American Society of Hematology

NIH Director Outlines NCATS Vision in a Commentary

Published on: July 06, 2011

In a Commentary published today in the journal Science Translational Medicine, the National Institutes of Health (NIH) Director Dr. Francis S. Collins set forth NIH's vision for advancing translational science. The Commentary provides a detailed description of the scientific goals and functions of the National Center for Advancing Translational Sciences (NCATS), a proposed new entity of NIH that will strive to reengineer the process of developing drugs, diagnostics, and devices.

The field of translational science currently stands at a critical juncture, according to Dr. Collins. In recent years, basic researchers have made tremendous progress in identifying the molecular causes of disease – discoveries that have revealed hundreds of potential new therapeutic targets. However, the rate at which these discoveries are moving from the lab to the clinic has not kept pace, and therapies exist for just 200 of the more than 4,000 conditions with defined molecular causes. Among the problems is that the translational pipeline is full of bottlenecks that slow the process and add expense. NCATS will seek to generate innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions with the goal of significantly shortening what currently takes about 15 years from molecular discovery to new therapy.

The Commentary lists several components of translational science that are "ripe for the new scientific approach offered by NCATS" and will likely be the subject of early targeted funding opportunities, based on "early discussions with a variety of stakeholders":

  • Therapeutic target validation
  • Chemistry
  • Virtual drug design
  • Preclinical toxicology
  • Biomarkers
  • Efficacy testing
  • Phase zero clinical trials
  • Rescuing and repurposing
  • Clinical trial design
  • Postmarketing research

Dr. Collins notes that NCATS will require participation and partnerships from academia, industry, patient advocacy groups, regulatory agencies, and philanthropies in order to drive forward the science of translational medicine. Dr. Collins also emphasizes that NIH will continue its strong support for basic science, which has been the bedrock of NIH's success over many decades. In fact, he suggests that the "virtuous cycle" between basic and applied research may be speeded up by the NCATS model, allowing new and interesting basic science problems to emerge.

Although the mission of the proposed NCATS – to advance the discipline of translational science and catalyze the development of novel diagnostics and therapeutics across a wide range of human diseases and conditions – is generally supported by the research community, the proposed transition and dissolution of NCRR has not been without some controversy. Some stakeholders question whether the NIH should get involved in research aimed at attracting the pharmaceutical industry, whether the new Center will be able to actually leverage outcomes of the translational research programs funded by the NIH, and whether this shift of focus toward translational science for creation of therapeutics will negatively affect investigator-initiated basic science research. Dr. Collins has tried to allay those fears by saying that the goals of creating the new Center are to facilitate – not duplicate – efforts in developing therapeutics, to complement – not compete – with the private sector, and to reinforce – not reduce – NIH's commitment to basic science research.

Earlier this month, Congressman Dennis Rehberg, Chairman of the House Appropriations Committee's Subcommittee on Labor, Health and Human Services, Education, and Related Agencies (R-MT) had written to Health and Human Services (HHS) Secretary Kathleen Sebelius expressing concerns about the NCATS)and suggested that the National Institutes of Health (NIH) "cease all action" related to establishing the new center. Representative Rehberg explained that his concerns stem from the relatively fast pace of the creation of NCATS and the "scant level of information provided to the Appropriations Committee thus far."

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