The gold standard of medical research, the randomized controlled trial, has been taking a bit of a beating lately. An entire issue of the journal Social Science and Medicine was recently devoted to it, with many articles pointing to shortcomings. Others have argued that randomized controlled trials often can’t address the questions that patients and physicians most want answered. But the randomized controlled trial remains a powerful tool. It’s still, perhaps, the best method for conducting explanatory research.
Promising health studies often don’t pan out in reality. The reasons are many. Research participants are usually different from general patients; their treatment doesn’t match real-world practice; researchers can devote resources not available in most physician offices.
Moreover, most studies, even the gold standard of randomized controlled trials, focus squarely on causality. They are set up to see if a treatment will work in optimal conditions, what scientists call efficacy. They’re “explanatory.”
Efficacy is important. But what we also need are studies that test if a treatment will work in the real world — if they have effectiveness. These different kinds of studies actually exist. They are called pragmatic trials, and a recent one might have helped serve as a brake as the opioid epidemic accelerated. That’s the topic of this week’s HCT.
The following originally appeared on The Upshot (copyright 2018, The New York Times Company). It also appeared on page B2 of the September 18, 2018 print edition. Research for this piece was supported by the Laura and John Arnold Foundation.
Among the standard complaints about the American health care system is that care is expensive and wasteful. These two problems are related, and to address them, Medicare has new ways to pay for care.
Until recently, Medicare paid for each health care service and reimbursed each health care organization separately. It didn’t matter if tests were duplicated or if a more efficient way of delivering care was available — as long as doctors and organizations were paid for what they did, they just kept providing care the way they always had.
But ordinary people do not think this way. We focus on solving our health problem, not which — or how many — discrete health care services might address it. New Medicare programs are devised to more closely align how care is paid for with what we want that care to achieve.
One of these programs is known as bundled payments. Instead of paying separately for every health care service associated with a medical event, you pay (or Medicare pays, in this case) one price for the entire episode. If health care providers can address the problem for less, they keep the difference, or some of it. If they spend more, they lose money. Bundled payment programs vary, but some also include penalties for poor quality or bonuses for good quality.
Medicare has several bundled payment programs for hip and knee replacements — the most common type of Medicare procedures — and associated care that takes place within 90 days. This includes the operation itself, as well as follow-up rehabilitation (also known as post-acute care). In theory, if doctors and hospitals get one payment encompassing all this, they will better coordinate their efforts to limit waste and keep costs down.
Do bundled payments work? They certainly appear promising, at least for some treatments. But it’s important to conduct rigorous evaluations.
Previous studies for Medicare by the Lewin Group and other researcherssuggest that Medicare’s Bundled Payments for Care Improvement program has reduced the amount Medicare pays for each hip and knee replacement.
But that doesn’t mean the program saved money over all.
One possible issue would be if, despite saving money per procedure, health care providers wastefully increased the number of procedures — replacing hips and knees that they might not otherwise. A related concern is if hospitals try to increase profits by nudging services toward patients who may not need a procedure as much as patients with more severe and more expensive conditions. An average joint replacement costs $26,000, split almost equally between the initial procedure and post-acute care. But more expensive cases can be $75,000 to $125,000 — a costly proposition for hospitals.
A recent study published in JAMA examined whether the volume of Medicare-financed hip and knee replacements changed in the markets served by hospitals that volunteered for a bundled payments program, relative to markets with no hospitals joining the program. It found no evidence that the bundled payment program increased hip and knee replacement volume, and it found almost no evidence that hospitals skewed their services toward patients whose procedures cost less.
“These results suggest bundled payments are a win-win,” said Ezekiel Emanuel, a co-author of the study. “They save payers like Medicare money and encourage hospitals and physicians to be more efficient in the delivery of care.”
But Robert Berenson, a fellow at the Urban Institute, urges some caution. “Studying one kind of procedure doesn’t tell you much about the rest of health care,” he said. “A lot of health care is not like knee and hip replacements.”
Michael Chernew, a Harvard health economist, agreed. “Bundles can certainly be a helpful tool in fostering greater efficiency in our health care system,” he said. “But the findings for hip and knee replacements may not generalize to other types of care.”
Christine Yee, a health economist with the Partnered Evidence-Based Policy Resource Center at the Boston Veterans Affairs Healthcare System, has studied Medicare’s previous efforts and summarized studies about them. (I and several others were also involved in compiling that summary.) “Medicare has tried bundled payments in one form or another for more than three decades,” Ms. Yee said. “They tend to save money, and when post-acute care is included in the bundle, use of those kinds of services often goes down.”
