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NIH's New Translational Chief on How to Solve Pharma's Woes

Translation chief. Christopher Austin is the first director of NCATS.

Maggie Bartlett, NHGRI

In September, Christopher Austin stepped into what some might view as a hot seat: the directorship of the National Institutes of Health's (NIH's) new center dedicated to translating science into applications. Approved by Congress last December, the $575 million National Center for Advancing Translational Sciences (NCATS) has raised concerns that NIH is venturing too far into drug development and will squander scarce federal dollars that could instead support basic research. The new center also stirred a firestorm of debate among NIH-supported researchers because it entailed breaking up an existing center at NIH.

Austin, 52, is a developmental neurogeneticist who spent 7 years at Merck before joining NIH to oversee translational programs at NIH's genome institute. In an interview last week with ScienceInsider, he seemed undaunted by public criticism of NCATS. Austin's remarks have been edited for clarity.

Q: Could you describe NCAT's mission in your own words?

C.A.: It comes down to the science by which fundamental discoveries are turned into tangible improvements in prevention and treatment and disease. That is a process which has never been subjected to the scientific method. That applies to individual steps in the translational process, which are remarkably empirical even today. Whether you talk about medicinal chemistry optimization or toxicity evaluation or how to recruit patients for clinical trials, these are remarkably trial-and-error processes. And as a result, they're very inefficient and they fail most of the time. And so that is the subject of NCAT's study.

On an operational level, there are three Ds: NCATS has to develop, demonstrate, and then disseminate improvements in the steps of the process. Those improvements could be technical ones, scientific ones, or they could be paradigmatic ones, collaboration ones, intellectual property issues, regulatory—they're not all strictly scientific.

Q: Former Merck CEO Roy Vagelos has said that if anyone thinks NCATS can achieve something that industry isn't doing already, "you believe in fairies."

C.A.: What Roy did at Merck in the '80s and '90s is truly legendary. He's very highly respected by everybody in the field.

At the same time, it is clear to me and to many people in the pharma industry that there are many things that pharma or biotech would like to do but can't do either because the return on investment is not great enough; there's a short term commercial imperative, which makes study of general principles difficult or impossible; or it doesn't fit the strategic goals of the company.

I don't think it's a matter that companies haven't thought of these things. It's just that these are things which don't generate revenue in the near term.

Q: What are the first things on your plate?

C.A.: The biggest one is the CTSA [Clinical and Translational Science Awards] program. It's about 80%, 85% of the NCATS budget. I'm devoting a huge amount of time to learning about that program. And I've really been extremely, extraordinarily impressed with the quality of the people and the work and the initiative of the people at the institutions [with CTSAs].

Q: The CTSAs are worried about what will happen to them.

C.A.: I think to some degree these misperceptions about what NCATS was going to do go back maybe a year and a half now to that famous article in The [New York] Times [suggesting that NCATS would develop drugs]. We're still living that down. It just was not an accurate portrayal of what NCATS was ever going to do. It led to a concern on the part of the CTSA PIs [principal investigators] that they would all be turned into drug developers or something.

It is true that that they're in a different institution than they were in before. [That institute's] mission was different from the NCATS mission. So [the CTSAs] will evolve with time. But that's a good thing; that's a healthy thing.

Q: How will you do new things with a flat budget?

C.A.: There are obviously new things we'd love to do that we will not be able to do without [new] funds. It's particularly hard for a new center that has a very ambitious mission with a lot of people counting on us. But half a billion dollars is still a lot of money and I think there's a lot we can do with it.

Q: How will you measure the success of NCATS?

C.A.: If you look at the NCATS mission, it talks about improving the process and improving efficiency. The problem is this is a really mushy area and there are a lot of things you could potentially measure. If you look at what's happened in pharma, and my old company was like this, there were really well-meaning efforts to set metrics which would make sense, and people met the metrics and the overall programs failed for a variety of reasons.

So there will not be one metric, there will be a portfolio of metrics—some long-term, some short-term, some intermediate outcomes, some high-risk, some low-risk. And in the end, we will have a composite score which hopefully will be reasonably positive.

We also want to have metrics that allow failure as long as you fail for the right reason and you publish why. We're not going to have number of publications as a meaningful metric. Of course we'll have papers, but that's not NCATS's mission.