The Idea-to-Drug Gap
The fundamental pact between medical researchers and the public is simple. The U.S. government spends billions of taxpayer's dollars on basic research each year, expecting at some point to reap the benefit of new therapies and medical tests that people can use.
Sure, the public needs to be patient as researchers painstakingly tease out details of how things work in living cells, genes, organs, and entire organisms, including humans. Experimentation requires a great deal of trial and error, and often ends in failure, though even failure can be as useful as success in testing hypotheses.
But what shall we make of a recent study in the prestigious journal Science that suggests a mean lag time of 24 years between the first publication of a therapeutic idea and the confirmation of that idea in a clinical trial on humans?
The study by a team at the University of Ioannina School of Medicine in Greece analyzed published studies of successful clinical trials for drugs and counted backwards to the year when the earliest journal publication on preparation, isolation, or synthesis appeared, or when the first patent was awarded. Researchers on the team also have appointments at Tufts Medical Center in Massachusetts and George Washington University in Washington.
The 24 years, by the way, does not include additional years it typically takes to complete successful human testing for drugs before they can be approved by the Food and Drug Administration. This additional time can mean a total of perhaps 30 years from idea to approved drug.
A previous study in 2004 by one of the co-authors, John Ioannidis, showed that most scientific findings touted in major journals as imminently feasible for treating humans never even make it to clinical trials.
Four years ago, his team scoured more than 25,000 studies in six journals published between 1979 and 1983; it found 101 studies that made particularly bold claims of imminent feasibility for treating humans. Yet only four of these have ended up as approved therapies, and only one-ACE inhibitors for high blood pressure-is widely used.
Other successful drugs apparently come from studies making less grandiose claims, or out of the labs of drug companies.
As the world faces economic turmoil, and what could be more limited resources expended on basic research, it seems as if we could get more bang for our shrinking bucks.
This has been the idea behind an effort launched in 2006 by Elias Zerhouni, who is about to step down as the director of the National Institutes of Health, to promote translational research-the study of ways to translate basic science into actual treatments-at the N.I.H. So far, the effort has spent or committed some $10 billion to programs in a consortium of 38 universities and research centers.
Last month, for instance, the N.I.H. awarded $22 million for the Albert Einstein College of Medicine and Montefiore Medical Center in New York City to create an institute for translational research.
The reasons given for the time lag and for the failure to follow up on initial discoveries are numerous and complex, though a few seem to pop up whenever the subject is brought up in papers, articles, or conferences.
One problem is how the culture of medical research tends to reward original and novel ideas and findings, but not the process of retesting these discoveries to see if they hold up. Research institutions gain funding for splashy new findings, and scientific journals garner headlines for publishing them.
The media plays its role by dutifully and often uncritically reporting-sometimes breathlessly-the latest astounding discoveries as fed to them by the journals. I have been known to do this myself, writing stories about findings that realistically are years-or decades-away from being tested in people, and others that will never make it out of the lab.
In other words, some studies make premature and exaggerated claims-or so says the team from Greece.
"As scientists, we should convey to our funders and the public the immense difficulty of the scientific discovery process," the authors of the recent study write. "Successful translation is demanding and takes a lot of effort and time even under the best circumstances; making unrealistic promises for quick discoveries and cures may damage the credibility of science in the eyes of the public."
Another issue is that no good system exists to connect the most promising labs and researchers with the companies that develop drugs into products. Technology transfer offices at Harvard, Stanford, and other leading universities have become more aggressive in recent years in proffering the findings of their researchers to companies and investors, yet there is no systematic vetting of studies to see which might be commercially viable.
The field of translational medicine means to address these problems and others in an effort to make good on the original deal between the research community and the public. The translational movement also has a philosophical and ethical component that aims to link medical researchers with other stakeholders, including physicians, ethicists, public health experts, policy makers, and the public.
Some progress is being made to closing the gaps between ideas and drugs, says Eric Topol, director of the new Scripps Translational Science Institute in La Jolla, California. "We need more funding and emphasis on translational science," he told me, "and we're up against a very entrenched system. But I am an optimist that we can make this work."VisitÂ Portfolio.comÂ for the latest business news and opinion, executive profiles and careers.Â Portfolio.com© 2007 Condé Nast Inc. All rights reserved.