One limitation shared by all of these studies is that they are voluntary: No hospital is required to participate. Nor are they randomized into the new payment system (treatment) or business as usual (control). Therefore we can’t be certain that apparent savings are real. Maybe hospitals that joined the bundled payment programs are more efficient (or can more easily become so) than the ones that didn’t.
Another new study in JAMA examines a mandatory, randomized trial of bundled payments. On April 1, 2016, Medicare randomly assigned 75 markets to be subject to bundled payments for knee and hip replacements and 121 markets to business as usual. This policy experiment, known as the Comprehensive Care for Joint Replacement program, will continue for five years. The JAMA study analyzed just the first year of data.
“In this first look at the data, we examined post-acute care because it is an area where there is concern about overuse,” said Amy Finkelstein, an M.I.T. health economist and an author of the study. “In addition, prior work suggested that it’s a type of care that hospitals can often avoid.”
The study found that bundled payments reduced the use of post-acute care by about 3 percent, which is less than what prior studies found. “Those prior studies weren’t randomized trials, so some of the savings they estimate may really be due to which hospitals chose to participate in bundled payment programs,” Ms. Finkelstein said. Despite reduced post-acute care use, the study did not find savings to Medicare once the costs of paying out bonuses were factored in. The study also found no evidence of harm to health care quality, no increase in the volume of hip and knee replacements, and no change in the types of patients treated.
“Savings could emerge in later years because it may take time for hospitals to fully change their behavior, “ Ms. Finkelstein said. In addition, the program’s financial incentives will increase over time; bonuses for saving money and penalties for failing to do so will rise.
On the other hand, Dr. Berenson said, health care providers could figure out how to work the system: “In three to five years, we may see volume go up in a way that offsets savings through reduced payments for a procedure. We’ll wait and see.”
Medicare put its best foot forward by using a randomized design. Not only were the markets selected in a randomized fashion, but providers in those markets were also required to participate. Though common in medical studies, randomization is rare in health care policy, as is mandatory participation. Nearly 80 percent of medical studies are randomized trials, but less than 20 percent of studies testing health system change are. Organizations that would be subject to the experiments often strongly resist randomizing health system changes and forcing providers to participate.
Unfortunately, the randomization of the Comprehensive Care for Joint Replacement program will be partly compromised in coming years. The Centers for Medicare and Medicaid Services announced last year that hospitals in only half of markets under the program would have to stay in it. Participation is voluntary in the other half, and only one-quarter of hospitals opted in.
Going to a partly voluntary program will make it harder to learn about longer-term effects, Ms. Finkelstein said, and to get at the answers we’re seeking.
The following originally appeared on The Upshot (copyright 2018, The New York Times Company).
We know it can be hard to persuade physicians to do some things that have proven benefits, such as monitor blood pressure or keep patients on anticoagulants. But it might be even harder to get them to stop doing things.
In May, a systematic review in JAMA Pediatrics looked at the medical literature related to overuse in pediatric care published in 2016. The articles were ranked by the quality of methods; the magnitude of potential harm to patients from overuse; and the potential number of children that might be harmed.
In 2016 alone, studies were published that showed that we still recommend that children consume commercial rehydration drinks (like Pedialyte), which cost more, when their drink of choice would do. We give antidepressants to children too often. We induce deliveries too early, instead of waiting for labor to kick in naturally, which is associated with developmental issues in children born that way. We get X-rays of ankles looking for injuries we almost never find. And although there’s almost no evidence that hydrolyzed formulas do anything to prevent allergic or autoimmune disease, they’re still recommended in many guidelines.
Those researchers had reviewed the literature on overuse in children before, looking at all the studies from a year earlier. They modeled the work on a set of papers in JAMA Internal Medicine that looked at overuse in adults through a review of the literature published in 2015, 2014 and 2013.
Overuse is rampant. And it can harm patients.
By the end of the 20th century, for example, research seemed to indicate that we wanted to keep patients in the intensive care unit in a tight range of blood glucose levels. The evidence base for these recommendations came from observational studies that showed that patients with such tight control seemed less likely to develop adverse outcomes like infections or hyperglycemia, and they seemed more likely to survive.
