Stanford Expert Lists Five Steps to Boost Impact of Comparative Effectiveness Research

STANFORD, Calif.--(Business Wire)--
Comparative effectiveness research is generating buzz these days, with the
recent economic stimulus package allocating $1.1 billion for these types of
studies. But a researcher at the Stanford University School of Medicine is
asking policymakers to take a step back and make sure that the plans for
comparative effectiveness research go deep enough to make a difference.

"The discussion that has taken place has been quite superficial and hasn`t
covered the range of changes that are needed for this type of research to be
meaningful," said Randall Stafford, MD, PhD, associate professor of medicine at
the Stanford Prevention Research Center.

The Obama administration sees comparative effectiveness research as a key
strategy for reforming the nation`s health-care system. The research would help
identify the treatment options that are the most effective for a given
condition. Many stakeholders, including health-care providers, consumer groups
and professional organizations, have also expressed enthusiasm at the prospect
of identifying new knowledge about how the effectiveness of one treatment
compares with others.

Despite this potential, Stafford and collaborator Caleb Alexander, MD, assistant
professor of medicine at the University of Chicago, highlight several challenges
that must be met if comparative effectiveness research is to be useful in
significantly improving the quality and affordability of health care. "This is
really a plea to delve into the details, to get beyond the slogan of
`comparative effectiveness` and to not lose the momentum gained to date,"
Stafford said.

Stafford and Alexander`s commentary, which will appear in the June 17 issue of
the Journal of the American Medical Association, outlines five ways to put more
meat on the bones of the discussions surrounding comparative effectiveness
research:

Generate the data more rapidly. The pain reliever Vioxx is the best-known
example of a drug originally aimed at a narrow patient population that became
widely prescribed before evidence of harm was discovered. Ultimately, Vioxx was
pulled from the market, but not before millions were exposed to these harms
without substantial benefits. To prevent similar mishaps, Alexander said that
obtaining comparative-effectiveness information earlier in the life of a new
drug or device is a priority.

Link the evidence to strategies proven to modify how physicians practice
medicine. Simply making the data available to physicians and patients isn`t
enough. "Unfortunately, we still want to believe that information alone will
change physician practice. Years of research, however, suggest there are more
potent influences on physicians, including their local culture of practice,"
Stafford said.

Broaden the agenda beyond drugs and devices. "It can`t just be a comparison of
this drug vs. that drug," Stafford said. "This misses important aspects of
practice and ends up exempting high-cost procedures from scrutiny." Researchers
should focus on comparisons that include lifestyle modifications, such as diet
and exercise, as well as alternative therapies that patients often implement on
their own. In addition, research is needed on the most effective ways of
delivering care. For instance, some studies show better chronic disease outcomes
with nurse case managers compared with physicians working alone.

Alter the regulatory environment. "Comparing a new drug against placebo doesn`t
make much sense if our goal is to compare different clinical strategies," said
Stafford, noting that placebo-controlled trials are the standard for drug
approval by the U.S. Food and Drug Administration. The threshold must be raised
for comparative effectiveness to work, he said. Stafford and Alexander suggested
that if a new medication isn`t tested head-to-head against similar drugs, its
labeling could be changed to say, for instance, "This drug has not been found to
be superior to the other calcium-channel blockers in the treatment of
hypertension." This requirement would provide useful information to patients and
physicians, as well as give manufacturers an incentive to perform more drug vs.
drug clinical trials.

Consider the cost implications. This is controversial because many fear that it
may lead to restrictions on higher-cost treatments, regardless of the
treatment`s effectiveness. Some proponents of comparative effectiveness research
have suggested not including cost as a factor. But as Stafford and Alexander
write in their commentary, "What good is comparative effectiveness if it cannot
be used to discern anything about value to clinicians, insurers, patients and
society?"

The discussions surrounding how to implement comparative effectiveness research
data into the health-care reform effort are still in the early stages, which is
why Stafford and Alexander hope their commentary will prod policymakers to
ensure that the discussions are as comprehensive as possible.

Stafford said previous reform attempts, such as the drive to develop clinical
guidelines in the 1990s for treating specific illnesses, failed because "our
approach to implementing them was simplistic and not sophisticated enough.
Unless we get it right with comparative effectiveness, it`s at risk of a similar
fate."

Stafford and Alexander support efforts to help physicians and patients make
better use of research results in determining which drugs, devices and other
treatment options are the most effective. "The drive for comparative
effectiveness has tremendous appeal. Who could argue against the idea of
generating knowledge about what works and what doesn`t?" said Alexander. But
they say broader changes are needed in the health-care system-including the
FDA`s process for approving new medications and devices-to yield the right kind
of data for such comparisons, and to ensure that patients, physicians and
medical organizations make the wisest possible use of their health-care dollars.

"Unless we start spending our resources more efficiently, our health-care system
won`t survive, let alone fully cover all of the people who are now uninsured or
underinsured," Stafford said.

The Stanford University School of Medicine consistently ranks among the nation`s
top 10 medical schools, integrating research, medical education, patient care
and community service. For more news about the school, please visit
http://mednews.stanford.edu. The medical school is part of Stanford Medicine,
which includes Stanford Hospital & Clinics and Lucile Packard Children`s
Hospital. For information about all three, please visit
http://stanfordmedicine.org/about/news.html.

Stanford University School of Medicine
Print Media:
Susan Ipaktchian, 650-725-5375
susani@stanford.edu
Broadcast Media:
M.A. Malone, 650-723-6912
mamalone@stanford.edu

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