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January 2010
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Index
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What is appropriate care - New
England Journal: August
2011
Therapy in
Juvenile Arthritis
Annals of Rheumatic disease july
Can Tumor Necrosis Factor
Inhibitors Be Safely Used in Pregnancy?
malignancy
and anti TNF drugs
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The Doctor’s Dilemma — What Is “Appropriate” Care?
NEJM | August 17, 2011 | Topics: Comparative Effectiveness,
Cost of Health Care
Victor R. Fuchs, Ph.D.
Most physicians want to deliver “appropriate” care. Most
want to practice “ethically.” But the transformation of
a small-scale professional service into a
technologically complex sector that consumes more than
17% of the nation’s gross domestic product makes it
increasingly difficult to know what is “appropriate” and
what is “ethical.” When escalating health care
expenditures threaten the solvency of the federal
government and the viability of the U.S. economy,
physicians are forced to reexamine the choices they make
in caring for patients.
In an effort to address this issue, physicians’
organizations representing more than half of all U.S.
physicians have endorsed a “Physician Charter” that
commits doctors to “medical professionalism in the new
millennium.” The charter states three fundamental
principles, the first of which is the “primacy of
patient welfare.” It also sets out 10 “commitments,” one
of which states that “while meeting the needs of
individual patients, physicians are required to provide
health care that is based on the wise and cost-effective
management of limited clinical resources.” How can a
commitment to cost-effective care be reconciled with a
fundamental principle of primacy of patient welfare?
The dilemma arises for two main reasons. First, recent
decades have witnessed a flood of new, expensive medical
technologies (drugs, imaging devices, surgical
procedures) that are of varying degrees of value to
patients. A few are true breakthroughs, with strong
favorable effects on mortality and morbidity. Others
make a meager contribution, at best, to health outcomes.
Moreover, technologies that may provide high value for
carefully selected patients are often used
indiscriminately for a much larger cohort of patients.
Second, health insurance, private or public, has become
so widespread that 90% of the country’s health care bill
is paid by third parties, not by the patient receiving
the service.
What is a conscientious physician to do? Some new cancer
drugs cost thousands of dollars per month for a single
patient. The bills for many surgical procedures run to
five or even six figures. Noninvasive imaging devices
can offer information to assist in diagnosis, at a
cumulative cost in the billions of dollars. U.S.
patients, on average, get almost three times as many
magnetic resonance imaging scans as Canadian patients;
there is no evidence that this large differential can be
explained by national differences in the medical
condition of patients or that it results in significant
national differences in health outcomes. So what level
of utilization deserves to be called “appropriate”?
If insurance were not widespread, many physicians would
be reluctant to order an expensive intervention unless
it offered a good chance of substantial benefit — that
is, unless it was cost-effective. Indeed, without
U.S.-style cost-insensitive insurance, many expensive
diagnostic and therapeutic innovations would not be
developed and brought to market.1 The
insured patient, on the other hand, will usually want
any and all care that might possibly be of net benefit,
regardless of cost. The physician may recognize that the
intervention under consideration is not cost-effective
but may recommend it anyway, for a variety of reasons:
to keep the goodwill of the patient, to protect against
a malpractice suit, or in the belief that the “primacy
of patient welfare” makes the denial of such care
“inappropriate” and “unethical.”
The doctor’s dilemma is the nation’s problem. Some
policy experts think that if patients had “more skin in
the game” — that is, had less insurance — the problem
would be solved. It would not. Even the most ardent
advocates of deductibles and copayments acknowledge the
need for an annual cap on patients’ payments, beyond
which insurance takes over completely. There is no
consensus on the right level for the cap, but it is
generally recognized that the average U.S. household,
with large debts and minimal financial assets, could not
handle much more than $5,000. But the extreme skew in
annual health care expenditures, with 5% of individuals
accounting for 50% of spending in any given year, means
that many health care decisions, and especially those
involving big-ticket interventions, will be made by and
for patients whose costs have exceeded the cap.
