We trust modern medicine to be evidence-based: grounded in the most current and reliable research available. But sometimes medicine gets it wrong, and clinicians engage in unproven, ineffective, expensive, and even harmful practices. Worse, these practices can become entrenched as standard protocols.
Good intentions
Clinicians are not immune from hope. And hope, bolstered by logic, intuition, and bio-plausibility—the idea that a practice aligns with known science—has occasionally led clinicians to accept new therapies in the honest expectation that they will be proven in time.
An example was the rush to embrace the use of corticosteroids for spinal cord injuries in the 1990s.
Approximately 18,000 Americans suffer a spinal cord injury each year. Depending upon the nature and location of the injury, patients may incur paraplegia or quadriplegia, injuries with tragic life limiting impacts that we see around us all too often.
A 1990 study of patients with acute spinal-cord injury found that those who received the corticosteroid methylprednisolone within eight hours of their injury scored significantly better on measures of motor function and sensation at six weeks and six months post injury than patients receiving a placebo.
Hopeful that subsequent research would support the first study, physicians adopted the use of methylprednisolone after traumatic spinal cord injuries in an attempt to prevent or limit paralysis.
Writing about Christopher Reeve in the New Yorker in 2003, Jerome Groopman, M.D., stated without equivocation that the 1990 study had shown “that a prompt dose of methylprednisolone could limit inflammation and spinal-cord damage.”
Subsequent studies did not bear this out. The MRC CRASH Study published in 2005 found “no evidence that the effect of corticosteroids differed by injury severity or time since injury.”
In 2011, the Mayo Clinic posted a video citing increased morbidity and “no true demonstrable benefit” from corticosteroids in its decision to remove them from Mayo’s protocols for spinal cord injuries. By 2013, the American Association of Neurological Surgeons’ Guidelines for the Management of Acute Cervical Spine and Spinal Cord Injuries recommended against the routine administration of methylprednisolone.
When doctors know best
Vinay Prasad, M.D., MPH, and Adam Cifu, M.D., have coined the term “medical reversal” to describe the “the phenomenon of a new trial — superior to predecessors because of better design, increased power, or more appropriate controls — contradicting current clinical practice.” For Prasad and Cifu, there is an important difference between the “the natural progression of supplanting old technologies with new ones and reversal in which a regularly used medicine, procedure, or device is identified as being ineffective or even harmful.”
The greatest number of reversals identified by Prasad and Cifu were specific to cardiovascular disease. One of the most famous of these concerns the use of percutaneous coronary intervention (PCI) or stenting in patients with chronic stable coronary artery disease (CAD).
By the end of the last century, PCI had come to be considered first-line therapy for patients with CAD. The procedure’s benefits and efficacy seemed evidenced by the fact that patients reported reduced angina pain after receiving stents. It was assumed that a procedure which opened a narrowed artery would lead to decreased mortality from heart attack, that improvement in anginal symptoms was a good surrogate for overall benefit.
The Clinical Outcomes Utilizing Revascularization and Aggressive Drug Evaluation study, (also known as the COURAGE Trial) study published in New England Journal of Medicine in 2007, showed that PCI in patients with chronic stable CAD did not decrease death rates compared to optimal medical management of atherosclerotic vascular disease. While stents were effective at relieving pain caused by blockages in arteries, a heart attack was as likely to result from the rupture of plaque in a less occluded artery.
Remarkably, the COURAGE Trial did not result in a marked reduction in the use of PCI. Rather, there was a continued increase in non-surgical coronary interventions in the years immediately following its publication. Many cardiologists held steadfast in their belief in the value of stents, confident that longer-term studies would support their assumptions.
In fact, subsequent studies supported the COURAGE Trial.
While inappropriate use of stents has declined in light of this mounting evidence, as recently as 2016, datafrom four American College of Cardiology National Cardiovascular Data Registries suggested that up to 50 percent of stent placements in stable patients may have been inappropriate.
Patient expectations
Patient demand can drive the continued use of disproven procedures, particularly when there are concurrent financial incentives for providers.
Arthroscopic partial meniscectomy (APM) is one of the most common orthopedic surgeries performed in the United States. But even though 750,000 patients undergo the procedure annually at a cost of up to $10,000, APM has not been shown to be a more effective treatment option than nonoperative approaches.
A study published in the NEJM in 2013 found that the outcomes for patients who had standardized physical therapy did not differ significantly from patients who had APM with postoperative physical therapy.