Researchers tested this recommendation prospectively in a randomized controlled trial in a surgical intensive care unit. The results, published in 2001, appeared to confirm the prior findings, that tight glycemic control saved lives.
The study wasn’t perfect. It wasn’t blinded, for instance, and there were downsides to the recommendations. About 5 percent of those who received the intensive therapy had severe hypoglycemia at least once. The mortality in the control group was higher than what might be expected. Finally, this was a study of mostly post-cardiac surgery patients, and it wasn’t clear how widely the findings could be generalized.
Nevertheless, this was a huge benefit, and given the severity of the population being treated (intensive care patients are usually very, very ill), many experts called for changes in treatment while further research was done.
That larger work was published in 2009. The study randomly assigned more than 6,000 patients admitted to an intensive care unit for more than three days — to either tight or traditional glucose control. This time, there was a significantly higher rate of death in the tight control group (27.5 percent vs. 24.9 percent), as well as a much higher rate of severe hypoglycemia (6.8 percent vs. 0.5 percent). These findings applied to patients over all and to subgroups (like surgical versus medical patients).
In light of this, guidelines changed again. Physicians were asked to stop the widespread tight glycemic control.
In 2015, some enterprising researchers set out to look at how this knowledge changed physician behavior. Beginning in 2001, they looked at how physicians adopted the recommendations to use tight glycemic control in patients admitted to intensive care units. Starting in 2009, they looked at how physicians absorbed new information telling them to stop.
From 2001 through 2012, they analyzed data on more than 377,000 admissions to 113 intensive care units in 56 hospitals. Before the first trial was published, in 2001, 17 percent of admissions used tight glycemic control. Beginning in that year, however, there was a slow but steady increase in its use. About 1.7 percent more patients were being treated with recommended practice each quarter.
It’s hard to change behavior, but over time, physicians did. By 2009, the use of tight glycemic control had increased to about 23 percent. Many might have hoped for more, but at least there was progress.
Starting in 2009, however, the reverse was recommended. Doctors were asked to stop. Tight glycemic control was associated not only with higher mortality, but also with more adverse events.
That didn’t happen. From 2009 through 2012, there was no decrease in tight glycemic control. The authors argued that “there is an urgent need to understand and promote the de-adoption of ineffective clinical practices.”
That is, of course, an understatement.
Choosing Wisely, an initiative of the American Board of Internal Medicine Foundation, is entirely focused on the identification of care that physicians routinely recommend but shouldn’t. Almost 600 different tests, procedures or treatments, collected over the last six years, are currently listed on their website. Almost all the recommendations basically say “don’t do” them.
This overuse doesn’t provide a benefit. It can lead to harms. It can also cost a lot of money.
The public shares some culpability. Americans often seem to prefer more care than less. But a lot of it still comes from physicians, and from our inability to stop when the evidence tells us to. Professional organizations and others that issue such guidelines also seem better at telling physicians about new practices than about abandoning old ones.
I asked Daniel Niven, the lead author of the 2015 study, why it’s so hard to persuade doctors to discontinue certain practices. He said physicians have a hard time unlearning what they have learned, even when there’s newer and better science available. He said, “Even if the new contradictory science is accepted, providers often struggle applying this information in their daily clinical practice, not because they don’t want to, but rather, because they work within a system that doesn’t adapt well to changing evidence.”
He also said doctors might need to be more thoughtful about prevention: “We need to take a more cautious approach to technology adoption, and learn from mistakes of early adoption of health care technologies based on little or low-quality clinical evidence. This way we can prevent the need to ‘break up’ with the practice when the high-quality evidence shows that it is ineffective.”
Overuse represents a significant problem. As policymakers look for ways to save money without harming quality in the health care system, reducing overuse seems as if it should be a top option.
Florida Governor Rick Scott tries to scare voters by claiming that Democrats are going to undertake socialist experiments with Medicare, which is in itself a socialized medicine program. Also, a surprising entrant into the opioid addiction treatment market, and in a surprise twist, the ACA marketplaces are showing signs of stabilizing. It’s Healthcare Triage news.
Last week’s hearing in Texas v. United States convinced a lot of observers, me included, that a district court judge in Texas is poised to declare that all or part of the Affordable Care Act is unconstitutional. When and if he does so, the judge might enter an injunction ordering the administration to stop enforcing the Act everywhere in the country.
