NIH-Wide Strategic Plan Fiscal Years 2016-2020: Turning Discovery Into HealthTo establish a framework for carrying out its mission and optimize return on public investment, NIH’s strategy will focus on four essential, interdependent objectives [documented as goals in this StratML rendition]. These objectives are: advance opportunities in biomedical research, foster innovation by setting NIH priorities, enhance scientific stewardship, and excel as a federal science agency by managing for results. National Institutes of HealthNIH_8c9e7852-57cc-11df-8407-a5537a64ea2aThe National Institutes of Health (NIH) is the United States' premier agency for biomedical research, which spans the broad spectrum of basic, translational, clinical, behavioral and social sciences research dealing with many aspects of biology and almost every human disease and disability.Francis S. Collins, M.D., Ph.D.Director, National Institutes of HealthDepartment of Health and Human ServicesNIH is an operating division of the Department of Health and Human Services (HHS), responsible for helping the Department realize its strategic goal of advancing scientific knowledge and innovation. NIH ICsTo accomplish this, NIH consists of 27 Institutes and Centers (ICs), along with Program Offices, which collectively are referred to as ICOs.NIH Program OfficesThese ICOs have individual strategic plans and specific research agendas, which are aligned with the legislative mandates that are often related to specific diseases or body systems. To support these missions, most of NIH's ICOs receive a specific appropriation from Congress, and support research and research training through extramural funding awarded to universities, academic health centers, and other research institutions. Most also conduct research and research training in their own intramural laboratories, the majority of which are located on the NIH's main campus in Bethesda, MD.NIH Grant RecipientsIn Fiscal Year (FY) 2014, NIH reviewed more than 51,000 research project grant (RPG) applications and awarded approximately 10,000 new and competing RPGs to institutions/organizations to support specific projects performed by designated investigators in areas representing their research interests and competencies. The average duration of an NIH grant award is about 4 years; funding the out years of a multi-year award is predicated on submission of an acceptable annual progress report. The total number of active grants in FY 2014 was more than 47,000.NIH Strategic Planning StaffThe NIH Office of the Director would like to thank the following stakeholders, committees, and staff for their time and effort in helping to develop this strategic plan: Stakeholder Communities of NIH; NIH Advisory Committee to the Director; Institute, Center, and Office Directors; NIH Director's Executive Committee ...NIH OD StaffCarla GarnettJill GeorgeNirupa GoelRebecca KolbergTara SchwetzNIH Strategic Plan Working GroupRonit AbramsonHugh AuchinclossElizabeth BadenMichelle BennettLaura BerksonJuliana BlomeDavid BochnerMaureen BoyleRosalina BrayPenny BurgoonJohn BurklowAndrew BurtonAnna CalcagnoCindy CaughmanLaura ColeChristine CooperNed CulhaneChristine CutilloJessica EscobedoMeredith FoxBekah GeigerTaylor GillilandSarah GlavinJohn GrasonPamela HarrisRebecca HawesGene HayungaJill HeemskerkJennifer HobinJonathan HorsfordLisa KaeserMary Beth KesterWendy KnospEllen LibermanJaron LockettJane LockmullerKarin LohmanDwayne LunsfordSheila NewtonMorgan O'HayreMarie ParkerAmy PattersonWilma Peterman CrossStephane PhilogenePatricia PowellMichele RankinBarbara RappSarah RhodesSamir SaumaPeggy SchnoorElka ScordalakesPaul ScottAllan ShippKeisha ShropshireEllen SilvaMegan SinghRachel SturkeKimberly Thigpen-TartSanta TumminiaDave VannierSusan VasquezSamantha WhiteBetsy WilderBridget Williams-SimmonsCarrie WolinetzBuck WongScientific discoveries lead to improved health. _2ab0a988-8aa7-11e7-a015-e3bd1f25b93bTo seek fundamental knowledge about the nature and behavior of living systems and to apply that knowledge to enhance health, lengthen life, and reduce illness and disability._2ab0b112-8aa7-11e7-a015-e3bd1f25b93bPeer ReviewNIH has long relied upon a multifaceted approach for funding decisions that involves peer review by scientific experts to determine scientific merit of a research proposal, review for program priority by a second set of scientific experts and thought leaders from the lay public-serving on ICO national advisory councils, individual ICO strategic plans, and, ultimately, the scientific expertise of ICO Directors, informed by their staff. NIH will continue and strengthen its commitment to a transparent, evidence-based process that encompasses these action-oriented principles: enhance the nimbleness needed to meet public health needs and capitalize upon scientific opportunity, using new portfolio analysis tools; incorporate burden of disease as an important, but not sole, factor; take advantage of opportunities presented by rare diseases to advance research; and consider the value of permanently eradicating a disease. TransparencyGoing forward, NIH will take additional steps to enhance the transparency of its decision process by making public a standard metric for funding each year. NIH will also harmonize approaches to decision making by ensuring ICOs set their individual paylines -- the funding cutoff point for grant applications based solely upon peer-review scores -- to provide maximum flexibility for use of the select pay option. Select pay refers to funds set aside to support grant applications that, based upon scores from peer review, do not fall within the payline, but that fill an important research gap and/or are of particular programmatic relevance to an ICO's scientific and health priorities. Final decisions on the use of select pay are made by the NIH ICO Directors following discussions with their Advisory Councils and appropriate ICO staff. NimblenessEnhance Nimbleness. NIH and all of its ICOs will nurture the nimbleness necessary to shift resources in response to unexpected scientific breakthroughs, to capitalize on scientific opportunities on the horizon, and to address emerging public health needs. Advancing human health requires taking advantage of scientific opportunities as they arise. It is important to recognize that different scientific fields mature at different rates, and the same amount of funding in two fields can lead to very different scientific returns.ReproducibilityReproducibility of research resultsDiversityFairnessCommunicationCoordinationTrans-DisciplinarityKnowledgeCollaborationBiomedical ResearchAdvance Opportunities in Biomedical Research_2ab0b428-8aa7-11e7-a015-e3bd1f25b93b1NIH CollaboratorsNIH encourages and, in many cases, collaborates with researchers from both the private and public sectors, including other HHS divisions, science agencies, philanthropic foundations, academia, and industry, to advance its mission of improving human health.HHS DivisionsScience AgenciesPhilanthropic FoundationsAcademiaIndustryFederal Science AgenciesAmong the federal science agencies that NIH often coordinates and works closely with are the Centers for Disease Control and Prevention (CDC), the Food and Drug Administration (FDA), the Agency for Healthcare Research and Quality (AHRQ), the HHS Office of the Assistant Secretary for Preparedness and Response (ASPR), the National Science Foundation (NSF), the Department of Energy (DOE), and the Defense Advanced Research Projects Agency (DARPA). Centers for Disease Control and Prevention (CDC)Food and Drug Administration (FDA)Agency for Healthcare Research and Quality (AHRQ)HHS Office of the Assistant Secretary for Preparedness and Response (ASPR)National Science Foundation (NSF)Department of Energy (DOE) Defense Advanced Research Projects Agency (DARPA)Over the next 5 years, NIH will capitalize upon a broad range of cross-cutting opportunities to move biomedical research forward in three highly important, interdependent areas: exploration of fundamental science, discovery of treatments and cures, and advancement of health promotion and disease prevention. These activities will be catalyzed by new approaches, strongly supported by NIH, that are aimed at speeding discovery across the biomedical research enterprise. This includes efforts to promote increased data sharing, to enhance the ability of scientists to pursue interdisciplinary studies, and to enable new types of partnerships... Health promotion and disease prevention clearly represent a critical facet of the NIH mission and its aim of improving the health of whole populations. It is imperative that NIH act upon opportunities to advance this vital area, which is complementary to the discovery of treatments and is integral to the entire biomedical research continuum.Fundamental ScienceExploration of fundamental science._2ab0b5ea-8aa7-11e7-a015-e3bd1f25b93b1.1Joseph GoldsteinIn the early 1970s, NIH-supported basic researchers Joseph Goldstein and Michael Brown studied families with very high cholesterol levels and discovered that cells have low-density lipoprotein (LDL) receptors that remove cholesterol from the blood. That Nobel Prize-winning work, coupled with the NIH-funded Framingham Heart Study’s landmark 1961 finding that high blood cholesterol is a major cardiovascular disease risk factor, set the stage for the first cholesterol-lowering statin drug in 1987. Michael BrownDavid BaltimoreLikewise, NIH-funded basic research was instrumental in the development of zidovudine (AZT), the first anti-retroviral drug approved for treating the human immunodeficiency virus (HIV)/acquired immune deficiency syndrome (AIDS). The drug was developed in the early 1960s to treat cancer, but failed to show efficacy. It faded from view until 1985, when others thought of using AZT as an AIDS drug because of its ability to inhibit reverse transcriptase, an enzyme that HIV uses to replicate. Reverse transcriptase was discovered by basic virology research several years before AIDS was identified. In 1975, NIH grantees David Baltimore and Howard Temin shared a Nobel Prize in Physiology or Medicine for that work. Howard TeminMartin ChalfieBasic innovation is also essential for the advancement of fundamental science because new technologies and methods can open whole new areas of scientific inquiry. In a tale of discovery spanning more than three decades and culminating in the 2008 Nobel Prize in Chemistry, NIH grantees Martin Chalfie, Osamu Shimomura, and Roger Tsien discovered a green fluorescent protein (GFP) in jellyfish and went on to develop GFP into a key tool for observing biological processes that were previously invisible to researchers. Osamu ShimomuraRoger TsienGenetic ResearchersMore recently, researchers have developed revolutionary customizable, gene-editing tools, such as CRISPR/Cas9. These technologies are enabling efforts to study genes in specific, targeted ways, often in real timeTechnologistsOn the other hand, new scientific challenges can inspire the creation of new technologies.Human Connectome ProjectCompelling recent examples of this innovative force in action include NIH's Human Connectome Project and the multi-agency Brain Research through Advancing Innovative Neurotechnologies® (BRAIN) Initiative, in which NIH plays a leadership role.Brain Research through Advancing Innovative Neurotechnologies® (BRAIN) InitiativeWith more than 100 billion cells and 100 trillion connections, the human brain remains one of science’s most daunting frontiers and one of medicine’s greatest challenges. To revolutionize understanding of how the brain enables the body to record, process, utilize, store, and retrieve vast quantities of information, BRAIN is supporting development of entirely new technologies, including some with the potential to benefit many other areas of biomedical research, such as single-cell analysis methods. NeuroscientistsEngineers, computer scientists, nanotechnologists, physicians, and neuroscientists will use these and other