This was supported by a study published in JAMA in 2022. Yet, even as recently as 2021 the American Academy of Orthopedic Surgeons maintained that APM was appropriate for the treatment of meniscal tears in patients with concomitant mild to moderate osteoarthritis who have failed physical therapy or other non-surgical treatments.
And patients have come to expect APM as an option, motivated, it might be supposed, by advertising which would have them believe that they are only one procedure away from returning to the tennis court or bike path.
Deliberate misrepresentation
Regrettably, some medical practices have been adopted as the result of misrepresented or falsified data.
One of the most egregious examples of collective medical mistake was the manner in which the medical community was manipulated into changing its perception of opioids in the 1990’s. A sincere and overdue conversation about untreated pain by trusted thought leaders was hijacked by profiteers, with deadly results.
Until the 1980’s, physicians had been particularly circumspect in their use of opioids for pain management, recognizing that the drugs posed a very real risk of addiction. As a result, the moderate to severe painexperienced by at least one third of cancer patients was often poorly managed, if managed at all. In response to what it recognized as an international health problem, World Health Organization issued revised guidelinesfor the management of cancer pain in 1986, opening the door for a new conversation about the use of opioids.
But the conversation was commandeered.
In The Tragedy of Needless Pain published in Scientific American in February 1990, Ronald Melzack argued that morphine taken solely to control pain was not addictive, asserting that “Addiction seems to arise in only some fraction of users who take the drug for its psychological effects.” As evidence, he cited a “study” by Jane Porter and
Hershel Jick, M.D. published in the New England Journal of Medicine in which only four of 11,882 patients using narcotic drugs in a hospital setting became addicted.
In 1995, James Campbell, president of the American Pain Society, proposed that pain be counted as the fifth vital sign. By 2001, the Joint Commission on the Accreditation of Healthcare Organizations (JCAHO, now The Joint Commission) had added pain assessment for all patient visits as one of its standards.
The timing was nearly perfect for Purdue Pharma’s introduction of Oxycontin, its patented Schedule II sustained-release oxycodone formulation in 1995.
Unlike other drugs in its category, Oxycontin only needed to be taken every 12 hours, not every four. But what really set it apart from its competitors was that Oxycontin debuted on the market with a unique label from the FDA reading, “Delayed absorption as provided by Oxycontin tablets is believed to reduce the abuse liability of a drug.”
For physicians balancing new responsibilities for pain management against longstanding wariness of opioids, Oxycontin seemed the perfect solution. According to the Government Accountability Office, by 2003, “nearly half of all OxyContin prescribers were primary care physicians.”
Regrettably, much of the data in which physicians had placed their trust wasn’t true or had been incorrectly represented. The Porter Jick study had been little more than a letter to the editor, not a randomized controlled trial.
The FDA had been misled in granting the special label, and Purdue Pharma had not shared feedback from clinicians and communities regarding the impact of the drug. According to the GAO, the drug’s safety warning “advising patients not to crush the tablets because of the possible rapid release of a potentially toxic amount of oxycodone may have inadvertently alerted abusers to methods for abuse.”
America’s opioid epidemic has taken hundreds of thousands lives, shattered families and communities and is estimated to cost over $1.5 trillion annually.
Avoiding and eliminating ineffective treatments
There is a growing movement to identify and eliminate ineffective medical treatments.
In January 2010, the New England Journal of Medicine published a Perspective piece by Howard Brody, M.D., Ph.D., in which the bioethicist and physician argued that “U.S. physicians could do a great deal to control costs if they were willing to practice more in accordance with evidence-based guidelines.”
Brody called upon fellow clinicians to identify the top five diagnostic tests or treatments within each medical specialty which were “among the most expensive services provided, and that have been shown by the currently available evidence not to provide any meaningful benefit to at least some major categories of patients for whom they are commonly ordered.”
Meant to engage physicians in the effort to reduce healthcare costs, Brody’s essay became the impetus for the National Internal Medicine Foundation’s Choosing Wisely initiative. The Choosing Wisely website offers advice for physicians and patients in identifying and choosing care that is evidence-based and cost effective.
Change can be difficult. And clinicians are not immune from the psychological discomfort of cognitive dissonance, which can force individuals to double down on their beliefs when confronted with contradicting evidence.
In an interview with PBS Newshour, one physician reassessing what he had been taught in his medical training observed that, “Well, first you have to have some humility. That’s hard right there. And then one has to change… if you have science that backs you up then you change.”