How Flawed Science Is Undermining Good Medicine

How Flawed Science Is Undermining Good Medicine

How Flawed Science Is Undermining Good Medicine

A surprising medical finding caught the eye of NPR’s veteran science correspondent Richard Harris in 2014. A scientist from the drug company Amgen had reviewed the results of 53 studies that were originally thought to be highly promising — findings likely to lead to important new drugs. But when the Amgen scientist tried to replicate those promising results, in most cases he couldn’t.

A surprising medical finding caught the eye of NPR’s veteran science correspondent Richard Harris in 2014. A scientist from the drug company Amgen had reviewed the results of 53 studies that were originally thought to be highly promising — findings likely to lead to important new drugs. But when the Amgen scientist tried to replicate those promising results, in most cases he couldn’t.

“He tried to reproduce them all,” Harris tells Morning Edition host David Greene. “And of those 53, he found he could only reproduce six.”

That was “a real eye-opener,” says Harris, whose new book Rigor Mortis: How Sloppy Science Creates Worthless Cures, Crushes Hope, and Wastes Billions explores the ways even some talented scientists go wrong — pushed by tight funding, competition and other constraints to move too quickly and sloppily to produce useful results.

“A lot of what everybody has reported about medical research in the last few years is actually wrong,” Harris says. “It seemed right at the time but has not stood up to the test of time.”

The impact of weak biomedical research can be especially devastating, Harris learned, as he talked to doctors and patients. And some prominent scientists he interviewed told him they agree that it’s time to recognize the dysfunction in the system and fix it.

“If it’s not operating at full steam … and not doing everything right,” Harris says, “it’s worth pointing that out and saying, ‘No. Think about this. Let’s make it better.’ ”

The following has been edited for clarity.

Tom Murphy was a healthy rugby player diagnosed with ALS in his 50s. …. With his doctor’s help he signs up for an experimental treatment with a drug called dexpramipexole, or “Dex.” At first, he’s very hopeful, and it seems to be helping him, but they run the tests and figure out that it actually doesn’t work. In fact none of the ALS drugs work. I focus on Tom Murphy because he’s a victim of the system here — of these failures.

What happened in the case of ALS was there were at least a dozen drugs that had been tried in a handful of small studies — way too small — of animals. And they all seemed to have some sort of promise — some of them went into very large clinical trials. We spent tens of millions of dollars developing these drugs, and they all failed. There’s a group in Cambridge, Mass. — the ALS Therapy Development Institute — that went back and reviewed all these studies and realized all the initial studies were wrong. They used very few mice. They weren’t thinking enough about the different genetics of the mice. And a lot of other problems. … This therapy institute came away thinking none of these drug candidates were really realistic.

On the ways the scientific enterprise in Charles Darwin’s time was very different

Darwin was very interesting. It took him decades to come up with his theory of evolution and he was not in a hurry — he was studying barnacles, he was studying birds, all sorts of things. He felt no pressure to publish until somebody came up with a similar idea, and he decided, ‘Hmmm … maybe I do want to be first. …” But we’re not in that world anymore. Things are very competitive, very fast-paced. So the competitive world of biomedicine is shaping this problem of evidence that can’t be replicated a lot.

On why the delight that’s long been an intrinsic part of science can disappear over time — and why that’s bad

I think a lot of people go into science out of a sense of wonder. But … as time goes on, people feel the career pressures, and they realize it isn’t just about exploring and having big ideas. They have to have research that helps them progress toward their first job, toward tenure, then the next grant, and so on. Those pressures are different from just, sort of, exploring and understanding fundamental biology…. And the less you’re focusing on delight, the less maybe you’re aiming at the truth and the more you are, inadvertently, often aiming at other goals — career goals, financial goals and so on. This may give you a fruitful life as an individual, but may produce less value to us as a society.

On how the public should respond when they hear of a big biomedical advance

I think it is good to question it. Every time you hear something like this, just remember, it’s all contingent — here is one study, and it may not stand the test of time. I think that’s healthy. … When scientists read the scientific literature, they realize, “Oh, probably half of this is wrong.” It’s just, not knowing which half — that’s the vexing part.

