A new player in drug research?

The proposed National Center for the Advancement of Translational Sciences would help develop medicines, but is it feasible?

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WASHINGTON, D.C.—An ambitious effort to create the National Center for the Advancement of Translational Sciences (NCATS) is raising plenty of eyebrows throughout the pharmaceutical industry and across Capitol Hill.

The brainchild of Dr. Francis S. Collins, director of the National Institutes of Health (NIH), the plan would lead to more than $700 million in research projects already under way at various institutes and centers being brought together at the new center. But officials hope that the prospect of finding new drugs will lure Congress into increasing the center's financing well beyond $1 billion.

Formation of the Center was approved last year, and President Barack Obama supported increasing the NIH's budget by $1 billion to get the program underway.

In a budget Obama released earlier this year, the president would give the NIH a 2.4 percent increase next year of $745 million over the 2010 level, bringing the total to $31.8 billion.

NCATS would "re-engineer the pipeline for diagnostics and therapeutics discovery and development," a budget document explains. NCATS would be built in part from a $485 million clinical research support program at the National Center for Research Resources (NCRR).

A 2006 law limiting the overall number of NIH departments meant that the creation of NCATS would require that another institute or center be sacrificed. NCRR, despite some opposition from the research community, would be abolished.

However, NCRR remains a line item in the 2012 budget, and NCATS is not there. That's because the agency is still figuring out where to put the other pieces of NCRR, Collins told reporters in early February.

With a new wave of budget-cutting sentiment taking hold in Washington, hopes of new money may be overly optimistic. Republicans in the House have promised to cut the kind of discretionary domestic spending that supports the health institutes, and officials are already bracing for significant cuts this year.

The U.S. House Appropriations Committee ramped up its attack on government spending on Feb. 11, releasing the text of a bill that it says represents the biggest round of spending cuts in U.S. history. The panel proposed cuts to the NIH budget, a 5 percent cut relative to 2010 spending levels. The cuts amount to $1.629 billion from the 2010 NIH budget.

U.S. Rep. Hal Rogers, R-Kentucky, is chairman of the committee. He issued a statement saying the cuts were motivated by concerns over the nearly $1.5 trillion federal deficit.

"The cuts in this continuing resolution are the result of difficult work by our subcommittees who have weeded out excessive, unnecessary and wasteful spending, making tough choices to prioritize programs based on their effectiveness and benefit to the American people," he said.

Sen. Daniel Inouye, D-Hawaii, chairman of the Senate Appropriations committee, slammed the GOP proposals in a separate statement.

"The priorities identified in this proposal for some of the largest cuts—environmental protection, healthcare, energy, science and law enforcementare essential to the current and future well-being of our economy and communities across the country," he said.

But Collins has hinted that he is willing to cannibalize other parts of the health institutes to bring more resources to the new center.

In a statement on the NIH website, Collins points out that the proposal for NCATS is that it will be assembled primarily from existing programs within the NCRR, the NIH Common Fund and the National Human Genome Research Institute (NHGRI).

Moreover, Collins points out that NCATS is not intended to be a drug company.

"It is a facilitator of translational research across the NIH and complementary to translational research already being conducted and supported on a large scale in the individual NIH institutes and centers," he explains. "NCATS will seek ways to leverage science to bring new ideas and materials to the attention of industry by demonstrating their value."

There are no plans to "cannibalize" the budgets or programs of other NIH institutes and centers to form NCATS, Collins asserts.

"NIH remains committed to continued support for basic, translational, and clinical research," Collins explains. "The new center will bring several existing efforts together in new ways to enhance the ability of all NIH institutes and centers to perform research that leads to the development of drugs, diagnostics, devices, vaccines and strategies for prevention."

The other programs that will become part of the new center include grants funded out of the Director's Common Fund, the Molecular Libraries Screening Network, Therapeutics for Rare and Neglected Diseases (TRND) and Rapid Access to Interventional Development (RAID).

Collins says a final vision for NCATS still is in the planning stages, with information being gathered from internal and external stakeholders. Until that information is systematically and objectively evaluated, the plan for NCATS remains a work in progress.

