Charting The Future Together

Objectives

The National Heart, Lung, and Blood Institute (NHLBI) seeks to prevent and reduce the burden of heart, lung, blood and sleep diseases, promote longer and more fulfilling lives, and advance personalised precision medicine by delivering the right treatment to the right person at the right time.[1][2]Its Strategic Vision provides a decade-long framework for research, training and education, organised around four mission-oriented goals: understand human biology, reduce human disease, advance translational research, and develop workforce and resources.[2][3]

• Understand normal biological function and resilience, including biological, behavioural, social and environmental influences on health, healthy ageing and adaptation throughout life.[1][4][5]
• Investigate newly discovered mechanisms underlying the onset and progression of heart, lung, blood and sleep diseases, with emphasis on preventing transition from health to disease.[1][6][7]
• Identify factors driving differences in health between populations, including biological, behavioural, environmental and socioeconomic influences on susceptibility, disease progression and outcomes.[1][8]
• Explain individual variation in disease biology and treatment response to support precise prevention, diagnosis and tailored interventions.[1][9][10]
• Develop and optimise diagnostic and therapeutic strategies to prevent, treat and cure disease, including gene editing, regenerative medicine, bioengineering, blood-product safety, transplantation, sleep health and palliative care.[1][11][12][13]
• Optimise clinical and implementation research so that discoveries are translated efficiently into clinical practice, programmes and population health impact.[1][14][15]
• Use data science to integrate genomic, clinical, environmental, behavioural, imaging, sensor and other information, improving disease classification, risk prediction and therapeutic discovery.[1][15][16]
• Develop, diversify and sustain a capable scientific workforce across the career continuum, while strengthening rigorous, reproducible and interdisciplinary research.[1][16][17]

Priority areas span fundamental, behavioural, clinical, population, implementation and data-science research.[18]The agenda supports investigator-initiated discovery while pursuing targeted opportunities in early disease development, health inequities, environmental exposures, cardiometabolic risk, molecular mechanisms, imaging, omics, novel therapeutics and advanced cellular technologies.[18][2][8][9]

Implementation

NHLBI will implement the Strategic Vision through a balanced research portfolio that combines investigator-initiated science with Institute-solicited initiatives and Funding Opportunity Announcements addressing emerging opportunities and knowledge gaps.[18][17]The Vision is intended as an adaptive, living framework that guides investment and activities without restricting scientific creativity.[18][2]

• Engage researchers, clinicians, patients, advocates, policymakers, professional groups and the public in ongoing priority-setting, implementation and periodic renewal of the Strategic Vision.[2][3][19]
• Use webinars, forums, think tanks, workshops and working groups to identify practical activities and incorporate new ideas as science and community needs evolve.[18][19]
• Seek advice from NHLBI staff, the National Heart, Lung, and Blood Advisory Council and the Board of External Experts when prioritising questions according to timeliness, feasibility and potential scientific benefit.[1][3]
• Apply advanced tools, including nanoscale imaging, single-cell analysis, systems biology, molecular imaging, deep phenotyping, high-throughput omics and predictive modelling, across studies from cells and animal models to communities and healthy populations.[4][5][6][7][10]
• Strengthen reproducibility through diverse investigational models, improved access to hard-to-culture and female-derived cells, and standardised cell-line protocols.[6]
• Integrate laboratory, clinical, population and community-centred implementation research to examine environmental, dietary, social and psychosocial exposures alongside genetic, epigenetic and molecular factors.[5][7][8][9]
• Embed clinical epidemiology registries, trial networks and implementation science programmes in learning health-care systems, using electronic health records, existing datasets and personal health data to identify participants and connect research with care.[14]
• Build shared data and biospecimen resources, including linked health-record, imaging, exposure, sensor, genomic and other omics data, supported by cloud-based platforms and scientific data commons.[15][20][16]
• Develop partnerships among basic, translational, patient-oriented, community and population researchers, alongside clinicians, engineers, bioinformatics specialists, academic institutions, non-profit organisations, industry and international collaborators.[15]
• Engage private-sector partners early in diagnostic and therapeutic development to facilitate commercialisation, and pursue multidisciplinary and multinational strategies for chronic diseases in developing countries.[15]
• Expand lifelong training, mentoring and career support for diverse learners and scientists, including skills in clinical research, drug development, biostatistics, computer science, bioinformatics, communication, cultural competence and commercialisation.[16][21][17]