On the risk that pointing out flaws in science will make people question its value

It’s always uncomfortable to point out problems, but it’s also essential. I mean, we are taxpayers — we are citizens, and we support this enterprise and we expect to reap its rewards. If it’s not operating at full steam … and not doing everything right, it’s worth pointing that out and saying, “No. Think about this. Let’s make it better.” Many prominent scientists agree with me and are concerned about this — and are thinking hard about how to make things better, from the top of NIH on down. There are solutions, and I talk about them in my book.

On why the Trump administration’s proposed cuts to NIH funding wouldn’t make things better

It’s a very appealing idea, obviously, to say, “Oh, well, let’s just identify the waste and root it out.” But that’s not the way science works. … If you cut the [$30 billion] budget of the National Institutes of Health, you’re going to shrink that already very small pool of money even smaller, and you’re going to increase the competitive pressures. You’re going to increase all these perverse incentives that put us in this position to begin with. So I think that would actually be devastating to biomedical research.

Half of Americans are responsible for only 3 percent of health care costs

Half of Americans are responsible for only 3 percent of health care costs

Half of Americans are responsible for only 3 percent of health care costs

Here’s a simple reason crafting health policy is so devilishly hard: Most Americans are pretty healthy and a few are really sick.

The top 1 percent of health-care spenders use more resources, collectively, than the bottom 75 percent, according to a new study based on national surveys. Slice the data a different way, and the bottom half of spenders all together rack up only about 3 percent of overall health care spending — a pattern that hasn’t budged for decades. This creates a fundamental inequality in the country’s health spending that is the crux of the challenge policymakers face: They need a system that works for people who are ill, but is attractive to those who are healthy and spend little on health care.

Here’s a simple reason crafting health policy is so devilishly hard: Most Americans are pretty healthy and a few are really sick.

The top 1 percent of health-care spenders use more resources, collectively, than the bottom 75 percent, according to a new study based on national surveys. Slice the data a different way, and the bottom half of spenders all together rack up only about 3 percent of overall health care spending — a pattern that hasn’t budged for decades. This creates a fundamental inequality in the country’s health spending that is the crux of the challenge policymakers face: They need a system that works for people who are ill, but is attractive to those who are healthy and spend little on health care.

The political debate over health care often focuses on how a new system will meet the needs of the sick: Will cancer patients or people with diabetes access and afford care when they need it? But the Health Affairs study, “Most Americans have good health, little unmet need and few health care expenses,” shows just how important the healthy people who spend very little on health care are. The message you draw from that, however, may depend on your politics.

“The key takeaway message really is most people are in good health; they don’t spend a lot of money, and yet it’s important to have them be part of our insurance system. If they’re left out of the system, we’re not going to have the funds to take care of people who are very sick,” said Marc Berk, a health policy researcher and contributing editor of Health Affairs who led the analysis.

But Tom Miller, a resident fellow at the American Enterprise Institute, disagreed. He said that the study is based on quick and incomplete snapshots of health and argued that it is yet another way to divert from the health-care discussion we should be having: about how to rein in spending. Using this data to argue about where to get premium dollars from — from the pockets of the well or the sick — simply allows the system to grow ever bigger and prop up an even-more-expensive medical system.

“We all get diverted by hoping we can hide the bill under someone else’s pillow,” Miller said. “I think that’s the political argument you hear — these low spenders, we’re scared to death they might catch on to the fact they’re getting taken to the cleaners” by being forced to buy expensive health insurance they don’t need.

According to House Speaker Paul Ryan earlier this month, the inequality in spending and sickness is the “fatal conceit of Obamacare.” Ryan used a graph that showed that chronically ill people are a narrow slice of the population, but a big driver of spending.

“The whole idea of Obamacare is the people … who are healthy pay for the people who are sick; it’s not working and that’s why it’s in a death spiral,” Ryan said.

