Alone of all the regular contributors to this blog, I am a surgeon. Specifically, I’m a surgical oncologist specializing in breast cancer surgery, which makes me one of those hyper-specialized docs that are sometimes mocked as not being “real” doctors. Of course, the road to my current practice and research focus was long and involved quite a few years doing general surgery, so it is not as though I am unfamiliar with a wide variety of surgical procedures. Heck, I’m sure I could do an old-fashioned appendectomy, bowel resection, or cholecystectomy if I had to. Just don’t ask me to use the da Vinci robot or, with the exception of the case of a cholecystectomy, a laparoscope, although, given the popularity of robotic surgery, I sometimes joke that I really, really need to figure out how to do breast surgery with the robot. After all, if plastic surgeons are using it for breast reconstruction, surely the cancer surgeon should get in on the action.
I keed. I keed.
Clinical trials of surgical procedures and placebo controls
I have, however, from time to time addressed the issue of science-based surgery, and this weekend seems like as good a time to do so again, given that I just came across an article in the BMJ reporting a systematic review of the use of placebos in surgical trials. It’s a year old, but worth discussing. Before I get to discussing the nitty-gritty of this particular trial, let me just note that the evaluation of surgical procedures for efficacy and safety tends to be more difficult to accomplish than it is for medications, mainly because it’s much harder to do the gold standard clinical trial for surgical procedures, the double-blind, placebo-controlled randomized clinical trial. The two most problematic aspects of designing such an RCT in surgery, as you might imagine, are the blinding, particularly if it’s a trial of a surgical procedure versus no surgical procedure, and persuading patients to agree. I’ll deal with the latter first, because I have direct personal experience with it. (more…)
Tree of Life – the first-known sketch by Charles Darwin of an evolutionary tree describing the relationships among groups of organisms (Cambridge University Library).
The Merriam-Webster Dictionary defines science as:
Knowledge about or study of the natural world based on facts learned through experiments and observation.
Knowledge as distinguished from ignorance or misunderstanding.
While this should distinguish science from pseudoscience, those who practice the latter often lay claim to the same definition. But one of the major differences between science and pseudoscience is that science advances through constant rejection and revision of prior models and hypotheses as new evidence is produced; it evolves. This is the antithesis of pseudoscience. At the heart of pseudoscience-based medicine (PBM) is dogma and belief. It clings to its preconceptions and never changes in order to improve. It thrives on the intransigence of its belief system, and rejects threats to its dogma. Despite the constant claims by peddlers of pseudoscience that SBM practitioners are closed-minded, we know that, in fact, PBM is the ultimate in closed-minded belief. Of course, those of us who claim to practice SBM aren’t always quick to adopt new evidence. We sometimes continue practices that may once have been the standard of care but are no longer supported by the best available evidence, or perhaps may even be contradicted by the latest evidence. Often this is a byproduct of habituated practice and a failure to keep current with the literature. While this is certainly a failure of modern medicine, it is not an inevitable outcome. It is not emblematic of the practice of medicine, as it is with PBM. When medicine is science-based, it strives for continual improvement based on modifications around emerging evidence. (more…)
If science-based medicine is unaffordable, then your care won’t be science-based. Prescription drug costs are one of the biggest concerns in health care today. There seems to be no upper limit on prices, with some new treatments costing over $1,000 per day. The arrival of new drugs to treat (and cure) hepatitis C has created a perfect pharmaceutical storm: highly effective treatments, a large population of potential patients, and huge per-patient costs. It’s renewing the debate about whether important medical treatments are being priced out of the reach of the patients that need them. It’s not just hepatitis. Cancer drug costs are rising as well, driven by more patients and new drugs that in some cases are transforming our expectations about what cancer drugs can do. And while many of us rely on some form of drug insurance to protect us from high drug costs, insurers are struggling with balancing coverage and premiums: A report by Express Scripts paints a grim picture:
An estimated 576,000 Americans spent more than the median household income on prescription medications in 2014. This population of patients grew an astounding 63% from 2013. Further, the population of patients with costs of $100,000 or more nearly tripled during the same time period, to nearly 140,000 people. The total cost impact to payers from both patient populations is an unsustainable $52 billion a year.
