A field guide to pointless medical research

A few years ago I wrote about a five-year-old Portland girl named Katie who was born with Smith-Lemli-Opitz syndrome, a rare disorder that causes severe developmental delays and a spectrum of birth defects.  I keep thinking about what her mother Kathy told me about her situation as a parent:

What’s been most difficult [she said] “is having no proven, real treatment out there for her, and having it all be in studies. But also it’s not knowing what to expect for her future, not having the answers you would have for a more common disease — whether she could be independent, what she may or may not be able to do as she grows into an adult.”

Although the syndrome’s root cause has been known since 1993 – and several major medical centers including Oregon Health & Science University formed a consortium in 2010 to expedite studies – researchers still have not published a randomized, controlled clinical trial of any treatment.  Only six small, nonrandomized studies appear in the National Library of Medicine’s PubMed database, and they don’t provide much in the way of answers.

The “race for a cure” is in many ways a fantasy. If only. Medical research is at best a precarious, plodding mountain climb with many contestants taking dead-end routes.  Some of the most acclaimed drug discoveries of the past decade took a median of 24 years from conception to become established as clinically useful treatments, according to an analysis reported a few years ago in the journal Science. The authors zeroed in on 101 studies published between 1979 and 1983 that were highly regarded and made strong claims about potential to cure illness. A decade later, only five had led to a practical treatment, and only one that was widely used. And 40% of the discoveries celebrated as “breakthroughs” later proved partially or completely ineffective. Among them: hormone replacement for preventing stroke and heart disease, and vitamin E for preventing heart disease.

At its worst, medical research is an enterprise that wastes tens of billions of dollars a year on studies that are repetitive, irreparably biased, kept hidden by industry sponsors, or designed with no regard to the desires of people who have to live with serious illness. I’m disgusted but no longer surprised by the estimate that 85% of the money poured into medical research is wasted,  which The Lancet reported in 2009.  The esteemed British journal has now published a major series of papers on the problem of waste in medical research, and I hope it will lead to a watershed. The series is freely accessible online (with registration) and worth reading in its entirety. Here are some of the highlights, which I’ve amplified with my observations and additional examples. Full article

§

Behind execution of Gary Haugen, controversy swirls around the drugs used and who administers them

The lethal injection era began in 1977, a year after the U.S. Supreme Court lifted a moratorium on the death penalty. Oklahoma lawmakers wanted a more humane and less expensive alternative to the electric chair or gas chamber. The head of anesthesiology at Oklahoma Medical School recommended the use of a short-acting barbiturate anesthetic in combination with a paralytic agent. Oklahoma’s chief medical examiner recommended the addition of a third drug, potassium chloride, used in bypass surgery to temporarily stop the heartbeat. Until recently, the drugs cost about $200 per execution but rose to more than $1,000 with companies such as Lundbeck cutting off the supply.

Oklahoma spent no time or money to study the effects of the three-drug combination. Nevertheless, it rapidly caught on in the U.S. Death penalty laws in Oregon and other states use nearly the same language Oklahoma used to specify how lethal injections are done, notes Fordham University law professor Deborah Denno. “By virtue of coming up with a method of execution that makes an inmate look serene, comfortable, and sleeping during the death process, the death penalty in this country was rescued…” Full article

§

Millions for rural health miss mark

Ashland plastic surgeon Scott Young offers face-lifts, liposuction and breast augmentation in a prosperous town known for its annual Shakespeare festival and its pricey real estate. The 52-year-old physician owns an 11,000-square-foot, 11-bedroom house and “spiritual retreat” with wraparound views of the scenic cliffs on his 1,700-acre spread a few miles north of town.

Young nevertheless qualified for financial aid as a rural doctor under a state program that awarded him a total of more than $21,000 in 2004 and 2005, state records show. Any licensed physician in Ashland – or any other location at least 10 miles from a city of 30,000 or more – is eligible.

The three-year-old program was supposed to help financially struggling doctors, especially those who deliver babies, survive in rural Oregon. With a projected price tag of $25 million, it is the state’s largest expenditure on efforts to improve access to rural doctors.

