HEALTH: Medical Guesswork - the health industry doesn't know which treatments really work

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BusinessWeek Magazine: COVER STORY

 

Medical Guesswork

 

From heart surgery to prostate care, the health industry knows little

about which common treatments really work.

 

The signs at the meeting were not propitious. Half the board members of

Kaiser Permanente's Care Management Institute left before Dr. David Eddy

finally got the 10 minutes he had pleaded for. But the message Eddy

delivered was riveting. With a groundbreaking computer simulation, Eddy

showed that the conventional approach to treating diabetes did little to

prevent the heart attacks and strokes that are complications of the

disease. In contrast, a simple regimen of aspirin and generic drugs to

lower blood pressure and cholesterol sent the rate of such incidents

plunging. The payoff: healthier lives and hundreds of millions in

savings. " I told them: 'This is as good as it gets to improve care and

lower costs, which doesn't happen often in medicine, " ' Eddy recalls.

" 'If you don't implement this,' I said, 'you might as well close up

shop. " ' The message got through. Three years later, Kaiser is in the

midst of a major initiative to change the treatment of the diabetics in

its care. " We're trying to put nearly a million people on these drugs, "

says Dr. Paul Wallace, senior adviser to the Care Management Institute.

The early results: The strategy is indeed improving care and cutting

costs, just as Eddy's model predicted.

 

For Eddy, this is one small step toward solving the thorniest riddle in

medicine -- a dark secret he has spent his career exposing. " The problem

is that we don't know what we are doing, " he says. Even today, with a

high-tech health-care system that costs the nation $2 trillion a year,

there is little or no evidence that many widely used treatments and

procedures actually work better than various cheaper alternatives.

 

This judgment pertains to a shocking number of conditions or diseases,

from cardiovascular woes to back pain to prostate cancer. During his

long and controversial career proving that the practice of medicine is

more guesswork than science, Eddy has repeatedly punctured cherished

physician myths. He showed, for instance, that the annual chest X-ray

was worthless, over the objections of doctors who made money off the

regular visit. He proved that doctors had little clue about the success

rate of procedures such as surgery for enlarged prostates. He traced one

common practice -- preventing women from giving birth vaginally if they

had previously had a cesarean -- to the recommendation of one lone

doctor. Indeed, when he began taking on medicine's sacred cows, Eddy

liked to cite a figure that only 15% of what doctors did was backed by

hard evidence. A great many doctors and health-care quality experts have

come to endorse Eddy's critique. And while there has been progress in

recent years, most of these physicians say the portion of medicine that

has been proven effective is still outrageously low -- in the range of

20% to 25%. " We don't have the evidence [that treatments work], and we

are not investing very much in getting the evidence, " says Dr. Stephen

C. Schoenbaum, executive vice-president of the Commonwealth Fund and

former president of Harvard Pilgrim Health Care Inc. " Clearly, there is

a lot in medicine we don't have definitive answers to, " adds Dr. I.

Steven Udvarhelyi, senior vice-president and chief medical officer at

Pennsylvania's Independence Blue Cross.

 

What's required is a revolution called " evidence-based medicine, " says

Eddy, a heart surgeon turned mathematician and health-care economist.

Tall, lean, and fit at 64, Eddy has the athletic stride and catlike

reflexes of the ace rock climber he still is. He also exhibits the

competitive drive of someone who once obsessively recorded his time on

every training run, and who still likes to be first on a brisk walk up a

hill near his home in Aspen, Colo. In his career, he has never been

afraid to take a difficult path or an unpopular stand. " Evidence-based "

is a term he coined in the early 1980s, and it has since become a

rallying cry among medical reformers. The goal of this movement is to

pierce the fog that envelops the practice of medicine -- a state of

ignorance for which doctors cannot really be blamed. " The limitation is

the human mind, " Eddy says. Without extensive information on the

outcomes of treatments, it's fiendishly difficult to know the best

approach for care.

