Keeping Myself Amused (While Nature Cures Me)

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.