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Introduction1

The process for translating basic science discoveries into clinical applications has historically involved a linear and lengthy progression from initial discovery to preclinical testing, regulatory evaluation and approval, and, finally, use in clinical practice. Despite significant advances in the understanding of human disease and clinical pharmacology, several stakeholders have recently expressed concern over the declining productivity of the translation of basic scientific discoveries into the practice of clinical medicine and the substantial cost of this process (Harrison et al., 2012). The low rate of translation from basic science to clinical application has been a source of frustration for many scientists, clinicians, investors, policy makers, and patients who hoped that investments in research would result in improved products and processes for patients (Mankoff et al., 2004). Although this is a general problem for scientific discoveries in the current translational pathway, these concerns also apply to genomic science. Specifically, some feel that the anticipated deliverables from the Human Genome Project have not yet materialized, and although understanding of human health and disease biology has increased, there has not been a

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1The planning committee’s role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and are not necessarily endorsed or verified by the Institute of Medicine, and they should not be construed as reflecting any group consensus.



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1 Introduction1 The process for translating basic science discoveries into clinical appli- cations has historically involved a linear and lengthy progression from initial discovery to preclinical testing, regulatory evaluation and approval, and, finally, use in clinical practice. Despite significant advances in the understanding of human disease and clinical pharmacology, several stake- holders have recently expressed concern over the declining productivity of the translation of basic scientific discoveries into the practice of clinical medicine and the substantial cost of this process (Harrison et al., 2012). The low rate of translation from basic science to clinical application has been a source of frustration for many scientists, clinicians, investors, policy makers, and patients who hoped that investments in research would result in improved products and processes for patients (Mankoff et al., 2004). Although this is a general problem for scientific discoveries in the cur- rent translational pathway, these concerns also apply to genomic science. Specifically, some feel that the anticipated deliverables from the Human Genome Project have not yet materialized, and although understanding of human health and disease biology has increased, there has not been a 1  The planning committee’s role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants, and are not necessarily endorsed or verified by the Institute of Medicine, and they should not be construed as reflecting any group consensus. 1

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2 EFFICIENCY AND EFFECTIVENESS OF GENOMIC SCIENCE TRANSLATION concomitant increase in the number of approved drugs for patients over the past 10 years.2 One of the major obstacles to translation is the lack of incorporation of ideas about clinical applications during the early stages of develop- ment of research projects. Not only is knowledge from outcomes and clinical or public health needs not routinely incorporated into the design of basic scientific research, but also the process of exchanging discoveries between basic and clinical scientists is not well defined. Improved com- munication to allow for clinical and patient needs to inform the priorities of basic science research can offer a clearer path toward improving clinical application. The realignment of academic incentives, the detection of inno- vative ways to fund translational research, and the generation or identifica- tion of alternative models that accurately reflect human biology or disease provide opportunities to work across sectors to advance the translation of genomic discoveries. To foster collaboration and the exchange of ideas among stakeholders and to improve the utilization of genomic research for practical applications, the Roundtable on Translating Genomic-Based Research for Health hosted a workshop on December 3, 2012, in Irvine, California, to explore ways to improve the efficiency and effectiveness of the translation of genomic science to clinical practice. The workshop convened academic researchers, industry representatives, policy makers, and patient advocates to explore obstacles to the translation of research findings to clinical practice and to identify opportunities to support improvement of the early stages of the process for translation of genetic discoveries (see the workshop objectives in Box 1-1).3 Workshop GOALS Wylie Burke, Roundtable co-chair and professor and chair of the department of bioethics and humanities at the University of Washington, stated that although the Roundtable has spent a significant amount of time addressing issues in genomic medicine, such as the implementation of genome sequencing in the clinic and related regulatory and evidentiary 2  In 2012, 39 new drugs were approved, which was a 16-year high. But as of October 8, 2013, the data suggest that there will be fewer new drug approvals in 2013 (at this point there were only 18: see Summary of New Drug Approvals and Receipts, 1938 to the Present, http://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/Summaryof NDAApprovalsReceipts1938tothepresent/default.htm and New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products of 2013, http://www.fda.gov/drugs/ developmentapprovalprocess/druginnovation/default.htm [accessed October 8, 2013]). 3  The workshop agenda, speaker biographical sketches, full statement of task, and registered attendees can be found in Appendixes A–D.

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INTRODUCTION 3 BOX 1-1 Workshop Objectives •  o examine how basic science can best be positioned to foster the successful T translation of early genomic discoveries. •  o explore the challenges to and identify potential opportunities for improving T the efficacy of the translation process. •  o define pathways for moving innovative basic science forward. T issues, the focus of the discussion at the workshop would be on the integra- tion of progress in basic science research with the translational pathway for clinical implementation. Burke specifically noted that the challenges, b ­ arriers, and opportunities would be key interdisciplinary topics for conver- sation and that the successful integration of research discoveries and clinical medicine relies on the communication and expertise of a diverse group of stakeholders in the process. The report that follows is a summary of the workshop and highlights the main discussion points that the workshop speakers and participants presented and considered. Chapter 2 introduces several of the broad issues associated with the process of the translation of research findings into improvements in health care. Chapter 3 focuses on how basic science research could be structured to promote translation. Chapter 4 examines the roles of industry and venture capital in the movement of discoveries along a translational pathway to the marketplace. Chapter 5 explores the role of advocacy groups in catalyzing translation, in part through the convening of the many stakeholders involved in the process. Finally, Chapter 6 provides additional discussions of data access, the management of the research enterprise, and the incentives that contribute to or hinder the translation of basic science research.

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