 

The human brain, Eddy explains, needs help to make sense of patients who

have combinations of diseases, and of the complex probabilities involved

in each. To provide that assistance, Eddy has spent the past 10 years

leading a team to develop the computer model that helped him crack the

diabetes puzzle. Dubbed Archimedes, this program seeks to mimic in

equations the actual biology of the body, and make treatment

recommendations as well as figure out what each approach costs. It is at

least 10 times " better than the model we use now, which is called

thinking, " says Dr. Richard Kahn, chief scientific officer at the

American Diabetes Assn.

 

WASTED RESOURCES

 

Can one computer program offset all the ill-advised treatment options

for a whole range of different diseases? The milestones in Eddy's long

personal crusade highlight the looming challenges, and may offer a

sliver of hope. Coming from a family of four generations of doctors,

Eddy went to medical school " because I didn't know what else to do, " he

confesses. As a resident at Stanford Medical Center in the 1970s, he

picked cardiac surgery because " it was the biggest hill -- the glamour

field. "

 

But he soon became troubled. He began to ask if there was actual

evidence to support what doctors were doing. The answer, he was

surprised to hear, was no. Doctors decided whether or not to put a

patient in intensive care or use a combination of drugs based on their

best judgment and on rules and traditions handed down over the years, as

opposed to real scientific proof. These rules and judgments weren't

necessarily right. " I concluded that medicine was making decisions with

an entirely different method from what we would call rational, " says Eddy.

 

About the same time, the young resident discovered the beauty of

mathematics, and its promise of answering medical questions. In just a

couple of days, he devoured a calculus textbook (now framed on a shelf

in his beautifully appointed home and office), then blasted through the

books for a two-year math course in a couple of months. Next, he

persuaded Stanford to accept him in a mathematically intense PhD program

in the Engineering-Economics Systems Dept. " Dave came in -- just this

amazing guy, " recalls Richard Smallwood, then a Stanford professor. " He

had decided he wanted to spend the rest of his life bringing logic and

rationality to the medical system, but said he didn't have the math. I

said: 'Why not just take it?' So he went out and aced all those math

courses. "

 

To augment his wife's earnings while getting his PhD, Eddy landed a job

at Xerox Corp.'s (XRX) legendary Palo Alto Research Center. " They hired

weird people, " he says. " Here was a heart surgeon doing math. That was

weird enough. "

 

Eddy used his newfound math skills to model cancer screening. His

Stanford PhD thesis made front-page news in 1980 by overturning the

guidelines of the time. It showed that annual chest X-rays and yearly

Pap smears for women at low risk of cervical cancer were a waste of

resources, and it won the most prestigious award in the field of

operations research, the Frederick W. Lanchester prize. Based on his

results, the American Cancer Society changed its guidelines. " He's smart

as hell, with a towering clarity of thought, " says Stanford health

economist Allan Enthoven.

 

Dr. William H. Herman, director of the Michigan Diabetes Research &

Training Center, has a competing computer model that clashes with

Eddy's. Nonetheless, he says, " Dr. Eddy is one of my heroes. He's sort

of the father of health economics -- and he might be right. "

 

Appointed a full professor at Stanford, then recruited as chairman of

the Center for Health Policy Research & Education at Duke University,

Eddy proved again and again that the emperor had no clothes. In one

study, he ferreted out decades of research evaluating treatment of high

pressure in the eyeball, a condition that can lead to glaucoma and

blindness. He found about a dozen studies that looked at outcomes with

pressure-lowering medications used on millions of people. The studies

actually suggested that the 100-year-old treatment was harmful, causing

more cases of blindness, not fewer.

 

Eddy submitted a paper to the Journal of the American Medical Assn.

(JAMA), whose editors sent it out to specialists for review. " It was

amazing, " Eddy recalls. " The tom-toms sounded among all the

ophthalmologists, " who marshaled a counterattack. " I felt like Salman

Rushdie. " Stanford ophthalmologist Kuldev Singh says: " Dr. Eddy

challenged the community to prove that we actually had evidence. He did

a service by stimulating clinical trials, " which showed that the

treatment does slow the disease in a minority of patients.

 

By 1985, Eddy was " burned out " by the administrative side of academia,

he says. Lured by a poster of the Tetons, he gave up his prestigious

post. He moved to Jackson, Wyo., so he could climb in his spare time. He

and a friend even made a first ascent of a new route on the Grand Teton,

now named after them. Meanwhile, he carved out a niche showing doctors

at specialty society meetings that their cherished beliefs were dubious.

