During Trump’s recent address to Congress he referred to 20 year-old Megan Crowley who has Pompe’s disease.
“Megan’s story is about the unbounded power of a father’s love for a daughter,” Trump said. “But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need.”
This statement, an unwarranted and factually-challenged attack on the FDA is all the more frightening when put into context. He has appointed to the FDA Jim O’Neill, who said in 2014:
“We should reform FDA so there is approving drugs after their sponsors have demonstrated safety—and let people start using them, at their own risk, but not much risk of safety. Let’s prove efficacy after they’ve been legalized.”
Not requiring evidence for efficacy for drugs would be an unmitigated disaster. It also makes not sense – the concept of “safety” cannot be entirely separated from efficacy. Drugs are evaluated and their use determined by risk vs benefit. You cannot do a risk vs benefit assessment if there is no data on benefit.
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An excellent article in ProPublica by David Epstein discusses the problem of doctors not adhering to the best evidence-based standards. The full article is worth a read, and I won’t just repeat it here, but I do want to highlight a few points which align well with what I have been writing here and at SBM for years.
The essential problem is that there is a disconnect between the best evidence-based standards and what is actually practiced out in the world. There are actually two problems here. The first is the scientific evidence itself. The second is the alignment of practice to this evidence.
Scientific evidence in medicine has a few challenges. There is publication bias, researcher bias, p-hacking, the decline effect, and problems with replication. What all of this adds up to is that there is a lot of published preliminary evidence, most of which is wrong in the false positive direction. There is a tendency, in my opinion, of adopting treatments prematurely.
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Researchers have published in PNAS promising results from a snail venom analogue used in the treatment of pain. This is exciting for a number of reasons, even if the current compounds under study do not pan out.
Pain is a difficult clinical problem. There are limited options for treating chronic pain and we can quickly run out of options if patients cannot tolerate certain classes of drugs. What we really need are entirely new classes of pain medication, and that is what this new approach promises.
There are essentially two neurological components to pain: there is the physical sensation, and then there is the emotional component. It is interesting to ask the question, why does pain hurt? There is nothing about the sensation itself that is inherently painful. Any sensation is just nerve cells firing and carrying signals to areas of the brain that interpret those signals. Pain hurts because pain pathways specifically connect to the emotional centers in the brain to create a negative experience.
For further background, clinically it is helpful to distinguish different types of pain. There is nociceptive pain, which is the nervous system appropriately sensing damage and generating protective painful sensations. There is also neuropathic pain, which is the nervous system malfunctioning and producing inappropriate pain that is not protective. We further divide pain into acute and chronic. Finally, we consider the context of the patient, such as whether or not they are terminal.
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The recent rapid development of CRISPR technology, which has made gene editing fast, affordable, and accurate, has rekindled the ethical debate about human gene editing. Last week a special panel put together by the National Academy of Sciences gave a “yellow light” to human germline gene editing – saying that such editing might be ethical once the risks were properly assessed.
Germline editing means that the changes would be part of the gametes, the sperm or egg, and would therefore be passed down to offspring. If gene editing were done to a fertilized then this would affect all cells, including the germ cells.
By contrast somatic cell editing would affect only adult cells and not be passed down to the next generation. Such editing would only affect the individual.
The ethical controversy over germline editing is that such changes essentially can become a permanent part of the human population.
What Changes are Acceptable?
The NAS report essentially lays out two criteria for human germline editing. The first is that research shows that such editing is safe in humans without any unintended consequences. They want to make sure that dangerous changes to the human genome will not enter the human population. This is, of course, a perfectly reasonable criterion.
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In a recent article for Slate, Jacob Grier argues that the science used to justify widespread bans on smoking in public places was flawed. Recent more robust research has show little to no health benefit from such laws, he argues. While he has a point regarding the arc of scientific evidence, I think he is going too far in the other direction in his conclusions about the science.
Second Hand Smoke
The current consensus of evidence is that there are health risks to second hand smoke, although they are statistically small. Debate centers around the magnitude of the effect, with few doubting that there is a negative health effect. Negative health effects include heart attacks, lung cancer, stroke, and exacerbation of asthma. On a population level, even small increased risks result in large numbers of excess deaths and negative health outcomes. The CDC estimates, for example, that second-hand smoke exposure results in 34,000 excess cardiac deaths each year.
Increased recognition of the health risks of passive smoke exposure lent significant political weight to anti-smoking efforts, resulting in a cultural shift over the last 30 years. As a result smoking has largely been banned in most indoor public places and many work places.
The empirical question on which Grier focuses is the impact of those smoking bans on health outcomes. He does a fairly thorough review of the literature, although I think his review is biased to make his point, that the health benefits of such bans have been overplayed and maybe don’t exist.
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We have an anti-vaccine president. One of my concerns about Trump the candidate was that one of his most consistent positions over the years was blaming vaccines for the alleged autism epidemic (there isn’t one, by the way). Once elected it did not take long for this to manifest as a policy priority. In January Trump met with RFK Jr. to discuss him heading an Orwellian commission on vaccine safety and scientific integrity.
At a recent meeting with educators, Trump continued to express his false belief in a “tremendous increase” in autism:
“Have you seen a big increase in the autism with the children?” Trump asked Jane Quenneville, the principle of a Virginia public school that specializes in special education. Quenneville responded that she had.
