Objective 6: Optimize clinical and implementation research to improve health and reduce disease

New methodologies, research frameworks, and resources need to be developed to refine the conduct of clinical studies and implementation research, while also improving the overall efficiency and effectiveness of the research enterprise. Current challenges include difficulties in recruitment of research participants and effective translation of knowledge gained from these studies into programs and policies that improve health at the clinic and community levels.

Envision a future in which we are able to...
  • Use novel platforms that link electronic health records and personal health data to facilitate the identification and enrollment of participants in clinical research and empower research volunteers to self-identify.
  • Accelerate the translation of discovery into practice by embedding HLBS research platforms (e.g., clinical epidemiology registries, clinical trial networks, and implementation science programs) within “learning” health care systems, thereby engaging the clinical care and patient communities; leveraging electronic health records, patient registries, and existing datasets; and creating an integrative interface between clinical research and practice.

Related Priorities

Compelling Question
What methods and technologies are effective for increasing awareness of and participation in clinical research, as well as awareness of and access to evidence-based diagnostics and therapeutics, including emerging approaches to care? (6.CQ.01)
Compelling Question
What clinical trial designs are best for studying the chronobiology of drug delivery? (6.CQ.02)
Compelling Question
How can we engage relevant stakeholders, including patients, private entities, and federal agencies, to improve the clinical research enterprise and address critical needs such as standardized informed consent and cost containment? (6.CQ.03)
Critical Challenge
Synergy and collaboration among people at the MD and PhD level for; basic science; translational, patient-oriented researchers; community and population scientists; and individuals from multiple disciplines (e.g., engineers, clinicians, subspecialists, generalists, bioinformatics experts, academics, nonprofit organizations, industry) are needed to enhance and expedite advances in HLBS research. (6.CC.01)
Critical Challenge
Improvements in clinical trial design, population estimations, project management, and other practices are needed to achieve timely trial completion. (6.CC.02)
Critical Challenge
Skills development and training are needed to improve the navigation of pre-clinical new drug phases of translational science. (6.CC.03)
Critical Challenge
Innovative approaches to private sector collaborations and partnerships are needed early in therapeutic and diagnostic product development to bridge the gap between academic discoveries and product commercialization. (6.CC.04)
Critical Challenge
Expanded resources for identifying therapeutic targets and agents, establishing proof of concept, and developing data for investigational new drug applications are needed to enable the early translation of research findings to clinical applications. (6.CC.05)
Critical Challenge
Creative use of the array of newly available data sources is needed in clinical trial design and conduct in order to improve efficiency, cost effectiveness, and generalizability. (6.CC.06)
Critical Challenge
Creative approaches to clinical trials in rare HLBS diseases are needed to successfully test strategies that will expand preventive and therapeutic options. (6.CC.07)
Critical Challenge
Standardized approaches and resources, including data and biospecimen repositories, should be developed to facilitate collaboration between basic, clinical, and population scientists in clinical trials and population studies. (6.CC.08)
Critical Challenge
Creative approaches are needed to effectively transcend silos (e.g., perinatal, pediatric, and adult divides in clinical and translational research). (6.CC.09)
Critical Challenge
Novel methodologies and improvements in existing methodologies are needed for implementation research that explores uptake of research findings into approaches, programs, and policies. (6.CC.10)
Critical Challenge
Multidisciplinary, multinational partnerships are needed to develop effective and sustainable strategies for combating chronic HLBS disorders in developing nations, which take into account the highly variable local epidemiology of HLBS disorders, the need for novel approaches to reducing disease burden, and the challenges of implementation in developing countries. (6.CC.11)

Overarching Objectives

  1. Understand normal biological function and resilience
  2. Investigate newly discovered pathobiological mechanisms important to the onset and progression of HLBS diseases
  3. Investigate factors that account for differences in health among populations
  4. Identify factors that account for individual differences in pathobiology and in responses to treatments
  5. Develop and optimize novel diagnostic and therapeutic strategies to prevent, treat, and cure HLBS diseases
  6. Optimize clinical and implementation research to improve health and reduce disease
  7. Leverage emerging opportunities in data science to open new frontiers in HLBS research
  8. Further develop, diversify, and sustain a scientific workforce capable of accomplishing the NHLBI’s mission