I recently participated in a debate opposing me to Professor Adam Cifu on the topic of “Evidence-based medicine in the age of COVID.” The debate took place on an episode of Dr. Chadi Nabhan’s Outspoken Oncology podcast. Dr. Saurabh Jha was the moderator and he did a great job keeping us on point and asking for important clarifications when needed. It was a fun and cordial moment and I found it intellectually fruitful. You can listen to it here or on any podcast platform. The discussion strengthened my conviction that the central issue about EBM is the conflation of the role of the physician with that of the clinical scientist.
That conflation was quite apparent in a recent online editorial published by Robert Yeh and colleagues on the topic of equipoise during the COVID-19 pandemic. Yeh at al. are accomplished academic cardiologists and outcomes researchers (Yeh was a guest on The Accad and Koka Report a couple of years ago).
I’ll get to their editorial in a moment, but equipoise is a term that I became aware of only in the last few years, mainly from mentions on MedTwitter. From those mentions I developed an intuitive sense of what equipoise must mean: a mental state of uncertainty about a treatment that prompts the medical community to seek a more definitive answer by way of a randomized controlled trial. For example, one might say “I’m not sure if hydroxychloroquine works to prevent or treat COVID-19. Based on the existing collective experience, there is equipoise about it. We need a clinical trial.”
That seems reasonably straightforward, but the editorial by Yeh et al. piqued my curiosity so I decided to look into the origin of the term and its introduction in the medical literature.
“The goal for me and for my clinical and research colleagues is to put ourselves out of a job as quickly as possible”. This is how Mikkael Sekeres ends his book “When Blood Breaks Down” based on true stories of patients with leukemia. I share Mikkael’s sentiments and have always stated that I’d be happy if I am out of a job caring for patients with cancer. To his and my disappointment, this wish is unlikely to ever come true, especially when dealing with leukemia.
With almost 15 years of experience, Sekeres possesses a wealth of knowledge and patient stories making him the ultimate storyteller taking us along an emotional journey that spanned hospital rooms, outpatient clinics, and even his car. We get to know Mikkael the person and the doctor and immediately recognize how difficult it is to separate these two from each other. With hundreds of patients he has cared for, Mikkael could choose which stories to share. He decides on 3 patients, each with a unique type of leukemia and a set of circumstances that makes their story distinct. While I don’t know for certain, his selection likely reflected his ultimate goal of writing this book. It was about sharing life lessons he had learned from his patients–lessons that we could similarly learn—but it was also about giving us a glimpse of history in medicine and the progress that has been made in treating leukemia.
We get to know the three main characters of the book very well. David is an older man with acute myeloid leukemia (AML), Joan is surgical nurse who suddenly finds herself diagnosed with acute promyelocytic leukemia (APL), and Mrs Badway is a pregnant woman who was in her 2nd trimester when she was diagnosed with chronic myeloid leukemia (CML). While learning about their illnesses and family dynamics, Sekeres educates us about the various types of leukemia and enlightens his readers about so much history that I found fascinating. I did not know that the Jamshidi needle that I have used on so many patients to aspirate their bone marrows was invented by an Iranian scientist. Maybe I should have known, but I didn’t, that FISH was developed at Yale in 1980 and the first description of leukemia has been attributed to a French surgical anatomist, Dr. Alfred Velpeau in 1827. Somehow, I always thought that Janet Rowley discovered the Philadelphia chromosome, but Sekeres corrects me when he pictured Peter Nowell and David Hungerford who discovered that chromosome in 1961. As a reader, you might be more drawn to the actual patient stories, but the geek in me enjoyed the history lessons, especially the ones I was unaware of. Sekeres inserts these pearls effortlessly and with perfect timing. He does that so seamlessly and naturally that you learn without realizing you are being taught.
There have been disturbing reports of hospitals firing doctors and nurses for speaking up about inadequate PPE. The most famous case was at the PeaceHealth St. Joseph hospital in Washington, where Dr. Ming Lin was let go from his position as an ER physician after he used social media to publicize suggestions for protecting patients and staff. At Northwestern Memorial Hospital in Chicago, a nurse, Lauri Mazurkiewicz warned colleagues that the hospital’s standard face masks were not safe and brought her own N95 mask. She was fired by the hospital. These examples violate a culture of safety and endanger the lives of both patients and staff. Measures that prevent healthcare workers from speaking out to protect themselves and their patients violate safety culture. Healthcare workers should be expected to voice their safety concerns, and hospital executives should be actively seeking feedback from frontline healthcare workers on how to improve their institution’s Covid-19 response.
Share power with frontline workers
According to the Institute for Healthcare Improvement, it is common for organizations facing a crisis to assume a power grab in order to maintain control. As such, it is not surprising that some hospitals are implementing draconian policies to prevent hospital staff from speaking out. While strong leadership is important in a crisis, it must be balanced by sharing and even ceding power to frontline workers. All hospitals want to provide a safe environment for their staff and high-quality care for their patients. However, in a public health emergency where resources are scarce and guidelines change daily, it’s important that hospitals have a systematic approach to keep up.