Understandably appalled at that possibility, the Maryland attorney general today filed a separate lawsuit in a Maryland district court. Among other things, he’s seeking an injunction requiring the continued enforcement of the law. Depending on how quickly the Maryland case moves, it’s possible we could see dueling injunctions—one ordering the Trump administration to stop enforcing the law, the other ordering it to keep enforcing.
That’s an unholy mess just waiting to happen. Now, it may not come to that. My best guess is that the Texas lawsuit will fizzle: any injunction will likely be stayed pending appeal, either by the Fifth Circuit or the Supreme Court, and the case is going nowhere on the merits. The Maryland lawsuit will likely prove unnecessary.
Even the risk of dueling injunctions, though, is a good argument for thinking that these national injunctions have gotten out of hand: one hard-right judge in Forth Worth shouldn’t have the power to suspend an Act of Congress. It’s an even better argument, more generally, for thinking that we’re channeling too many of our political disputes through the courts.
That’s the title of a new perspective piece from Rachel Sachs and me in the New England Journal of Medicine.
The Trump administration has refused to allow Massachusetts to experiment with a closed formulary, which would give the state some leverage in price negotiations over low-value drugs. But, as we explain, CMS has offered no written explanation whatsoever for its refusal.
From a legal perspective, that’s a problem. Administrative law requires agencies to provide reasons for their actions. Judges will usually defer to those reasons, but an unexplained action won’t stand up in court. If Massachusetts sues, the agency’s decision is at least as vulnerable as other actions that judges have recently invalidated, including CMS’s approval of Kentucky’s Medicaid work requirement. Indeed, CMS’s willingness to push the legal envelope on work requirements stands in stark contrast to its timidity when it comes to Massachusetts’ request to experiment with a closed formulary.
In subsequent statements to the press, HHS Secretary Alex Azar and CMS Administrator Seema Verma have suggested that granting the waiver would be illegal and would give states too much power in price negotiations.
These after-the-fact justifications track an argument that the pharmaceutical industry pressed in its campaign against the Massachusetts waiver. In its view, the establishment of a closed formulary would violate a “grand bargain” it struck with Congress as part of the 1990 legislation. The industry’s view of that bargain is that it agreed to sell drugs to Medicaid programs at a discount, and, in exchange, states would have to cover every FDA-approved drug.
Azar and Verma appear to believe that departing from the law’s purportedly neutral baseline would be unlawful, unfair, or both. But no part of the 1990 law limits CMS’s waiver authority over the portion of the Medicaid statute that governs prescription drugs. That’s an important omission. Congress knows how to restrict the scope of waivers when it wants to; indeed, it has done so in other parts of the Medicaid statute (for example, under the section covering cost-sharing requirements). For its part, CMS recently granted Oklahoma’s request to waive a different part of the same statutory provision that Massachusetts now seeks to waive, suggesting that the agency’s legal objections are a pretext.
What’s more, the grand bargain has already been violated—but not by Massachusetts. Since 1990, changes in pricing strategies, the proliferation of orphan drugs and complex biologics, and the decreasing rigor of the FDA approval process have placed an enormous strain on state Medicaid budgets. Massachusetts’ request reflects a thoughtful effort to restore the balance established by that bargain in light of an evolving pharmaceutical market. In rejecting the request, the Trump administration appears to have sided with the drug industry over the states.
You can read the whole thing here.
Today Melissa Garrido, Austin Frakt, and I published a piece in JAMA Forum about the growing number of Geriatric Emergency Departments in the U.S.
Older patients present with unique physical, pharmacological, cognitive, and social needs. These Geriatric EDs aim to provide the staff, physical design, and resources required to promote better health outcomes for older adults. Some EDs had taken steps toward these measures, but a new accreditation process created by the American College of Emergency Physicians aims to stringently evaluate the capacity of EDs that aim to serve older adults.
You can read the full text on JAMA Forum.
Elsa Pearson is a Policy Analyst with Boston University’s School of Public Health. She tweets at @epearsonbusph.
Austin and I coauthored an article on the impending physician shortage, out in STAT today. We reason that while a physician shortage is likely, the best solution may not be more physicians.
Specifically, we argue for expanded autonomy for non-physician providers and standardized scope of practice laws nationwide. The number of nurse practitioners, physician assistants, etc. is growing rapidly and they could play a critical role in improving access to care amid a physician shortage.
We also suggest reforming the current resident physician training program – graduate medical education – for the sake of efficiency and simplicity.
