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Smarter Clinical Trials for Faster Drug Development

August 23, 2004

An August 4 New York Times1 article describes a new approach to clinical trials. The idea, (sometimes called "experimental medicine") is to use modern imaging, genetic, and other technology early, starting with the first patient who ever takes an experimental drug, to get solid indications of whether or not it is working. Researchers and companies make sure the drug is getting to where it is needed in the body, that it has the biochemical effects intended, and that the dose is appropriate -- eliminating losers early and focusing attention and resources on more promising drug candidates. The traditional approach uses the first human trials to pick the largest dose most patients can tolerate (which may be more than needed for medical efficacy, but is likely to become a standard dose for further research and for approval), and only then runs other trials to start testing whether the drug does anybody any good.


AIDS is barely mentioned in the article -- perhaps because it is no longer in the forefront of clinical-trial design. In AIDS and other diseases the biggest block to finding new treatments seems to be the gap between where academic research stops and where drug development begins. Many good ideas get published in journals, but usually the researchers who developed them do not do human testing, and no one does the next, relatively inexpensive steps to show which ideas have solid potential for practical development now. And usually only one company has the legal rights to a compound, so if anyone else has a good idea for using it, they (and the public as well) are often out of luck. Or nobody has exclusive rights, so no company gets involved. Clearly the current system works very poorly in turning scientific advances into new kinds of treatment, and fundamental rethinking is needed.


  1. Andrew Pollack. "In drug research, the guinea pigs of choice are, well, human." The New York Times, August 4, 2004. Note: to find the article online you can search for "Garabadian" the name of a patient in a cancer trial, on -- however the Times requires payment for online articles older than one week.

ISSN # 1052-4207

Copyright 2004 by John S. James. Permission granted for noncommercial reproduction, provided that our address and phone number are included if more than short quotations are used.

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