" At each meeting I would do the same exercise, " he says. He would ask

doctors to think of a typical patient and typical treatment, then write

down the results of that treatment. For urologists, for instance, what

were the chances that a man with an enlarged prostate could urinate

normally after having corrective surgery? Eddy then asked the society's

president to read the predictions.

 

The results were startling. The predictions of success invariably ranged

from 0% to 100%, with no clear pattern. " All the doctors were trying to

estimate the same thing -- and they all gave different numbers, " he

says. " I've spent 25 years proving that what we lovingly call clinical

judgment is woefully outmatched by the complexities of medicine. " Think

about the implications for helping patients make decisions, Eddy adds.

" Go to one doctor, and get one answer. Go to another, and get a

different one. " Or think about expert testimony. " You don't have to hire

an expert to lie. You can just find one who truly believes the number

you want. "

 

More important, the lack of evidence creates a costly clash. Americans

and their doctors want access to any new treatment, and many doctors

fervently believe such care is warranted. On the other hand, those

beliefs can be flat wrong. As a consultant on Blue Cross's insurance

coverage decisions, Eddy testified on the insurer's behalf in

high-profile court cases, such as bone marrow transplants for breast

cancer. Women and doctors demanded the treatment, even though there was

no evidence it saved lives. Insurers who refused coverage usually lost

in court. " I was the bad guy, " Eddy recalls. When clinical trials were

actually done, they showed that the treatment, costing from $50,000 to

$150,000, didn't work. The doctors who pushed the painful, risky

procedure on women " owe this country an apology, " Eddy says.

 

Is medicine doing any better today? In recognizing the problem, yes. But

in solving it, unfortunately, no. Take prostate cancer. Doctors now

routinely test for levels of prostate-specific antigen (PSA) to try to

diagnose the disease. But there's no evidence that using the test

improves survival. Some experts believe that as many cancers would be

detected through random biopsies. Then, once cancer is spotted, there's

no way to know who needs treatment and who doesn't. Plus, there is a

plethora of treatment choices -- four kinds of surgery, various types

of implantable radioactive seeds, and competing external radiation

regimens, notes Dr. Eric Klein, head of urologic oncology at the

Cleveland Clinic. " How is a poor patient supposed to decide among

those? " he asks. Most of the time, patients don't even know the options.

 

VESTED INTERESTS

 

" Because there are no definitive answers, you are at the whim of where

you are and who you talk to, " says Dr. Gary M. Kirsh at the Urology

Group in Cincinnati. Kirsh does many brachytherapies --implanting

radioactive seeds. But " if you drive one and a half hours down the road

to Indianapolis, there is almost no brachytherapy, " he says. Head to

Loma Linda, Calif., where the first proton-beam therapy machine was

installed, in 1990, and the rates of proton-beam treatment are far

higher than in most other parts of the country. Go to a surgeon, and

he'll probably recommend surgery. Go to a radiologist, and the chances

are high of getting radiation instead. " Doctors often assume that they

know what a patient wants, leading them to recommend the treatment they

know best, " says Dr. David E. Wennberg, president of Health Dialog

Analytic Solutions.

 

More troubling, many doctors hold not just a professional interest in

which treatment to offer, but a financial one as well. " There is no

question that the economic interests of the physician enter into the

decision, " says Kirsh. The bottom line: The conventional wisdom in

prostate cancer -- that surgery is the gold standard and the best chance

for a cure -- is unsustainable. Strangely enough, however, the choice

may not matter very much. " There really isn't good evidence to suggest

that one treatment is better than another, " says Klein.

 

Compared with the skepticism Eddy faced in the 1990s, many physicians

now concur that traditional treatments for serious illnesses often

aren't best. Yet this message can be hard for Americans to believe.

