This month the National Institutes of Health (NIH) began putting into place the biggest reorganization of the sprawling $30.6 billion enterprise in a decade. It is launching a new $575 million National Center for Advancing Translational Sciences (NCATS) that aims to tackle bottlenecks in drug development and speed the translation of basic discoveries into therapies.
The NCATS plan sparked much debate when it was proposed last year. Some industry scientists said they feared that NIH was moving away from basic research into drug development. Many researchers were also concerned about losing programs within another NIH center that was to be dismantled, the National Center for Research Resources (NCRR). In the end, Congress allowed the changes but attached some conditions. For example, last month's spending bill that established NCATs requests several reports and outside studies of the center's activities and stipulates that it cannot support expensive, late-stage clinical trials of the kind typically done by industry.
In an interview with ScienceInsider last week, National Institute of Mental Health (NIMH) Director Thomas Insel, the acting director of NCATS, dismissed reports that industry is skeptical. Industry leaders told NIH that they would "love to have help" with improving their R&D efforts, Insel said. NCATS will continue initiatives begun last year to find new uses for old drugs, develop a drug toxicity chip, and work with industry to validate "targets," the protein or cell structure that drugs aim to modulate. NIH Director Francis Collins outlined other possible directions, from virtual drug design to research on marketed drugs, in an article in Science Translational Medicine last summer.
As NCATS takes shape, perhaps those most uncertain about the future are directors of programs funded by the Clinical and Translational Science Awards (CTSAs), part of NCRR. These large grants support translational research at some 60 academic medical centers. The $487 million program makes up most of NCATS's budget (the only new money Congress approved is $10 million for the center's Cures Acceleration Network). Some CTSA directors fear that components that don't involve therapeutics development, such as behavioral research and community engagement, will be trimmed.
Insel said that NIH wants to preserve the range of CTSA activities but adds that the program should expect to "evolve."
According to Insel, the CTSAs in the new center will be overseen by a division of clinical innovation led by acting director Josephine Briggs, who will also remain in her current post as director of the National Center for Complementary and Alternative Medicine. A division of preclinical innovation will house a $44 million set of mostly intramural programs, including small-molecule screening and drug development for rare diseases. Until now, those programs were managed by the National Human Genome Research Institute (NHGRI) under the direction of Christopher Austin; he will serve as director of the NCATS preclinical division. (Some NCATS programs also have funding from NIH's Common Fund, a pot of money in the NIH director's office.)
Insel's edited remarks follow.
Q: Why does NIH need NCATS when many institutes at NIH do translational research already?
T.I.: I'm going to answer that question with my NIMH hat on, because I do a fair amount of translational science within NIMH. But what NCATS is going to do is not what we're doing at NIMH.
We need a place to actually look at the whole process of translation in a way that can consider how it might be reengineered, consider how we can make a difference by partnering with both advocacy groups and with industry. Consider where the opportunities are for great innovation that are not just related to schizophrenia or autism or bipolar illness but that are really generic. They go across all of medicine.
And rather than doing that 27 times, it might be good to have one place where we say, "Let's solve this problem. Let's really look at this in a very careful way and actually do something that we've never been able to as an institute which is to do experiments on translation itself." This is a really exciting moment to begin to look at this for the first time.
Q: Why do you think NCATS was so controversial last year when it was proposed?
T.I.: I'm not even going to go there. I wasn't part of that fight. And I'm not even going to begin to contemplate what the issues were.
Q: Some industry scientists have expressed skepticism about NCATS. They're worried that it will lure scientists away from basic research. They're also worried that NIH is going to do drug development.
T.I.: There was a lot of concern about that at some points in the past. We brought in a lot of people from the outside. We had this working group of the NIH Director's advisory committee; Maria Freire [president of the Lasker Foundation] chaired that. I don't think those were the issues that we heard most often. There was actually quite the opposite. What we heard was that nobody was particularly happy with the way that R&D is happening whether it's in the public sector or the private sector.
We never heard anybody come forward and say, "You know what, we have figured out how to do this, it's working great." In fact what we heard was, this isn't working very well anywhere, we'd love to have help.
Q: The translational awards to academic medical centers make up most of the NCATS budget, but a lot of what they do doesn't seem to fit with the mission of NCATS. Should they be ready to change?
T.I.: I think if there's a change, it's going to be the possibility that going forward not every CTSA will be exactly the same thing whether it's in community engagement or whether it's in first-in-man trials. We're going to look at being able to, to the extent possible, cultivate what they do best.
Q: How are you going to pay for new things?
T.I.: Well, there's actually close to $500 million here [for the existing CTSA program], so there's actually quite a bit of funding to do some exciting science. I don't think there's any question about not being able to do new things. But I think part of what we'll have to is to help these centers develop flexibility that they don't feel that they have right now. I was there at the beginning of the Roadmap [former NIH Director Elias Zerhouni's set of cross-NIH initiatives] when we were setting all this up and we always understood that the CTSAs were a fantastic opportunity to reengineer clinical research in academic health centers and provide a new home for this.
But I don't think anybody felt that this was a 3-year or a 5-year project or that it would be done and finished. It was always envisioned as an evolving program.
Q: Will NCATS fund clinical trials?
T.I.: The CTSAs do not have the funds to run large-scale clinical trials. That's not the way they're supported. They're supporting infrastructure.
Q: But aren't there components of NCATS that can fund trials?
T.I.: That could happen but that's on a very small scale. You're talking about relatively small dollar amounts for both TRND [Therapeutics for Rare and Neglected Diseases] and right now for CAN as well.
Q: Will NCATS have an intramural program?
T.I.: There are two answers to that question. The first answer is yes. The division of preclinical innovation is largely intramural. But having said that, one of the things that I'm most excited about in playing this interim role is it's a chance to try some experiments. One of the questions we're now grappling with is, "Can we get away from this rigid separation between intramural and extramural?"
It becomes really interesting when you think about something like [the rare diseases program] which is technically an intramural program, but 90% of what they do is done with extramural collaborations. They don't have a big intramural faculty that's on a tenure track, they don't have a large number of FTEs [staff positions]; it's a very different way for NIH to operate.
So we're looking at NCATS as a place to raise questions about the relationship of intramural to extramural and how we can get them to cross talk and provide a much more dynamic kind of relationship than what we've had up until now.
Q: Is the search for a permanent director moving along?
T.I.: It is. I'm co-chairing that search with [NHGRI Director] Eric Green. Eric and I have been assisted by an outstanding search committee which includes both NIH and non-NIH people. We've had a great response to the various ads and solicitations we've put out there. We're looking forward to interviewing candidates in the very near future.
Q: On one blog somebody had speculated that you could be a candidate.
T.I.: I can answer that very clearly. I have a job which is keeping me very busy at NIMH and I'm very committed to that and I'm not a candidate for being director of NCATS.
*11 January: An earlier version of this item stated that Christopher Austin will serve as the acting director of the NCATS division of preclinical innovation; he will be the director.