Trump continued: “So what’s going on with autism? When you look at the tremendous increase, it’s really such an incredible — it’s really a horrible thing to watch, the tremendous amount of increase. Do you have any idea?”
“The autism?” Really? Continue Reading »
Pew recently published a survey looking at the attitudes of Americans regarding the safety and effectiveness of vaccines. The results are not surprising, but there are some interesting bits in the data.
The headline main results are that 88% of those surveyed thought that the benefits of vaccines outweigh the risks, while only 10% thought the risks outweigh the benefits. Further, 82% supported mandatory vaccinations for healthy school children.
This is both good and bad news. It means a solid majority of Americans understand that vaccines are safe and effective. However, the minority who doubt the safety of vaccines are enough to cause problems. Also, those numbers are a bit worse when you dig into the data.
Parents of young children, age 0-4, were more negative about vaccines, with only 81% stating that the benefits outweigh the risks, compared to 91% of those with older children and 90% of those with no children. Parents of young children are the ones deciding if they get vaccinated. The reason for this is likely that parents with young children are facing the decision of whether or not to vaccinate and are looking for information online. They are therefore more likely to come across anti-vaccine propaganda. Parents of young children may also be easier to scare than more experienced parents.
These numbers are important because of herd immunity – if enough of the population is vaccinated, then a disease outbreak cannot find enough susceptible hosts to spread and will therefore peter out quickly. This will keep the disease from being endemic, meaning that it is self-sustaining in the population. Continue Reading »
Recently scientists published initial results from an ambitious project to reproduce the results of 50 influential cancer studies. The first five studies resulted in one clear failure to replicate, two partial replications, and two with uninterpretable results.
This is how science works. No one study is definitive, because there are simply too many ways to generate spurious results (even without fraud and with the best intentions). Replication is the final arbiter – any result that is real should consistently reproduce. Results that are spurious will be inconsistent.
These are the core lessons that I have been repeating here and on SBM – most studies are flawed and their results are unreliable. Most false studies are false positive. Even experienced and well-meaning researchers can fall victim to p-hacking and other subtle errors. You can only arrive at a reliable conclusion by looking at a mature and robust research program involving numerous studies and replications. The various replication projects that are under way are confirming this overall impression.
Let’s turn to one of my favorite examples: acupuncture. Acupuncture involved sticking thin needles into specific areas of the body in order to provoke specific clinical benefit, such as pain reduction. There have been several thousand studies of acupuncture. When you review all the research you find, put simply, that acupuncture does not work, for anything. There is no specific effect here, one that is reliably found when appropriately controlled for. The entirety of the research is highly consistent with the conclusion that acupuncture is nothing more than a theatrical placebo.
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A recent systematic review of the alleged health benefits of curcumin show that, yet again, hype based on “traditional use” is not a reliable guide.
Curcumin is a spice that makes up about 5% of turmeric, a yellow spice used in many curries. It is also a traditional herbal treatment. The health claims made for curcumin are numerous – WebMD has this entry:
Other preliminary lab studies suggest that curcumin or turmeric might protect against types of skin diseases, Alzheimer’s disease, colitis, stomach ulcers, and high cholesterol. Based on lab studies, turmeric and curcumin might also help treat upset stomach, scabies, diabetes, HIV, uveitis, and viral infections.
The systematic review had two main findings:
No double-blinded, placebo controlled clinical trial of curcumin has been successful. This manuscript reviews the essential medicinal chemistry of curcumin and provides evidence that curcumin is an unstable, reactive, nonbioavailable compound and, therefore, a highly improbable lead.
Let’s take the second point first, bioavailability. In order for a drug to be useful when taken orally it has to have adequate bioavailability. This means it needs to be relatively stable, it has to be absorbed in adequate amounts through the GI tract, and then it has to survive a first pass through the liver and be distributed in the body in such a way that it gets to its target tissue is sufficient concentration to have a clinical effect.
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There are several phases to a serious gaffe. First, there are the conditions which led to or allowed the gaffe to occur. Then there is the gaffe itself. There is then the response of the person or institution who committed the gaffe – which is perhaps the most important phase . Finally, there may or may not be a long term correction of the underlying conditions.
Over the weekend, as I discussed yesterday, Dr. Daniel Neides, Medical Director and Chief Operating Officer of the Cleveland Clinic Wellness Institute, published an anti-vaccine screed on cleveland.com. I won’t bother debunking the anti-vaccine pseudoscience again, just read my post from yesterday. You can also read David Gorski’s excellent take down at Science-Based Medicine.
Suffice it to say the column was full of misinformation and common anti-vaccine tropes that are long debunked. The publication of a dangerous anti-science rant by Cleveland Clinic’s Director of the Wellness Center was, of course, the gaffe.
The conditions that led to that gaffe are obvious, and something about which my SBM colleagues and I have complained for years- the infiltration of alternative medicine pseudoscience into academic medical institutions. Cleveland Clinic has an “integrative medicine” center headed by Mark Hyman, who co-authored an anti-vaccine book. The Wellness Center itself embraces all sorts of vitalistic nonsense and pseudoscience.
As David says – no one should be surprised when the director of a center that embraces quackery publishes an article that embraces quackery. Continue Reading »