leading-edge technologies to work together to achieve BRAIN’s goal of measuring real-time cognition, emotion, perception, and behavior at the scale of complex neural networks in living organisms -- all at the speed of thought. A bold plan for the BRAIN initiative, extending over a 12-year period, was recently put forward by an expert working group of neuroscientists, and serves as the current blueprint for this project. Ultimately, the foundation of understanding developed by the BRAIN Initiative® will help reveal the underlying pathology in a vast array of brain disorders and provide new therapeutic avenues to treat, cure, and prevent neurological and psychiatric conditions, such as Alzheimer's disease, autism, schizophrenia, depression, epilepsy, and addiction. EngineersComputer ScientistsNanotechnologistsPhysiciansTo achieve its mission, NIH must support the many types of fundamental scientific inquiry that are so essential to the progress of biomedicine. Fundamental science includes basic biological research that generates the knowledge of how living systems work at the molecular, cellular, and organismal level. Such knowledge is the foundation for translational and clinical studies that, over time, can lead to major medical advances. Because the private biopharmaceutical sector funds only a limited amount of basic research, NIH-supported research serves as the world’s leading source of foundational knowledge of relevance to both the public and private sectors of biomedicine. History shows that major biomedical advances frequently spring from unexpected sources. As anyone familiar with the story of penicillin's discovery knows, it is impossible to predict exactly what a basic researcher may uncover and what positive health benefits may eventually arise from such fundamental discoveries. Furthermore, no one can foresee what threads of foundational knowledge will be woven together to produce a new breakthrough, which could open up entirely new fields or pave the way for new technology that will enable researchers to tackle questions once beyond the reach of biomedical science. Most of the examples cited in this section are those in which a basic science discovery has led to a significant clinical advance -- an advance that could not have been foreseen at the time of the original basic research. One dramatic example is the story of how fundamental advances in cell biology led to development of a class of drugs widely used to lower the risk of cardiovascular disease. DataEncourage rapid, open sharing of data._2ab0b996-8aa7-11e7-a015-e3bd1f25b93b1.1.1Data ScientistsData science also holds tremendous potential, not only for enhancing the efficiency of the conduct of science, but also for increasing the impact of fundamental science, along with many other areas of biomedical research. To this end, NIH will serve as a focal point for catalyzing this historic research opportunity, continuing to leverage its roles as an influential convener and major funding agency to encourage rapid, open sharing of data and greater harmonization of scientific efforts.HarmonizationEncourage greater harmonization of scientific efforts._2ab0bc34-8aa7-11e7-a015-e3bd1f25b93b1.1.2Big DataSupport of research to address computational challenges in accessing, managing, analyzing, integrating, and mining the huge amounts of data._2ab0be1e-8aa7-11e7-a015-e3bd1f25b93b1.1.3Biomedical ScientistsNIH will also maintain and expand its support of research aimed at addressing new computational challenges in accessing, managing, analyzing, integrating, and mining the huge amounts of data, often referred to as "Big Data," being generated by biomedical scientists.SimulationConduct more experiments via computer simulation._2ab0c292-8aa7-11e7-a015-e3bd1f25b93b1.1.4One hope is that advances in bioinformatics and computational biology will lead to basic researchers conducting more experiments via computer simulation (in silico), with the ensuing results being used to generate and test novel hypotheses that will be rapidly shared with the broad research community.PortfolioSupport a broad, balanced portfolio of basic research across a wide range of scientific disciplines._2ab0c5a8-8aa7-11e7-a015-e3bd1f25b93b1.1.5From FY 2016-2020, NIH will support a broad, balanced portfolio of basic research across a wide range of scientific disciplines, a portfolio that will be complemented by vigorous support of innovations in technology and data science. By maintaining and strengthening its already impressive foundation of fundamental science, biomedical research will be poised to identify and capitalize upon potential opportunities for revolutionary breakthroughs with the potential for preventing, treating, and curing disease.Treatments & CuresDiscover treatments and cures._2ab0c7c4-8aa7-11e7-a015-e3bd1f25b93b1.2To achieve its mission, NIH is strongly committed to supporting the process of turning advances in fundamental scientific knowledge into treatments and cures. When integrated with existing knowledge about cells, systems, and organisms, insights generated by this innovative work will provide a new conceptual framework for therapeutic development that is based on a deeper understanding of biological systems and how, depending upon context, these complex mechanisms interact to influence health and disease.Diseases & TherapiesUnderstand the bases of diseases and identify potential therapeutic targets._cb02470c-8b9c-11e7-b76f-edbffedb5b8c1.2.1This process begins with basic research discoveries in biology, disease, or behavior that serve to further understanding of the basis of a disease and to identify potential therapeutic targets. Cell or tissue samples, animal models, and/or computer simulations are then used to design and test candidate approaches for diagnostics, devices, treatments, and/or cures. If the candidate approaches prove to be safe and effective in this pre-clinical testing, the experimental treatments and/or cures are then moved into human clinical trials, where they are tested for safety and efficacy. It must be emphasized that advances in these areas are closely interconnected and often do not progress in a linear manner. In fact, sometimes the process even circles back on itself in a "virtuous cycle," with applied research informing new ideas in basic research.Randomized TrialsEnhance the speed and efficiency of randomized trial._cb0253f0-8b9c-11e7-b76f-edbffedb5b8c1.2.2The randomized trial is the gold standard by which clinical researchers determine the safety and/or effectiveness of interventions that are thought to have potential to improve human health. NIH, which currently devotes approximately 10% of its budget to supporting clinical trials, has a distinguished history of funding landmark trials that have led to a wide spectrum of interventions. Such interventions have included coronary bypass surgery, treatments for breast cancer, lifestyle improvements to prevent diabetes, approaches for lowering blood pressure, screening methods for lung cancer, hormone replacement therapy in postmenopausal women, and anti-retroviral drugs in people with, or at high risk for, HIV infection. In recent years, NIH developed an increasing interest in fostering approaches to enhance the speed and efficiency with which trials are conducted, as well as to learn more about the role of "pragmatic trials," which are trials of direct interest to patients and clinicians.Molecular CommonalitiesStudy disease commonalities at the molecular level._cb025b48-8b9c-11e7-b76f-edbffedb5b8c1.2.3Alzheimer's PatientsFor example, thanks to an innovative public-private partnership, an experimental drug originally developed to fight cancer is now being tested for Alzheimer's disease (AD) in human clinical trials. The compound, called saracatinib, is particularly exciting because it acts through a different mechanism than other AD experimental therapies.Traditionally, diseases have been researched and treated within an organ-based framework, e.g., diseases of the heart, the eye, the gastrointestinal tract, and so forth. Today, thanks to fundamental research, researchers have learned that many apparently different diseases have commonalities at the molecular level. These molecular similarities have led us to think in new ways about the roots of disease and open the door to identifying therapies that work across different organ systems and disease states. These shifts in thinking have profound implications for the future of scientific research and, ultimately, for the future of medicine. Tools and technologies that offer opportunities to screen rapidly for similarities among seemingly disparate diseases, as well as seemingly disparate drugs, are providing opportunities to repurpose existing drugs for use in conditions other than those for which they were originally developed.Rare & Unique DiseasesStudy rare and unique diseases._cb0264e4-8b9c-11e7-b76f-edbffedb5b8c1.2.4As important as this new emphasis on cross-cutting molecular mechanisms may be, there remains much that can be learned by studying the rare or unique. Especially for rare diseases caused by mutations in a single gene, the identification of a specific molecular defect through DNA sequencing can point directly to possible treatment strategies. Still, even in such cases, the road from discovery to treatment may be long, as evidenced by the two decades between discovery of the gene for cystic fibrosis (CF) and FDA approval of the first drug that directly affects a CF-causing molecular defect. For more common disorders, finding rare individuals carrying a genetic protective factor can provide critical clues to new therapeutic strategies. For example, a search for genes involved in cholesterol metabolism turned up a few healthy individuals with a rare gene variant that leads to very low levels of cholesterol and a very low incidence of cardiovascular disease. Further studies showed this gene variant reduces production of a protein called PCSK9, setting off a race among pharmaceutical firms to develop a new class of drugs that lower cholesterol by blocking this protein. Many experts think there are more such drug targets out there waiting to be discovered through molecular characterization and stratification of common diseases and disease risk factors; NIH is assembling the right research teams and resources to find such targets.CancerEnlist a patient's own immune system in the fight against cancer._cb026aca-8b9c-11e7-b76f-edbffedb5b8c1.2.5James AllisonLike the previous examples of statin and HIV drugs, it must be emphasized that cancer immunotherapy owes its success to decades of NIH-funded fundamental science. In fact, a recent analysis of a cancer immunotherapy approach pioneered by NIH grantee James Allison, who is a 2015 Lasker Award winner, documented the contributions of 7,067 scientists over more than a century, with many working on basic research with no clear connection to cancer. Cancer PatientsProgress toward treatment and cures is certainly not limited to high-throughput screening and DNA sequencing. Consider the example of cancer immunotherapy. In the early 1970s, basic research, spearheaded in large part by NIH-funded scientists, led to the development of methods to splice fragments of DNA together, giving birth to the field of biotechnology. When merged with fundamental advances in molecular immunology, this set of technologies made it possible to begin pursuing ideas for cancer immunotherapy -- a radical new approach that involves enlisting a patient's own immune system in the fight against cancer. In one promising strategy, T cells are collected from patients and engineered to produce special surface proteins, called chimeric antigen receptors. This work has already saved the lives of children with acute lymphoblastic leukemia and adults with chronic lymphocytic leukemia and refractory multiple myeloma.Antibiotic-Resistant BacteriaCombat antibiotic-resistant bacteria._cb026f98-8b9c-11e7-b76f-edbffedb5b8c1.2.6Scientific innovation is also central