Martin Shkreli this month became the face of what’s wrong with the U.S. drug industry.
A congressional committee hauled in the former chief of Turing Pharmaceuticals and asked him to justify boosting the price of a lifesaving drug by 5,000 percent, charging $750 per pill for a drug that had sold for $13.50.
Shkreli declined to answer. The 32-year-old, dubbed by the New York media as the “teen wolf of Wall Street,” only smirked.
“It’s not funny, Mr. Shkreli,” a congressman told him during the hearing. “People are dying.”
You may never need the drug Shkreli is selling — Daraprim, which treats a disease called toxoplasmosis. But Summit County physicians and pharmacists said it’s important that patients know that the drug industry has changed.
Skyrocketing prices of some drugs are just a part of it. Hundreds of common drugs doctors once depended on aren’t available because of production problems, shortages of raw materials or myriad other issues that have no easy solution.
Officials at Summa Health System and Akron Children’s Hospital said this month that none of their patients has been adversely affected by the shortages so far.
“But unless action is taken by our government, unless there’s a change, it’s inevitable,” said Dr. Jeffrey Hord, director of Children’s Hospital’s division of hematology-oncology.
Production problems
About 25 miles north of Akron sits Ben Venue Laboratories. If Shkreli is the face of outrageous drug pricing, Ben Venue in recent years has been the symbol of why drug shortages happen.
In November 2011 — following complaints about metal particles ending up in Doxil, a chemotherapy drug Ben Venue made — the federal Food and Drug Administration released a scathing inspection report that revealed problems in the Bedford drugmaker’s production areas.
Among other things, FDA inspectors found rusty tools, mold on walls and a small barrel that was apparently used by employees as a toilet. Tests revealed the barrel was filled with urine.
Ben Venue voluntarily closed to clean up its act, which may have made inspectors happy. But the closure caused sudden shortages of many drugs, causing a crisis in the pediatric oncology community.
“We sat on pins and needles,” said John Lepto, director of pharmacy at Akron Children’s.
He feared the hospital would run out of Cytarabine, the only chemotherapy drug that works on some kinds of cancer in children.
Trouble started in 2010, when there were three U.S. manufacturers of the lifesaving drug, including an arm of Ben Venue. One of the three had manufacturing issues and recalled the Cytarabine it made.
At the time, Ben Venue’s Bedford Laboratories produced about 50 percent of the Cytarabine used by the U.S.
To cover the shortage, it bumped up production. And when Ben Venue closed, it took 84 percent of the U.S. supply chain of the pediatric cancer drug with it, according to published reports at the time.
“We’d place back orders and held our breath … it was very touchy at times, but we never ran out,” Lepto said.
The shortage eased, but only after the FDA took unusual action: It quickly permitted imports of Cytarabine from Europe.
Such an approval process can often take years and isn’t worth it for overseas manufacturers because of the cost of testing to prove to the FDA that their drugs are safe and effective.
The story of Ben Venue continues to resonate with the medical community, because it illustrates how fragile the U.S. drug supply can be.
Most shortages are caused by manufacturing and quality problems, according to the FDA.
“And what you’re seeing in drugs is happening in other industries,” said Chris Snyder, a Cleveland Clinic pharmacist whose sole job is to track drug shortages and help manage them.
The FDA’s crackdown at Ben Venue is akin to what’s happened with the Environmental Protection Agency and Volkswagen after federal officials determined the automaker had cheated on emission results, he said.
Auto buyers can always turn to a Ford, Honda or some other car.
But doctors, Snyder said, sometimes don’t have that option.
Over the past decade, there’s been a consolidation among U.S. drugmakers. Often, there’s only one or two drugmakers producing a particular medication. If a production line goes down, there’s no way to quickly pick up the slack.
So what do doctors do?
Drug rationing
The Cleveland Clinic recently allowed a reporter from the New York Times, who is also a physician, to talk to its staff about making decisions in an era of drug shortages.