As critics have pointed out, this was part of how the Affordable Care Act was designed, and it is how insurance traditionally works. The auto insurance of people who don’t get into car crashes helps foot the bill for those who do. The premiums from homeowners who never file a claim help underwrite the insurance payouts for those whose houses burn down. It’s the same for health, and a major challenge in the exchanges, where people buy individual plans with government subsidies, has been getting enough healthy people to sign up to keep the premiums reasonable for everyone.

A conservative vision of health care would have people take more responsibility for their own health care costs, but the graph above presents policy challenges as well, because it suggests that solution wouldn’t just be a matter of making people at all levels smarter shoppers. It would saddle those with the bad luck of being sick with costs that could quickly become untenable.

To different people, the pattern suggests very different policy implications: Berk thinks the data are a powerful argument that everyone needs to pay in to the system; to Miller, it suggests the opposite.

“We should probably leave a lot more people alone,” Miller said. “There’s unexpected catastrophic coverage, but don’t micromanage every detail of coverage for people who are just going to be fine.”

Virtual Doctor Visits May Not Reduce Healthcare Spending

Virtual Doctor Visits May Not Reduce Healthcare Spending

Virtual Doctor Visits May Not Reduce Healthcare Spending

Consultations with doctors by phone or video conference appear to be catching on, with well over a million virtual visits reported in 2015.

The convenience of ‘telehealth’ appeals to people seeking care, and the notion that it costs less than an in-office visit would makes it attractive to employers and health plans.

Consultations with doctors by phone or video conference appear to be catching on, with well over a million virtual visits reported in 2015.

The convenience of ‘telehealth’ appeals to people seeking care, and the notion that it costs less than an in-office visit would makes it attractive to employers and health plans.

But a new study suggests that although telehealth services may boost access to a physician, they don’t necessarily reduce healthcare spending, contrary to assertions by telehealth companies.

The study, published 6 March in the journal Health Affairs, shows that telehealth prompts individuals to seek care for minor illnesses that otherwise would not have induced them to visit a doctor’s office.

Telehealth has been around for more than a decade, but its growth has been fueled more recently by the ubiquity of smartphones and laptops, says study investigator Lori Uscher-Pines. She is a policy researcher at the RAND Corporation, a nonprofit think tank based in Santa Monica, California.

These virtual consultations are designed to replace more expensive visits to a doctor’s office or emergency room. On average, a telehealth visit costs about $79, compared with about $146 for an office visit, according to the study. But the researchers found that virtual visits generate additional medical use.

“What we found is contrary to what [telehealth] companies often say,” Uscher-Pines says. “We found an increase in spending for the payer.”

The researchers found that only 12 percent of telemedicine visits replaced an in-person provider visit, whereas 88 percent represented new demand.

Face to face:

The researchers examined 2011-13 utilization data of 300,000 people enrolled in the Blue Shield of California Health Maintenance Organization (HMO) plan offered by the California Public Employees Retirement System (CalPERS), which covers current and former state employees and their families. CalPERS’ Blue Shield HMO started offering telehealth services, available 24/7 to its beneficiaries, in April 2012.

The researchers focused on virtual visits for respiratory illnesses, which include sinusitis, bronchitis, pneumonia and tonsillitis, among others.

Although a single telehealth visit for a respiratory illness costs less than an in-person visit, it often results in more follow-up appointments, lab tests and prescriptions, which increases spending in the long run. Liability concerns may prompt telehealth physicians to recommend that a patient go in for a face-to-face appointment with a doctor, the researchers note.

The researchers estimated that annual spending for respiratory illnesses increased about $45 per telehealth user, compared with patients who did not take advantage of such virtual consultations.

Jason Gorevic, chief executive officer at Teladoc, the operator that provides telehealth services for CalPERS Blue Shield members, says the new study doesn’t square with Teladoc data showing the cost savings of telemedicine.

According to 2016 data, Gorevic says, only 13 percent of Teladoc visits represent new medical use. He notes that the RAND study uses older data, and that many things have changed since then — including the technology, the rate at which these services are being adopted and patient engagement.