By PRANAV PURI, PUNEET KAUR, and MARCUS WIGGINS, MBA
As current medical students, the ongoing COVID-19 pandemic represents the most significant healthcare crisis of our lifetimes. COVID-19 has upended nearly every element of healthcare in the United States, including medical education. The pandemic has exposed shortcomings in healthcare delivery ranging from the care of nursing home residents to the lack of interoperable health data. However, the pandemic has also exposed shortcomings in the residency match process.
Consider the United States Medical Licensing Examination (USMLE) Step 1. A 2018 survey of residency program directors cited USMLE Step 1 scores as the most important factor in selecting candidates to interview. Moreover, program directors frequently apply numerical Step 1 score cutoffs to screen applicants for interviews. As such, there are marked variations in mean Step 1 scores across clinical specialties. For example, in 2018, US medical graduates who matched into neurosurgery had a mean Step 1 scores of 245, while those matching into neurology had a mean Step 1 score of 231.
One would assume that, at a minimum, Step 1 scores are a standardized, objective measure to statistically distinguish applicants. Unfortunately, this does not hold true. In its score interpretation guidelines, the National Board of Medical Examiners (NBME) provides Step 1’s standard error of difference (SED) as an index to determine whether the difference between two scores is statistically meaningful. The NBME reports a SED of 8 for Step 1. Assuming Step 1 scores are normally distributed, the 95% confidence interval of a Step 1 score can thus be estimated as the score plus or minus 1.96 times the standard error (Figure 1). For example, consider Student A who is interested in pursuing neurosurgery and scores 231. The 95% confidence interval of this score would span from 215 to 247. Now consider Student B who is also interested in neurosurgery and scores 245. The 95% interval of this score would span from 229 to 261. The confidence intervals of these two scores clearly overlap, and therefore, there is no statistically significant difference between Student A and Student B’s exam performance. If these exam scores represented the results of a clinical trial, we would describe the results as null and dismiss the difference in scores as mere chance.
With the exceptions of pediatrics and obstetrics/gynecology, women make up fewer than half of all medical specialists. Representation is lowest in orthopedics (8%), followed by my own specialty, urology (12%). I can testify that the numbers are changing in urology – women are up from just 8% in 2015, and the breakdown in our residency program here at Indiana University is now about 20% of the 5-year program.
One reason for the increase is likely the growth of women in medicine – 60% of doctors under 35 are women, as are more than half of medical school enrollees. I also credit a generational shift in attitudes. The female residents I work with do not anticipate hostility from men in the profession and they expect male patients to give them a fair shake. They may be right – their male contemporaries are more egalitarian than mine – but challenges still exist in our field.
Urologists see both men and women, but the majority of patients are male. Urology focuses on many conditions that only affect men such as enlarged prostate, prostate cancer, and penile cancer. Furthermore, stone disease is more common in men, as are many urologic cancers such as bladder cancer and kidney cancer. So the greatest challenge for young women in urology is to gain acceptance among older men who require examination of their genital region and often need surgery. I’m hopeful that women entering urology today can meet that challenge, largely because we have already made significant progress. For the barriers we still face, leading urologists have blazed a clear path to follow with these three guideposts.
If you think the grim coronavirus death toll is causing health care workers everywhere to always wash their hands, think again.
A recent research letter published in The Journal of Hospital Infection examined whether it’s “possible to achieve 100 percent hand hygiene compliance during the Covid-19 pandemic.” The medical center involved in the research, Queen Mary Hospital in Hong Kong, had reached a pre-Covid-19 hand hygiene rate of over 75 percent.
Yet the hospital’s goal of complete compliance proved surprisingly elusive. In one pediatric ward devoted to suspected or confirmed Covid-19 patients, doctors and nurses followed hand hygiene rules 100 percent of the time, but in another ward with similar patients and staff, compliance was 83 percent, or about one-fifth less.
Given Covid-19’s risk to providers as well as patients, this was “unexpected,” the researchers admitted.
The Queen Mary study supports what infection control experts have long maintained: awareness isn’t enough. Doctors and nurses, particularly during a pandemic, understand that hand hygiene is “the most important intervention” to reduce the staggering death toll from infections, as the American Journal of Infection Controlput it.
The Smithsonian National Museum of Natural History has reported its biggest number of visitors in more than 2 ½ years. There’s a string of new Broadway musicals that are well-attended every night. It’s safe to shop in malls, eat out in restaurants and go to movie theaters again.
Of course, this has all been made possible by an effective vaccine against COVID-19 that was widely administered in the fall of 2021. Vaccinated citizens of the world are now confident that it’s safe to go out in public, albeit with appropriate precautions.
However, U.S. residents who have health problems are facing a new challenge. Five years ago, in 2017, the median wait time of new patients for doctor appointments was six days. In 2022, the wait time is six months or more.
The reason for this is no mystery. While life has started to return to what we think of as the new normal, the U.S. healthcare system has taken an enormous financial hit, and primary care practices have been especially affected. Many primary care physicians have closed their practices and have retired or gone on to other careers. Consequently, the shortage of primary care has been exacerbated, and access to doctors has plummeted. Urgent care centers, retail clinics and telehealth have not filled this gap.
