James D. Heckman, Editor-in-Chief, The Journal of Bone and Joint
Surgery
In his thoughtful editorial published this year in Spine
(2006;31:1-3), Dr. Weinstein, Editor-in-Chief, clearly portrays a
"broken" health-care system in the United States. A major
component of the problem is the inefficiency and ineffectiveness of clinical
research, and he outlines a novel and potentially extremely effective
solution, a National Clinical Trials Consortium. The editorial is reproduced
here for your consideration.
We find ourselves working in a healthcare system in which $1.9 trillion
dollars are spent to have the U.S. rank 37th in world
health.1 Clearly,
our health expenditures don't necessarily coincide with better outcomes. There
is evidence to suggest our healthcare system is fundamentally broken. The
Dartmouth Atlas studies have provided ample evidence that our healthcare is
irrationally distributed, and that patients in high utilization areas had more
inpatient treatment, more diagnostic tests, and increased use of
specialists.2
Frequently, technologies or diagnostic tests are brought to market
(increasingly, directly marketed to patients) despite a paucity of good
studies providing solid evidence of proven efficacy and or effectiveness. We
continue to bring new technologies into practice with studies that are often
underpowered and without clearly defined endpoints. Unfortunately these
studies can provide a false sense of security to our practicing physicians and
to the patients to whom these treatments are directly marketed. Inadequate
follow up and failure to report negative findings can undermine the very
scientific process that has advanced medicine.
Naturally, given their investment, industry-supported trials are driven by
the need for swift FDA approval in order to get a device or drug to market.
However, we know that industry-supported projects yielded a nearly 73%
positive result, whereas unfunded research had a much more even distribution
of results, with positive at 46.2%, neutral at 45.6% and negative at 8.2%.
Therefore, we should also pay close attention to the affiliations of those in
academia that perform research. At university campuses around the country we
are seeing a greater influence of large corporations in the naming of science
centers, departments, professorships, and, ironically, schools of ethics. So
carefully reading the disclosure statements of articles reporting the results
of a trial is imperative to interpreting and understanding the results. Thus,
caution is justified in adopting new technology that has not been evaluated
with the rigor that affords us more than a modicum of confidence that we are
doing what is best for our patients.
Dr. Elias Zerhouni has outlined a new initiative at the NIH called the
Clinical and Translational Science Awards (CTSA), targeting some of the most
basic systemic barriers in medicine as practiced
today.4 Recognizing
that we currently depend on two problematic systems to perform scientific
research-the first being companies with much to gain financially from a
positive result that their trials of effectiveness should be critically
assessed and the second being that many overworked clinicians and basic
scientists are being asked to cram the three careers of teacher, practitioner,
and researcher into one lifetime. The CTSA hopes to partner with institutions
who are already "grappling with the complex challenges posed by the
clinical and translational science of the 21st century." This program is
an acknowledgment that the changes necessary to reform all aspects of health
care are profound, difficult, and complex.
Such change doesn't involve easy or quick fixes, or shiny new technology.
Only the earnest collaboration of everyone who cares deeply about the state of
our health care environment will bring this about. To this end, I'm
recommending formation of a National Clinical Trials Consortium (NCTC)
(Figure 1). This should be
formed and run by physicians and surgeons and their PhD colleagues. It should
have an oversight board from independent professional societies, and
appropriate specialty societies as well as public members. This consortium
would be supported by industry, payers, FDA, NIH, and others, including the
public, with vested interest in the clinical research enterprise. There should
also be a data safety and monitoring board with public members. The primary
function of the consortium would be to promote and direct high-quality
clinical trials less susceptible to conflict of interest, affording them more
face validity and almost certainly less bias.
Such a structure would be both tangibly and conceptually constructive.
Funding of an independent consortium cannot and will not eradicate all
conflicts, but would provide checks and balances to minimize true as well as
apparent conflicts. Answering the most pressing and relevant clinical
questions would be the priority. The NCTC would fund innovative practicing
physicians and PhDs with collective expertise in those aspects of clinical
trials most prone to weakness: study design consistent with ones' stated
hypothesis and specific aims; protocol development; data collection and data
monitoring; complex longitudinal statistical analysis; intention-to-treat
analysis and appropriate adjustments in an as-treated analysis; and
appropriate oversight by an independent data and safety monitoring board.
Another advantage of such a well-organized consortium would be more rapid
enrollment and completion of projects with required open dissemination of
results to all relevant public and private groups. This structure would serve
to increase credibility and validity and possibly lessen medical
liability.
We must avoid using a technique or device on our patients only to find out
weeks, months, or years later that it failed to achieve what we had understood
from available literature. The NCT consortium could also be used for
postmarket surveillance in collaboration with our industry partners, FDA,
Medicare, Medicaid, and independent private payers. The hope remains that a
National Clinical Trials Consortium with broad geographic representation would
allow us to bring the focus of our profession back to serving our diverse
patient needs while minimizing the costs incurred by the current extensive
network of various individual(s) trial group(s) conflicted in mission and
purpose.
Barriers
There are many potential barriers to establishing the utopian clinical
trials consortium. First, we must have the will to do this. We must avoid
further fragmentation. We must trust one another and we must commit the time
and resources necessary to accomplish this most important endeavor. Second, we
must proactively change the current process by which technology is adapted by
surgeons and colleagues to the benefit of our patients without any unintended
detriment. In so doing we will be empowered to regain our focus, diminishing
the noise of reimbursement and medical liability demands that currently drown
out everything else. Breaking down barriers is not easy. Knowing the right
thing is hard enough; doing the right thing is even harder. Most of us find
reasons to avoid it: "I'm too busy" is a common complaint.
"This will cut into my practice." "This interferes with my
physician/patient relationship." "My results are already good and
I don't need more data."
Reality
External forces are now coming to bear on these issues. Ford automotive
company and General Motors stock has been relegated to "junk bond"
status, in part related to the high cost associated with their medical
benefits. Recently, the American automobile industry has changed its strategy
for long-term benefits-employees now share in their long-term health
expenditures. Failure to do so would be prohibitive for the U.S. auto industry
and its capacity to remain competitive in world markets. United Airlines, a
leader in air transportation, is another example where the toll of ever
increasing healthcare cost is pushing their company and this industry toward
bank-ruptcy with a corporate pension plan at risk. Today, fuel cost is partly
responsible but escalating healthcare costs have been omnipresent and loom
larger each year. Clearly, our major industries can no longer afford to pay
for health care that doesn't work, and we can't afford to provide care without
scientific evidence to support our work. Medicare and other large private
payers can no longer afford to pay for medications, diagnostic tests,
surgeries, or other treatments that are not proven to be effective.
Winston Churchill said "Americans do the right thing after they have
tried everything else." I hope in our case that will not be true. There
are many examples today in which well-designed clinical trials have benefited
our profession and more importantly our patients. One recent example is in
patients with persistently active rheumatoid arthritis, the combination of
etanercept and methotrexate was safe and well tolerated and provided
significantly greater clinical benefit than methotrexate
alone.5
Being transparent in our mission, vision and our research is the best
medicine we can give to society. A National Clinical Trials Consortium offers
industry, our profession and our patients the results we all want, an
altruistic, selfless means to determine the most effective treatment
alternatives. Our patients are having to take more and more responsibility for
their own healthcare. As true partners, we along with our patients, industry,
government and the private sector can have a major impact in moving our
nation's health forward.