Strategic Plan Introduction

Overview of NCATS

The National Center for Advancing Translational Sciences (NCATS), one of the 27 Institutes and Centers (ICs) at the National Institutes of Health (NIH), is transforming translational science to get more treatments to more patients more quickly. NCATS relies on the power of data, new technologies and teamwork to develop, demonstrate and disseminate innovations that reduce, remove or bypass costly and time-consuming bottlenecks in translational research.

Rather than targeting a particular disease or area of fundamental science, NCATS focuses on what is common across diseases and the translational process. The Center conducts and supports research on both the scientific and operational aspects of translation to lead to more predictive and successful development of new medical interventions, such as drugs, diagnostics, and medical devices, for all human diseases.

NCATS’ efforts by design complement and empower those of other NIH ICs, academic scientists, the private sector and the nonprofit community. By emphasizing collaboration, innovation, deliverables and team science, the Center serves as a catalyst to enable others in the translational research ecosystem to work more effectively.

Translation and Translational Science

Defining Translation and Translational Science

  • Translation is the process of turning observations in the laboratory, clinic and community into interventions that improve the health of individuals and the public — from diagnostics and therapeutics to medical procedures and behavioral changes.
  • Translational science is the field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process.

NCATS studies translation on a system-wide level as a scientific and operational problem.

Translational Science Spectrum

Translational Science Spectrum

The translational science spectrum, as illustrated in the graphic above, represents each stage of research along the path from the biological and chemical basis of health and disease to interventions that improve the health of individuals and the public. The spectrum is not linear or unidirectional; each phase builds upon and informs the others. At all stages of the spectrum, NCATS develops new approaches, demonstrates their usefulness, and disseminates the findings. Patient involvement is a critical feature of all phases in translation.

Basic Research — Basic research involves scientific exploration that can reveal fundamental mechanisms of biology, disease or behavior. Every stage of the translational research spectrum builds upon and informs basic research. NCATS scientists typically do not conduct basic research; however, insights gained from the Center’s studies along the translational spectrum can inform basic research.

Preclinical Research — Preclinical research connects the basic science of disease with human medicine. During this stage, scientists develop model interventions to further understand the basis of a disease or disorder and find ways to treat it. Testing of medical interventions is carried out using cell or animal models of disease; samples of human or animal tissues; or computer-assisted simulations of drug, device or diagnostic interactions within living systems.

Clinical Research — Clinical research includes studies to better understand a disease in humans and relate this knowledge to findings in cell or animal models; testing and refinement of new technologies in people; testing of interventions for safety and effectiveness in those with or without disease; behavioral and observational studies; and outcomes and health services research. The goal of many clinical trials is to obtain data to support regulatory approval for an intervention.

Clinical Implementation — The clinical implementation stage of translation involves the adoption of interventions that have been demonstrated to be useful in a research environment into routine clinical care for the general population. This stage also includes implementation research to evaluate the results of clinical trials and to identify new clinical questions and gaps in care.

Public Health — In this stage of translation, researchers study health outcomes at the population level to determine the effects of diseases and efforts to prevent, diagnose and treat them. Findings help guide scientists working to assess the effects of current interventions and to develop new ones.

Center Organization

NCATS’ divisions and offices span the entire spectrum of translational science. Through programs in its Division of Preclinical Innovation, the Center drives advances in early stages of the translational process, from target validation to first-in-human studies. Through its Division of Clinical Innovation, NCATS supports clinical and translational research, creating and sharing the expertise, tools and training needed to develop and deploy effective treatments in people. Cross-cutting programs in rare diseases, translational technologies, strategic alliances and other emerging areas address scientific and operational barriers to enable faster and more effective interventions that tangibly improve human health.

Bridging the Gap: Scientific and Operational Challenges and Opportunities in Translational Science

Several thousand genetic diseases affect humans, of which only about 500 have any treatment. A novel drug, device or other intervention can take about 14 years and cost $1 billion or more to develop, and about 95 percent never make it past clinical trials. Even when a new drug or other intervention is developed and shown to be effective in clinical trials, many years may pass before all patients who could benefit from it are identified and treated.

Numerous scientific and organizational roadblocks can limit the speed of progress. Obstacles along the path to translation include:

  • Lack of understanding about the science of translation and insufficient rigor in conducting translational research, leading to unpredictability and frequent failure of possible interventions
  • A shortage of qualified translational investigators
  • Organizational structures and incentives that do not encourage the teamwork essential to translational science
  • Inflexible and inefficient clinical trial implementation and low participation in studies
  • A lack of data interoperability
  • Insufficient tools and technologies to predict toxicology and efficacy in safety assessment
  • A shortage of qualified biomarkers for application in disease diagnosis and measurement of treatment response
  • Inadequate development and measurement of appropriate clinical outcome measures or endpoints, including patient reported outcomes