Tuesday, December 20, 2011

What is NIH's Role in Bridging the Valley of Death?

The spending bill referenced in our post yesterday will, if signed into law, establish the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health. Good news for our medical research and development enterprise, and for the millions of patients who need treatments and cures, the inclusion of funding for NCATS in the omnibus will help to speed the translation of basic discoveries into real world applications that will improve public health.

Last month at our
Partnering for Cures conference, a distinguished group of panelists from academic, advocacy, and government institutions examined the challenges and opportunities to bridging the "valley of death" between preclinical development and clinical research, and specifically explored the impact and appropriateness of NCATS’ role in this effort. Moderator

Anna Barker of Arizona State University (formerly of the
National Cancer Institute), challenged the panel to consider the rapid advancement of accessible information - who is the appropriate convener to push forward translational research?
Kathy Hudson of NIH explained the nuts and bolts of NCATS: instead of focusing on individual projects, NCATS will identify bottlenecks in the pipeline that slow the research process for all players. Hudson acknowledged that it is an "awkward time" given the difficult economic climate, but emphasized that the goal of this new center is to collaborate to address shared problems - not to duplicate what is already happening in the pharma/biotech industry.
University of Pennsylvania professor Garret FitzGerald agreed that NCATS will be helpful to "shine a light on problems," and pointed to the erosion of human capital in translational research. NCATS could play a role in rebirthing the discipline through the training of heterogeneous skills, he noted, which could be especially important in the era of comparative effectiveness research. FitzGerald argued that this type of focus would be politically uncontroversial and relatively inexpensive.

Academic scientist and entrepreneur Peter Lansbury said that government had a huge role to play, particularly in neurodegenerative disease. He noted that there a special need for tools that could help facilitate clinical trials for these types of diseases, where progression is erratic and often slow.
Louis DeGennaro of the
Leukemia & Lymphoma Society - which earlier this year signed an cooperative R&D agreement with the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the University of Kansas Medical Center to accelerate the development of potential clinical therapies for rare blood cancers - mentioned that an often overlooked topic during discussions of solution development is the role advocacy and disease advocacy groups play. The "fiduciary responsibility" he and his colleagues feel for patients served by LLS has driven them to develop creative partnerships to engage in translational research.
Chris Varma, Entrepreneur-in-Residence at Third Rock Ventures, noted that although the amount of life science venture capital funding continues to increase, a smaller quantity of individual investments are being made overall. Given this environment, NCATS "makes a lot of sense" in driving efficacy and reducing risk, especially when it comes to identifying failures early on in the development process, he pointed out.

The panelists suggested a few priority areas for NIH's engagement in this area including

  • Get the funding and support for NCATS to successfully get up and running
  • Develop human capital - increasing the expertise, capabilities, and attractiveness of the research discipline
  • Keep focus on patients when bridging the gap from academia to clinical trials
  • Take an analytical approach to the data available for new research

Value of Establishing the NIH Center on Advancing Translational Sciences to Patients

Statement of Margaret Anderson, Executive Director, FasterCures on Value of Establishing the National Center on Advancing Translational Sciences at the NIH to Patients

December 20, 2011 (Washington) – The spending bill President Barack Obama is expected to sign into law later this week will establish the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health. The inclusion of funding for this initiative bodes well for our nation's medical research and development enterprise and for millions of patients who need new treatments and cures now. FasterCures has been a vocal proponent for the creation of this effort to speed the translation of basic discoveries to real world applications that will improve public health.

The bill includes an increase for the NIH ($299 million over last year) and approximately $576.5 million for NCATS. Of this amount, 2.3 percent is new funding while the remainder brings together resources from other NIH Centers for programs such as the Therapeutics for Rare and Neglected Diseases (TRND) and the Clinical and Translational Science Awards (CTSA). The bill allows NIH to spend up to $10 million to support the Cures Acceleration Network (CAN) which creates new funding mechanisms at the NIH through which contracts will be awarded to expedite the development of “high need cures.” In all, the bill provides NIH with $30.7 billion in funding.

The goal of NCATS is to save time in the way we pursue and conduct medical research and development across disease areas. To patients with limited or no meaningful treatment options, saving time means saving lives. The creation of NCATS demonstrates our strong national commitment to medical research by ensuring advances achieved in basic science are translated into new therapies, better prevention strategies, and cures more efficiently.

As envisioned, NCATS would create tools to speed the development of new therapies, and make these tools widely available, thereby fostering efficiencies. This vision provides an opportunity for the patient and disease communities to work together on addressing a problem that cuts across diseases.

NCATS would also serve to strengthen the translational efforts underway at many NIH Institutes. Many NIH Institutes support both basic and translational research portfolios in an effort to move discoveries through the pipeline more quickly. Mid-sized and smaller Institutes that may not possess significant expertise in drug development would benefit greatly from having access to a central hub at NIH where such expertise exists.

At FasterCures, we fully support NIH’s efforts to create NCATS. Thanks to our vitally important national investment in scientific discovery, we know more about disease and biology than ever before. We must now maximize the return on this investment and have systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients’ health and quality of life.

We believe this new Center will strengthen our nation’s medical innovation infrastructure, helping spur economic growth and create jobs in the life sciences industry, and most importantly, it will accelerate the process of turning scientific discoveries into much needed medical solutions.