to the quest to find new ways of combating the growing threat of antibiotic-resistant bacteria, which each year infect more than 2 million Americans and kill at least 23,000. For example, an ingenious microfluidic system that can trap and sort single cells has enhanced efforts to mine one of nature's richest sources of potential antibiotics: dirt. Certain microorganisms that naturally live in soil produce antibiotic-like compounds that are highly toxic to other microbes. Thanks to their improved ability to "dig through dirt," NIH-funded researchers recently uncovered a new class of antibiotic drugs with the power not only to kill a wide range of infection-causing bacteria, but to kill them in a way that may reduce the problem of antibiotic resistance. Development & TestingAccelerate the development and testing of potential treatments and cures._cb0278b2-8b9c-11e7-b76f-edbffedb5b8c1.2.7Discovery of potential therapeutic targets and candidate therapies are essential first steps in the development of new treatments and cures, but they are far from the only steps. The transition of scientific discoveries to human clinical trials has become increasingly costly and time consuming, with a great number of candidate therapies failing to cross what has been dubbed the "Valley of Death." NIH-funded research will play an increasingly important role in identifying hurdles in this process, as well as generating approaches for accelerating the development and testing of potential treatments and cures. Chronic Conditions & ComorbiditiesUnderstand of the cumulative and synergistic impacts that multiple chronic conditions and comorbidities._cb027d58-8b9c-11e7-b76f-edbffedb5b8c1.2.8Interagency Pain Research Coordinating CommitteeOn behalf of HHS, NIH has established the Interagency Pain Research Coordinating Committee, which has generated a National Pain Strategy and facilitated collaborations aimed at advancing fundamental understanding of pain and improving pain-related treatment. Also, as part of its effort to push research beyond a strictly organ-based view of health and disease, NIH will encourage efforts to study the interactions of various diseases and conditions. The aim is to gain a better understanding of the cumulative and synergistic impacts that multiple chronic conditions and comorbidities can exert upon the human body, thereby informing efforts to develop therapeutic and preventive approaches for these complex challenges. Among the many comorbidities in need of additional research is pain.Disease Prevention & ManagementDevelop of more precise, individualized ways of managing and preventing disease._cb0280f0-8b9c-11e7-b76f-edbffedb5b8c1.2.9National Cancer InstituteOne example of pharmacogenomics is the National Cancer Institute (NCI)-Molecular Analysis for Therapy Choice (NCI-MATCH) clinical trial, which will build a foundation for the oncology component of the multi-agency Precision Medicine Initiative® (PMI), in which NIH has a lead role. In this trial, involving up to 3,000 patients with different types of advanced solid tumors and lymphomas, researchers will analyze a patient's tumor for "actionable" genetic abnormalities and use that information to select molecularly targeted drug(s) most likely to work for that particular patient.To speed the movement of discoveries from the lab to the clinic, NIH will also accelerate and expand upon its efforts to encourage development of more precise, individualized ways of managing and preventing disease. Known collectively as precision medicine, these emerging approaches for preventing, diagnosing, and treating disease take into account individual variability in genes, environment, and lifestyle. While individualized, molecularly based strategies are in use for some conditions, including cancer, HIV/AIDS, and hepatitis C, more research is needed to realize precision medicine’s promise for all conditions. Among the frontiers in this area is pharmacogenomics, which studies how an individual's genetic makeup (or the genetic makeup of a tumor) affects response to drugs. The goal of such research is to enable health-care providers to prescribe the right drug at the right dose at the right time for each patient.Other ApproachesPursue other innovative approaches with precise therapeutic potential._cb02887a-8b9c-11e7-b76f-edbffedb5b8c1.2.10Other innovative approaches with precise therapeutic potential include gene therapies, including approaches aimed at correcting vision and hearing loss; cell-based therapies; and gene editing systems.Vision & HearingCorrect vision and hearing loss._cb028d2a-8b9c-11e7-b76f-edbffedb5b8c1.2.10.1Cell-Based TherapiesDevelop cell-based therapies._cb02920c-8b9c-11e7-b76f-edbffedb5b8c1.2.10.2Gene EditingDevelop gene editing systems._cb0299a0-8b9c-11e7-b76f-edbffedb5b8c1.2.10.3Sickle Cell Disease PatientsIn a recent proof-of-concept study, NIH-funded researchers paired the latter two technologies to develop a potential cure for sickle cell disease, a painful, life-threatening disorder caused by mutations in the beta globin gene. To accomplish this, the researchers generated induced pluripotent stem cells (iPSCs) derived from the white blood cells of people with sickle cell disease; used CRISPR/Cas9 gene editing to replace the mutant gene; and then converted the iPSCs into normal red blood cells. If the technology proves safe and effective in additional pre-clinical and clinical tests, gene-corrected red blood cells could be generated from individuals with sickle cell disease and used for transfusions, reducing need for donor blood and providing hope for an eventual cure. Clinical StudiesFoster and reward innovations in the design, execution, and management of clinical studies._cb029f86-8b9c-11e7-b76f-edbffedb5b8c1.2.11Clinical and Translational Science Awards ProgramOne of the primary ways in which NIH will encourage innovation in the clinical research enterprise is through its support of the Clinical and Translational Science Awards program, which is a national network of institutions engaged in developing and testing new approaches for clinical research and training. Along with advances in basic and translational research, advances in clinical research are essential to NIH's efforts to catalyze the development of treatments and cures. To move clinical science forward, NIH will seek to foster and reward innovations in the design, execution, and management of clinical studies.Health Data StandardsCreate and implement health data standards in electronic health records and health information exchange systems._cb02a36e-8b9c-11e7-b76f-edbffedb5b8c1.2.12To promote the effective research use of clinical data, NIH will engage in efforts to create and implement health data standards in electronic health records and health information exchange systems.Trial DesignsDevelop alternative clinical trial designs._cb02aae4-8b9c-11e7-b76f-edbffedb5b8c1.2.13The agency will also back the development of alternative clinical trial designs that permit flexibility, while maintaining the utmost priority of patient safety.Research MethodologiesImprove clinical research methodologies._cb02af8a-8b9c-11e7-b76f-edbffedb5b8c1.2.14FDAAHRQCDCNIH will also work closely with its sister HHS agencies, including FDA, AHRQ, and CDC, to improve clinical research methodologies in a variety of important areas, such as identifying new approaches for combating antibiotic-resistant bacteria and timely reporting of clinical trial results. These and other steps will increase the rate at which clinical research findings inform current areas of scientific inquiry and stimulate entirely new avenues for biomedical research, which could in turn spark ideas for further treatment and prevention strategies.Therapeutic DevelopmentAddress obstacles that lie at various points throughout the therapeutic development process._cb02b372-8b9c-11e7-b76f-edbffedb5b8c1.2.15Despite the many exciting scientific opportunities for speeding the development of treatments and cures, significant challenges remain. Over the next 5 years, NIH will support research aimed at addressing a wide range of obstacles that lie at various points throughout the therapeutic development process.ConnectionsForge new connections across research disciplines._cb02bc1e-8b9c-11e7-b76f-edbffedb5b8c1.2.15.1NIH will strive to forge new connections across research disciplines to advance understanding of molecular mechanisms and discovery of treatments and cures for a wide range of illnesses. Systems-based and interdisciplinary approaches are vital to making progress toward treatments tailored to individual patients.InnovationsCatalyze innovations._cb02c0ba-8b9c-11e7-b76f-edbffedb5b8c1.2.15.2To improve the efficiency, relevance, and accuracy of preclinical research, NIH will catalyze powerful innovations, including molecule cross-coupling methods that will open a vast new frontier of "chemical space" and human 3D organoid technologies that will be better than animal models.Translational SciencesTransform and accelerate the translational process._cb02c60a-8b9c-11e7-b76f-edbffedb5b8c1.2.15.3National Center for Advancing Translational SciencesThrough its National Center for Advancing Translational Sciences, NIH will continue to support efforts to transform and accelerate the translational process, using science to find new ways to bridge the gaps and get more treatments to more patients more quickly.Clinical TrialsSpeed and streamline clinical trials._cb02ce0c-8b9c-11e7-b76f-edbffedb5b8c1.2.15.4NIH will also work to speed and streamline clinical trials by encouraging the use of molecular knowledge to select the individuals most likely to respond to experimental therapies, and promoting respect for research volunteers though steps such as the updating of the Common Rule protections for human subjects research.Molecular KnowledgeEncourage the use of molecular knowledge to select the individuals most likely to respond to experimental therapies._cb02d2d0-8b9c-11e7-b76f-edbffedb5b8c1.2.15.4.1Research VolunteersPromote respect for research volunteers though steps such as the updating of the Common Rule protections for human subjects research._cb02d6e0-8b9c-11e7-b76f-edbffedb5b8c1.1.15.4.2Research VolunteersHealth & DiseaseAdvance health promotion and disease prevention._2ab0cc88-8aa7-11e7-a015-e3bd1f25b93b1.3CDCWhile NIH supports its own distinct and robust research portfolio, it collaborates with CDC, AHRQ, the Health Resources and Services Administration (HRSA), and other HHS agencies involved in complementary activities related to health promotion and disease prevention, including efforts in dissemination and implementation.AHRQHealth Resources and Services Administration (HRSA)HHS AgenciesWest AfricaRecent collaboration between CDC and NIH on surveillance and initiation of clinical trials of candidate vaccines against Ebola virus disease in West Africa is one noteworthy example.Opioid Drug UsersLikewise, NIH, in collaboration with other HHS agencies, is playing a key role in the implementation and dissemination of the HHS Secretary's new multipronged, evidence-based initiative to combat the use of opioid drugs. Along with basic research and research aimed at developing treatments and cures, NIH supports research to promote health; to prevent diseases, disorders, conditions, or injuries; and to detect and/or prevent progression of asymptomatic disease. This broad and deep research portfolio encompasses studies of biology, behavior, environment, and health-related policies. Among the many advances in this area are identification and assessment of risk and protective factors; screening and identification of at-risk individuals/groups, (e.g., human papilloma virus testing for cervical cancer screening); development and evaluation of risk-reduction strategies; and translation, implementation, and dissemination of preventive interventions, (e.g., Sudden Infant Death Syndrome campaign). PrioritiesEmphasize research in key areas._2ab0cf4e-8aa7-11e7-a015-e3bd1f25b93b1.3.1Over the next 5 