Los Angeles Times
Medicine in the dark
By Michael Hochman and Danny McCormick
Some doctors treat patients with early-stage prostate cancer with radiation.
Others favor surgery, while some advocate only close monitoring. Which
approach is most successful? No one knows.
When it comes to diabetes management, doctors don't have answers to key
questions: At what point should insulin be started? Is it safe to lower the
blood sugar to normal levels? What is the best way to monitor blood sugar
Similarly, endocrinologists don't know what is the best way to treat
patients with hyperactive thyroids. Doctors in Europe typically use
medications, while those in the U.S. more frequently give radioactive
iodine. Only limited evidence is available to guide the decision.
It may seem perplexing that there is so much uncertainty about these
relatively simple questions. All of the above treatments have been around
for decades. Shouldn't we have definitive answers by now?
In this week's issue of the Journal of the American Medical Assn., we report
the results of a study that may help explain why we don't. In the study, we
analyzed 328 medication studies recently published in six top medical
journals and found that just 32% were aimed at determining which available
treatment is best. The rest were either aimed at bringing a new therapy to
market or simply compared a medication with a placebo. Whether the therapy
was better or worse than other treatments was simply not addressed.
Research involving new therapies is of course crucial for medical progress,
but there is also a need for research that compares the effectiveness of the
rapidly growing array of existing therapies and approaches.
So why, then, did only a third of medication studies focus on helping
doctors use existing therapies more effectively? The answer lies in the fact
that pharmaceutical companies fund nearly half of all medication research,
including the lion's share of large clinical trials. For obvious reasons,
commercially funded research is primarily geared toward the development of
new and marketable medications and technologies. Once these products have
won approval for clinical use, companies no longer have incentives to study
exactly how and when they should be used.
In support of this claim, we found that 87% of the comparative effectiveness
studies we analyzed were funded entirely or in part by non-commercial
sources, such as nonprofit foundations or government institutions. In
addition, 91% of studies comparing medications with non-pharmacologic
therapies (such as surgery or lifestyle changes) received non-commercial
funding, as did 94% of studies comparing different medication strategies
(such as different blood sugar targets in patients with diabetes) and 90% of
studies comparing the safety profiles of medications. Non-commercial sources
funded 100% of studies comparing the cost- effectiveness of different
treatments, though only 2% of the studies we reviewed included such
Congress recently appropriated more than $1 billion in the American Recovery
and Reinvestment Act to promote comparative effectiveness research. This is
a good first step, but the money will need to be spent carefully. We believe
studies that address fundamental clinical decisions -- such as when to use
medications versus surgery or how to use therapies more effectively --
should be favored over those that simply compare two alternative
medications. There is also clearly a need for more research on the
comparative safety and costs of different treatments. And although many
researchers are thankful for the new research funds, it may soon become
apparent that $1 billion is far from sufficient.
Reform is also necessary to ensure that commercially funded research is
designed in a way that is more helpful to doctors. Our study showed that
two-thirds of commercially funded randomized trials compared medications
with a placebo rather than with another active therapy. Though placebos are
appropriate when no alternative therapies are available, in many of the
trials we examined, we suspect alternative therapies could have been used
instead. For this reason, we believe that regulatory agencies such as the
Food and Drug Administration should only approve new therapies that have
been shown to be at least as good as existing therapies whenever such
alternatives exist. Alternatively, though more controversial, some experts
have proposed that pharmaceutical companies should be allowed to fund -- but
not design -- clinical studies.
As medical science advances, clinical decision-making will only become more
complex. Only by expanding public funding for comparative effectiveness
research can we hope to put existing medical treatments and healthcare
services to their best use. Doing so would ensure that national research
priorities are determined by patient needs rather than by corporate agendas.
Michael Hochman, MD, is an assistant professor of clinical medicine at USC's
Keck School of Medicine. Danny McCormick, MD, MPH, is an assistant professor
of medicine at Harvard Medical School.
Comment: Rather than using excerpts from the JAMA article by Hochman and
McCormick as today's qotd, their op-ed in today's Los Angeles Times provides
an even better summary of their findings along with their astute comments.
Their op-ed obviates the need for me to provide any additional commentary.
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