Q&A with Francis S. Collins - Inside the National Institutes of Health - BioPharm International


Q&A with Francis S. Collins
Inside the National Institutes of Health

BioPharm International Supplements
Volume 24, Issue 9, pp. s4-s10

BioPharm: And what is the second project underway?

Collins: The other program, which is going to be sponsored jointly by NIH and the Wellcome Trust, is called Human Health and Heredity in Africa, otherwise known as H3Africa. This is a bold effort to create a network of research activities that will look at environmental and genetic risk factors for both infectious and noninfectious diseases in the continent. It will adopt some of the same strategies that have been carried out more recently here in the US and other developed nations, but try to find out—in the cradle of humanity—how can we make the most of some of the new technologies that allow us to do genomics and environmental science and understand the causes of illness.

This will mean building capacity to do that kind of research, including information networks to share data and to carry out computational analyses. It will include setting up phenotyping capabilities to look at clinical consequences of various exposures and genetic risk factors. It will also mean establishing biorepositories to be able to store the samples that will be collected on what we expect will be thousands of individuals in Africa who will be the research participants in this bold new effort that will undoubtedly stretch over many years.

BioPharm: Have you found that countries and other groups are more willing to collaborate in recent years, and, if so, what do you think has led to that change?

Collins: I certainly do think that collaborative spirit is expanding in a wonderful way and it's driven, in part, by scientific opportunities. [For example, with our new consortium for rare-disease research], we are discovering the molecular causes at a prodigious rate. There are now some 4000 rare diseases for which we know, at a pretty detailed level, what the actual molecular problem is that leads to that illness, many of these being genetic diseases caused by mutations in the genome.

And, so, if you want to see that knowledge applied in terms of developing new therapeutics, that is something that is not trivial. You certainly don't want to waste the opportunity to bring groups together that might be able to do this faster, and you don't want to duplicate efforts and waste resources.


BioPharm: In previous statements you've mentioned that innovation should not be limited to the work that's done for developing nations. How can we best capture the technology and knowledge that's used for developing-nation treatments and apply them to drug development and discovery in every region of the world?

Collins: I think of innovation very much as a two-way street. We shouldn't think that new inventions or new creative ideas come forth only in the US or in Europe. We can also learn, in what's being called 'reverse innovation', about how to adopt new ideas that are being developed in low-income countries. A particularly good example is the use of cellphone technologies for medical purposes....

For instance, we have a technology that's being developed and tested in Africa which is a simple method of assessing whether, in fact, individuals who are being treated for tuberculosis or HIV/AIDS are compliant with the therapy. So, you have a pill box which essentially is hooked up to the cellphone network, and every time the pill box gets opened, it sends a signal to the clinic where that patient is being cared for so that you can tell, was it, in fact, opened, and was it at the appropriate time of day?

BioPharm: That technology provides a great way for doctors to follow through when they're not close by.

Collins: Exactly, and that, already, has spurred ideas about how we might adopt those same approaches here in the US for people who have, for instance, diabetes or hypertension. Medical compliance, we know, is critical for success in treating many conditions, and yet we haven't really had a good way of monitoring it.

BioPharm: Some other conversations going on in the pharmaceutical industry right now have to do with translational research, and NIH is a big supporter of that. How is NIH selecting projects to pursue in this area and how far can it take the projects, for example, through proof of concept in animals or Phase I clinical trials?

Collins: This ties into the conversation we were having about rare diseases [as part of the new consortium's goals to find treatments for 200 diseases by the year 2020] and the goal of having new therapeutics in a reasonable time. As you know, the current situation is a little scary when it comes to making such promises because, after all, the average time it takes from identification of a potential drug target to ultimate approval of that therapy is about 14 years, and the failure rate is about 98%. We think the time has come to look at that process the way that an engineer would and see if there are ways that we could optimize some of the steps that currently are slow, expensive, and likely to fail. That is part of NIH's effort now in putting together a new entity called the National Center for Advancing Translational Sciences.

We certainly will do this in a way that is complementary, and not competitive, with what the private sector would like to do, but we do expect that this kind of initiative may make projects that previously appeared too risky start to look attractive. So some of this is the idea of derisking projects which the private sector might otherwise not feel were economically attractive.

And certainly, when NIH sees opportunities for therapeutic development, working through the 27 institutes and centers that have a lot of knowledge about these areas, we will try to move projects forward to the point where they've become commercially viable and then license them out as quickly as possible in order to get them over the finish line.

Again, the goal here is to try to take advantage of remarkable scientific opportunities that might otherwise lie untouched but also to recognize the economic realities which means that companies, in general, are not going to go after projects that they don't think ultimately will become profitable.

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