" When there is more than one medical option, people mistakenly think

that the more aggressive procedure is the best, " says Annette M. Cormier

O'Connor, senior scientist in clinical epidemiology at the Ottawa Health

Research Institute. The message flies in the face of America's

infatuation with the latest advances. " As a nation, we always want the

best, the most recent technology, " explains Dr. Joe Thompson, health

adviser to Arkansas Governor Mike Huckabee. " We spend a huge amount

developing it, and we get a big increase in supply. " New radiation

machines for cancer or operating rooms for heart surgery are profit

centers for hospitals, for instance (see BW Online, 07/18/05, " Is Heart

Surgery Worth It? "

http://www.businessweek.com/magazine/content/05_29/b3943037_mz011.htm?chan=tc).

Once a hospital installs a shiny new catheter lab, it has a powerful

incentive to refer more patients for the procedure. It's a classic case

of increased supply driving demand, instead of the other way around.

" Combine that with Americans' demand to be treated immediately, and it

is a cauldron for overuse and inappropriate use, " says Thompson.

 

The consequences for the U.S. are disturbing. This nation spends 2-1/2

times as much as any other country per person on health care. Yet

middle-aged Americans are in far worse health than their British

counterparts, who spend less than half as much and practice less

intensive medicine, according to a new study. " The investment in health

care in the U.S. is just not paying off, " argues Gerard Anderson,

director of the Center for Hospital Finance & Management at Johns

Hopkins' Bloomberg School of Public Health. Speaking not for

attribution, the head of health care at one of America's largest

corporations puts it more bluntly: " There is a massive amount of

spending on things that really don't help patients, and even put them at

greater risk. Everyone that's informed on the topic knows it, but it is

such a scary thing to discuss that people are not willing to talk about

it openly. "

 

Of course, there are plenty of areas of medicine, from antibiotics to

early detection of certain tumors, where the benefits are huge and

incontrovertible. But if these effective treatments are black and white,

much of the rest of medicine is a dark shade of gray. " A lot of things

we absolutely believe at the moment based on our intuition are

ultimately absolutely wrong, " says Dr. Paul Wallace, of the Care

Management Institute.

 

The best way to go from intuition to evidence is the randomized clinical

trial. Patients with a particular condition are randomly assigned to

competing treatments or, if appropriate, to a placebo. By monitoring the

patients for months or years, doctors learn the relative risks and

benefits of the treatment being studied.

 

But such trials take years and cost many millions of dollars. By the

time the results come in, science and medicine may have moved on, making

the findings less relevant. Moreover, patients in a clinical trial

usually aren't representative of real people, who tend to have complex

combinations of diseases and medical problems. And patients often don't

stick with the program.

 

Such difficulties are highlighted by an eight-year study of low-fat

diets that cost upward of $400 million. Most subjects failed to stick to

the low-fat regimen, making it tough to draw conclusions. In addition,

the study failed to take stock of different kinds of fats, some of which

are now known to have beneficial effects. Many trials fall into similar

traps. So it's no surprise that up to one-third of clinical studies lead

to conclusions that are later overturned, according to a recent paper in

JAMA.

 

Even when common treatments are proved to be dubious, physicians don't

rush to change their practice. They may still firmly believe in the

treatment -- or in the dollars it brings in. And doctors whose oxen get

gored sometimes fight back. In 1993, the federal government's Agency for

Health Care Policy & Research convened a panel to develop guidelines for

back surgery. Fearing that the recommendations would cast doubt on what

the doctors were doing, a prominent back surgeon protested to Congress,

and lawmakers slashed funding for the agency. " Congress forced out the

research, " says Floyd J. Fowler Jr., president of the Foundation for

Informed Medical Decision Making. " It was a national tragedy, " he says

-- and not an isolated incident. The agency's budget is often targeted

" by special interest groups who had their specialty threatened, " says

Arkansas' Dr. Thompson.

 

With proof about medical outcomes lacking, one possible solution is

educating patients about the uncertainties. " The popular version of

evidence-based medicine is about proving things, " says Kaiser's Wallace,

" but it is really about transparency -- being clear about what we know

and don't know. " The Foundation for Informed Medical Decision Making

produces booklets, videotapes, and other material to put the full

picture in the hands of patients. Health Dialog markets the information

to providers and companies, addressing back pain, breast cancer, uterine

fibroids and bleeding, coronary heart disease, depression,

osteoarthritis, and other conditions.