years, NIH's health promotion and disease prevention efforts will place particular emphasis on research in several key areas: studying healthy individuals across the lifespan; applying technological advances in early detection, diagnosis, and prevention; and utilizing evidence-based interventions to reduce health disparities.Healthy IndividualsStudy healthy individuals across the lifespan._cb02dfc8-8b9c-11e7-b76f-edbffedb5b8c1.3.1.1Healthy IndividualsTechnological AdvancesApply technological advances in early detection, diagnosis, and prevention._cb02e48c-8b9c-11e7-b76f-edbffedb5b8c1.3.1.2Health DisparitiesUze evidence-based interventions to reduce health disparities._cb02e888-8b9c-11e7-b76f-edbffedb5b8c1.3.1.3Healthy Development & AgingPromote research on healthy development and aging._2ab0d19c-8aa7-11e7-a015-e3bd1f25b93b1.3.2Environmental influences on Child Health Outcomes (ECHO) InitiativeOne example of this is NIH’s new Environmental influences on Child Health Outcomes (ECHO) initiative. To understand how things can go wrong in the human body, it is essential to understand how things work when everything goes right. For example, studies of normal embryonic development have informed efforts to understand, prevent, and treat birth defects caused by genetic and a broad range of environmental factors. ChildrenNIH will promote research on healthy development and aging, as well as on understanding disease susceptibility and prevention across the life span. A lifetime of benefits will result from efforts to establish healthy behaviors early in life and to identify and prevent the mechanistic antecedents to chronic conditions that begin during pre-, peri-, or post-natal periods of development.Microbial CommunitiesTrack the composition of microbial communities over the course of an individual's life._2ab0d57a-8aa7-11e7-a015-e3bd1f25b93b1.3.3In another example of research aimed at health promotion and disease prevention, NIH will expand efforts to track the composition of microbial communities over the course of an individual’s life. Such action is motivated, in part, by the explosion in understanding of the role played by the microflora in the development of the immune system. Development & AgingSupport research into the basic mechanisms of development and aging in healthy individuals._2ab0d944-8aa7-11e7-a015-e3bd1f25b93b1.3.4Healthy IndividualsTo make similar advances in other areas, NIH will continue to support research into the basic mechanisms of development and aging in healthy individuals. This will include intensifying studies of "resilience" -- that is, to understand why some individuals’ bodies age more slowly and/or are better able to resist disease risks posed by particular genetic, lifestyle, and/or environmental factors. NIH will also strive to develop tools to enhance measurement of physical, social and environmental exposures, as well as to assess the impacts of such exposures on development, health, and longevity. In addition, NIH-funded research will explore why people make unhealthy or risky choices, generating valuable information for devising risk reduction and/or early intervention strategies. Prevention & ScreeningEstablish a base of scientific knowledge that can be used to develop prevention and screening strategies tailored to individuals._2ab0db92-8aa7-11e7-a015-e3bd1f25b93b1.3.5Technological innovations will also be instrumental for research aimed at making advances in the early detection, diagnosis, and prevention of disease. At the forefront of this effort will be the NIH-led PMI cohort. Taking advantage of emerging biomedical tools and technologies, such as availability of electronic health records, DNA sequencing, and exposure monitoring, PMI's longitudinal research cohort of 1 million or more U.S. volunteers will establish a base of scientific knowledge that can be used to develop prevention and screening strategies tailored to individuals at the most opportune times across the course of their lives. Individual CharacterizationTake advantage of the latest methods and approaches in data science to characterize individuals._2ab0e06a-8aa7-11e7-a015-e3bd1f25b93b1.3.6PMI will also take advantage of the latest methods and approaches in data science, including advances in large-scale databases, computational tools, and -omics methodologies to characterize individuals.Mobile TechnologiesTest whether mobile technologies are useful._cb02f206-8b9c-11e7-b76f-edbffedb5b8c1.3.7In addition, PMI will offer researchers the ability to test whether mobile technologies are useful in adapting preventive strategies to individuals' needs and preferences, enhancing delivery of interventions, and improving monitoring of compliance and outcomes.Individual StrategiesAdapt preventive strategies to individuals' needs and preferences._cb02f71a-8b9c-11e7-b76f-edbffedb5b8c1.3.7.1InterventionsEnhance delivery of interventions._cb02fb5c-8b9c-11e7-b76f-edbffedb5b8c1.3.7.2MontoringImprove monitoring of compliance and outcomes._cb03035e-8b9c-11e7-b76f-edbffedb5b8c1.3.7.3Prevention DataMerge, integrate, and analyze data from a wide variety of sources with implications for prevention._cb030944-8b9c-11e7-b76f-edbffedb5b8c1.3.8PMI will pioneer efforts to merge, integrate, and analyze data from a wide variety of sources with implications for prevention, including basic biological data, health status information from electronic health records, individual data on environmental exposures, geospatial data on community environmental exposures, and so on. Asymptomatic Diseases/DisordersDevelop better methods for screening, assessing, and identifying those at risk for onset or progression of asymptomatic diseases/disorders._2ab0e34e-8aa7-11e7-a015-e3bd1f25b93b1.3.9DiabeticsOne notable success in the realm of prevention of a common, chronic disease is the NIH-led Diabetes Prevention Program (DPP) trial, which involved overweight or obese U.S. adults with prediabetes. DPP researchers found that exercise and dietary changes leading to modest weight loss (5%-7% of body weight) could prevent or delay development of type 2 diabetes.Obese PeopleFurthermore, the prevention program was shown to be effective in both men and women and all racial/ethnic groups studied, including those disproportionately burdened by obesity.NIH will also build upon ongoing efforts to develop better methods for screening, assessing, and identifying those at risk for onset or progression of asymptomatic diseases/disorders.Early TreatmentIdentify diseases and conditions in early, more readily treatable states._2ab0e588-8aa7-11e7-a015-e3bd1f25b93b1.3.10Also needed are molecular, cellular, and imaging technologies that provide greater power to identify diseases and conditions in early, more readily treatable states before they progress to symptomatic or metastatic disease.Point-of-Care TechnologiesDevelop point-of-care technologies._cb030d72-8b9c-11e7-b76f-edbffedb5b8c1.3.11To further facilitate early diagnosis and detection, NIH will encourage the development of point-of-care technologies that lead to less costly, more rapid results, and improved patient outcomes. MetricsProvide clinicians and researchers with access to efficient, precise, and valid patient-reported measures of health and well-being. _2ab0ea10-8aa7-11e7-a015-e3bd1f25b93b1.3.12CliniciansResearchersNIH will also cultivate efforts to provide clinicians and researchers with access to efficient, precise, and valid patient-reported measures of health and well-being. For example, NIH currently supports the Patient-Reported Outcome Measurement Information System® (PROMIS®), which is using measurement science to create a state-of-the-art assessment system for self-reported health. VaccinesSupport vaccine development._2ab0ed3a-8aa7-11e7-a015-e3bd1f25b93b1.3.13Vaccine Research CenterVaccine and Treatment Evaluation UnitsVaccines -- one of biomedicine's most powerful tools for preventing and eradicating disease -- also are heavily reliant upon NIH-funded research and innovation. Over the next 5 years, NIH will take advantage of its intramural Vaccine Research Center, along with its network of Vaccine and Treatment Evaluation Units located across the nation, to support the full spectrum of vaccine development from early discovery to clinical evaluation for a wide variety of infectious diseases. Particular emphasis will also be placed upon innovative approaches, such as a universal influenza vaccine and other DNA-based vaccines, to improve protection and optimize production.Interdisciplinary CollaborationFacilitate collaboration across biomedical, behavioral and social sciences, as well as disciplines not traditionally considered to involve health._2ab0ef7e-8aa7-11e7-a015-e3bd1f25b93b1.3.14Biomedical ScientistsBehavioral ScientistsSocial ScientistsArchitectsTransportation PlannersUrban PlannersNIH also will promote health and encourage disease prevention by facilitating collaboration across biomedical, behavioral and social sciences, as well as disciplines not traditionally considered to involve health, such as architecture, transportation, and urban planning. Although many behaviors that increase disease risk have been identified, more effective approaches to promoting behavior change are still needed. Basic, behavioral, and social sciences research can inform new strategies for preventing distinct conditions caused by high-risk behaviors that share an underlying basis. One example is the wide range of cancers and other diseases associated with use of various forms of tobacco. Health DisparitiesDevelop evidence-based interventions to reduce health disparities._2ab0f442-8aa7-11e7-a015-e3bd1f25b93b1.3.15WomenFor instance, an NIH-supported study of women who received housing vouchers that enabled them to move from high-poverty to low-poverty neighborhoods found that such women were less likely to be obese or have diabetes than similar controls.Importantly, NIH will continue to pursue research aimed at developing evidence-based interventions to reduce health disparities. Such efforts will address the importance of understanding social determinants of health, disease, and disability; disproportionate disease risk; and opportunities for progress in prevention. Social DeterminantsUnderstand social determinants of health, disease, and disability. _cb031736-8b9c-11e7-b76f-edbffedb5b8c1.3.15.1Disease RiskUnderstand disproportionate disease risk._cb031c68-8b9c-11e7-b76f-edbffedb5b8c1.3.15.2PreventionUnderstand opportunities for progress in prevention._cb0320be-8b9c-11e7-b76f-edbffedb5b8c1.3.15.3InterventionsDisseminate evidence-based interventions to promote health and prevent disease._cb0328d4-8b9c-11e7-b76f-edbffedb5b8c1.3.16NIH-funded research will also evaluate methods to disseminate evidence-based interventions to promote health and prevent disease -- with particular emphasis on comorbid conditions -- in a variety of community health and clinical settings, as well as identify barriers to adoption of such interventions.Strategic PlanDevelop a strategic plan to guide efforts to address minority health and health disparities._cb032f1e-8b9c-11e7-b76f-edbffedb5b8c1.3.17MinoritiesUnderstanding mechanisms that lead to disparities in health outcomes by race/ethnicity and socioeconomic status will require multi-disciplinary collaboration of population, clinical, and basic scientists. An NIH-wide assessment of current minority health and health disparities research using standardized coding will inform the development of a strategic plan to guide this emerging scientific area. Innovation & PrioritiesFoster Innovation by Setting NIH Priorities_2ab0f76c-8aa7-11e7-a015-e3bd1f25b93b2In order for NIH to achieve its mission, it must serve as an effective and efficient steward of public resources. To advance these efforts over the next 5 years, NIH will focus intensely on prioritization. The process of setting NIH's research priorities must balance the opportunities presented by the best science, public health needs, and the unique ability of NIH to address challenges