"However, one thing is certain: NIH will continue to seek out new ways to advance our common cause—improving human health through science—even in difficult budgetary times. Every NIH institute and center is pursuing novel, imaginative plans of its own, and the creation of NCATS is just one of many exciting initiatives that we, working collectively, hope to achieve this year," Collins concludes.

Trade groups react to NCATC proposal

At least one industry trade organization views Dr. Francis S. Collins effort to create the National Center for the Advancement of Translational Sciences (NCATC) as a step in the right direction.

For Pharmaceutical Research and Manufacturers of America (PhRMA) Senior Vice President David E. Wheadon, collaboration—including industry, NIH and academia—is one element driving innovation in drug development, particularly early-stage development—and "'bold and ambitious' proposals, such as Dr. Collins,' will be key to how we collectively progress in discovering novel compounds for addressing patients' unmet medical needs."

"PhRMA agrees that we need more innovative new medicines to help patients," he says. "This recognition is important as it supports the role that new medicines play in addressing healthcare problems."

The biopharmaceutical research sector invested an estimated $65.3 billion to discover and develop new medicines in 2009 alone, a figure that dwarfs what it spends on marketing in total. Yet, the challenges and risks remain real. In fact, the odds of success are steep—just one approved medicine, on average, for every 10,000 promising molecules initially studied.

"We're encouraged by Dr. Collins' goal, described in this publication and others, of taking a sharper focus on early-stage research in the hope of improving the ultimate success rates in drug development," Wheadon says. "The fact remains that biopharmaceutical research companies today and in the future will play a pivotal role: Our companies create the vast majority of new medicines from start to finish and, for the remainder, in close collaboration with academia and NIH, fulfill the critical final phase that transforms promising molecules into actual medicines for patients."

Dr. Mark O. Lively is a biochemist at Wake Forest University School of Medicine. He is the immediate past president of the Federation of American Societies for Experimental Biology (FASEB) and has been a member of the FASEB board of directors since 2004. He served as FASEB president from July 2009 to June 2010, during which time he met many of the NIH institute directors as well as members of the NIH administration, including Collins. Since 2009, he has have been a member of the NIH National Advisory Research Resources Council (NARRC) and is a newly appointed member of the NIH Council of Councils.

Lively admits that as a biochemist, he isn't truly familiar with the details of successfully bringing a drug to market. That noted, he says the fundamental problem that he sees with pharma currently (which he bases on discussions with former execs with Merck and Pfizer) is that they "lack a steady stream of basic scientific discoveries that would give them the information that they need to identify and pursue high quality targets and leads that have a high probability of making it through the development pipeline. NIH is able to fund the basic discoveries that pharma cannot support because of the risk. In my opinion, the role of NIH is support the best basic research possible."

Lively says it remains unclear exactly what the NCATS center will do, as its mission statement has not been drafted or revealed publicly if it has been formulated. He points out that a January article in the New York Times left many with the impression that NCATS will be a drug discovery unit at NIH with a budget approaching $1 billion.

"Some even seem to think it will be a laboratory at NIH, perhaps even part of the intramural program," he says, "This is surely incorrect. First, it will most likely function as an NIH unit with a budget to fund extramural grants focused on the themes that Dr. Collins laid out in the 'Fact from Fiction' posting on the NIH website."

Collins admits that the final budget for the proposed center is unknown at the present time.

"For the most part, the budget and staff for each relocated program will remain with that program," he says on the website. "Thus, the overall budget for NCATS will be the sum of the imported programs—an amount much smaller than the several billion dollars currently being spent on translational research by existing Institutes and Centers."

"Drug discovery and development is not a high priority of the large majority of these programs whose total funding is projected to be $500 million," Lively says. "So, that accounts for the bulk of the funds projected to initially go to NCATS."

The plan currently being debated NIH corridors and likely on Capitol Hill certainly isn't without its drawbacks. If the NIH is to focus resources on therapeutic discovery and development, it must be done with a careful evaluation of the impact that could have on other programs, Lively says.

"It is clear to all that the NIH budget is unlikely to increase in the current economic environment. Important efforts to reduce deficit spending will likely constrain the NIH budget for years to come," he says. "NIH must ask if new investment in a therapeutics development initiative is the best way to use limited resources. Paylines for individual investigator grants are now dropping below the 10th percentile in some NIH institutes."

As a result, Lively maintains that an October start probably isn't very realistic.

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