 

In studies where one group of patients hears the full story while other

patients simply receive their doctors' instructions, a key difference

emerges. The well-informed patients opt for more invasive, aggressive

approaches 23% less often, on average, than the other group. In some

cases, the drop is much bigger -- 50% to 60%. " Patients typically don't

understand that they have options, and even if they do, they often

wildly exaggerate the benefits of surgery and wildly minimize the

chances of harm, " says Ottawa's O'Connor, a leader in this field of

so-called decision aids.

 

Eddy's computer simulation could help more patients attain appropriate

care. His approach is to create a SimCity-like world in silicon, where

virtual doctors conduct trials of virtual patients and figure out what

treatments work. After getting funding from Kaiser Permanente in 1991,

Eddy hired a particle physicist, Len Schlessinger, who knew how to write

equations describing the complex interactions in biology. The pair

selected diabetes as a test case. In their virtual world, each simulated

person has a heart, liver, kidneys, blood, and other organs. As in real

people, cells in the pancreas make insulin, which regulates the uptake

of glucose in other cells. And as in the real disease, key cells can

fail to respond to the insulin, causing high blood-sugar levels and a

cascade of biological effects. The virtual patients come down with high

blood pressure, heart disease, and poor circulation, which can lead to

foot ulcers and amputations, blindness, and other ills. The model also

assesses the costs of treating the complications.

 

Eddy dubbed the model Archimedes and tested it by comparing it with two

dozen real trials. One clinical study compared cholesterol-lowering

statin drugs to a placebo in diabetics. After 4-1/2 years, the drugs

reduced heart attacks by 35%. The exact same thing happened in Eddy's

simulated patients. " The Archimedes model is just fabulous in the

validation studies, " says the University of Michigan's Herman.

 

STANDARD OF CARE

 

The team then put Archimedes to work on a tough, real problem: how best

to treat diabetes in people who have additional aliments. " One thing

not yet adequately embraced by evidence-based medicine is what to do for

someone with diabetes, hypertension, heart disease, and depression, "

explains Kaiser's Wallace. Doctors now typically try to treat the most

pressing problems. " But we fail to pick the right ones consistently, so

we have misdirected utilization and a great deal of waste, " he says.

Kaiser Permanente's Dr. Jim Dudl had a counterintuitive suggestion. With

diabetics, doctors assume that keeping blood sugar levels low and

consistent is the best way to ward off problems such as heart disease.

But Dudl wondered what would happen if he flipped it around, aiming

treatment at the downstream problems. The idea is to give patients a

trio of generic medicines: aspirin, a cholesterol-lowering statin, and

drugs called ACE inhibitors.

 

Using Archimedes and thousands of virtual patients, Eddy and

Schlessinger compared the traditional approach with the drug

combination. The model took about a half-hour to simulate a 30-year

trial, and showed that the three-drug combination was " cost- and

life-saving, " says Kaiser's Wallace. The benefits far surpassed " what

can be achieved with aggressive glucose control. " Kaiser Permanente docs

switched their standard of care for diabetes, adding these drugs to

other interventions. It is too early to declare a victory, but the

experience with patients seems to be mimicking Eddy's computer model.

" It goes against our mental picture of the disease, " says Wallace. But

it also makes sense, he adds. " Cardiovascular disease is the worst

complication of diabetes -- and what people die of. " Eddy readily

concedes that this example is a small beginning. In its current state of

development, Archimedes is like " the Wright brothers' plane. We're off

the sand and flying to Raleigh. " But it won't be long, he says, " before

we're offering transcontinental flights, with movies. "

 

The modeling approach allows each of us, in essence, to have an

imaginary twin. We can use our twin to predict what our lives and state

of health are likely to be with different lifestyles and approaches to

care. Companies could create virtual clones of each employee, predicting

what will occur with current care or with added prevention or treatment

programs. " They can see what happens to such things as the complications

suffered by diabetics, the lost time from work, the amount of angina or

the rate of heart attacks, the number of deaths, and the cost of new

employees if one dies, " Eddy explains. " Our mission is that in 10 years,

no one will make an important decision in health care without first

asking: `What does Archimedes say? " '

 

Curing Without Cutting

 

Can you trust your doctor's recommendation to have surgery for an aching

back? Make sure you have all the facts. Evidence says surgery does not

fix the problem over the long term any better than time, physical

therapy, and exercise. Indeed, says University of North Carolina's Dr.