Evidence generation will use longitudinal tracking of disease onset and progression, biomarkers of exposures and treatment response, clinical outcomes, registries and integrated datasets.[6][7][14][20]Education and employment data will also be collected to identify metrics and predictors of workforce and training-programme success.[17]However, the available text does not specify a document-wide performance indicator set, reporting timetable, formal evaluation design, accountability framework, implementation budget, financing allocation or costed delivery plan.[2][3][14][17]

Monitoring & Evaluation

The Strategic Vision treats priority-setting as a continuing, adaptive process rather than a fixed implementation plan. It relies on ongoing engagement with the research community to identify, reconsider and refine compelling research questions as scientific opportunities and needs change.[18][2][3][19]Formal document-wide indicators, targets, reporting cycles, evaluation designs, surveillance arrangements and accountability mechanisms are not specified.[2][3][14][17]

• Refresh strategic priorities periodically through a living Strategic Visioning process, retaining submitted ideas for reconsideration when circumstances or opportunities evolve.[2][3][19]
• Engage researchers, clinicians, patients, advocates, policymakers, professional groups, advisory bodies and the wider public in priority-setting and implementation discussions.[1][2][3][19]
• Use webinars, workshops, think tanks and working groups to translate broad priorities into specific activities and to sustain dialogue with the scientific community.[18][19]
• Track disease onset and progression across the life course, including the influence of early exposures, interventions and lifestyle changes on transitions from health to chronic or acute disease.[6]
• Assess biomarkers of environmental exposure, disease onset and progression, treatment response, blood-product stability, transfusion success, sleep dysfunction, bleeding risk and cardiopulmonary disease.[7][10][12][13]
• Use deep phenotyping, genomics and other omics, imaging, clinical outcomes and cohort studies to investigate genetic variants, gene knockouts and differences in disease biology and treatment response.[7][9][10]
• Embed electronic health records, patient registries, clinical epidemiology registries, trial networks, datasets, biospecimen repositories, exposure data and personal sensor information within learning health-care systems to strengthen evidence generation.[14][15][20]
• Improve clinical-research effectiveness through timely and efficient trials, stronger project management, improved population estimation and generalisability, and translation of findings into programmes and policies.[15]
• Collect and analyse education and employment data across scientists’ careers to define success metrics and predictors for individuals and training programmes.[17]
• Strengthen training in clinical research, pre-clinical drug development, biostatistics, computer science and bioinformatics to support the evidence base and research workforce.[15][20]

Costing & Financing

No budget, costed workplan, financing allocation, funding-gap estimate, resource-mobilisation target or economic assumption is provided for the Strategic Vision or its research priorities.[2][3][14][16][17]The Vision nonetheless identifies substantial resource needs across research infrastructure, workforce development, therapeutic innovation and data-enabled clinical research.[15][16][21]

• Support investigator-initiated discovery science as the principal mechanism for funding research, complemented by Institute-solicited initiatives and Funding Opportunity Announcements addressing emerging opportunities and knowledge gaps.[18][17]
• Expand resources for identifying therapeutic targets and agents, establishing proof of concept and preparing data for investigational new drug applications.[15]
• Develop infrastructure for coordinated data access, scientific data commons, planned sample analyses, integrated omics and registry datasets, and advanced bioinformatic and biostatistical methods.[16]
• Invest in workforce exposure, lifelong training, mentoring, cultural competence and interdisciplinary skills for diverse and disadvantaged learners and researchers.[16][21][17]
• Develop large-scale production capacity for designer blood products, alongside advanced implants, diagnostics and therapies, although associated costs and financing arrangements are not specified.[13]
• Improve clinical-research cost-effectiveness and address cost containment through better data use, trial efficiency and project management.[14][15]
• Develop private-sector partnerships early in diagnostic and therapeutic product development to support commercialisation.[15]
• Recognise that heart, lung, blood and sleep diseases impose major health and economic burdens in the United States, while rare disorders create substantial burdens for affected people.[18]