in human health that would otherwise go unmet. These priorities, which will require NIH's constant review and adjustment, must be flexible and based on the best science of the moment; formulas and fixed percentages are inconsistent with NIH's efforts to carry out its mission in an effective and efficient manner that is driven scientifically. Funding DecisionsApply a multifaceted approach for funding decisions._2ab0f9a6-8aa7-11e7-a015-e3bd1f25b93b2.1NIH has long relied upon a multifaceted approach for funding decisions that involves peer review by scientific experts to determine scientific merit of a research proposal, review for program priority by a second set of scientific experts and thought leaders from the lay public serving on ICO national advisory councils, individual ICO strategic plans, and, ultimately, the scientific expertise of ICO Directors, informed by their staff. NIH will continue and strengthen its commitment to a transparent, evidence-based process that encompasses these action-oriented principles: enhance the nimbleness needed to meet public health needs and capitalize upon scientific opportunity, using new portfolio analysis tools; incorporate burden of disease as an important, but not sole, factor; take advantage of opportunities presented by rare diseases to advance research; and consider the value of permanently eradicating a disease. MetricsPublicize a standard metric for funding each year. _cb033374-8b9c-11e7-b76f-edbffedb5b8c2.2Going forward, NIH will take additional steps to enhance the transparency of its decision process by making public a standard metric for funding each year.Select PayEnsure ICOs set their individual paylines to provide maximum flexibility for use of the select pay option._cb033d56-8b9c-11e7-b76f-edbffedb5b8c2.3NIH ICO DirectorsFinal decisions on the use of select pay are made by the NIH ICO Directors following discussions with their Advisory Councils and appropriate ICO staff. NIH will also harmonize approaches to decision making by ensuring ICOs set their individual paylines -- the funding cutoff point for grant applications based solely upon peer-review scores -- to provide maximum flexibility for use of the select pay option. Select pay refers to funds set aside to support grant applications that, based upon scores from peer review, do not fall within the payline, but that fill an important research gap and/or are of particular programmatic relevance to an ICO’s scientific and health priorities.Nimbleness. Enhance Nimbleness._cb03427e-8b9c-11e7-b76f-edbffedb5b8c2.4Ebola VictimsA recent example of how NIH's rapidly responsive flexibility has served to address an urgent public health crisis is its pivotal role in the development and accelerated clinical testing of a vaccine against the deadly Ebola virus.BRAIN Initiative®Likewise, NIH's nimbleness in the face of unexpected scientific breakthroughs has enabled it to take a leadership role in the BRAIN Initiative®, which has the ambitious goal of producing the first dynamic picture of the human brain, showing how individual cells and complex circuits interact in both time and space. NIH and all of its ICOs will nurture the nimbleness necessary to shift resources in response to unexpected scientific breakthroughs, to capitalize on scientific opportunities on the horizon, and to address emerging public health needs. Advancing human health requires taking advantage of scientific opportunities as they arise. It is important to recognize that different scientific fields mature at different rates, and the same amount of funding in two fields can lead to very different scientific returns.BreakthroughsShift resources in response to unexpected scientific breakthroughs. _cb0346d4-8b9c-11e7-b76f-edbffedb5b8c2.4.1OpportunitiesShift resources to capitalize on scientific opportunities on the horizon. _cb034f3a-8b9c-11e7-b76f-edbffedb5b8c2.4.2NeedsShift resources to address emerging public health needs. _cb0355a2-8b9c-11e7-b76f-edbffedb5b8c2.4.3Grant ProgramsExplore the strengths and weakness of different types of grant programs._cb03604c-8b9c-11e7-b76f-edbffedb5b8c2.5To help inform these decisions, NIH will explore the strengths and weakness of different types of grant programs, along with other funding mechanisms, to identify optimal approaches. Among the nimble approaches currently at NIH's disposal are Other Transaction Authority, which enables support of high-risk, milestone-driven research supported through the NIH Common Fund and various ICOs; fast-track review of Small Business Innovation Research and Small Business Technology Transfer awards, in which Phase I and Phase II grant applications are reviewed together, reducing funding gaps between phases; and various scientific challenge prizes, which include competitions to encourage development of novel methods for analyzing individual cells; point-of-care diagnostics for antibiotic-resistant infections; and new products or services to harness the power of Big Data to improve health.SurveillanceEnhance surveillance of the scientific landscape._cb03657e-8b9c-11e7-b76f-edbffedb5b8c2.6Of course, it requires more than investment to drive biomedical progress -- scientific opportunities also need to be present. To enhance surveillance of the scientific landscape, NIH will utilize a network of internal and external expertise, and will continue to develop, improve, and use new tools for portfolio analysis to identify scientific opportunities, high-performing areas of research, and areas of potential overlap among ICOs. NetworksUse a network of internal and external expertise._cb036a24-8b9c-11e7-b76f-edbffedb5b8c2.6.1ExpertsPortfolio AnalysisDevelop, improve, and use new tools for portfolio analysis._cb0372d0-8b9c-11e7-b76f-edbffedb5b8c2.6.2OpportunitiesIdentify scientific opportunities._cb037834-8b9c-11e7-b76f-edbffedb5b8c2.6.1High-Performance ResearchIdentify high-performing areas of research._cb037dde-8b9c-11e7-b76f-edbffedb5b8c2.6.2OverlapsIdentify areas of potential overlap among ICOs._cb038824-8b9c-11e7-b76f-edbffedb5b8c2.6.3NIH ICOsSocial Media & Nontraditional SourcesTrain staff in the effective analysis of social media trends and other nontraditional sources of information._cb038dce-8b9c-11e7-b76f-edbffedb5b8c2.7NIH StaffTo further empower its ability to monitor highly active or emergent areas of public health concern and scientific opportunity, NIH will train staff in the effective analysis of social media trends and other nontraditional sources of information. BurdenConsider Burden of Disease._cb039274-8b9c-11e7-b76f-edbffedb5b8c2.8The relative burden that various diseases place upon human health and wellbeing will serve as a crucial, but not the only, consideration in aligning NIH's research priorities with public health needs.DataCollect high quality, comparable data on the burden of disease._cb039b2a-8b9c-11e7-b76f-edbffedb5b8c2.8.1CDCTo this end, NIH will work with its many partners, including CDC, to strengthen the collection of high quality, comparable data on the burden of disease and will integrate analyses of such data into its priority setting process.Priority AnalysisIntegrate analyses of disease burden data into our priority setting process._cb03a17e-8b9c-11e7-b76f-edbffedb5b8c2.8.2It must be emphasized that there currently are multiple types and sources of disease burden data, and these data vary depending on whether researchers measure death or disability, direct or indirect economic costs, and domestic or global populations. However, none of these measures incorporates the cost of conducting basic research, which is essential for finding interventions. Another important variable is the degree to which subjective judgments factor into the process, making it extremely difficult to compare all diseases and conditions with a single measurement. Additional caveats regarding the use of current disease burden datasets to establish research priorities include the lack of patient-derived assessments and inconsistent accounting of the burden on caregivers. Finally, it is imperative to keep in mind that current burden of disease does not necessarily predict future burden of disease. Rare DiseasesAdvance Research Opportunities Presented by Rare Diseases. _cb03a64c-8b9c-11e7-b76f-edbffedb5b8c2.8.3FDAIn recent years, FDA approvals of "orphan drugs" to treat diseases and disorders affecting fewer than 200,000 people in the United States have been increasing. However, effective treatments are lacking for many rare diseases, constituting an important public health need that NIH research still needs to address.People Afflicted by Rare DiseasesBesides helping individuals affected by rare diseases, such research can provide insights that spill over into other, more common diseases and greatly enhance understanding of healthy physiology.Older PeopleFor example, studies of the molecular mechanisms involved in a very rare premature aging condition called progeria have revealed valuable insights into the normal aging process. Rare Disease ResearchersNIH is uniquely situated to tackle the challenges, as well as capitalize on the opportunities, presented by rare diseases over the next 5 years. In contrast to the typical situation in private industry, public funding enables researchers to pursue scientific questions, such as those posed by rare diseases, on the basis of opportunity, not just perceived market value.If NIH had used burden of disease as the sole determinant for setting its priorities over the past century, rare disease research, in all likelihood, would have been seriously neglected. While any given rare disease affects a relatively low number of people, such conditions represent a significant health problem when they are considered together, affecting some 25 million Americans collectively.EradicationConsider the Value of Permanently Eradicating a Disease._cb03b04c-8b9c-11e7-b76f-edbffedb5b8c2.9Achieving the complete cure or eradication of any disease is one of the ultimate goals of medical research. While each year brings NIH-funded science closer to improved treatments for any number of diseases and conditions, the ability to completely remove the threat of a single disease from the face of the Earth is a rare opportunity. Just think of the monumental effort it took to eradicate smallpox and how, even after decades of intense vaccination campaigns, the world is just now on the verge of eliminating polio.HIV/AIDSEnd the worldwide scourge of HIV/AIDS._cb03b59c-8b9c-11e7-b76f-edbffedb5b8c2.9.1People Infected with HIV/AIDSOffice of AIDS ResearchBiomedical research stands at another such pivotal moment today: the very real possibility of entirely eliminating HIV/AIDS. Decades of robust investment in HIV/AIDS research has resulted in extraordinary improvement in the health of infected individuals, and NIH now plans to support the best science to eliminate HIV/AIDS as a public health threat domestically and globally. While the traditional, non-statutory 10% set aside for HIV/AIDS research, overseen by the Office of AIDS Research, was an appropriate response to the crisis more than two decades ago, NIH no longer sees the value in this formula-driven approach. That does not mean taking the foot off the accelerator, however. To seize this unique moment, NIH will prioritize its research efforts to end the worldwide scourge of HIV/AIDS and usher in the first AIDS-free generation in more than half a century. Not only does this research strategy hold out the hope of eliminating the death and suffering caused by this worldwide epidemic, it makes good economic sense: every new case of HIV diagnosed in the United States (currently, about 50,000 per year) translates into a lifetime cost of approximately $350,000 for treatment with antiretroviral drugs. Getting to zero new cases of HIV/AIDS would save our nation an estimated $17.5 billion annually. TransparencyBe judicious and transparent in decisions about investing the monies entrusted to us by the American public._cb03ba4c-8b9c-11e7-b76f-edbffedb5b8c2.10As