Nortin M. Hadler, pain clinics are full of people who have had back

surgery and now are worse off. Geographic data suggest that such

procedures may be a fad. In people with identical symptoms, operations

like spinal fusion are performed 20 times as often in some parts of the

U.S. as in others. " Spinal fusion is the most variable condition in all

of medicine, " says Dr. James N. Weinstein, editor of Spine magazine and

chair of orthopedic surgery at Dartmouth.

 

Leave Those Ears Alone

 

In the 1950s, kids routinely got their tonsils taken out. Then

physicians such as Dr. Jack L. Paradise of the University of Pittsburgh

School of Medicine showed that the procedure brought no benefits to most

children. In a study published last August, Paradise took on another

common treatment: implanting tubes to drain the fluid in children's ears

-- thought to hamper hearing and slow language development. Children

with fluid do tend to have more speech problems. But Paradise believes

the two conditions have a common cause: poor living conditions.

" Medicine is fraught with error when people assume correlation is

causality, " he says. So Paradise did a study of 6,000 babies. By age

three, 429 had persistent fluid in their ears. Half got ear tubes, the

other half didn't -- and there was no difference in outcomes between the

two groups. Paradise's advice to parents of such kids: " Don't just do

something. Sit there. " Many doctors still perform the surgery, however.

" People are reluctant to believe our results, " Paradise says. Why? " You

get paid for operating and not paid for not operating. "

 

Bypass That Operation?

 

Each year doctors perform 400,000 bypass surgeries and 1 million

angioplasties, where mesh tubes are placed in diseased arteries to hold

them open. While most people believe that such surgery is life-saving,

the available data say otherwise. Except for about 3% of people with

severe heart disease, treatment with drugs alone works just as well to

extend life and prevent heart attacks as surgery does. " Cardiologists

like to open up arteries, " says Dr. David D. Waters, chief of cardiology

at San Francisco General Hospital. " But there is no evidence that

opening up chronically narrowed arteries reduces the risk of heart

attack. " Harvard Medical School's Dr. Roger J. Laham figures that at

least 400,000 angioplasties a year are unnecessary. " I'm sure we are way

overtreating our patients, " he says. Surgery carries big risks, such as

mental declines after bypass operations. The overuse is exacting a big

toll on individual patients and the health-care system, argue such

experts as Dr. Nortin M. Hadler, professor of medicine at the University

of North Carolina at Chapel Hill.

 

A Lumpectomy May Do It

 

For Jeanine Whitney, the diagnosis of breast cancer last June was bad

enough. But when her doctor told her that her best chance was an

immediate mastectomy, " I cried for 24 hours. I felt that part of my

womanhood would have been taken, " says Whitney, who works at an air

conditioner factory in Rushville, Ind. Her employer, American Standard

Cos., had a program to provide workers with unbiased information about

the risks and benefits of potential treatments. Thanks to the program,

Whitney learned that there was no evidence that a mastectomy would have

a better outcome than a lumpectomy, provided the tissue around the lump

was clear of cancer. Twenty years after treatment, the outcomes were the

same, according to studies. " It was a total surprise, " she recalls. She

requested a lumpectomy, which was carried out in July, followed by seven

weeks of radiation and six of recovery. Now, Whitney is grateful that

she was able to get the information she needed to buck her doctor's

recommendation. If Whitney had had to make a decision without that, she

says she would have " ended up in the psychiatric ward. " Treatments are

based largely on rules and traditions, not scientific evidence

 

" I've spent 25 years proving that what we lovingly call clinical

judgment is woefully outmatched by the complexities of medicine, " says

Dr. David Eddy, heart surgeon turned mathematician and health-care

economist.Think about the implications for helping patients make

decisions " Go to one doctor, and get one answer. Go to another, and get

a different one. " Or think about expert testimony. " You don't have to

hire an expert to lie. You can just find one who truly believes the

number you want. "

 

 

http://www.businessweek.com/magazine/content/06_22/b3986001.htm

 

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