The newspaper story opened with what was happening in operating rooms on the hospital’s main Cleveland campus because of a national shortage of aminocaproic acid.
A Cleveland Clinic anesthesiologist routinely relied on aminocaproic acid to prevent hemorrhages during open-heart surgeries, the story said, but determined the hospital only had enough of the drug for patients at the highest risk of bleeding complications.
Doctors used “military-style triage” on the rest, the story said, and patients never knew they weren’t getting the preferred treatment because they were asleep during surgery.
How often rationing happens in patient care here and across the country is unclear.
Drug shortages are the new normal, according to more than a dozen doctors, pharmacists and educators interviewed for this story.
Doctors and pharmacists at Summa Health and Akron Children’s said they would inform patients if their care could be compromised.
But before they reached that point, health care workers said they would search hospitals and suppliers for the needed drug, consider making the drug themselves or figure out an alternative to get patients the care they need.
Julie M. Aultman, a clinical ethicist who teaches family and community medicine at Northeast Ohio Medical University, said that may not be enough.
“They’re looking at it from risk management — whether a person has been harmed,” Aultman said. “We have to look beyond that.”
Patients may have to pay more for alternative treatments if a regular drug isn’t available. Other drugs may be less effective. There could be unknown side effects, even if an alternative drug contains the same components, said Aultman, who sits on separate ethics committees at four area hospitals.
One of those committees recently had to think through a shortage of Intravenous Immunoglobulin, or IVIG.
The FDA has approved IVIG for treatment of neuropathy. But doctors also use IVIG off-label — in a way not approved by the FDA — to treat patients with several conditions, including pediatric HIV.
Patient privacy laws prevented Aultman from giving too many details. But she said the committee wrestled with several scenarios. Could patients wait longer between dosages, maybe buying enough time for more IVIG to become available? Should neuropathy patients trump pediatric HIV patients because of the original intent of the drug?
“These are tough questions,” Aultman said.
Planning ahead
In recent weeks, the American Society of Health-System Pharmacists listed more than 160 drugs in short supply, from penicillins and heart medications to meningococcal vaccines and oncology drugs. But on Feb. 19, Summa didn’t have a single drug in short supply, said John Feucht, Summa’s system director of pharmacy services.
“We’re trying to be proactive,” he said. “We’ve come up with a strategy and a procedure.”
Summa — like Akron Children’s and the Cleveland Clinic — has increased, sometimes doubled, how many drugs it stores on-site.
It also tracks shortages with spreadsheets, often meeting daily or weekly to strategize supply and alternatives.
Most patients will never be harmed by a shortage, said Dr. Erik Steele, chief medical officer of Summa.
“I don’t want patients worried that what’s in the bag isn’t the right thing,” Steele said, “or worrying that there’s some hidden problem and you’re getting something less.”
Patients’ wallets are much more likely to be hurt by the shortages and by other problems in the system.
Martin Shkreli’s small pharmaceutical company — which overnight jacked up the price of a 62-year-old drug by 5,000 percent — isn’t the only business cashing in on reliable, cheap drugs.
Last year, another small drug company boosted the price of the generic drug to treat pinworms — a common parasitic infection among U.S. children — 15-fold, he said.
“I once was able to tell a mom to get something very affordable to help her child,” Steele said. “Now [those pills] basically cost as much as rent.”
Neither the FDA nor other federal agencies have authority to interfere with drug pricing or how much of a certain drug is made, if it’s made at all.
But Steele agreed with Akron Children’s Hord: The federal government must find ways to intervene or there’s no fixing America’s broken drug system.
“If the government is for anything, it’s for resolving this kind of thing.”
Drug prices are a concern for everyone, he said, but don’t fret about shortages.
“If you’re taking your medications, wearing your seat belt, getting your mammogram,” he said, “this can be pretty low on your list of worries.”
Amanda Garrett can be reached at 330-996-3725 or agarrett@thebeaconjournal.com.