NIH takes these and other factors into account in setting biomedical research priorities, it is imperative that the agency be judicious and transparent in decisions about investing the monies entrusted to it by the American public. Going forward, NIH will consider using the previously described HIV/AIDS research prioritization process as a potential model approach for shaping its research focus in other scientific areas.ROIIncrease the rate of return that NIH-supported research delivers to the nation._cb03c316-8b9c-11e7-b76f-edbffedb5b8c2.11Through concerted efforts to improve and refine key priority-setting activities, the agency is committed to increasing the already high rate of return that NIH-supported research delivers to the nation in the form of scientific advances and improved human health.Scientific StewardshipEnhance Scientific Stewardship_2ab0ff1e-8aa7-11e7-a015-e3bd1f25b93b3To achieve its mission and maintain its role as the world's premier biomedical research agency, NIH must support the best scientific ideas and brightest scientific minds while, at the same time, earning and maintaining public trust. NIH’s role as a steward of public resources also requires not only supporting innovative research, but also fostering innovation across the entire research enterprise by enhancing individual and collective scientific stewardship. NIH must live up to the commitment that every dollar is being spent in a way that maximizes long term public benefit. Over the next 5 years, NIH will take several significant steps to strengthen and sustain its most valuable resource -- the scientific workforce -- and to strive for the highest level of scientific integrity, public accountability, and social responsibility in the conduct of science.Biomedical Research WorkforceRecruit and Retain an Outstanding Biomedical Research Workforce._2ab10234-8aa7-11e7-a015-e3bd1f25b93b3.1To ensure that the nation cultivates a thriving and talented next generation of scientists, NIH will seek ways to strengthen the biomedical research work force.Early Stage InvestigatorsImprove the outlook and opportunities for new and early stage investigators._2ab104a0-8aa7-11e7-a015-e3bd1f25b93b3.1.1Early Stage InvestigatorsSuch efforts will include improving the outlook and opportunities for new and early stage investigators through policies and grant mechanisms that are designed to support investigators at the outset of their careers. NIH will continue to take steps to enable exceptional early career scientists to flourish independently by bypassing the traditional postdoctoral training period, to bridge the gap from early- to mid-career investigator, and to normalize grant success rates between early stage investigators and more experienced investigators. For example, within the next 4 years, NIH will be evaluating its Early Independence Awards program to gauge its success in fostering independent and productive research careers.IDeA ProgramBroaden the geographic distribution of NIH funding for biomedical research._2ab10996-8aa7-11e7-a015-e3bd1f25b93b3.1.2Another way in which NIH will aim to ensure that all of America develops and maintains an outstanding biomedical research workforce is through its Institutional Development Award (IDeA) program. By broadening the geographic distribution of NIH funding for biomedical research, the IDeA program fosters health-related research and enhances the competitiveness of researchers at institutions located in states in which the aggregate success rate for grant applications to NIH has historically been low. Such activities also benefit unique populations -- such as rural and medically underserved communities -- in Puerto Rico and the 23 states that are currently part of the IDeA program.Training EvaluationContinually evaluate the effectiveness of our scientific training programs and efforts._2ab10d10-8aa7-11e7-a015-e3bd1f25b93b3.2Physician-ScientistsData Science WorkforceBasic ScientistsClinical ScientistsNIH will also continually evaluate the effectiveness of its scientific training programs and efforts. For example, it will identify and target specific areas of biomedical research in which workforce training should be tailored to meet growing needs, including revitalizing physician-scientist training, fostering recruitment to expand the data science workforce, and promoting the cross-training of basic scientists, clinical scientists, and physician-scientists to facilitate the development of inter- and cross-disciplinary research teams and to stimulate translational research.Training InnovationPromote innovative training programs._2ab10f86-8aa7-11e7-a015-e3bd1f25b93b3.2.1Graduate StudentsPostdoctoral ScholarsFurthermore, NIH will promote innovative training programs to prepare trainees for the wide spectrum of career options that will be available in tomorrow's biomedical research workforce. For example, the recently established Broadening Experiences in Scientific Training (BEST) program will enhance training for graduate students and postdoctoral scholars to prepare them for careers outside of conventional academic research. Ultimately, a stable, predictable funding stream is needed to attract and retain new talent for the research workforce.Workforce DiversityEnhance Workforce Diversity._2ab1149a-8aa7-11e7-a015-e3bd1f25b93b3.3NIH strongly believes that diversity in the biomedical research workforce is critical to producing new scientific discoveries. From NIH's vantage point, racial and ethnic diversity is paramount. It is also important to pursue diversity in other areas, including sex and gender, socioeconomic status, geographic location, and disability status. RecommendationsImplement the recommendations of the Advisory Committee to the Director's Working Group on Diversity in the Biomedical Research Workforce._2ab117e2-8aa7-11e7-a015-e3bd1f25b93b3.3.1African-American/Black ApplicantsNIH Chief Officer for Scientific Workforce DiversityBUilding Infrastructure Leading to Diversity (BUILD) InitiativeExamples of new NIH programs that are part of this strategy are the BUilding Infrastructure Leading to Diversity (BUILD) initiative, which has the long-term goal of catalyzing cultural changes at academic institutions so that talented students from groups historically underrepresented in biomedical research are well-prepared to enter research careers, and ...NIH National Research Mentoring Network (NRMN)the NIH National Research Mentoring Network (NRMN), which will facilitate the development of robust mentoring relationships by pairing scientific leaders with early career scientists from underrepresented groups across the nation. A unique attribute of these programs is that they are being run as a "trial" with a data-coordinating center, collecting common measures across all programs. In this manner, NIH will be able to identify subsets of "best practices" and then swiftly apply them across the network. These best practices will also be used to inform enhancement of other programs that are designed to enhance diversity of the biomedical research workforce.In an effort to understand why the biomedical workforce does not reflect the diversity of the Nation, NIH sponsored a landmark study that demonstrated a disparity in R01 funding to African-American/Black applicants. Importantly, NIH has launched a broad range of efforts to redress this untenable situation, including a thorough analysis of potential biases in peer review and experiments in anonymized review. Under the leadership of its first Chief Officer for Scientific Workforce Diversity, NIH will work to implement the recommendations of the Advisory Committee to the Director's Working Group on Diversity in the Biomedical Research Workforce. This comprehensive strategy aims to enhance scientific workforce diversity, engaging partners from academia and industry to achieve diversity at all stages of biomedical research career trajectory.Rigor and ReproducibilityEnsure Rigor and Reproducibility._2ab11a6c-8aa7-11e7-a015-e3bd1f25b93b3.4As a global leader of biomedical research, NIH has a responsibility for maintaining and bolstering the public’s confidence in research results. To uphold this responsibility, NIH will take the lead in promoting new approaches toward enhancing the rigor of experimental design, analysis, and reporting. These efforts are not aimed at rare instances of research misconduct or willful deception, which require separate oversight mechanisms, but are intended to improve the biomedical research community’s overall culture and training to encourage best practices for rigorous scientific methods.Science JournalsImprove rigor and reproducibility with science journal editors._2ab11f76-8aa7-11e7-a015-e3bd1f25b93b3.4.1Science Journal EditorsNIH recently initiated several activities aimed to encourage transparency and reproducibility of research results. Over the next 5 years, NIH will build upon these activities, which include: discussing ways to improve rigor and reproducibility with science journal editors; establishing principles and guidelines for reporting preclinical research; emphasizing the importance of studying sex differences and incorporating sex as a variable in preclinical research; developing training modules and curriculum for the next generation of scientists on approaches to enhance reproducibility of their research; ensuring compliance with policies for open access to the published literature and data sharing; and continuing to expand the studies included in the NIH-supported ClinicalTrials.gov results database to improve dissemination of clinical trial results. Moving forward, NIH will continue to develop and promote other innovative initiatives to promote scientific rigor across the entire biomedical research enterprise. Preclinical ResearchEstablish principles and guidelines for reporting preclinical research._2ab122f0-8aa7-11e7-a015-e3bd1f25b93b3.4.2Sex DifferencesEmphasize the importance of studying sex differences and incorporating sex as a variable in preclinical research._2ab12674-8aa7-11e7-a015-e3bd1f25b93b3.4.3Training & CurriculumDevelop training modules and curriculum for the next generation of scientists on approaches to enhance reproducibility of their research._2ab12bb0-8aa7-11e7-a015-e3bd1f25b93b3.4.4Open AccessEnsure compliance with policies for open access to the published literature and data sharing._2ab12ef8-8aa7-11e7-a015-e3bd1f25b93b3.4.5DisseminationContinue to expand the studies included in the NIH-supported ClinicalTrials.gov results database to improve dissemination of clinical trial results. _2ab13178-8aa7-11e7-a015-e3bd1f25b93b3.4.6Reporting ProcessReduce Administrative Burden._2ab136fa-8aa7-11e7-a015-e3bd1f25b93b3.5NIH GranteesScientistsNIH is committed to streamlining its reporting processes to reduce the administrative burden on its grantees as much as possible, while maintaining the agency's necessary oversight role. These actions have been informed by recommendations from a 2015 report from an ad hoc committee of The National Academies of Sciences, Engineering, and Medicine, "Optimizing the Nation's Investment in Academic Research: A New Regulatory Framework for the 21st Century, Part 1"; and a 2012 report from the National Research Council's Committee on Research Universities, "Research Universities and the Future of America: Ten Breakthrough Actions Vital to Our Nation's Prosperity and Security." NIH has already made changes to many steps throughout the grant award process to optimize the system as much as possible, but there is no easy, one-size-fits-all solution. Over the next 5 years, NIH will continue to evaluate opportunities to streamline and automate this process to allow scientists to focus their attention first and foremost on their research.Funding DecisionsOptimize Approaches to Inform Funding Decisions._2ab13a56-8aa7-11e7-a015-e3bd1f25b93b3.6Grant ApplicantsAt NIH, the crucial, initial assessment of a grant application's scientific merit is conducted through the agency's highly respected peer review