More MS news articles for Aug 2001

Human Trials: Scientists, Investors, and Patients in the Quest for a Cure

http://www.medscape.com/medscape/GeneralMedicine/journal/2001/v03.n04/mgm0731.01.shim/mgm0731.01.shim.html

By Susan Quinn
Perseus Publishing
Copyright 2001
295 pages
ISBN: 0-7382-0182-0
US $26.00

Reviewed by: David S. Shimm, MD, FACP
[MedGenMed, July 31, 2001. © Medscape, Inc.]

Popular accounts of pioneering scientists and physicians have long fascinated the public. Indeed, the somewhat 1-dimensional picture of the dedicated researcher or clinician struggling against disease, not to mention skeptical or jealous colleagues, is a compelling one. However, although books such as Paul DeKruif's Microbe Hunters; Devils, Drugs and Doctors, by Howard Wilcox; and Magic, Myth and Medicine, by John Camp, do mention their protagonists' struggles, they have a fairy tale "and they all lived happily ever after" denouement -- Paul Ehrlich discovers salvarsan and Marie Curie gets the Nobel Prize.

In Human Trials: Scientists, Investors, and Patients in the Quest for a Cure, however, Susan Quinn, who has written biographies of physicist Marie Curie and psychologist Karen Horney, gives the reader a Paul Harvey-esque "here's the rest of the story" look at clinical research -- specifically, a look at the tensions among venture capitalists, start-up biotechnology companies, and clinical researchers.

The book tells the story of Howard Weiner, a professor of neurology at Harvard conducting research on multiple sclerosis (MS), who develops a technique for inducing immunologic tolerance by oral administration of antigen. He forms a company, Autoimmune, Inc., which attempts 2 clinical trials -- one a trial of myelin basic protein to treat MS, and the other a trial of collagen to treat rheumatoid arthritis (RA). In neither trial does he show a significant difference between the experimental agent and placebo, due to an unusually high placebo response rate. This leads to the failure of Autoimmune, Inc.

Quinn's depiction of the science behind her story seems appropriate for the book's audience. Her description of immunologic tolerance gives the reader sufficient background to understand where Dr. Weiner is trying to go with his technology, without confusing the reader or providing excessive detail. Her portrayals of individual MS and RA patients enrolled in the clinical trials are engaging and add a human dimension to her story. Some of her second-level characters -- such as Weiner's laboratory colleague, Ruth Maron, and Autoimmune's CEO, Bob Bishop -- are also interesting. Particularly compelling portions of the book ("the rest of the story") include Quinn's narrative of Weiner's meeting with venture capitalists on behalf of Autoimmune, Inc., and her depiction of the competitive atmosphere that exists among the clinical researchers when they discover how much money each is being paid for accruing patients to clinical trials.

While Quinn may draw a deft portrait of Ruth Maron and Bob Bishop, her depictions of the protagonist and some of the bit players seem more 1-dimensional. Perhaps Dr. Weiner is a candidate for beatification, but Quinn's saintly description of him does not ring entirely true. While she does cite some narcissistic passages from his personal journal, which he has apparently kept at least since medical school --perhaps to show that her saint has feet of clay, Weiner does not appear as a real, 3-dimensional individual. At the other end of the spectrum, for example, Quinn's passing description of a spokeswoman for a consulting company as "a bleached blonde in her thirties with bright red lipstick" is gratuitously demeaning and distracting.

Quinn's discussion of the payment of capitation fees to clinical researchers was not entirely satisfying. Here, she discusses the financial benefits of doing clinical research for one academic physician:

The one thing that gives Sewell [a Harvard rheumatologist] some autonomy and fiscal clout within the increasingly burdensome bureaucracy is clinical research.... Lea Sewell, situated at a Harvard teaching hospital that is a magnet for patients with unusual diseases, manages to attract a lot of drug company research at a decent per-patient fee. Even after she pays out a large percentage of the money to cover office space, laboratory, and administrative costs, Sewell manages to support a part time research nurse and to augment her own salary from her clinical research. (pp. 217-218)

And here is her description of a private practice physician who does pharmaceutical industry-sponsored clinical trials:

... there is no doubt that clinical trials are also sustaining to [Dr.] Ron Rapoport and his operation. "The income from studies is valuable," he acknowledges. Rapoport has an office staff of five, including his wife Jenny, a former surgical nurse who comes in three days a week. And he has three children... who go to private school. "That's another thing the studies have helped," he says. "I can afford to send them to private school. And if I did not do studies or give lectures, it might not be affordable."

Rapoport... is also attracted to the entrepreneurial side of trial work. He has a card that promotes his clinical research... "It's not just making the money that's exciting," he explains, "it's making a successful venture." (p. 234)

Quinn has shown how performing industry-sponsored clinical trials provides extra money above and beyond expenses for Dr. Sewell, in academic practice, and for Dr. Rapoport, in private practice. For Dr. Sewell, this money helps to defray some of her overhead and to augment her salary, giving her some leverage with the university and hospital administration as well as some direct personal gain. Dr. Rapoport clearly acknowledges the personal financial benefits of performing clinical trials.

Clearly, drug company capitation payments are more than simply compensation for overhead, analogous to the overhead paid along with NIH grants, as some have suggested. It is frustrating that in one paragraph Quinn glosses over the very real possibility that clinical researchers, squeezed by decreasing patient revenue due to managed care and by decreasing peer-reviewed research money, might be a little too persuasive or encouraging with regard to clinical trials (pp. 228-229). Because of possible financial benefit to the clinical researcher, danger lies in the fact that he or she might (consciously or unconsciously) steer a patient toward a clinical trial when conventional treatment, or even no treatment at all, might be in a patient's best interest. While physicians make more for doing more under fee-for-service reimbursement, this situation differs from instances in which physicians are paid for enrolling patients into clinical trials. Patients understand that doctors are paid for performing clinical activities, and if they believe that the physician might be suggesting a particular course of treatment for financial reasons, they can question him or her, just as they would question an auto mechanic suggesting expensive repairs. However, since most patients are not even aware of capitation payments to clinical researchers, they would not even have reason to consider that a recommendation to enter a trial might have financial motivations. In a book directed at the lay public, more attention to this issue would have been very valuable.

In summary, Quinn has written a very readable accounting of modern clinical research. She covers the science and medicine in understandable language, and perhaps most important, she discusses the financial and regulatory issues in a way that makes the reader understand that drug development has come a long way from the times of Paul Ehrlich and Alexander Fleming.
 

David S. Shimm, MD, FACP, is in private practice at Porter Hospital in Denver, Colorado. Until recently, he was a Professor in the Department of Radiation Oncology at the University of Arizona. He has co-edited Conflicts of Interest in Clinical Practice and Research (Oxford University Press, 1999).

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