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A NATIONAL CLINICAL RESEARCH ENTERPRISE – WHAT ARE THE ODDS?

Clinical research in America today is a “nonsystem” desperately in need of repair – or more accurately, a complete overhaul. That was the conclusion of more than a dozen members of the Clinical Research Roundtable (CRR) – writing independently and not under the rubric of the CRR – in an article earlier this year (JAMA 2004;291(9):1120–1126).

“The clinical research effort in the United States must be seen for what it is – a fragmented cottage industry comprising multiple stakeholders, some of whose funding is derived from governmental sources and others from industry, with no overarching vision, no cohesive organizational framework, and at times not even a common forum for dialogue or active collaboration,” declared the authors, led by Harvard Medical School Director of Clinical Research William F. Crowley, MD.

“Most important, this existing but outdated infrastructure, put in place nearly 40 years ago to support a much narrower and less professional spectrum of clinical research activities, is currently functioning on overload.”

PERSPECTIVE FROM NEUROLOGY

Few people with any involvement in clinical research today would challenge this conclusion. “I think they've identified something that's very important,” University of Rochester Professor of Neurology Ira Shoulson, MD, told Neurology Today. Dr. Shoulson chairs the Huntington Disease and Parkinson Disease Study Groups and helped to found the American Society for Experimental Neurotherapeutics (ASENT), a group that pursues a very similar agenda within neurology specifically.

“Clinical research has been thought of in a compartmentalized fashion – at best, by industry working together, which rarely happens, and by academic medical centers working together, which sometimes happens, and by government agencies working together, which sometimes happens. But there's very little going on between those entities.”

“I think we're actually doing phenomenally good basic research in this country and around the world, but the translation of that basic research into therapies is flawed,” agrees Christopher Gallen, MD, PhD, Vice President of Clinical Operations and Clinical Research and Development at Wyeth Research.

“Investigators' levels of training and expertise vary widely. Our tools and techniques vary – something as simple as data systems vary from lab to lab and company to company, and are so non-standardized that it's impossible to develop high levels of expertise,” he said. “Seriously addressing the national clinical research infrastructure is an idea whose time has come, and I applaud the authors for bringing it to national attention.”

RESPONSE: PUBLIC-PRIVATE PARTNERSHIP

Possibly more controversial, however, is the authors' proposed solution: a public-private partnership dubbed the National Clinical Research Enterprise (NCRE), funded by a contribution of 0.25 percent of the annual operating budgets of all health care stakeholders and bringing together all those stakeholders to help set a coherent and cogent public agenda for clinical research.

The NCRE should, the JAMA article suggests, do three things: provide a national forum for participatory discussions, integrated deliberations, and collective actions involving all governmental and private stakeholders in clinical research; set and maintain national standards to improve the safety and efficiency of clinical investigation simultaneously; and coordinate activities, create incentives, and directly fund construction of an up-to-date, seamlessly integrated, and continuously improving national clinical research infrastructure.

It sounds exciting and visionary, but is it feasible? “I think theoretically it does make sense. We need to get people at the same table, and there really is no such table right now,” said Dr. Shoulson. “The idea of a public-private research round table under the banner of a National Clinical Research Enterprise makes a lot of sense.”

“This certainly sounds like a comprehensive, all-encompassing solution, and I have no reason to question the feasibility,” said NINDS Clinical Trials Group Director John Marler, MD. He noted that it parallels efforts to re-engineer clinical research within the NIH.

“We're trying ourselves to transform the way we do clinical research and increase our capabilities to speed the movement of research discoveries from bench to bedside. I think we're all trying to work on the same problem, and these efforts seem very compatible to me.”

WHAT WOULD WORK OR NOT

Some elements of the proposed enterprise will be easier to achieve than others, Dr. Shoulson noted. “I think the national forum is feasible, that it can be established, and that over time people will become comfortable participating and solving clinical research problems in that way,” he said. “But to set and maintain national standards to improve the safety and efficiency of clinical research, now that's going to take much more effort. To establish standards, you must agree on what the deficiencies are, and you have to trust your partners. That's going to take time.”

And what about that proposed 0.25- percent budgetary set-aside from all stakeholders? Some wonder about that number and just how many organizations will reach into their pockets to provide it.

“Taking 0.25 percent from stakeholders' budgets is difficult to enforce, and I doubt that people would do that,” said Alan Faden, MD, Editor-in-Chief of the ASENT journal, NeuroRx®, and Professor of Neuroscience, Neurology, and Pharmacology at Georgetown University School of Medicine in Washington, DC. “Where does that number come from? I'd like to see a number based upon specific needs: it will take this much to support MD salaries, this much to support training programs, this much to facilitate the translation from bench to bedside, and so forth, and within that context try to get people to buy into programs. I don't think it'll happen like this, by setting aside a fixed amount of money and then deciding how to spend it. What would entice stakeholders to buy in to that?”

Dr. Shoulson is more optimistic. “While funding is nothing you can mandate, certainly, it is something to set down as a goal. I think the organizations I'm associated with would think it was worthwhile to contribute,” he said. “Everybody's going to say, ‘We don't have any more money,’ but unless you say this is important, no one's going to come forward and do it.”

From the industry point of view, said Dr. Gallen, the funding proposal has potential – although it needs to be clarified. “It wasn't exactly clear how the funding mechanism would work, and there are many details of it that would need to be figured out,” he said. “But it's clear that an investment needs to be made, and a coordinated investment would probably be more efficient than separate solutions. The concept of coordinated funding, I think, is one that could be supported.”

Dr. Faden suggests that any national clinical research enterprise needs to establish specific dollar amounts of funding required to accomplish its goals. “That would include things like setting up training courses for physicians, both in-training and practicing physicians. But most importantly, it would require some mechanism for ‘buying out’ time for physicians to devote to clinical research,” he said. “No matter how you do it, someone has to buy that time equivalency. It's a major hurdle.”

MERGING SYSTEMS: ANOTHER CHALLENGE

The question of dollars is just the beginning of the challenge facing any entity that tries to develop a coordinated national approach to clinical research. “It's a monumental undertaking. Think of a merger between two companies: it takes tens of thousands of people-hours just trying to get the systems from two different companies to speak the same language,” Dr. Gallen observed. “Now, think about disparate health care companies, government, and academia, all trying to come together. It's a big task; they've bitten into an elephant, and a lot of details are still missing. But in general, the idea is very good, and could pay for itself significantly if it actually worked to increase efficiency and productivity.”

Dr. Shoulson points to ASENT as a model of success. “This committed group, just in the area of experimental therapeutics in neurology, is a microcosm,” he said. “If you broaden it to all of clinical research, I think you'll find that people are very interested in doing this. And when you get people sitting down at the same table representing diverse entities, they'll find a small common point they can start a discussion on. It could be very small to begin with, but we just need a little success working together on a small common problem to get things moving.”

“As neurologists, we owe it to our specialty and our patients to do everything we can to support clinical research and the successful development of important new treatments,” said Dr. Marler. “A few years of hard work could yield some wonderful results.”

Figure

Dr. Ira Shoulson: “Clinical research has been thought of in a compartmentalized fashion – at best, by industry working together, which rarely happens, and by academic medical centers working together, which sometimes happens, and by government agencies working together, which sometimes happens. But theres very little going on between those entities.”

Figure

Dr. Christopher Gallen: “I think were actually doing phenomenally good basic research in this country and around the world, but the translation of that basic research into therapies is flawed.”

Figure

Dr. Alan Faden: “Taking 0.25 percent from stakeholders budgets is difficult to enforce, and I doubt that people would do that.”