WASHINGTON, DC—Finding innovative research vectors for major diseases, furthering partnerships with industry, and ironing out conflicts of interest among scientists are some of the many pressing goals facing NIH. Testifying before Congress last month, NIH Director Francis Collins, MD, PhD, discussed those issues, as well as how the agency allocates its research dollars.

Areas of Opportunity

When Collins first took the helm of NIH last year, he targeted areas of the biomedical research landscape that he believes are ripe for major advances and could yield the greatest benefits in the coming years. One of those areas is an investment in high-throughput technology, such as genomics. “We have seen invented, in just the last four or five years, a number of dramatically powerful high-throughput technologies that give us the chance to ask comprehensive questions about how life works. What are all the proteins in the cell? What are all the components of the immune system? What are all the steps in development?” Collins said to the House Energy and Commerce Subcommittee on Health last month. “We couldn’t really ask those questions before and now with technologies like genomics and imaging, computational biology, nanotechnology, we can do those things, and the chance to apply them is really unprecedented in its scope.”

The Common Fund, which was put into place by the 2006 NIH Reauthorization Act, has helped target work in this area. The fund provides support for research that crosses the boundaries of the different institutes within NIH. “[NIH will be] investing substantially in these areas because this is an example of the kind of research that touches on all diseases, ” Collins noted.

Another goal for the agency is to boost the research community as a whole. “We depend upon these wonderfully bright minds to make discoveries and we need to encourage the best and brightest of the next generation to join us. We need to inspire innovation and improve the diversity of our workforce,” Collins said.

The flat-lined budget that NIH struggled with for much of the second half of the last decade had a palpable impact on the agency’s ability to draw in talent. Collins pointed out that when the Recovery Act brought an additional $10.4 billion dollars to the agency’s coffers, young researchers began looking to NIH again. When NIH rolled out its Challenge Grant program, they expected 5,000 applications at most and instead received 20,000.

Clearing the Way from Bench to Bedside

One of the complex challenges facing the agency is finding better ways to partner with industry. “[NIH has] the chance now to play an ever-larger role in the front end of drug development pipelines and we aim to push that agenda as quickly as we can in order to take basic science discoveries to the bedside at an unprecedented pace.”

Legislators noted complaints about regulatory bottlenecks at FDA and about which drug targets should be prioritized. One way that NIH and FDA are working together on this issue is with the NIH/FDA Leadership Council which will meet on a regular basis.

Another effort to streamline the pipeline is a plan that NIH is developing with FDA to foster better regulatory science. “What if you have a clinical trial where everybody in the trial is on slightly different combination therapeutics, where combination therapy is the name of the game? We need science to understand that,” Collins declared.

Filling in the Conflict of Interest Loopholes

The relationship between NIH and industry suffered during the early part of the last decade as a series of conflicts of interest between researchers and industry became public. NIH has since strengthened its conflict of interest policies to the point where Collins is sure of its reliability. “I am determined that NIH should lead in this area, and that is why we are in the midst of proposing a new set of regulations that will require more complete disclosure from NIH-funded investigators about their dealings with industry.”

He cited a “particularly egregious case” of an investigator failing to cite conflicts of interest that came to light last month. In 2008, Charles Nemeroff, a psychiatrist doing research at Emory University, was discovered to have received large sums of money from pharmaceutical companies for work in the same general area as his NIH-funded research. NIH stripped him of the grant he was receiving at that time.

Nemeroff left Emory University and went to the University of Miami, where under current rules he was allowed to apply for new grants, since NIH’s arrangements are with institutions, not individuals.Collins noted that Nemeroff had not received any further grants, and said that NIH was going to fix this problem. “The notice of proposed rulemaking about conflicts of interest gives us an opportunity to address cases where there are violations of NIH and university disclosure rules. We can clarify our options for enforcement actions against both individuals and institutions.”

Choosing Research Priorities

Deciding how to funnel funding within the agency has always been a juggling act, and will likely remain so. A simple process of giving the most money to the diseases that create the most social burden is not sufficient, Collins declared. “Burden is one factor. But if we only study common diseases that affect hundreds of millions of people, then what about rare diseases? If a rare disease strikes your family, it probably doesn’t matter to you that it’s rare.”

Collins cited gastric cancer as an example of one of those relatively rare diseases. “It’s growing in its frequency and we don’t know why, but it is still a rare cancer. Should we ignore it and just study the common cancers?”

Research into these rare diseases sometimes has a far greater impact than initially expected and often sheds light on common diseases. “We would not right now have the statin drugs had it not been for the study of a rare disease called familial hypercholesterolemia,” Collins noted.

As for how NIH decides where to direct funding, much of that work is done through a two-level peer review system. “The first level is to look at scientific excellence of a proposal but the second level looks at program needs, and there is an opportunity for experts to come to our advisory councils from universities and companies all over the country and sometimes the world to give us advice,” Collins said.

Asked if research using embryonic stem cell lines would continue to grow, Collins stated that it almost certainly would. While only 73 stem cell lines are now approved for federal funding, there are 100 more in the approval pipeline.

When questioned about whether the scientific community would feel more secure if the decision to open federal funding to new stem cell lines was codified in legislation, instead of laid out in an executive order, Collins told legislators that he was not in a position to say. He added, “I think the executive order has provided the scientific community with a great deal of excitement in terms of the ability to do experiments that were previously not allowed.”

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