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