“In fact, other more comprehensive studies — using six times the amount of claims data including the same population as the [RAND] study — have found tremendous value of telehealth, with consistently repeatable results,” Gorevic says. These other studies have shown that telehealth decreases overall health care spending, he says. But Uscher-Pines says the RAND findings are not surprising.

When RAND researchers studied retail clinics last year, they found that making access to healthcare more convenient triggers new use and additional costs. That study found 58 percent of visits to in-store clinics represented new use of medical services rather than a substitute for doctor office visits.

Yet the fact that telehealth services are more affordable per visit than a trip to a physician’s office shows that there is still a pathway to cost savings, Uscher-Pines says.

Target groups:

To achieve cost savings, telehealth services would have to replace costlier visits, the researchers say. Insurers could increase telehealth visit costs for patients to deter unnecessary use. Another way to increase the health system value of virtual doctor visits is to target specific groups — such as those who often use emergency rooms for less severe illnesses. An emergency room visit costs an estimated $1,734.

“You could take these people in the emergency department and offer them this cheaper option. That would be a direct replacement,” Uscher-Pines says.

Gorevic says that a challenge for telehealth is engaging consumers, so the comparatively low fees provide a financial incentive.

“Because a telehealth visit is much cheaper than an in-person visit, the cost sharing should be reflective of that,” he says.

Marcus Thygeson, senior vice president and chief health officer at Blue Shield, which also provides virtual doctor visits through Teladoc, in a statement said that “increased convenience can increase utilization, so overall healthcare costs may increase or stay the same. Blue Shield supports the use of telemedicine to improve access for both primary and specialty healthcare, especially in rural communities.”

The researchers noted several limitations to the RAND study. For example, they examined only one telehealth company and studied only visits for respiratory illnesses. In addition, the individuals whose data were scrutinized had commercial insurance, and it is possible the use of telehealth would differ among people with government insurance, high-deductible plans or no insurance at all, the researchers wrote.

 

This story was produced by Kaiser Health News, which publishes California Healthline, an editorially independent service of the California Health Care Foundation.

This story originally appeared on Kaiser Health News. It has been slightly modified to reflect Spectrum’s style.

Health-care costs, not new iPhones, are the reason you’re broke

Health-care costs, not new iPhones, are the reason you’re broke

Health-care costs, not new iPhones, are the reason you’re broke

Republican Rep. Jason Chaffetz provoked an outcry after saying that, under the GOP’s proposed health-care plan, poorer Americans could somehow afford insurance by choosing to forgo new iPhones.

“Americans have choices. And they’ve got to make a choice. So maybe, rather than getting that new iPhone that they just love and they want to go spend hundreds of dollars on, maybe they should invest that in health care,” Chaffetz said Tuesday. “They’ve got to make those decisions themselves.”

Republican Rep. Jason Chaffetz provoked an outcry after saying that, under the GOP’s proposed health-care plan, poorer Americans could somehow afford insurance by choosing to forgo new iPhones.

“Americans have choices. And they’ve got to make a choice. So maybe, rather than getting that new iPhone that they just love and they want to go spend hundreds of dollars on, maybe they should invest that in health care,” Chaffetz said Tuesday. “They’ve got to make those decisions themselves.”

His comment came off to many like a high-tech twist on what self-made millionaire David Bach calls “The Latte Factor,” the notion that many people are broke because they spend too much on trivialities like coffee.

Other experts, however, point out that the real reason so many Americans are financially insecure isn’t because companies like Starbucks and Apple seduce them into spending more than they should on small luxuries. Rather it’s because wages are stagnant, and the cost of necessities such as rent, student loans and, of course, health care are increasing.

Twitter users were quick to explain that the kind of Americans who qualify for the Medicaid expansion are not the ones dreaming about the iPhone 7. And while smartphones may cost a lot, medical care in America is expensive on a whole different scale.

It is true that Americans, especially young ones, spend a lot of money on their smartphones, perhaps even more than many of them can comfortably afford.