Because of the long waiting times for primary care appointments, many more people now seek care in emergency departments (EDs). The waiting rooms of these EDs are overcrowded with people who have all types of complaints, including chronic and routine problems as well as emergencies. And this is not just a common sight in inner-city areas, as it once was; it’s now the same pretty much everywhere.
Last month marked the 400th anniversary of the birth of John Graunt, commonly regarded as the father of epidemiology. His major published work, Natural and Political Observations Made upon the Bills of Mortality, called attention to the death statistics published weekly in London beginning in the late 16th century. Graunt was skeptical of how causes of death were ascribed, especially in times of plagues. Evidently, 400 years of scientific advances have done little to lessen his doubts!
A few days ago, Fox News reported that Colorado governor Jared Polis had “pushed back against recent coronavirus death counts, including those conducted by the Centers for Disease Control and Prevention.” The Centennial State had previously reported a COVID death count of 1,150 but then revised that number down to 878. That is but one of many reports raising questions about what counts as a COVID case or a COVID death. Beyond the raw numbers, many controversies also rage about derivative statistics such as “case fatality rates” and “infection fatality rates,” not just among the general public but between academics as well.
Of course, a large part of the wrangling is due not only to our unfamiliarity with this new disease but also to profound disagreements about how epidemics should be confronted. I don’t want to get into the weeds of those disputes here. Instead, I’d like to call attention to another problem, namely, the somewhat confused way in which we think about medical diagnosis in general, not just COVID diagnoses.
The way I see it, there are two concepts at play in how physicians view diagnoses and think about them in relation to medical practice. These two concepts—one more in line with the traditional role of the physician, the other adapted to modern healthcare demands—are at odds with one another even though they both shape the cognitive framework of doctors.
Something didn’t seem right to epidemiologist Eric Weinhandl when he glanced at an article published in the venerated Journal of the American Medical Association (JAMA) on a crisp fall evening in Minnesota. Eric is a smart guy – a native Minnesotan and a math major who fell in love with clinical quantitative database-driven research because he happened to work with a nephrologist early in his training. After finishing his doctorate in epidemiology, he cut his teeth working with the Chronic Disease Research Group, a division of the Hennepin Healthcare Research Institute that has held The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) contract for the United States Renal Data System Coordinating Center. The research group Eric worked for from 2004-2015 essentially organized the data generated from almost every dialysis patient in the United States. He didn’t just work with the data as an end-user, he helped maintain the largest, and most important database on chronic kidney disease in the United States.
For all these reasons this particular study published in JAMA that sought to examine the association between dialysis facility ownership and access to kidney transplantation piqued Eric’s interest. The provocative hypothesis is that for-profit dialysis centers are financially motivated to keep patients hooked to dialysis machines rather than refer them for kidney transplantation. A number of observational trials have tracked better outcomes in not-for-profit settings, so the theory wasn’t implausible, but mulling over the results more carefully, Eric noticed how large the effect sizes reported in the paper were. Specifically, the hazard ratios for for-profit vs. non-profit were 0.36 for being put on a waiting list, 0.5 for receiving a living donor kidney transplant, 0.44 for receiving a deceased donor kidney transplant. This roughly translates to patients being one-half to one-third as likely to get referred for and ultimately receiving a transplant. These are incredible numbers when you consider it can be major news when a study reports a hazard ratio of 0.9. Part of the reason one doesn’t usually see hazard ratios that are this large is because that signals an effect size that’s so obvious to the naked eye that it doesn’t require a trial. There’s a reason there are no trials on the utility of cauterizing an artery to stop bleeding during surgery.
But it really wasn’t the hazard ratios that first struck his eye. What stuck out were the reported event rates in the study. 1.9 million incident end-stage kidney disease patients in 17 years made sense. The exclusion of 90,000 patients who were wait-listed or received a kidney transplant before ever getting on dialysis, and 250,000 patients for not having any dialysis facility information left ~1.5 million patients for the primary analysis. The original paper listed 121,000 first wait-list events, 23,000 living donor transplants and ~50,000 deceased donor transplants. But the United Network for Organ Sharing (UNOS), an organization that manages the US organ transplantation system, reported 280,000 transplants during the same period.
The paper somehow was missing almost 210,000 transplants.
From the vantage point of our self-quarantined shrunken universes, we cannot see even the immediate future, let alone what our personal and professional lives will look like some years from now.
Factories are closed, luxury department stores are in bankruptcy, hospitals have stopped performing elective procedures and patients are having their heart attacks at home, unattended by medical professionals. New York office workers may continue to work from home while skyscrapers stand empty and city tax revenues evaporate.
Quarantined and furloughed families are planting gardens and cooking at home. Affluent families are doing their own house cleaning and older retirees are turning their future planning away from aggregated senior housing and assisted living facilities.
In healthcare, procedure performing providers who were at the pinnacle of the pecking order sit idle while previously less-valued cognitive clinicians are continuing to serve their patients remotely, bringing in revenues that prop up hospitals and group practices.