This isn’t just an issue in the United States. Prescription drug costs are climbing around the world, because we’re effectively all in this together: We all rely on private companies to bring new drugs to market, and we’re largely buying the same drugs from the same small group of companies. Because ready access to safe and effective prescription drugs is so important to the practice of medicine and the delivery of health care, the pharmaceutical industry is heavily regulated – not just by the FDA, but by regulators worldwide. Yet despite the dual requirements of regulatory disclosure and the financial obligation to be transparent (as many pharmaceutical companies are publicly-held), little is known about how much it costs to bring drugs to market, and how manufacturers arrive at their selling prices. Pharmaceutical manufacturers claim that high drug costs reflect the high costs of research and development (R&D), and provide the incentives for companies to invest heavily and take risks, when many drugs may never make it to market. Are they correct? (more…)
Nootropics are an emerging class of drugs that are designed to enhance cognitive function. They are part of a broader category of drugs known as performance and image enhancing drugs (PIED) which are used for enhancement of memory and cognition, sexual performance, athletic performance or musculature (also called “lifestyle” drugs).
It will probably come as no surprise to regular readers of SBM that nootropics and PIED are being abused and hyped without adequate evidence. One of the primary problems is that they are sold as supplements or as drugs, often over the internet without adequate regulation. One simple fix is to properly classify these drugs as drugs, and to properly regulate them as drugs.
Many of the cognition-enhancing “supplements” on the market make all the usual claims about “natural” enhancement – meanwhile they predictably contain just vitamins, herbs which have not been shown effective, perhaps nootropics (see below), and often a stimulant, like caffeine. The only drug in the mix which is likely to have a noticeable effect by the user is the stimulant.
A cycling enthusiast asked me about helmets. It seems compellingly obvious to me that a head impacting the pavement without a helmet is likely to sustain more damage than a head protected by a helmet. He challenged that, citing a BMJ article by Ben Goldacre that questioned whether the evidence showed that helmets do any good. He said I was making a non-evidence-based assumption and challenged me to actually look at the evidence, so I did.
Goldacre says there is a:
complex contradictory mess of evidence on the impact of bicycle helmets. Like most places where there’s controversy and disagreement, this is a great opportunity to walk through the benefits and shortcomings of different epidemiological techniques, from case control studies to modeling.
He proceeds to give a lesson in epidemiology. He points out that there are a lot of emotion involved, and that epidemiologic studies, because of their inherent imperfections, are probably not capable of resolving the debate.
There are basically two questions:
- What is the effect of wearing a helmet for the individual?
- What is the effect of a public policy that promotes or requires helmet use?
Last year, I did several posts on what I consider to be a profoundly misguided and potentially harmful type of law known as “right-to-try.” Beginning about a year and a half ago, promoted by the libertarian think tank known as the Goldwater Institute, right-to-try laws began popping up in state legislatures, which I likened to Dallas Buyers Club laws. Both Jann Bellamy and I wrote about how these laws are far more likely to do harm than good, and that is a position that I maintain today. The idea behind these laws is to give terminally ill patients access to experimental drugs—in some cases drugs that have only passed phase I testing—that might help them. It’s an understandable, albeit flawed argument. After all, it’s perfectly understandable why terminally ill patients would fight for drugs that give them hope, and it’s just as understandable why politicians and the public would see such a goal as a good thing. In practice, as I will explain again in the context of this update, such laws are far more likely to harm patients than help them. Indeed, as you will see, in the year since the first wave of right-to-try laws have passed, not a single patient that I can find has obtained access to experimental drugs under a right-to-try law, much less been helped by them.
Unfortunately, given how effectively “right to try” has been sold on grounds of providing terminally ill patients hope and as a matter of personal freedom, it’s clear that this wave is not going to abate. Since Colorado passed the very first right-to-try law almost exactly a year ago today, a total of 17 more states now have passed passed similar legislation, the most recent being Tennessee, and 22 others have introduced legislation. It’s a good bet that right-to-try will pass in all of those states, because, as I’ve explained many times before and in many interviews, if you don’t understand clinical trial ethics and science, opposing the concept of right-to-try comes across like opposing Mom, apple pie, and the American flag, and leaves opponents open to false—but seemingly convincing—charges of callousness towards the terminally ill on the order of enjoying drop kicking puppies through flaming goalposts.
The Lyme tick
As the saying goes, when you do not have the facts, argue the law. This tried and (?) true approach was successful in New York where a law was passed protecting those who are, shall we say, creative in treating patients with Lyme and ‘chronic’ Lyme.
