Healthcare, especially in the United States, is expensive.
It’s
been
reported
(in 2012) to be 17.9% of the US gross domestic product (GDP), costing $8362 per
person. Despite the United State’s reputation of having predominantly private
healthcare, in 2012 the US government actually spent $4437 per person – making
US government healthcare spending second only to Luxembourg, Monaco, and Norway
– all countries with universal healthcare. In 2010, 93 million Americans (31 % of
the population) received government health insurance – 44.3 million through
Medicare and 48.6 million through Medicaid.
Being Canadian, I did not know the difference between
Medicare and Medicaid, even though I’ve been living in the United States since
2005. So I decided to ask some of my American friends about this. I send out
the question – “What’s the difference between Medicare and Medicaid?” – to a
handful of people with advanced degrees in various medical research
fields, and all covered by private health insurance. Invariably, their first
response was, “one is for old people and the other
is for poor people,” sometimes including caveats such as, “I think. I could be
wrong.”
It turns out that they were right, but there are some
additional distinctions between the two. Medicare is a federally run insurance
program that accounts for 13% of the federal budget. Those covered by this program
– including people over 65 and younger disabled people or dialysis patients –
contribute to a trust fund from which medical bills are paid. Medicare is also
funded through a 2.9% payroll tax levied on employers and workers. In contrast,
Medicaid is run both at the federal and state level, representing 7% of the
federal and 16.8% of the states budgets, and covers ‘low-income’ people of
every age. In this case, medical bills are paid from federal, state, and local
tax funds.
I then asked a couple of my American friends, “did you know
that in 2012 the Canadian government spent $3104/person on healthcare, which is
$1333/person less than the US
government spent in the same year?” I got some of the expected, somewhat cynical
responses, but one of my friends – a remarkably thoughtful person – gave me a
new, more positive perspective, which he prefaced with, “I’m NOT going to
defend the US healthcare system and I think most of the defenses are ridiculous.”
Basically, his take is that the US pays more because it is a
consumer driven country that has traditionally placed great value on science and
healthcare. He based this argument on three factors. First, the US capitalist
economy allows doctors to easily make a lot of money. Second, the US is a huge
country compared to say, Canada, with numerous hospitals, research
universities, and training institutions. Finally, the barriers to training in
the US are lower than much the rest of the world.
“We end up with a ton of doctors who can compete to make
money – because people, generally, will pay anything not to die – and prices go
up,” he said. “Additionally, because we shoulder a lot of the world’s medical
training and biomedical research, we effectively subsidize both medical
training and medical treatment for countries around the globe. So if we were to
adopt a ‘perfect’ system, we would still pay more than, say, Canada, because we
have an additional set of costs attached.”
I have never thought about the US healthcare system in this
way before. It was nice to hear something positive about a system that gets so
much bad press, even though I think many criticisms are well-deserved. But there
is so much more to US healthcare than the distinction between Medicare versus
Medicaid. If you dig deeper, you encounter an “alphabet soup” of abbreviations that
represent a wide variety of healthcare-related philosophies and institutions.
The most well known is, of course, the US Food and Drug
Administration (US FDA) and, in particular, the
Center
for Drug Evaluation and Research (CDER), which is the largest center within
the FDA. CDER’s mission is to ensure that therapeutic drugs used in the US are safe
and effective, and it is required that, prior to developing a clinical trial
program for a potential new drug, an
Investigational
New Drug (IND) application be submitted to CDER. Once the IND application is in effect it’s time to start
clinical trials, with the aim of establishing the safety and effectiveness of your
new drug. Once this evidence is acquired, your company – the drug sponsor – submits
the chemical, pharmacological, medical, and statistical data to the FDA as a
New Drug Application (NDA). A group of
CDER-appointed physicians, statisticians, chemists, pharmacologists, and other
scientists review the NDA and, if approved, the drug manufacturer and the FDA
establish specific language to describe dosage, route of administration, and
any other information to be included on the drug’s label. The FDA also reviews applications for the marketing of generic drugs (through the
Abbreviated
New Drug Application, ANDA), non-prescription drugs (
over-the-counter drugs, OTC), and biological products (through a
Biologic License Application, BLA – my favorite
acronym so far).
[Just for fun, I threw these abbreviations (US FDA CDER IND
NDA ANDA OTC BLA) into an online “anagram solver.” These two were my favorites:
“A candelabra addicts fund nod,” and “A cabana candid fuddled snort.” Hmm. I
don’t think there’s a hidden message here. Perhaps I should forge ahead.]