Another popular “solution” is to eliminate care that
does more harm than good — that is, “unnecessary” care.
Such elimination would be desirable, but the potential
savings from this source are smaller than is usually
claimed. It is true that after the fact, many
interventions turn out to be useless or even harmful for
some patients. But the heterogeneity of patient
populations and uncertainty about the response of
individual patients to an intervention means that it is
often difficult or impossible to determine in advance
which ones will prove to help particular patients and
which will turn out to have been unnecessary.
There is no escaping the fact that many interventions
are valuable for some patients even if, for the
population as a whole, their cost is greater than their
benefit. Under what circumstances are they likely to be
ordered, and when are they likely to be withheld? The
context within which the physician practices, his or her
assumption about the behavior of other physicians, and
the economic and health consequences of ordering all the
care that might do some good versus practicing
cost-effective medicine will affect the physician’s
choice. If the physician is paid on a fee-for-service
basis and the patient has open-ended insurance, the
scales are tipped in favor of doing as much as possible
and against limiting interventions to those that are
cost-effective. In that setting, who would benefit from
the resources that are saved by practicing
cost-effective medicine is not obvious to the physician.
In contrast, if the physician is practicing in a setting
that has accepted responsibility for the health of a
defined population and the organization receives an
annual fee per enrollee, the chances of the physician’s
practicing cost-effective medicine are substantially
increased, even though all patients are insured. The
physician’s colleagues are practicing the same way, and
the resources saved can be used for the benefit of the
defined population, which includes the physician’s
patient. In Canada, which has universal insurance, per
capita spending on health care is only 55% of the U.S.
level because there is a limited overall budget, and all
physicians in the system recognize the need for prudence
in making decisions about care.
In short, when physicians are collectively caring for a
defined population within a fixed annual budget, it is
easier for the individual physician to resolve the
dilemma in favor of cost-effective medicine. That
becomes “appropriate” care. And it is an ethical choice,
as defined by philosopher Immanuel Kant, because if all
physicians act the same way, all patients benefit.2
Disclosure forms provided
by the author are available with the full text of this
article at NEJM.org.
Source Information
From Stanford University, Stanford, CA.
References
-
Weisbrod B. The health care quadrilemma: an
essay on technological change, insurance,
quality of care, and cost containment. J
Econ Lit 1991;29:523-552Web
of Science
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Kant I. Critique of practical reason and
other writings in moral philosophy. Beck LW,
trans. Chicago: University of Chicago Press,
1949.
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Aggressive Combination Drug Therapy in Very Early Polyarticular
Juvenile Idiopathic Arthritis (ACUTE–JIA): a multicentre
randomised open-label clinical trial
Ann Rheum Dis 2011;70: 1605-1612
Abstract
Objectives In
juvenile idiopathic arthritis (JIA), the efficacy of
very early disease-modifying drug therapy, synthetic or
biological, is not well known. Three alternative
strategies were compared for treating recent‑onset
polyarticular JIA.
Methods In
a 54-week multicentre open-label clinical trial, 60
disease-modifying antirheumatic drug (DMARD)-naive
patients aged 4–15 years were randomly assigned into
three treatment arms. The efficacy of infliximab plus
methotrexate (TNF) was compared to that of two synthetic
therapies: methotrexate alone (MTX) and DMARD
methotrexate, sulphasalazine and hydroxychloroquine in
combination (COMBO). Primary endpoint was American
College of Rheumatology paediatric 75% improvement (ACR
Pedi 75). Secondary endpoints were inactive disease and
safety.
Results In
59 patients, mean (±SE) age at baseline was 9.6±0.4
years, duration of JIA 1.9±0.2 months and number of
active joints 18±1. ACR Pedi 75 was achieved in 100%
(19/19) of patients receiving TNF, 65% (13/20) on COMBO
(95% CI 44% to 86%) and 50% (10/20) on methotrexate (95%
CI 28% to 72%) p<0.0001. Thirteen patients receiving TNF
(68%; 95% CI 47% to 89%) achieved inactive disease,
whereas eight (40%; 95% CI 22% to 63%) on COMBO and five
(25%; 95% CI 6% to 44%) on methotrexate did (p=0.002).