The bill protects those from investigation of misconduct:
based solely on treatment that is not universally accepted by the medical profession.
The major bone of contention has been extended courses of IV antibiotics for ‘chronic’ Lyme, but there are a wide variety of treatments that may be used by so called “Lyme literate” doctors.
The number of therapies divorced from reality that are offered to Lyme patients is remarkable. I do not know if chronic Lyme patients have greater use of pseudo-medical therapies than other pseudo-diagnoses, but I am impressed by the offerings in a recent review:
Results. More than 30 alternative treatments were identified, which fell into several broad categories: these included oxygen and reactive oxygen therapy; energy and radiation-based therapies; nutritional therapy; chelation and heavy metal therapy; and biological and pharmacological therapies ranging from certain medications without recognized therapeutic effects on Borrelia burgdorgeri to stem cell transplantation. Review of the medical literature did not substantiate efficacy or, in most cases, any rationale for the advertised treatments.
Conclusions. Providers of alternative therapies commonly target patients who believe they have Lyme disease. The efficacy of these unconventional treatments for Lyme disease is not supported by scientific evidence, and in many cases they are potentially harmful.
In February, 2015, the American Chiropractic Association House of Delegates ratified “Six Key Elements of A Modern Chiropractic Practice Act.” For what it’s worth, this means that the “Six Elements” are part of the official “Public Policy” of the ACA.
1. “Chiropractic Physician” and “Chiropractic Medicine” as the Regulatory Terms of Licensure.
2. Scope of Practice Determined by Doctoral and Post-Doctoral Education, Training and Experience Obtained Through Appropriately Accredited Institutions.
3. Full Management, Referral and Prescription Authority commensurate with contemporary chiropractic education for Patient Examination, Diagnosis, Differential Diagnosis and Health Assessment.
4. Full Management, Referral and Prescription Authority commensurate with contemporary chiropractic education for the Care and Treatment of Neuromusculoskeletal and Other Health Conditions or Issues.
5. Full Authority for the Delivery of Information, Advice, Recommendations and Counseling Regarding General Health Matters, Wellness and Health Optimization.
6. Full Authority and Adaptable Requirements for the Management and Training of Health Care Teams and the Participation in Collaborative or Integrative Health Care Groups.
There are few home-runs in medicine. Most of our choices have some sort of trade-off – drugs have side effects, interventions have risks, and many treatments have marginal benefits. Sometimes, however, medical science hits one out of the park and develops a treatment that is safe, effective, cost effective, and convenient. Any dispassionate view of the evidence can only lead to one conclusion, leading to the absence of any legitimate scientific or medical debate.
I think the human papilloma virus (HPV) vaccine falls into this category. We have learned that many oral and genital cancers are caused by a sexually transmitted virus, HPV. Getting vaccinated against several strains of this virus prior to becoming sexually active effectively protects against infection by the virus, and dramatically reduces the risk of these cancers. Think about it – we can prevent cancer with a vaccine. This is a no-brainer.
A recent study shows:
HPV DNA was detected in 90.6% of cervical, 91.1% of anal, 75.0% of vaginal, 70.1% of oropharyngeal, 68.8% of vulvar, 63.3% of penile, 32.0% of oral cavity, and 20.9% of laryngeal cancers, as well as in 98.8% of cervical cancer in situ (CCIS). A vaccine targeting HPV 16/18 potentially prevents the majority of invasive cervical (66.2%), anal (79.4%), oropharyngeal (60.2%), and vaginal (55.1%) cancers, as well as many penile (47.9%), vulvar (48.6%) cancers: 24 858 cases annually. The 9-valent vaccine also targeting HPV 31/33/45/52/58 may prevent an additional 4.2% to 18.3% of cancers: 3944 cases annually.
The new 9-valent vaccine covers more strains. The study suggests that there are over 28,000 cases of cancer each year in the US that could be prevented by this vaccine.
Homeopathy is arguably the silliest form of alternative medicine: the published studies show no evidence of anything beyond nonspecific contextual effects, and the underlying premise is incompatible with the existing body of scientific knowledge. Homeopathy has increasingly been questioned or denounced by organizations in several countries, most recently in FDA hearings in the US.
I recently spoke at the QED conference (Question, Explore, Discover) in Manchester, England. Another speaker, Michael Marshall, gave a talk on homeopathy and the National Health Service. He presented information that was new to me and that I thought was worthy of sharing with SBM readers. (more…)