Introducing the NIH’s National Center for Advancing Translational Sciences: Improving Outcomes from the Same Inputs

The U.S. government has recognized that the process of translating scientific discoveries into new medical solutions is ripe for innovation, and acknowledged the need to enhance their commitment to this effort.
The recently created National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), seeks to make the development of new, life-saving diagnostics and therapeutics more efficient, less expensive, and less risky.
Working across institute boundaries to gather existing translational initiatives into an integrated scientific enterprise, it will focus on studying the pathway to commercialization and highlighting bottlenecks in the drug development pipeline.


bridges, vol. 33, May 2012 / OpEds & Commentaries
By Margaret Anderson

Margaret AndersonStem cells. Genes linked to Alzheimer's, autism, or diabetes. Cancer drugs tailored to treat an individual tumor.
Every day we see stories in the media about the latest medical "breakthroughs" that could lead to cures for dreaded diseases. And yet, too often these breakthroughs are not translated into therapies that can improve health and save lives.

The fact is that many basic discoveries barely get to start the journey down the therapeutic development pipeline, often getting get stuck in translation because they lack the funding, incentives, and technical expertise to advance any further. Known by many as the "Valley of Death" this gap in resources and support for the kind of research that moves basic science down the path toward in-human clinical trials impedes medical progress.

Companies today are spending more than $1 billion, and an average of 15 years, on research and development for each new approved drug or biologic1 , yet 80 to 90 percent of research projects fail before they ever get to be tested in humans2 . And the number may be even higher: By industry's estimates, only 1 in 10,000 potential therapeutic compounds make it through the drug development pipeline to the marketplace3 .

Whether it be limited resources or restrictive policies that are stalling progress, patients are paying the price in life-or-death consequences.

Bridging the “Valley of Death” in drug development: The long way from the laboratory bench to the patient’s bedside. (Source: Parkinson’s Action Network and FasterCures)


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Threading the Needle

Recently, universities have shown increasing interest in stretching their work into the "Valley of Death," and companies are rethinking their alliances with academics and nonprofits as sources of innovative products to fill their pipelines. Disruptive innovators from across the medical research enterprise are poised to change the face of R&D by employing novel approaches to partnership building, data exchange, clinical trials, and funding. Even the government, recognizing that the process of translating scientific discoveries into new medical solutions is ripe for innovation, has acknowledged the need to enhance their commitment to this effort.

Academic research and industry partners have long noted the strong, symbiotic relationship between funding in basic research by the National Institutes of Health (NIH) and subsequent investment and innovation in the private economy. In fiscal year 2010, NIH spent $26.6 billion on research awards4, and a study by the National Bureau of Economic Research concluded that a dollar of NIH support for research leads to an increase of private medical research of roughly 32 cents5.

NIH Director Francis Collins said it best: "If there was ever a time when we all need to get together in the same room and figure out how to take this wonderful scientific opportunity and apply the most bold, audacious principles of innovation to make those products happen, it is now." It was under his vision that NIH recently created the National Center for Advancing Translational Sciences (NCATS). With a budget of about $575 million, derived primarily from preexisting NIH programs, NCATS seeks to make the development of new, life-saving diagnostics and therapeutics more efficient, less expensive, and less risky.

Approved by Congress as part of the fiscal year 2012 spending bill and signed into law by President Obama on December 23, 2011, NCATS will work across institute boundaries to gather existing translational initiatives into an integrated scientific enterprise that studies the pathway to commercialization and highlights bottlenecks in the drug development pipeline.

Described by Acting Director Dr. Thomas Insel as "disease-agnostic," the vision of the center is to "try and improve outcomes from the same inputs."

Collins and Insel have both noted that the purpose of NCATS is to "facilitate – not duplicate – other translational research activities supported by NIH; to complement – not compete with – the private sector; and to reinforce – not reduce – NIH's commitment to basic research."

At its core, NCATS aims to ensure that Americans recognize a better return on the enormous investment of their tax dollars in the form of improved health and cures for disease. A number of programs that are already underway demonstrate NCATS' potential:

  • Discovering New Therapeutic Uses for Existing Models, a pilot program designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance therapeutic development. The initiative will give researchers access to more than 20 compounds initially developed by AstraZeneca, Eli Lilly and Co., and Pfizer.
  • Research Electronic Data Capture (REDCap), a secure Web application that enables investigators to create standardized surveys, easily transfer data, and export data into a variety of statistical programs;
  • The Learning Collaborative, a partnership between the Therapeutics for Rare & Neglected Diseases (TRND) program at NCATS, University of Kansas Cancer Center, and the Leukemia & Lymphoma Society that aims to target repurposed drugs and novel therapies for the treatment of rare blood cancers, and work with industry partners to develop them.
  • An NIH/FDA/DARPA collaboration that aims to develop a tissue chip that mimics human physiology to screen for safe, effective drugs.

However, along with the rest of the NIH, NCATS' funding is threatened by looming across-the-board cuts for all federal agencies if a budget agreement is not reached for 2013. According to United for Medical Research, this sequester would result in a 7.8 percent cut in funding for research supported by NIH, including 33,000 fewer jobs across the U.S. and a $4.5 billion decrease in economic activity.

NIH is crucial to retaining and bolstering U.S. leadership in biomedical innovation, and most importantly, NIH investment can yield outcomes that save lives. That is why we must maintain funding for the institutes, and specifically for NCATS – to improve our odds of getting to cures faster, cheaper, and better.

Patients are Waiting

The creation of NCATS is an important step toward realizing the unprecedented scientific opportunities that exist today and saving time in the way we translate discoveries into effective products that improve human health. For patients whose treatment options are limited or nonexistent, saving time means saving lives.

There is no time to waste.

 ***

The author, Margaret Anderson, is executive director of FasterCures/The Center for Accelerating Medical Solutions.

 

References:

1 Pharmaceutical Research and Manufacturers of America (PhRMA). 2011. Profile 20011. Washington, DC: PhRMA.
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2
NIH Therapeutics for Rare and Neglected Diseases Program announces initial drug development projects. (n.d.). Retrieved March 12, 2012, from National Center for Translational Therapeutics: <http://nctt.nih.gov/27544933>
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3
Pharmaceutical Research and Manufacturers of America (PhRMA). 2008. Profile 2008. Washington, DC: PhRMA
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4
Ehrlich, Everett. "An Economic Engine: NIH Research, Employment, and the Future of the Medical Innovation Sector," United for Medical Research (UMR). March 2012.
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5
Margaret E. Blume-Kohout, Krishna B. Kumar, and Neeraj Sood, "Federal Life Sciences Funding and University R&D," National Bureau of Economic Research Working Paper 15146, July 2009.
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