Some medical care may not help. Alternatives may not either.

‘Why Doctors Still Offer Treatments That May Not Help’, by Austin Frakt in the New York Times, August 27, 2019, is a well-done article that, despite its title, is fairly optimistic. He reports that while about 50% of current medical treatments no not have good evidence supporting them, only 3% have evidence showing them to be harmful, and another 6% unlikely to be helpful. This means about 41% (or only 41%, if you prefer) have good evidence that they are helpful.

Frakt cites some of the research on health interventions that do not work, or may even harm, quoting leading researchers Vinay Prasad and Adam Cifu (although he does not mention John Ionannidis, arguably the father of the field). He discusses the important “Choosing Wisely” campaign, initiated by the American Board of Internal Medicine Foundation, that asks each medical specialty society to identify at least 5 things often done in their specialty which usually should not be done. There is not, however, always consensus. For example, among its 20 recommendations (a high number that continues to grow, perhaps because of its discipline’s broad scope), the American Academy of Family Physicians (AAFP) recommends against imaging (including MRI and CT) for uncomplicated back pain, as does the American College of Physicians (Internal Medicine) (see ‘Medicine should not be primarily a business: choosing appropriate care for all, not excess testing for some, August 19, 2018).  Yet, neither the American Academy of Orthopaedic Surgeons, or the various groups of radiologists who perform these procedures, make this recommendation. Of course, both stand to benefit financially from doing these procedures.

It is concerning to think that, at least in some cases, financial benefit may influence the selection of some medical societies’ “Choosing Wisely” recommendations. Sadly, it is not a surprise, and financial benefit is, I believe, the reason for the use of many unproven treatments. Another important reason is an understandable reluctance for providers to abandon therapies that they have used for many years and believe to be successful. After all, if 50% of treatments do not have good evidence that they help or harm, many probably often do help, even if most of the evidence is anecdotal. Much more malicious, however, is the intensive marketing of new drugs and therapies to physicians by drug and device makers with a strong financial profit motive.

Frakt provides a litany of therapies-since-found-not-to-work-or-even-do-harm, including hormone replacement for post-menopausal women. The only drug he specifically names is Vioxx®, the “miracle” anti-inflammatory pulled from the market when it was found to cause heart disease (at least publicly found; the manufacturer actually knew it before it was released!). He also mentions “tight blood sugar control in critically ill patients” (and actually, probably most patients). This certainly relates to drug. The plethora of new, expensive, and marginally advantageous diabetes drugs is a testimony to their profitability. Ironically, it is often the same physicians who are unwilling to give up on treatments that are unproven or even proven to be of no benefit or of harm who are the first to begin using new (and, redundantly) more expensive drugs heavily promoted by the manufacturers.

Of course, it is not just diabetes drugs that are expensive. Compared to some of the newer drugs for rare diseases they seem like a bargain. In “The $6 million drug claim”, Times writers Katie Thomas and Reed Abelson discuss a woman who has $1 million in drugs to treat her condition in her refrigerator! While in her case they were paid for by her husband’s union, the cost can still be backbreaking: “At one point in 2018, for every hour that one of the union’s 16,000 members worked, 35 cents of his or her pay went to Alexion to cover the Pattersons’ prescriptions.” And what about those who have poor insurance, or no insurance? Or a union that goes bankrupt paying for them? Alexion, of course, does well, though.

A new law allows “gravely ill” patients the “right to try” drugs that the FDA has not approved. It sounds good; as a friend told me “I figure if I’m going out and they want to try something that might kill me I haven’t got much to lose. At best I’m cured. At worse, I go out but knowledge is gained.” Except for maybe dying sooner or more painfully, or possibly going bankrupt. It is not uncommon for doctors to suggest something new, unproven, or even a treatment that has already failed, one more time. Not everyone in this country has good insurance, and if your insurance company appropriately refuses to pay because there is no evidence of efficacy (yes, they are sometimes right!), you are on the hook for the bill. You may be dying, but your family may need the money you were saving for retirement. Thus, there may be a great deal to lose.

A certain percentage of people will take all this to mean that mainstream medicine is not to be trusted. This is definitely an overreaction, although a healthy skepticism is warranted, especially when the treatment is incredibly expensive and it is obvious that somebody (or some corporation) is making a killing on it. Some people will decide to pursue only what used to be called “alternative” medicine. These practices, often with hundreds or thousands of years of experience, are often called “complementary” and are now frequently offered along with mainstream medical therapies in “integrative” practices. But the same cautions must apply; because something is different, or traditional, does not make it necessarily either effective or safe.

For example, while people sometimes believe it is safer to choose only “natural” medicine, because a therapy grows in nature does not mean it is necessarily safe. Earlier in my career, medicine virtually abandoned the use of digitalis, made from the natural plant foxglove, to treat heart disease, after it had been used for decades. The key point is that if a substance works biologically and has “good” (i.e., desired) effects it can also have “bad” (i.e., undesired, or “side”, effects). This is true if it is straight from the plant, modified and standardized, or completely created in the laboratory.

Another real danger is what Frakt calls “wishful thinking”. Being optimistic and hoping that things will turn out well is good, and often useful when confronting serious illness. But when this transitions into the realm of “magical thinking”, being convinced that something good will happen to you because you want it to (or vice versa, that something bad will not happen because you don’t want it to), or believing a treatment will work because it is “natural”, or because your doctor recommended it in the absence of evidence, it is a real risk. “Magical thinking” is normal in three year olds, but dangerous in adults.

So what to do? Be open to new treatments, but do not reject the old, whether “traditional” or medical. Ask for evidence for treatment, such as when just changing a drug from an inexpensive standard (say, metformin for diabetes) to a new, costly one. Learn to understand probability, and ask for numbers. Do not reject anything out of hand, but do not believe that something will work just because you want it to.

Stay skeptical but not intransigent. Look for the evidence. And look also, when something is expensive, for the profit motive. Cui bono? It may not always be you.