel Clinical Trial Designs to Maximize Evidence Generation The traditional approach for clinical trials -- studying one disease in one population using one treatment -- is neither amenable to nor sustainable for genetics-enabled drug development, said Brooks. Accordingly, sponsors have begun implementing novel designs for genetically enabled clinical ­ rials, such as umbrella and basket trials (e.g., Lung-MAP and STARTRK-2, t respectively)
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From page 89...
... COLLABORATION TO ADVANCE PRECISION MEDICINE Public–Private Partnerships The complex challenges associated with genetics-enabled drug development cannot be addressed by a single entity or organization, Nisenbaum said. There is a need for more interaction between biopharmaceutical companies, which often struggle with the economic and business cases for integrating genomics into drug development, and the foundations and patient advocacy groups that are gathering patients, genotyping them, and channeling them into clinical trials, a workshop participant said.
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From page 90...
... New and established innovative trial models might also help to lessen the risk of participation in precision medicine by companies, as the infrastructure for engaging in these models is already in place, PatrickLake said. 2  Workshop participants were referred to the proceedings of a prior Roundtable on Genomics and Precision Health workshop that discussed precompetitive collaboration for genomicsbased discovery in drug development (IOM, 2011)
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From page 91...
... This strategy might encourage participation from a more diverse patient population, and researchers could more extensively leverage real-world information in EHRs. Indeed, the PMI's All of Us Research Program will employ a multidisciplinary approach, noted Burchard, that ­ will include genetic testing for potential use in genetics-enabled studies or to increase the genetic diversity of clinical trial populations.
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Key Terms

  • precision medicine
  • clinical trials
  • clinical trial
  • patient engagement
  • trial sites

  • trial designs
  • diverse populations
  • clinical drug
  • advance precision
  • genetic testing

  • causal biology
  • rare diseases
  • trial sponsors
  • patient advocacy
  • patient input

  • patient populations
  • genetic diversity
  • ical research
  • genetic diseases
  • launching trial

  • trial populations
  • enabled clinical
  • trial accrual
  • including diverse
  • screening panel

  • laura nisenbaum
  • operationalize data
  • innovative methods
  • biopharmaceutical companies
  • maximize evidence


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