In addition to the FDA, there are advisory committees of
outside experts who provide the FDA with recommendations and independent
opinions. One example is the
Oncologic Drugs Advisory Committee (ODAC), which
consists of authorities in a variety of fields,
including general, pediatric, hematologic, and immunologic oncology. There are
also a variety of institutions that aim to regulate healthcare more broadly. One
is called the
National Comprehensive Cancer Network (NCCN), which is a non-profit
alliance of leading cancer centers. Another is the
Agency for HealthcareResearch and Quality (AHRQ), one of twelve agencies within the US Department of
Health and Human Services (USDHHS), which sets standards for coverage and acts
as a “consumer watchdog.” One final example is the
US Preventive ServicesTask Force (USPSTF), an independent panel of experts that assesses the merits
of a variety of preventative treatments.
So how do these institutions regulate health care and pharmaceutical
development? Well, all this alphabet soup really comes down to three critical
letters: EBM. This abbreviation stands for “evidence-based medicine,” which is a
cornerstone of the US healthcare system.
One definition of
EBM is, “the conscientious, explicit, and judicious use of current best
evidence in making decisions about the care of individual patients.” Or, in the
words of an anonymous Wikipedia author, “by introducing scientific methods –
particularly the methods of the population sciences – in clinical decision
making, EBM has driven a transformation of clinical practice in medicine.”
Sounds pretty simple, right? Doctors should just do what the
data say. But can we agree on what the data say? Based on my personal
experiences at the University of Chicago, scientists find it challenging to
agree with one another when interpreting data. And how do we determine what
data are “best” when comparing, say, anecdotal evidence from leading medical
practitioners with data from company sponsored clinical trials? In other words,
how do we assess the available evidence and come up with a consensus regarding
practical applications for health care providers?
This is not the only conundrum faced by modern healthcare. A
second issue involves the “off-label” use of drugs. Pharmaceutical companies
develop new drugs for the treatment of very specific indications. In this
context, “indication” means, according to the
online
medical dictionary, “an appropriate therapeutic treatment for a given
condition.” For example, let's say an imaginary disease, Unicornitis, is the approved indication for
the use of an imaginary drug called NoHorn. So, the NoHorn drug label would state that Unicornitis is an FDA-approved indication, and if NoHorn is prescribed for an indication
not listed on its label (say, Rhinoitis), this would qualify as “off-label” use.
It’s illegal for pharmaceutical companies to promote any
drug for off-label purposes; however, once a drug has been approved by the FDA
for one indication, physicians are free to prescribe that drug for any other indication.
According to Wikipedia, “this off-label prescribing is most commonly done with older,
generic medications that have found new uses but have not had the formal (and
often costly) applications and studies required by the FDA to formally approve
the drug for these new indications.” But, if pharmaceutical
companies cannot recommend or even discuss their drugs for off-label use, how
can doctors get the information they need for off-label prescribing?
This brings me back to alphabet soup. The main goals of the
aforementioned health care institutions (NCCN, USPSTF, etc.) are to (1) evaluate
the scientific evidence supporting the use of specific drugs for given
conditions, (2) provide guidelines for health care practitioners regarding the
appropriate use of therapeutic drugs, and (3) generate insight as to whether treatment
costs should be covered by health insurance providers. For example, the “NCCN
Compendium®,” which is recognized by Medicare and Medicaid as an authoritative
reference for oncology coverage policy, contains information regarding NCCN’s
evaluation of oncology drugs in terms of their safety and efficacy. In
addition, when an oncology drug is listed in the NCCN Compendium®, it is generally
reimbursable, even for off-label use.
The USPSTF has a similar system, where medical services are
given a letter grade (A, B, C, D or I), with each grade representing a
different USPSTF recommendation. The USPSTF also assigns levels of certainty
(high, moderate, or low), which reflects the likelihood that the USPSTF
assessment is correct. They have also developed a system to rank evidence quality,
with the best level (Level I) reflecting evidence obtained from at least “one
properly designed randomized controlled trial.”
How well does this all work? Well, I don’t feel qualified to
criticize either Evidence-Based Medicine or US healthcare. Others,
however, have done so. I think one of the most interesting criticisms of EBM is
that it downplays the individual opinions of experienced medical practitioners, with the implication that there is more to “knowing” and decision-making
than can be found in formal evidence. But this is getting into some heavy epistemology
and, since my purpose in writing this essay was to educate myself about the US
healthcare system and not to solve its problems (thank goodness), I will end
with two quotations. The first, by Thomas Jefferson, goes as follows: “If
people let the government decide what foods they eat and what medicines they
take, their bodies will soon be in as sorry a state as the souls who live in
tyranny.” And, finally, some wise words from Voltaire: “The art of medicine
consists of amusing the patient while nature cures the disease.” How
interesting, Monsieur, perhaps you should design a clinical trial to test that
hypothesis?
The opinions here are
mine alone, unless otherwise noted in the text. Any errors in this essay are also mine
alone. To be clear: I am by no means an expert in this field, nor is it my
intention to criticize anyone or anything.