The average millennial has less than $1,000 in a savings account. Even 33 percent of older millennials, ages 25-34, have nothing saved at all. Yet, between the hardware and the monthly plan, smartphones can cost users about $1,000 a year, and 85 percent of millennials have one. Nielson reports that “these young consumers are the largest segment of smartphone owners.”

The costs associated with your smartphone may indeed be part of the reason you aren’t able to save much for the future. They’re still insignificant in comparison to the costs associated with health care.

By 2016, annual average health-care costs had spiked to over $10,000 per person and $25,000 for families of four. That’s about half the median household income in 2016 ($56,000). And that’s for people fortunate enough to have coverage.

Compared to that, as Twitter users were also quick to point out, a phone really is about as big a deal as an occasional latte.

While young people could indeed figure out ways to be smarter about their smartphones — by switching to cheaper monthly plans and upgrading to new models less often, for example — the onus is still on Congress to help all Americans figure out how to afford health care without going bankrupt. Otherwise, even if millennials can figure out how to save for retirement, many of them won’t live long enough to enjoy it.

Virtual-reality system for the elderly wins health care prize

Virtual-reality system for the elderly wins health care prize

Virtual-reality system for the elderly wins health care prize

Critics have long faulted U.S. medical education for being hidebound, imperious and out of touch with modern health-care needs. The core structure of medical school—two years of basic science followed by two years of clinical work—has been in place since 1910.

Now a wave of innovation is sweeping through medical schools, much of it aimed at producing young doctors who are better prepared to meet the demands of the nation’s changing health-care system.

Virtual reality is quickly gaining steam in the gaming industry. But an MIT startup is now aiming the technology at a different demographic, putting it to use as a health care tool for the elderly.

At last night’s MIT Sloan Healthcare Innovations Prize pitch competition, Rendever earned the $25,000 grand prize for creating a virtual-reality platform that gives residents in assisted-living facilities the chance to explore the world virtually. The platform also provides cognitive therapy and tracks movement data to aid in early diagnosis of dementia.

“We’re using virtual reality to improve the way we age, so you don’t become isolated, don’t become depressed, and you can keep your mind happy and healthy,” said Rendever co-founder and CEO Dennis Lally, an MIT Sloan School of Management student who launched the startup with classmate Reed Hayes.

Rendever was one of eight finalist teams — winnowed down from 50 applicants — to pitch health care innovation ideas to a panel of expert judges and a capacity audience last night at the MIT Wong Auditorium in the Tang Center. The competition, part of the 14th annual MIT Sloan Healthcare and BioInnovations Conference (being held today in the MIT Media Lab), is organized by the student-run MIT Sloan Healthcare Club to promote innovation and entrepreneurship in the health care space.

A second-place prize of $4,000 went to Need-a-Knee, a team of MIT mechanical engineering undergraduates developing an inexpensive leg prosthetic that allows users to sit cross-legged — an important part of culture in countries such as India, where current models don’t allow for such flexibility.

Day Zero Diagnostics, a team from the Harvard-MIT Health Sciences and Technology program, earned a $1,000 audience-choice prize for developing a diagnostic tool that makes the detection of specific bacterial infection in hospitals much faster, reducing wait times from days to hours.

Other competing teams were: CareMobile Transportation, an Uber-like medical-transportation startup that employs social workers to transport and care for people with disabilities; Manus Robotics, a wearable robotic gripper that helps stroke survivors with impaired hand functions; Patients Like This, an analytics tool that improves mental health patient outcomes by using electronic health record data; Neurosleeve, a computerized glove that measures electrical function of nerves in the hand to diagnose carpal tunnel syndrome and other conditions; and #1, a wearable device and app system that treats urinary incontinence by tracking pelvic muscle training and measuring moisture associated with incontinence.

