In Defense of Clinical Drug Trials
Published: July 18, 2013
To the Editor:
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In “Do Clinical Trials Work?” (Sunday Review, July 14):
Clifton Leaf cites a large clinical trial of Avastin for brain cancer that showed no survival benefit. He claims that we know that some patients responded to Avastin because they “significantly beat the average.” But the trial was negative presumably because a similar number in the control group “responded” in this way to placebo. Mr. Leaf suggests that we learned little from this trial, but in fact we learned that for this disease the drug does not work.
The failure to know whether treatments that don’t work over all might help some patients is not a failure of clinical trials, but rather reflects our limited biologic understanding, the focus of basic research.
Innovations like Bayesian trials at M. D. Anderson and point-of-care randomization at Stanford help make trials more efficient and easier to embed in practice. The reason to make them a wider part of care is that millions of people are alive or better off today because of them: clinical trials work.
STEVEN GOODMAN
CHRISTINE LAINE
ELISEO GUALLAR
Stanford, Calif., July 15, 2013
CHRISTINE LAINE
ELISEO GUALLAR
Stanford, Calif., July 15, 2013
Drs. Goodman and Laine are editors of, respectively, the journals Clinical Trials and the Annals of Internal Medicine. Dr. Guallar is associate editor of the Annals of Internal Medicine.
To the Editor:
Clinical trials have been the foundation of progress against cancer for more than 50 years. The challenge in developing new treatments is the complex biology of cancer and the diversity of patients, sometimes making it difficult to match the right drug to the right patient.
In the clinic, the rapid launch of very large studies of middling drug candidates driven by the hope of short-term profits is not the most efficient and effective way to make meaningful advances for patients.
While faster and more adaptive trial designs like the I-SPY trial are one important approach to the problem, they can address only some of the limitations. A fundamental problem continues to be that only 3 percent to 5 percent of adult cancer patients in the United States participate in clinical trials.
We need to learn from every single patient receiving treatment on or off clinical trials. Informed by the input of “big data” learning health systems like the American Society of Clinical Oncology’s CancerLinQ, we can determine what treatments work best in real-world clinical practice, so that clinical trials can be smarter, smaller, faster and more broadly relevant.
CLIFFORD A. HUDIS
New York, July 15, 2013
New York, July 15, 2013
The writer is president of the American Society of Clinical Oncology. A member of the I-SPY data safety monitoring committee, he is the committee’s unpaid former chairman.
To the Editor:
Clifton Leaf asks whether clinical trials work. In suggesting solutions to various trial challenges, he reports on a study design that seems potentially highly prone to bias.
Frequently, trial results are not usable because of bias, which can significantly distort study results, or because of insufficient reporting to determine study quality. Many instances of reversals are a matter of the difference in outcomes that can arise when comparing results of studies at low risk of bias with studies of the same intervention at high risk of bias.
Unfortunately, most physicians lack skills in evaluating studies for bias and relevancy. This can result in harmful consequences to patients and is one of the reasons enthusiastic use of Vioxx ended up harming so many patients.
Over all, there is a need for better study design, execution, reporting and scientific critical appraisal skills by researchers and health care decision makers.
SHERI ANN STRITE
MICHAEL E. STUART
Seattle, July 15, 2013
The writers, co-founders of Delfini Group, health care consultants, are the authors of “Basics for Evaluating Medical Research Studies: A Simplified Approach (And Why Your Patients Need You to Know This).”
To the Editor:
In 2005 I received a diagnosis of Waldenstrom’s macroglobulinemia, a lymphoma defined as “rare and incurable.” I chose to enroll in a clinical trial and was most fortunate to attain the ideal result: I became and remain cancer-free.
Even more important, my oncologist and his research team have discovered the genetic mutation at the root of this disease in 90 percent of patients tested. New drugs are in development, new clinical trials are in progress, and the results so far are thrilling. I believe that a cure is in reach.
Although cancer can express itself very differently from one person to the next, as scientists zero in on its causes and develop new treatments, more patients will be needed to participate in clinical trials. With luck, the number of success stories will skyrocket.
At the end of the day, medicine, like art, is a creative process, and very much a team effort.
KAREN LEE SOBOL
Boston, July 16, 2013
Boston, July 16, 2013
The writer is the author of “Twelve Weeks: An Artist’s Story of Cancer, Healing and Hope.”
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