Report of the Director National Institutes of Health Fiscal Years 2012 & 2013 Report of the Director National Institutes of Health Fiscal Years 2012 & 2013 Preface This is the fourth National Institutes of Health (NIH) Biennial The chapter begins with a brief introduction describing Report, which is required by Section 403 of the Public the full continuum of biomedical research at NIH. The Health Service (PHS) Act. Appendix A provides the language research continuum moves from basic research to in the PHS Act that is relevant to this report. NIH’s goal is for preclinical translational research, clinical research, and this report to serve as a useful reference for understanding finally postclinical translational research. In partnership NIH activities and operations, and the agency welcomes with the other agencies of the U.S. Department of Health feedback on it. and Human Services (HHS), NIH aims to bring the rich evidence base of its research into clinical and community practice, ultimately turning discovery into health. The path Chapter Organization in the continuum is not strictly linear, because all steps of Chapter 1 opens with a statement from the Director of biomedical research can inform and relate to other areas. NIH assessing the state of biomedical and behavioral research. It then describes NIH structure, policies, and The introduction is followed by a summary of the NIH procedures, focusing on the operations of the extramural research portfolio relating to these research stages, across and intramural research programs; mechanisms for strategic all of the Institutes and Centers (ICs) and Office of the planning (including the activities and processes of the Director (OD) program offices. The summary includes Division of Program Coordination, Planning, and Strategic specific examples that illustrate how NIH research at each Initiatives and its management of the Common Fund); and stage of the continuum augments human knowledge and various cross-cutting activities not covered in the chapters improves public health. Chapter 2 also describes how that follow, such as programs that provide the platform NIH ensures the uptake of research results by clinical for discovery, including training and career development practitioners and the public. The chapter concludes with activities and science literacy efforts. a description of NIH-funded research technologies, which provide innovative tools that are used within multiple steps Chapter 2 provides an overview of the NIH research in the continuum and often provide the means for exchange portfolio. The chapter includes the following sections: of information. • Identifying Public Health Needs—Epidemiology Chapter 3 addresses NIH research activities from the perspective of diseases, disorders, and adverse health • Basic Research conditions. The chapter includes the following sections: • Preclinical Translational Research • Cancer • Clinical Research • Neuroscience • Postclinical Translational Research • Life Stages, Human Development, and Rehabilitation • Information at the Service of Health • Chronic Diseases and Organ Systems • Harnessing Technology ii Preface • Autoimmune Diseases The appendices present reference documents and supporting data: • Infectious Diseases and Biodefense • Public Health Emergency Preparedness • Appendix A provides excerpts from the PHS Act that set the legal mandate for this Biennial Report and the • Minority Health and Health Disparities inclusion of certain content within it. These topics, many of which are categories specified in • Appendix B contains excerpts of the Report of the the PHS Act (see Appendix A), are grouped together to Advisory Committee on Research on Women’s Health. address the intent of the statute by presenting information on diseases, disorders, and adverse health conditions in • Appendix C provides the Common Fund Strategic a standardized format. Each topic is addressed in a Planning Report, 2013. separate section. • Appendix D lists and provides links to the missions and strategic plans of the NIH ICs and the missions of the OD Chapter 4 addresses NIH Centers of Excellence, which program offices. are diverse in focus, scope, and origin. The NIH Centers of Excellence described in this report are a subset: those • Appendix E consists of data on the primary NIH research established by statutory mandate. This chapter provides training program, the National Research Service Award overviews, progress reports for Fiscal Years (FYs) 2012 and program, the National Library of Medicine training 2013 (covering programmatic and research activities and programs, and NIH graduate medical education activities. outcomes), recommendations, evaluation plans, and future • Appendix F contains excerpts of Monitoring Adherence directions for the six congressionally mandated NIH Centers to the NIH Policy on the Inclusion of Women and of Excellence programs, which are described in the order of Minorities as Subjects in Clinical Research. their establishment: • Appendix G provides catalogs of disease registries and • Alzheimer’s Disease Centers (1984) other data systems. • Claude D. Pepper Older Americans Independence • Appendix H provides information on actions undertaken Centers (1989) to carry out scientific frameworks on recalcitrant cancer. • Senator Paul D. Wellstone Muscular Dystrophy • Appendix I lists NIH funding levels for chronic diseases Cooperative Research Centers (2001) and organ systems. • National Institute on Minority Health and Health • Appendix J contains a list of acronyms that are used in Disparities Centers of Excellence (2001) this Biennial Report. • Rare Diseases Clinical Research Network (2003) • Autism Centers of Excellence (2006) Report of the Director, National Institutes of Health FYs 2012 & 2013 iii Contents Preface ............................................................................................................................................................................................... i Chapter 1: About NIH ..................................................................................................................................................................... 1 Statement of the Director ................................................................................................................................................................ 1 NIH’s Mission .................................................................................................................................................................................. 4 Overview of NIH Structure and Organization ................................................................................................................................... 5 Extramural and Intramural Research Programs ............................................................................................................................... 8 Extramural Research Program .................................................................................................................................................... 8 Intramural Research Program ................................................................................................................................................... 11 Providing the Platform for Discovery ............................................................................................................................................. 13 Research Training and Career Development .............................................................................................................................. 13 Science Education and Literacy ................................................................................................................................................ 22 Chapter 2: Overview of NIH Research ........................................................................................................................................ 25 Introduction .................................................................................................................................................................................. 25 Identifying Public Health Needs – Epidemiology ............................................................................................................................ 27 Basic Research ............................................................................................................................................................................ 34 Preclinical Translational Research .................................................................................................................................................50 Clinical Research .......................................................................................................................................................................... 53 Postclinical Translational Research ................................................................................................................................................ 59 Information at the Service of Health .............................................................................................................................................. 62 Harnessing Technology.................................................................................................................................................................71 Chapter 3: Research in Diseases, Disorders, and Health Conditions ....................................................................................