system. Still, there is always room for further optimization. During FY 2016-2020, NIH will step up efforts to make its peer review and post-grant award system even stronger by: enhancing diversity and fairness; optimizing the process for promoting interdisciplinary and team science; and voicing an expectation that all NIH grantees serve on NIH peer-review study sections when asked, thus ensuring that every researcher “gives back” to the scientific enterprise as a whole. Diversity & FairnessEnhance diversity and fairness._2ab13d6c-8aa7-11e7-a015-e3bd1f25b93b3.6.1Interdisciplinarity & TeamsPromote interdisciplinary and team science._2ab142da-8aa7-11e7-a015-e3bd1f25b93b3.6.2Peer ReviewsExpect all NIH grantees serve on NIH peer-review study sections when asked. _2ab14622-8aa7-11e7-a015-e3bd1f25b93b3.6.3NIH GranteesPortfolio Analysis & Strategic PlanningShare best practices in portfolio analysis and strategic planning._2ab148c0-8aa7-11e7-a015-e3bd1f25b93b3.6.4NIH ICOsIndividual ICOs play a key role in funding decisions through their Program Officers, who field grant applicants' inquiries and manage specialized portfolios of grants ...NIH National Advisory Councils... their National Advisory Councils, which provide a second level of review for scientific merit; and ...NIH ICO Directorstheir Directors, who have the ultimate authority over funding decisions.NIH LeadersNIH leadership will also encourage sharing of best practices in portfolio analysis and strategic planning among ICOs.Funding ApproachesExplore different funding approaches._2ab14ece-8aa7-11e7-a015-e3bd1f25b93b3.6.5To help inform these decisions, NIH will continue to explore the efficacy of different funding approaches -- comparing mechanisms to ascertain if their strengths and weaknesses and analyzing whether there is an optimal threshold of funding for research groups via RPG mechanisms.PortfolioMaintain a diverse and broad portfolio._2ab15266-8aa7-11e7-a015-e3bd1f25b93b3.6.6Since it is virtually impossible to predict where the next great breakthrough will emerge, NIH places a heavy emphasis on maintaining a diverse and broad portfolio. However, NIH currently uses the number of projects that it supports as the key metric in assessing program breadth.Investigators & ProjectsTest whether portfolio breadth would be best achieved through an increase in the number of investigators as opposed to the number of projects._2ab1552c-8aa7-11e7-a015-e3bd1f25b93b3.6.6.1Research GroupsInvestigatorsEmergent data suggest that on average, there are optimal levels of funding for research groups, beyond which, there is only minimal increase in return. NIH must therefore decide if portfolio breadth would be best achieved through an increase in the number of investigators that it supports as opposed to the number of projects it supports. Over the next 5-year period, pilot programs will be put in place to test this important question. InnovationEncourage Innovation._2ab15a72-8aa7-11e7-a015-e3bd1f25b93b3.7As part of its responsibility to be a wise steward of the resources provided by the American public, NIH will catalyze innovative research through novel funding mechanisms, groundbreaking initiatives, and creative policy approaches.Risks & RewardsPromoting high-risk, high-reward research._2ab15dd8-8aa7-11e7-a015-e3bd1f25b93b3.7.1NIH Common FundNational Institute of General Medical SciencesNational Cancer InstituteNational Institute on Drug AbuseNational Institute of Environmental Health SciencesOne major way in which NIH will accomplish this is by promoting high-risk, high-reward research through intensely competitive programs that fund individual investigators with the most promising cross-cutting research or ideas. Such programs include the NIH Common Fund's New Innovator, Pioneer, and Transformative Research Awards, the National Institute of General Medical Sciences' Maximizing Investigators' Research Award, the National Cancer Institute's Outstanding Investigator Award, the National Institute on Drug Abuse's Avenir Awards, and the National Institute of Environmental Health Sciences' Method to Extend Research in Time Awards. There is growing evidence that such approaches are working, with a 2012 independent review of Pioneer awards concluding that these awards resulted in higher impact and more innovative research relative to the traditional R01 award.Emerging TechnologiesSupport innovative short-term initiatives that take advantage of emerging technologies to focus resources on specific high-impact, trans-NIH basic research questions._2ab1606c-8aa7-11e7-a015-e3bd1f25b93b3.7.2BRAIN Initiative®NIH Common FundNIH will also support innovative short-term initiatives that take advantage of emerging technologies to focus resources on specific high-impact, trans-NIH basic research questions, such as those posed by the BRAIN Initiative® and various Common Fund initiatives.Design & Data SharingEncourage innovative clinical trial design and data-sharing activities._2ab165b2-8aa7-11e7-a015-e3bd1f25b93b3.7.3FDAOn the translational and clinical front, NIH will encourage innovative clinical trial design and data-sharing activities through collaborative efforts with the FDA and other stakeholders.Communication & CoordinationFacilitate communication and coordination among clinical researchers about new trial designs and best practices._2ab16940-8aa7-11e7-a015-e3bd1f25b93b3.7.4Clinical ResearchersIn addition, NIH will continue to facilitate communication and coordination among clinical researchers about new trial designs and best practices, whether at the level of policies for funded researchers, interdisciplinary working groups, or clinical trial networksPartnershipsEnhance Impact through Partnerships._2ab16be8-8aa7-11e7-a015-e3bd1f25b93b3.8To increase the reach of NIH-funded research, NIH will leverage its resources by partnering with other organizations in the public and private sectors. Not only do such partnerships enable NIH to make maximum use of finite resources, they can lead to sector-spanning synergies that result in creative new ways of fulfilling NIH's mission.Trans-Disciplinary KnowledgeCapitalize upon trans-disciplinary knowledge._2ab17110-8aa7-11e7-a015-e3bd1f25b93b3.8.1This will include building on current efforts within NIH to capitalize upon trans-disciplinary knowledge, as well as fostering mechanisms to establish new collaborations.CollaborationFostering mechanisms to establish new collaborations._2ab174ee-8aa7-11e7-a015-e3bd1f25b93b3.8.2Drugs, Technologies & PracticesTranslate NIH research findings into new drugs, technologies, and evidence-based practices for improving health._2ab17778-8aa7-11e7-a015-e3bd1f25b93b3.8.3FDACDCAHRQHRSASAMHSAFederal AgenciesFor example, NIH recently partnered with other federal agencies to develop the National Strategy for Combating Antibiotic-Resistant Bacteria.National Institute of Allergy and Infectious DiseasesAlso, NIH's National Institute of Allergy and Infectious Diseases leads an interagency workgroup on developing therapeutics and vaccines against the emerging global public health threat posed by the Middle East Respiratory Syndrome Coronavirus (MERS-CoV). Other federal agencies represented include CDC, FDA, Department of Defense, and Biomedical Advanced Research and Development Authority. Department of DefenseBiomedical Advanced Research and Development AuthorityTissue Chip for Drug Screening ProgramOther outstanding examples of cross-agency collaboration are the Tissue Chip for Drug Screening program, in which NIH, FDA, and DARPA are collaborating to develop 3D human tissue chips that mimic human physiology.DARPAInteragency Artificial Pancreas Working GroupOn another highly innovative front, the Interagency Artificial Pancreas Working Group, in which the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Biomedical Imaging and Bioengineering, and the Eunice Kennedy Shriver National Institute of Child Health and Human Development are working with FDA, patient advocates, and industry toward development of an artificial pancreas, which could be the most revolutionary advance in diabetes care since the discovery of insulin. National Institute of Diabetes and Digestive and Kidney DiseasesNational Institute of Biomedical Imaging and BioengineeringEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentPatientsPatients, disease advocacy organizations, and community members at the local, state, and federal levels also are playing an increasingly significant role in spurring advances in biomedical research. Consequently, NIH will embrace these and other members of the public as active partners in the research enterprise, with the aim of generating more effective -- and more relevant -- research outcomes.Disease Advocacy OrganizationsCommunity MembersVolunteersThis will include seeking input from diverse volunteers at all stages of the research process, from study design to data collection and analysis. The PMI cohort will be among the NIH-led efforts pioneering this highly interactive, proactive participation model.As part of this activity, NIH will work closely with FDA, CDC, AHRQ, HRSA, SAMHSA, and other HHS and federal agencies that will provide the necessary knowledge and expertise to help translate NIH research findings into new drugs, technologies, and evidence-based practices for improving health.OutreachReach out to previously underrepresented groups._2ab17c50-8aa7-11e7-a015-e3bd1f25b93b3.8.4Underrepresented GroupsBesides forging partnerships with individuals, NIH's new model for research will underscore the importance of reaching out to previously underrepresented groups, consulting with communities, providing equal access to research studies and results, protecting patient privacy, and conducting research in an ethical and responsible manner.ConsultationConsulting with communities._2ab17f7a-8aa7-11e7-a015-e3bd1f25b93b3.8.5AccessProvide equal access to research studies and results._2ab18218-8aa7-11e7-a015-e3bd1f25b93b3.8.6PrivacyProtect patient privacy._2ab186d2-8aa7-11e7-a015-e3bd1f25b93b3.8.7PatientsEthics & ResponsibilityConduct research in an ethical and responsible manner._2ab18a10-8aa7-11e7-a015-e3bd1f25b93b3.8.8Public-Private PartnershipsCultivate public-private partnerships with health-related industries._2ab18cae-8aa7-11e7-a015-e3bd1f25b93b3.9Health-Related IndustriesSmall BusinessesVenture Capital CompaniesBiotech CompaniesPharmaceutical CompaniesAccelerating Medicines PartnershipSuch efforts, exemplified by the Accelerating Medicines Partnership, will bring together the expertise and resources necessary to address the gap in the development pipeline between scientific discovery and the commercial marketplace, with the goal of turning breakthrough basic science discoveries into useful drugs and other biomedical products more quickly.Foundation for the National Institutes of HealthHelping to facilitate NIH's public-private partnerships is the Foundation for the National Institutes of Health (FNIH), which Congress established in 1990 as an independent non-profit charged with supporting NIH's mission.Biomarkers ConsortiumFor example, FNIH manages The Biomarkers Consortium, which brings together industry, patient advocacy organizations, academia, and government agencies and institutes to develop and support research aimed at qualifying promising biological markers for use in diagnosing disease, predicting therapeutic response, or improving clinical practice. Current members include NIH, FDA, the Pharmaceutical Research and Manufacturers of America (PhRMA), the Centers for Medicare & Medicaid Services (CMS), and the Biotechnology Industry Organization (BIO), along with more than 30 companies and not-for-profit organizations. NIHFDAPharmaceutical Research and Manufacturers of America (PhRMA)Centers for Medicare & Medicaid Services (CMS)Biotechnology Industry Organization (BIO)Another important area of partnership that NIH will seek to encourage over the next 5 years is cultivating public-private partnerships with health-related industries, including small