The Building Block of Drug Discovery - Pharmaceutical Executive


The Building Block of Drug Discovery
Director Francis Collins sees NIH as a catalyst for translating biomedical innovations into effective therapies, but skeptics fear it's harder than it looks. It's all in the DNA of its partnerships

Pharmaceutical Executive

FDA Partnership

The new Center also will support NIH collaboration with FDA. Collins acknowledges the importance of ensuring that federally funded research produces information needed to gain FDA approval of a test therapy and has formed a "real partnership" with FDA commissioner Margaret Hamburg to achieve this objective. A year ago this month, the two agencies announced a formal collaboration, with an NIH/FDA Joint Leadership Council providing a forum for communication between FDA and NIH research sponsors and a process for agency staffers to discuss clinical protocol questions.

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." Ellen Sigal, chair of Friends of Cancer Research, agrees that a mechanism for the two agencies to query each other on endpoints and adaptive trial designs should be very useful. "Anything that anyone can do to help FDA make decisions based on science is incredibly important," adds Richard Klausner.

Conflicts and Collaboration

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. Pharma and academic researchers fear that the strict standards will discourage joint projects, just as broad "Sunshine" policies governing pharma payments to doctors threaten to undermine industry relations with health professionals. Yet Collins maintains that collaborative relationships can't even appear to provide personal financial benefit to academic partners funded by NIH, or that could raise doubts about the validity of resulting research.

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."

Looming Budget Squeeze

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." He notes that translational science will be just a tiny part of NIH's $32 billion budget, at most 1 percent of its funds. And translation, he adds, "gets a lot of traction with Congress as well as the public."

Collins values industry input and expertise in implementing his vision for a more effective and collaborative approach, recently luring Rajesh Ranganathan from the Novartis Institutes for Biomedical Research to be a senior advisor. And he's meeting with pharmaceutical executives to discuss practical issues related to drug repurposing efforts, and to see "who wants to play," explains Christopher Austin, who came to NIH in 2003 from Merck to help Collins tackle many of these same issues arising from the human genome project; Austin heads TRND and the chemical genomics center, both on the list of activities for the new translational sciences center.

Collins cheered the advisory panel's recommendation to establish his new entity as a "momentous occasion" because it was based on "scientific arguments" and not politics. Drug development is at "a critical juncture," he told the committee. Filling the biomedical pipeline requires a whole new approach, he said, and "not an incremental tweaking of the system." 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."


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