Patients on TNF spent a mean 26 weeks (95% CI 18 to 34)
with inactive disease, longer than did those receiving
COMBO (13 weeks; 95% CI 6 to 20), or methotrexate (6
weeks; 95% CI 2 to 10). Serious adverse events were
rare.
Conclusion In
early polyarticular JIA, targeting to achieve minimally
active or inactive disease, infliximab plus methotrexate
was superior to synthetic DMARD in combination and
strikingly superior to methotrexate alone.
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Can Tumor Necrosis Factor Inhibitors Be Safely Used in
Pregnancy?
The Journal of Rheumatology
2010
vol. 37 no.
1 9-17
.
Objective.
We review available safety data for use of currently approved
tumor necrosis factor (TNF) inhibitors during pregnancy and
lactation and suggest guidelines for use of these agents among
women of reproductive age.
Method.
Although regulatory agencies encourage the inclusion of pregnant
women and those of child-bearing age in randomized controlled
trials, pregnant and lactating women have universally been
excluded from studies because of unknown or potential risks to
the fetus. Thus, strong evidence-based treatment recommendations
during pregnancy are usually lacking and safety information is
derived from voluntary reports of adverse events during
post marketing surveillance or via uncontrolled, observational
studies, reviewed here.
Results.
Uncommon adverse pregnancy outcomes observed with TNF inhibitor
therapy appear to approximate those seen in women not receiving
such therapy and may include premature birth, miscarriage, low
birth weight, hypertension, and preeclampsia. There are rare
reports of fetal malformations or congenital anomalies in
patients exposed to TNF inhibitors during conception or
pregnancy. However, the incidence of these events appears to be
far below the 3% rate of congenital anomalies in the general
population.
Conclusion.
If the activity or disease severity
precludes the cessation of a TNF inhibitor and/or DMARD,
uncontrolled observations suggest that conception and early
pregnancy are not adversely affected by use of TNF inhibitors.
Nearly 70% of pregnant patients can discontinue their TNF
inhibitor early in the pregnancy (or with determination of
pregnancy) without augmenting maternal or fetal risks.
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Malignancies associated with tumour necrosis factor
inhibitors in registries and prospective
observational studies: a systematic review and
meta-analysis
Abstract
Objectives This
project was undertaken to assess the risk of
malignancy in patients with rheumatoid
arthritis treated with tumour necrosis
factor inhibitors (TNFi) in clinical
practice, as recorded in prospective,
observational studies.
Methods The
authors undertook comprehensive searches of
MEDLINE, EMBASE, the Cochrane Database of
Systematic Reviews and American College of
Rheumatology, European League against
Rheumatism and British Society for
Rheumatology conference abstracts according
to a prespecified protocol.
Results The
searches identified 2039 full-text papers
and 1979 conference abstracts, of which 21
full texts and eight abstracts met the
inclusion criteria. The pooled estimate for
the risk of all-site malignancy from seven
studies was 0.95 (95% CI 0.85 to 1.05). Two
studies reported there was no evidence that
longer exposure to TNFi agents increased the
risk of malignancy. In patients with
previous malignancies there was a higher
risk of a new/recurring malignancy. This
risk was not increased further by exposure
to TNFi, although CI were wide. Results from
four studies showed that patients treated
with TNFi have a significantly increased
risk of developing a non-melanoma skin
cancer (1.45, 95% CI 1.15 to 1.76). In
addition, patients are at an increased risk
of developing melanoma, as the pooled
estimate from two studies was 1.79 (95% CI
0.92 to 2.67). The pooled estimate for the
risk of lymphoma was 1.11 (95% CI 0.70 to
1.51).
Conclusions This
systematic review and meta-analysis shows
that TNFi treatments do not increase the
risk of malignancy, particularly lymphoma.
However, they do appear to increase the risk
of skin cancer, including melanoma.
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