PharmExec Blog

NIH Director Speaks Out on the Institutes' New Remit

As the National Institutes for Health hits the headlines over its remit to help develop new medicines, Director Francis Collins talks to Pharm Exec about the new initiatives he sees as critical for innovation, industry and public health.

Francis Collins devoted more than 15 years in the lab to deciphering the human genome. Ten years later, as Director of the National Institutes of Health (NIH), he finds himself in another unique position: to deliver the promise of the genome through the translation of research into technologies, products, and services that are clinically relevant and commercially marketable.

A critical task for Collins this year is to convince Congress to provide healthy funding for NIH amidst plans to cut federal outlays. Flat NIH budgets during the last decade created a huge pent-up demand for funds, which quickly absorbed the extra $10 billion provided by the 2009 stimulus package. Even though the White House sought a 3 percent increase for NIH for 2011, Congress never approved it, and most federal agencies are currently operating at last year’s funding levels.

Collins pressed for Committee approval of the new translational sciences center in December 2010 to be able to include it in the administration’s 2012 budget plan and thus obtain initial funding this fall. But the lack of new money for NIH means that most of the $700 million that would be needed to support translational sciences will have to come from existing funds. That makes the research community nervous, as well as pharma companies that want NIH to continue its focus on basic research.

Speaking to Pharm Exec, Collins says the role for NIH is to “de-risk” the process of drug development and to leverage its own human and financial capital to foster better partnerships with Big Pharma and biotech. He sees a rapidly changing biomedical research landscape as the prime driver behind stronger NIH partnerships with the pharmaceutical industry. He’s optimistic that new genetic discoveries can chart pathways for discovering new medical treatments, and that the emergence of more well-validated genes will be useful in “identifying drug targets in unprecedented numbers.”

At the same time, biotech and pharma companies face serious financial challenges in funding research projects. “These are not easy times for the development of new therapeutics,” Collins allows, noting that venture capital is limited and many biotech companies are struggling to stay afloat.

“So it’s kind of the best of times, and worst of times,” he observes. The scientific enterprise is yielding up a lot of new ideas about therapeutics. Yet, “the traditional private sector efforts to capitalize on that are taking a hammering.” NIH, thus, is “an attractive alternative” for supporting new partnerships between academic researchers and industry.

Collins has an eye out for tax provisions, patent incentives, and regulatory assistance that can encourage such collaboration. And he emphasizes that NIH won’t move into full drug development, but will support projects just long enough for them to become attractive for a biotech or pharma company to pick up. For a completely new molecular entity to treat a common disease, “I suspect the interest in the private sector would be very high, very early,” he says. Conversely, a treatment for a rare disease that affects only a few hundred patients is not likely to generate much interest until further along. NIH is prepared to contribute to promising projects as far down the line as it has to—“but no further,” he maintains.

Bench to Biotech
The NIH translational sciences center envisioned by Collins would house a spectrum of programs now scattered among different NIH entities, according to the report to NIH’s Scientific Management Review Board, which was established two years ago to examine and recommend changes in the agency’s organization and structure. Current translational research programs cover the development waterfront, many funded by the director’s Common Fund, a $500 million kitty that Collins administers to support cutting-edge, trans-NIH projects. Instead of shifting these activities to an existing NIH institute, the advisory group thought it best to establish a new organization to emphasize the importance of these changes.

Several NIH activities may remain outside the new Center, such as the Pharmacogenomics Research Network (PGRN), a group studying how genes affect patient response to medicines for cancer, heart disease, asthma, nicotine addiction, and other conditions. The panel also decided to leave NIH’s Clinical Center as a distinct entity, but with strong links to the Center.

FDA partnership
The new Center also will support NIH collaboration with FDA. Under the collaboration, NIH is funding several research projects designed to assist product development and enhance regulatory review. The first round of grants, which total $10 million over three years, support research on predicting harmful effects of nanoparticles in drug delivery, new models for safety and efficacy screening of drug candidates, innovative statistical methods and study designs for clinical trials, and a novel strategy for predicting eye irritation in clinical studies.

Collins acknowledges that the funding is very modest, but more than what FDA has for this kind of research. FDA chief scientist Jesse Goodman regards FDA’s collaboration with NIH as a real “culture change, and recognition that FDA’s engagement in the development of medical products is very important.”

Conflicts & Costs
One assignment for the new Center is to reduce barriers to public/private collaboration that can be created by conflict-of-interest (COI) concerns and intellectual property (IP) issues. Collins is working to put “NIH’s COI house in order” with more stringent and more transparent financial reporting requirements.

Equally challenging is the task of balancing open access to research data against IP protection. NIH wants to establish models for collaboration agreements and licensing arrangements, although Collins sees most discoveries from public/private collaborations residing in precompetitive space; exclusive licensing agreements should be “limited to circumstances where it’s really needed as an incentive for product development,” he says. Collins made waves in September 2010 by announcing that NIH would license a key protease inhibitor to the UNITAID HIV medicines patent pool, a move designed to spur pharma companies to take similar action.

R&D Support = Lower Prices?
Drug pricing is another issue that emerges in discussions about NIH/pharma collaborations. Collins doesn’t see NIH trying to influence drug prices per se, blanching at remembrance of the “reasonable pricing” debate that made industry very skittish about partnering with NIH back in the ’90s. Yet, he also is concerned about the trend toward more high-cost therapies. “The market can’t bear $93,000 for compounds across the board that give you four months of additional quality of life,” the NIH director told Science magazine in May. “That’s just not going to be sustainable.”

Instead of pressing for lower prices, Collins looks to reduce upstream R&D costs—and resulting product prices—by providing data that supports smaller clinical trials and by rescuing abandoned compounds. “If we could simply improve success rates by a factor of two—you know, only a 90 percent failure rate—that would be a phenomenal achievement,” he enthuses. Throwing out unpromising projects is key to improving the process, he adds: “People should be rewarded for having the courage to do that.”

Taking Risks
The dark cloud over these and other NIH initiatives is an ever tighter funding climate for federal agencies. “That’s what keeps me awake at night,” Collins says. “We have such exciting scientific opportunities now, but we’re really in a squeeze as far as our ability to support them.”
While trying to figure out what NIH can afford to do, Collins insists the wrong move is to “just hunker down and not try anything new.”

Pushing for such a significant organizational change at NIH in a very short time is certainly risky. But NIH “is about innovation,” he emphasizes. “It’s about taking risks.”

The full version of this interview will be published in February’s Pharmaceutical Executive.

This entry was posted in FDA, Gene therapy, Guest Blog, healthcare, IP, leadership, R&D, Regulatory and tagged , , , , , , . Bookmark the permalink. Trackbacks are closed, but you can post a comment.

One Comment

  1. Posted January 27, 2011 at 8:21 am | Permalink

    It would be helpful to know how the NIH plans to address and support research involving non-US sites and investigators.

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