Socializing, therapy, and diagnostics

In the team’s winning pitch, Rendever said its system includes multiple virtual-reality headsets, custom software, and a tablet. The software syncs headsets together, so users can join together in a virtual world, visiting childhood homes, exotic locales, sports games, or a relative’s wedding across the world. The headsets can be controlled simultaneously by caregivers, using the tablet. All content is also custom-made by Rendever, based on 20 million gigabytes of content mined from the internet.

Apart from providing a socializing tool, the system can double as a form of reminiscence therapy, which involves discussing past experiences, with aid of photographs, familiar items, and music — or virtual cues, in the case of Rendever.

According to trial studies, Rendever’s system has increased overall resident happiness at the Brookdale Senior Living Community in Massachusetts by 40 percent, Lally said. The startup is currently working with the MIT AgeLab to validate those statistics.

Rendever also hopes to leverage virtual-reality data to aid in diagnosing dementia, which currently relies on qualitative studies and expert opinion. “We’re creating a data-driven [diagnostics] solution,” Lally said.

Users are asked to work through real-life simulations, such as making dinner, completing a series of tasks. In the process, Rendever’s software collects thousands of data points per minute on movement, reaction time, and executive function. All this can lead to earlier interventions for dementia patients and help measure the efficacy of treatments.

“We can track very precisely how someone moves through this space, and what they were doing,” Hayes, Rendever’s chief operations officer, told MIT News. “We also built a machine-learning model that’s currently being trained to help find patterns [characteristic of] someone who has early-stage dementia versus someone of healthy mind.”

Rendever’s prize money will help fund research and development, with a second trial study kicking off soon. Hayes attributes the team’s win to tapping into an underserved need. “Everyone has a parent or grandparent who’s getting older … and not much can be done when they can’t leave the house,” he said. “The solution we built is a fun way for them to re-experience the world again, to be explorers. We’re bringing that to a demographic that has lost the ability [to explore].”

Last year, Rendever entered the pitch competition — its first entrepreneurship contest ever — and made it to the semifinals round. By refining the business and technology through MIT classes at the Martin Trust Center for MIT Entrepreneurship — under the watchful eye of managing director Bill Aulet — the team came out on top, Reed said. “It’s this awesome story for us to come back [to the competition] and win,” he said.

Pushing ideas forward

There were only three prizewinners last night. But MIT Sloan student Maddie Thoms, co-president of the MIT Sloan Healthcare Club that organized the competition, said the aim is to help all the entrepreneurs further refine their ventures. “It’s not just about one or two teams walking away with a good prize. We’re hoping to push a bunch of teams forward in their endeavors,” she said.

In the weeks leading up to the pitch competition, teams were offered mentorship, networking opportunities, and a one-day workshop on developing business pitches. The competition consisted of two semifinal rounds, where judges provided further coaching.

Teams were each required to have at least one MIT or Harvard University student. Criteria for choosing competing teams included determining the novelty of the invention, its technical feasibility, any hurdles in governmental and other regulations, and whether the team has acquired customers or made its first sale.

With the competition, organizers also hope to strengthen the health care community at MIT, in neighboring Kendall Square, and across Boston by fostering connections between teams, judges, and local mentors and investors. Thoms said that networking helps “get the right ideas connected with the right people, ventures, or partner companies.”

Competing amongst similar startups helped second-place team Need-a-Knee solidify a business plan, said team member Matthew Cavuto, a mechanical engineering student and a 2017 Marshall Scholar. The team built a rotating disc with a simple push-button spring-pin mechanism at the knee joint of a low-cost prosthetic. Pushing a button on the disc releases a pin inside, which lets users rotate the disc and snap it into place at an angle to sit cross-legged. In India, for instance, people sit this way for praying, eating, and other activities, but current inexpensive prosthetics aren’t designed for the position.

Most engineering for the prosthetic was completed in one semester, Cavuto said, and the competition helped demonstrate how major global problems can be tackled quickly. “There are real needs that can be solved in a fairly short amount of time in the developing world, and there’s a big market … that’s not usually seen here in the United States,” he said. The team’s $4,000 prize money will go toward International Organization for Standardization testing, which will certify it for safety and compatibility with humans.