The U.S. has the best health care in the world but access to it is fading fast. Reforming graduate medical education may be needed to prepare for future workforce demands, but independent practice for non-physician practitioners is likely at the crux of an immediate solution.
If anyone’s been paying close attention (and, really, there are far better things to do), they’d have noticed that I’ve been tweeting a lot less lately and have blogged well below my historical rate for some time. So, what the hell have I been doing?
Putting aside that work got busier, which is true, I have been doing something else in my non-work time. I’ve been playing and thinking about playing trumpet. A lot. Turns out there’s a generalizable lesson in this experience, which I’ll get to.
I did a ton with the trumpet growing up, starting at age 10. It was a big part of my life and opened a lot of interesting doors. I played at a presidential inaugural ball with my high school jazz band, in Carnegie Hall with a youth orchestra, and traveled to the Soviet Union (1986) with another orchestra.
Despite these appearances of success, it was my summer at Interlochen that convinced me I had no business making an attempt at a profession in music. The talent there was astounding. As good as I was, or thought I was, I could not compete. Not to do so was not a hard decision. I had plenty of other interests.
And yet, this instrument is very important to me. I feel my best playing and having played. That makes practice easy. It’s not a chore. It’s therapy. Everything else, from cerebral concerns to physical ones melt away. This is only something I’m noticing now, at least consciously. Maybe it also happened when I was younger and I wasn’t fully aware of it. Maybe that’s why I worked so hard at it.
After more than a decade of barely playing — during which I started a family and blogged my brains out — I decided to return to the horn in a serious way about a year ago. I took lessons. With few exceptions, I’ve practiced every day, as much as my feeble facial muscles could stand.
It didn’t take long to discover that the limitations I had as a kid — the ones that, in part, convinced me not to go pro — were still there. I hit the same walls.
But I’m smarter now. I know that if there is an apparent limit to how well I can do something it’s almost certainly because I’m not doing it in the right way, not because I’ve hit some natural, innate limit. This is the big lesson from Anders Ericsson’s work, what I learned from his book Peak (highly recommended). Yes, you have to put in the work (this is the much misunderstood 10,000 hours “rule”), but there’s something else required to push through apparent limits.
Part of the process of doing so is to question basic assumptions. What the evidence was telling me was that some approaches I took to playing were wrong. No amount of doing it more would help. I had to do it differently.
I researched trumpet playing in a way I never could have as a kid. The internet is a fantastic thing! I got tons of ideas, the best ones from my teacher. I experimented. I spent time in front of a mirror finding facial muscles I had trouble activating. In meetings I practiced subtly moving just those muscles. This is subtle enough that few would notice.
Meanwhile, I suffered two massive collapses of ability in the last year, playing quite well (for me) one week to hardly being able to play at all for weeks after. These were clear signs I was doing something or some things wrong.
And then finally, I figured it out (again, with help). I won’t bore you with the details, but do want to make a point about this: there are very subtle things about how facial muscles are used that are nearly impossible to discern visually or, sometimes (and in my case) even by how one sounds. You’ve got to do some pretty creative things and a lot of experimentation to figure out what’s wrong and how to do what’s right.*
That’s the bigger point. To keep getting better at something like this, and anything, really, is a creative process. You have to find ways to break through the plateaus. Sometimes it means you have to unlearn bad habits, even ones you didn’t realize were bad. At some point brute force doesn’t work. You have to find another way in. Smashing your head against a wall has its uses, but it’s not the right tool all the time or even most of the time.
This, to me, is the deeply fascinating part of the story, and the generalizable part. It starts with the simple lesson that if after substantial effort something isn’t going right, do it differently. It’s not the world. It’s you.
Naturally, which of the infinite different ways is not clear. If it were, you’d already be doing it. Very likely the right way is one of the different ways that you’ve never conceived, that you don’t even think is possible. Finding the almost unfindable is where the creativity is.
It’s also hard, which is why a teacher is important. Still, there’s a ton one has to do on one’s own. So long as your ego is not involved (i.e., you don’t judge yourself badly for not having found the secret yet), this is pretty fun and interesting … at least for me … and a lot more so than Twitter of late.
* For my fellow brass/wind players, I have to acknowledge that embouchure is not the entire ball game. Naturally, there’s a lot about breathing one has to get right too. I’m putting that aside because that wasn’t the big wall I was hitting. But, yes, I’ve learned a lot about breathing too.