Wednesday, February 12, 2014

The NIH Accelerating Medicines Partnership—Francis Collins Sings a Few Bars

I had wanted to respond to a news release some days ago about the Accelerating Medicines Partnership announced by the NIH and 10 drug companies, but before I got around to it, I caught a tiny bit of this interview on The Diane Rehm Show on my car radio:
--in which not only did Dr. Francis Collins, head of the NIH, talk about the AMP, but also sang about it self-accompanied on his guitar (which, he explained, suitable for the former head of the Human Genome Project, has a double helix inlaid in mother-of-pearl on its fretboard).

I’ll let you enjoy the music if you wish on the audio and turn straight to the written transcript.

Here’s how Dr. Collins explains the AMP:

So more than 1,000 new drug targets have emerged in the last five years from that kind of study, but it's very hard to sift through them and pick out which ones are going to be the real home runs that we're all looking for…. this is full 50/50 skin-in-the-game kind of collaboration, $230 million committed to this over five years, half of that coming from NIH, half of that coming from the 10 companies that are participating. The scientists from both sectors will sit around the same table and work together to make this happen in a fully open access atmosphere…. We already have detailed research plans for these disease areas that have been worked out over the past year with very clear milestones that have to be met. This is not one of those where everybody goes off and plays in the lab. We are really serious here about making real progress.

Well, in this phase of trying to identify the next generation of drug targets, everybody agrees that this is precompetitive, and that is that all the information has to be openly shared both within this consortium and for anybody else who's watching. The competitiveness, Diane, kicks in once you've identified, oh wow, that particular molecule is a really promising target for the next generation of Alzheimer's therapy…. Then every company will run off and do what they do really well… And we want them to do that. That's good competition. That means that things move quickly and there's lots of ways that you can get to yes.

Let me see if I can make sense of this from the standpoint of the view of current pharmaceutical science that I proposed in HOOKED. I suggested then that the current drug pipelines were so dry because there are only a limited number of molecules that do useful things in the human body without killing us. Not that there would never again be a “golden age” of major advances in drug therapy; but the new golden age would not come from drug companies doing cranked-out research on their assembly line—it would require breakthroughs in our basic understanding of disease, discoveries that the NIH and academic centers are better able to make than industrial labs. So Take Home Message #1, I hear Dr. Collins saying that we’re poised to enter another age of advancement, that basic science research has now identified a bunch of exciting new possibilities—locks that we now need to discover the keys to.

Take Home Message #2 seems to be that the “translational science” model, which NIH has embraced in the last decade, tells them that we’re now ready for a more accelerated and focused phase of discovery, when NIH scientists and Pharma scientists can both participate in a program that will quickly move (as the translational mantra has it) from (lab) bench to bedside. At this point, as a complete non-expert, I would enter a note of caution. I generally support the translational science model (full disclosure: a small portion of my salary is currently funded under a translational science grant, to address the ethical issues). However, there are times in the life cycle of a discovery when you can safely go on the fast track and other times when you’d be better off “playing in the lab” just a little bit longer. I hope the NIH gurus are right that their particular list of diseases—Alzheimers, diabetes, and autoimmune disease—is truly ready for this hurry-up approach.

OK, so that’s the science part; what about the potential-conflict-of-interest part, the rocks on which so many previous scientific ships have broken up and sank? Says Dr. Collins:

With regard to the drug companies, I know that people are concerned about what their motives are. Again, they make pills. NIH doesn't. … We believe, by working together, we can speed up the process of getting the right answers. … You know, five years ago, I don't think this [collaboration] would have been possible. I think it's a combination of scientific opportunity that is so exciting but so overwhelming that no company can tackle it on their own, plus their own anxieties about the failures that are all too common, even now, in drug trials where you've spent hundreds of millions of dollars and you get to the end of that Phase III trial and it didn't work. They've had enough of that, and they're anxious to try something different.

So two things here—first, with a demand for open access, NIH figures they have a safeguard against one of the main dangers of industry collaborations of the past; and second, they believe that the drug industry today is in a different place and more willing to play nicely in the sandbox. Interestingly, Dr. Collins made a comment similar to one I have made previously in this blog—

[Rehm] I'm interested that Johnson & Johnson hired Yale University to oversee the sharing of clinical trial data. What can you tell us about that? [Collins] I think that's fascinating. So the person at Yale University, Mr. Harlan Krumholz, who's a cardiologist, who is a wonderful leader in this whole idea about getting information out there where everybody can see it -- and he has a lot of credibility so people will believe that if Harlan is involved that what Johnson & Johnson proposes to do is the real deal and not just some window dressing. I think it's great.

Final conclusion I’d offer: NIH seems to be going into the AMP with decent reasons and with eyes open, but so far, Pharma has an excellent track record of managing to have such folks for lunch nonetheless. I hope Dr. Collins is right and this time it will be different, and if so, we may have yet another good model of positive collaboration to work with. Stay tuned.

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