businesses, venture capital companies, biotech companies, and large pharmaceutical companies.Clinicians & Professional SocietiesStrengthen ties and forge new partnerships with clinicians and professional societies._2ab19172-8aa7-11e7-a015-e3bd1f25b93b3.10CliniciansProfessional SocietiesAHRQHealthcare ProfessionalsPhysicians, nurses, and other healthcare professionals, both individually and collectively, are essential for the design and implementation of NIH-supported research studies, the timely dissemination and implementation of evidence-based practices into healthcare, and the education of fellow clinicians, patients, and the general public about evidence-based interventions and treatments.PhysiciansNursesNIH, in collaboration with AHRQ and other HHS agencies, will also seek to strengthen its existing ties to and forge new partnerships with clinicians and professional societies.Global Health ChallengesReach out to partners that are integral to its efforts to address global health challenges._2ab194a6-8aa7-11e7-a015-e3bd1f25b93b3.11In addition to partnerships within the United States, NIH has a responsibility to reach out to partners that are integral to its efforts to address global health challenges and improve the health of all humankind. Such actions are not only consistent with our nation's scientific and humanitarian values, but are frequently in our own best interest because infectious diseases do not respect national boundaries. Forging such partnerships involves negotiating international collaborations with nongovernmental organizations, private industry, and governments of other nations. These partnerships may serve to promote biomedical science, for example, by sharing samples and data, or by building research capacity, such as is being done in the Human Heredity and Health in Africa (H3Africa) initiative and the Medical Education Partnership Initiative in sub-Saharan Africa. Other globally oriented partnerships promote the implementation of research results, such as NIH's milestone-driven projects with the Bill & Melinda Gates Foundation to reduce premature births, improve maternal and infant nutrition, develop models to accelerate drug discovery for tuberculosis, design vaccines against HIV and other infectious diseases, and devise affordable point-of-care diagnostic technologies. Risk ManagementEngage in Proactive Risk Management Practices._2ab19758-8aa7-11e7-a015-e3bd1f25b93b3.12To meet the evolving needs of an ever-changing and increasingly challenging biomedical research environment, NIH's risk management abilities will continue to grow and mature over the next 5 years. Using standardized approaches, NIH must systematically assess its administrative processes, operational procedures, and scientific programs, for potential risks that could lead to failure. The identified risks must be prioritized and then proactively addressed by applying appropriate human and monetary resources to minimize, monitor, and control the potential impact of these risks to the NIH mission. Over the next 5 years, NIH will pursue these and many forward-looking measures to reinvigorate our role as a visionary, yet careful, steward of the resources entrusted to us by the American people. Such actions will ensure that the U.S. biomedical research enterprise remains firmly on the pathway to a bright and sustainable future. ResultsExcel as a Federal Science Agency by Managing for Results_2ab19cee-8aa7-11e7-a015-e3bd1f25b93b4As a public science agency, NIH is obligated to use transparent, scientific approaches in its decision making. Ultimately, NIH is accountable to the American people, who have every right to expect all of their government agencies not only to perform, but also to excel. To fulfill this responsibility in a thoughtful manner that goes beyond one-size-fits-all solutions, NIH will build upon its strong tradition of excellence by managing for results in the following ways: Science of ScienceDevelop the "Science of Science."_2ab1a04a-8aa7-11e7-a015-e3bd1f25b93b4.1NIH will take greater leadership in developing and validating the methodologies that are needed to evaluate scientific investments. For example, new approaches to portfolio analyses have been devised that allow for a rapid assessment of potential overlap and gaps.BenchmarkingCompare each ICO to one another as well as agencies and foundations for which grant portfolio data is available._2ab1a310-8aa7-11e7-a015-e3bd1f25b93b4.1.1NIH ICOsOver the next 5 years the portfolio of each ICO will be compared to one another as well as those agencies and foundations for which grant portfolio data is available.Bibliometric MeasuresPromote bibliometric measures._2ab1a87e-8aa7-11e7-a015-e3bd1f25b93b4.1.2NIH has also promoted more robust bibliometric measures through development of disambiguation tools and a normalized citation metric termed the Relative Citation Ratio (RCR). In addition, the agency is considering outside bibliometric approaches, such as those developed by the Eigenfactor® Project.Value AssessmentIdentify better tools to assess the value of each grant._2ab1ac02-8aa7-11e7-a015-e3bd1f25b93b4.1.3LaboratoriesHowever, more tools are clearly required to help NIH better assess what value each grant in its portfolio provides and to test whether the mechanisms that it is employing for supporting investigators is optimal. For example, several recent studies have suggested that there is a limit to the value added in providing more and more funding to a single laboratory. Outputs & OutcomesBalance Outputs with Outcomes._2ab1aec8-8aa7-11e7-a015-e3bd1f25b93b4.2By their nature, outputs are easier to measure and have a shorter lag time between the onset of the activity and the "result." In contrast, outcomes, which should have some effect on the external environment, are much harder to measure, in part, because the lag time between the activity and the "result" is longer and often unpredictable. Further, because outputs are easier to measure, organizations often use them without full consideration of the perverse incentives they may be creating. Much of NIH's investment is made through its grant portfolio. In an attempt to evaluate the "success" of a grant, a wide range of scientific outputs can now be assembled, each with its own inherent flaws. Nevertheless, the use of bibliometrics that account for variations in publication and citation practices among different scientific disciplines can provide a preliminary indication of a program’s "value." The number of patents and/or investigational new drug (IND) applications filed can also be a surrogate for program worth, but again, these take time, and by themselves are not necessarily predictors of outcomes that may take much longer to realize. For example, who would have assigned a very high value to early research on thermophilic microorganisms in the late 1960s that ultimately led to the discovery of Taq polymerase and the development of the polymerase chain reaction (PCR) technique that fueled the biotech revolution? For evaluation of NIH-supported training, a standard approach is to count the number of trainees in tenure-track or tenured positions at universities around the world. This is a relatively facile measure in the era of social media. But this has led to a systematic undervaluation of trainees who have gone on to important careers in industry, policy development, intellectual property adjudication, or teaching, to list a few. NIH has recently made providing postdocs and graduate students with a broad exposure to career options as a critical part of a successful training program, and will now align its stated goals with the measures used to assess the programs that undergird the efforts. Improving the health of the nation and the world is the ultimate outcome that NIH aims to achieve. And over the last several decades, it is clear that NIH-supported research has had a major positive impact on human health. Major prizes, such as the Laskers and Nobels, are also an indication of how the world views major advances in science, and NIH has been responsible for supporting the work of a very large number of those honored with such prizes over the last few decades. But within the 5-year horizon contemplated for this Strategic Plan, it would be difficult to chart a course toward widespread population benefit from NIH investments in research -- the timelines are just too long.Workforce AnalysesConduct Workforce Analyses._2ab1b3f0-8aa7-11e7-a015-e3bd1f25b93b4.3UniversitiesResearch InstitutesPolicy Think TanksEducational SystemIn general, workforce analysis has proven to be challenging for many fields, but NIH has created a static representation of the Ph.D. workforce and is currently working on a dynamic model that can be used in concert with all relevant stakeholders (universities, research institutes, industry, the federal government, policy think tanks, and the K-community college educational system) to better predict the number of Ph.Ds. and postdoctoral fellows that would be optimal for NIH to support. Particularly vexing is the continued decline of physician-scientists. A recent snapshot has been generated of the M.D., M.D.-Ph.D. census, but more work must be done to design interventions that will increase the number of these invaluable members of the workforce. While NIH has engaged in many research partnerships, it will also formulate and evaluate new approaches to engaging physician-scientists through inclusion of professional organizations, academic health center leadership, and, of course, the trainees themselves. Peer ReviewContinuously Review Peer Review._2ab1b788-8aa7-11e7-a015-e3bd1f25b93b4.4There are many other elements of peer review that demand continuous evaluation. As science becomes more interdisciplinary in nature, new approaches to review need to be tested and validated, including asynchronous, electronic reviews and two- or three-stage "editorial board" models. In addition, the cost/benefit ratio of each must be evaluated. NIH will also continue to seek measures to compare the "performance" of each study section.Rigor & ReproducibilityEvaluate Steps to Enhance Rigor and Reproducibility. _2ab1ba6c-8aa7-11e7-a015-e3bd1f25b93b4.5Science AgenciesJournal PublishersProfessional SocietiesUniversitiesFoundationsNIH, in partnership with fellow science agencies, journal publishers, professional societies, universities, foundations, and a number of other stakeholder groups, has launched a series of initiatives to enhance the rigor and reproducibility of the conduct of science and reporting of scientific results. Each of these initiatives will be evaluated over the next 5 years for beneficial effects, as well as for any unanticipated, negative consequences.Administrative BurdenReduce Administrative Burden._2ab1bf94-8aa7-11e7-a015-e3bd1f25b93b4.6CongressThere are a wide range of administrative burdens placed upon NIH's stakeholders, and several recent studies have enumerated many of these. In approaching this issue, NIH first must classify each burden with regard to origin -- some are mandated in law; others are rooted in policy; and still others can be traced to historic custom. NIH's goal over the next 5 years will be to reduce or, wherever possible, eliminate those burdens that arise from custom and/or policy. NIH will also work with Congress to ascertain which of the burdens arising from laws can be modified to provide some relief.Risk ManagementTrack Effectiveness of Risk Management in Decision Making._2ab1c3d6-8aa7-11e7-a015-e3bd1f25b93b4.7Winston Churchill once said: "Never let a good crisis go to waste." When unexpected issues arise, it is important to do a formal analysis of not only what events occurred, but also why they occurred. In this manner, NIH's risk management system can be continuously adapted to include new elements that had not previously been considered or even anticipated. 2016-10-012020-09-302017-08-27https://www.nih.gov/sites/default/files/about-nih/strategic-plan-fy2016-2020-508.pdfOwenAmburOwen.Ambur@verizon.net