But an analysis by The Oregonian shows that the program is sending millions of dollars to specialists in places where doctors abound and residents have a choice of hospitals. Of more than 1,100 doctors who met the criteria to qualify for support in the program’s first two years, fewer than 200 worked in communities with serious unmet medical needs, as defined by the state’s Office of Rural Health. More than 200 recipients worked in cities or suburbs within 15 miles of two or more hospitals. Full article

§

Doctor and invention outlast jeers and threats

John Epley’s stooped shoulders and gentle eyes gave him a turtlish look. He wore a thickly knotted necktie and wrinkled sport coat. No amount of combing could tame the stubborn cowlick in his short hair.

His audience of ear surgeons muttered skeptically and shook their heads. Few at the October 1980 meeting in Anaheim, Calif., believed Epley’s claim to have developed a cure for the most common cause of chronic vertigo. In any given year, tens of thousands of people seek treatment for the disorder’s strange, crippling attacks. Provoked by a casual tilt or turn of the head, the victim’s surroundings whirl. The eyeballs twitch involuntarily. Nausea overwhelms the senses. On-and-off bouts may torment a sufferer for years.

Physicians were baffled. The best they could offer as treatment was a drastic last resort: surgically destroying nerves to the inner ear, impairing patients’ balance and possibly their hearing.

Epley proposed an elegant alternative.

His talk concluded with a demonstration, a young woman acting as his patient. Epley and his research collaborator, audiologist Dominic Hughes, began by tilting the woman flat on her back, her head hanging over the end of an exam bench. Hughes cradled her head in his hands and rotated it about 45 degrees to his right, then he and Epley rolled the woman’s head and shoulders back to the left in a counterclockwise move that ended with her face down. In a final move, Hughes and Epley lifted the woman to a sitting position.

And that was it… Full article

§

In health care, sometimes less is really more

The appeal of computer-aided cancer screening seems obvious. A sophisticated program searches X-ray images for subtle signs of breast cancer and flags them for confirmation by a radiologist. Developers of the technology claim it can improve the accuracy of standard mammography, which misses up to 20 percent of tumors.

But the largest independent study of computer-aided mammography found that it not only failed to increase cancer detection rates significantly, it also boosted the number of false-positive results and prompted a 20 percent increase in the rate of invasive biopsies.

The finding is just one example of a widespread problem in the health care system: the tendency to embrace new technology without waiting for proof that it’s better than older, cheaper, time-tested solutions.

“Everybody stands to make a lot of money using the newest drugs and devices,” says Dr. John Santa, director of the Consumer Reports Health Ratings Center in Yonkers, N.Y. “The whole business model is oriented toward rewarding the new and different.” Full article

§

Baby seems caught in hospital rivalry

Six months into her pregnancy, 22-year-old Melissa Peterson learned that her unborn son had a life-threatening heart defect.

A specialist at Oregon Health Sciences University in Portland told the Roseburg woman that without an operation soon after birth, her baby boy would die.

The pediatric cardiologist offered three choices: forgo surgery and provide comfort care until death; register for a heart transplant operation and hope for a suitable donor; or send the baby soon after birth to a surgeon in San Francisco for a risky and complicated operation known as the Norwood procedure.

What the OHSU doctor neglected to say, according to Peterson, was that a rival group of cardiologists and heart surgeons across town at Legacy Emanuel Children’s Hospital regularly performs the Norwood procedure, attracting referrals from as far away as Alaska.

“I was very upset when I found out. I just think I should have been given the choice,” said Peterson, who is unmarried and supports herself on a minimum wage job at a shoe store.

Her story highlights how murky the process of getting a medical referral can be for patients, whose choices may be limited by behind-the-scenes competition among rival doctors and hospitals. And because of medical providers’ long-standing reluctance to release performance results, such as mortality and infection rates, referral choices are often based on best guesses and subjective judgments about reputation rather than objective evidence, consumer groups contend. Full article