NCATS: “Disruptive Innovation” at NIH

More than 650 participants tuned in to a Webinar on March 16 hosted by FasterCures with National Institutes of Health Director Francis Collins, who gave the latest updates on the progress on the proposed National Center for Advancing Translational Sciences (NCATS), answered questions, and sought to dispel myths about this effort. Participants were from 38 states and represented all medical research sectors – more than 229 patient advocates from 100 different organizations, nearly 200 academics from 78 research institutions, 62 from biotechnology and pharmaceutical companies, 82 from various government agencies, and dozens of other stakeholders from the venture capital community to policy influencers.

Calling NCATS “a disruptive innovation, in a good way,” Collins said the motivation for the new Center is “the need to view the drug development pipeline as a scientific problem, ripe for experimentation and process engineering.” He noted that now is the time to act as opportunities for new drug targets to be tackled successfully have been growing well beyond capacity of the private sector to take on by itself and that the appetite for public-private partnerships is high. Engaging in translational research is not new for NIH, he said. A 2010 survey showed 550 translational research projects going on at NIH, 65% preclinical and 35% clinical, and a recent New England Journal of Medicine article reported that 153 new therapies have been discovered through research carried out in public-sector research institutes, most of which have been supported by NIH.

But he also acknowledged that the productivity of the medical research system overall is declining, and stated his belief that NIH – by facilitating and supporting partnerships, providing resources, and enhancing training – has an increasingly important role to play in moving the results of promising research it funds down the pathway toward new treatments. “The opportunity for new targets is growing well beyond industry’s ability to address them all. … In addition to neglected diseases, for many common diseases there are ‘neglected targets,’” which NCATS can help de-risk and make attractive for investment. He also believes that NCATS could be “quite catalytic” in helping repurpose abandoned compounds, and mentioned that NIH will be holding a meeting with biotech and pharmaceutical executives in April to discuss such ideas.

Collins was emphatic that NCATS is intended to complement, not compete with, the private sector. “We are not turning NIH into a drug development company,” he said, contrary to some headlines that the initiative has generated. He also took care to say that there is no intent to take focus and resources from NIH’s basic science mission and programs. As a matter of fact, he believes NIH’s investment in translation can empower basic research.

FasterCures executive director Margaret Anderson moderated this virtual meeting, noting that NIH’s effort to make translational research a priority is significant and meaningful for the thousands of patients with a deadly and debilitating disease for which there are no viable treatments or cures. Especially when you consider industry’s estimate that for every 5,000 compounds tested, only five make it to clinical trials, and only one is ever approved by the Food and Drug Administration (FDA). Patients are anxious for – and deserve – better returns on our nation’s research investment.

Collins appealed for all research stakeholders to be reasonable about expectations for this new effort, saying that NCATS should not be judged by the number of drugs ultimately approved. He ended by saying, “This is science, so there will be surprises along the way. But there is an opportunity to do something historic here.”

NIH is actively seeking feedback on the proposed structure and functions of NCATS at a new Website. An archive of the FasterCures Webinar is available for viewing.