As Life-Saving Childhood Cancer Drugs Run Short, Patients and Doctors Face Impossible Choices

While a shortage of vincristine has grabbed headlines, doctors say other drugs are in short supply too. And they fear the effects on patients.

What to Know

  • Shortages of life-saving medicines have been increasing for years, according to the Food and Drug Administration.
  • These shortages acutely affect childhood cancer patients, like Eliot Hayes, a Wayne, Pennsylvania teen who has lymphoma.
  • When shortages occur, patients suffer as doctors and hospitals are forced to ration drugs.

The nurses and doctor swarmed into the young patient's room, grabbing and pulling as they worked. Out came the IV. In came epinephrine and hydrocortisone to keep his heart pumping. 

The drug in Eliot Hayes' IV was supposed to help cure his cancer. But in this moment, it was killing him.

Soon after the medicine had started pumping into his veins, Hayes felt something was wrong. As he lay in bed, face growing redder by the second and throat quickly closing, a three-word whisper escaped his lips: "I can't breathe."

The medical staff worked quickly. Less than a minute after the episode began, the 18-year-old felt sick, but he was alive.

Eliot Hayes had just suffered anaphylactic shock as a result of an allergic reaction to one drug used to treat his B-cell lymphoblastic lymphoma. It was clear he needed another option.

Another drug, Erwinaze, was the answer -- if only the hospital could find enough of it to treat him.

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Hayes Family
Eliot Hayes was being treated for lymphoma when one of the drugs he needed was in short supply.

Jazz Pharmaceuticals, the Ireland-based company that sells and distributes Erwinaze, alerted doctors and patients in September that one of its batches had been contaminated by "particulate matter" and was in short supply.

"How is it possible? Why is there a shortage? What is going on?" Sophie Hayes, Eliot's mother, recalled thinking when she was told the treatment for her son would have to be delayed.

The Hayes family, from Wayne, Pennsylvania, experienced first-hand a problem that doctors say is too common and worsening: shortages of childhood cancer drugs, exacerbated by the dwindling number of suppliers and problems in production.

"It does make us nervous, knowing that if there is just one supplier of a given medicine, any hiccup or disruption in manufacturing would lead to a shortage that would affect patients," said Dr. Spencer Mangum, a pediatric oncologist at Nemours/Alfred I. duPont Hospital for Children in Delaware, where Hayes is being treated.

For Some, Shortages of More than 8 Years

The pediatric chemotherapy drug vincristine grabbed headlines when Teva Pharmaceuticals stopped manufacturing it in July, a move the U.S. Food and Drug Administration described as a "business decision." That left Pfizer as the sole manufacturer and caused the FDA to declare a shortage.

Teva Pharmaceuticals did not return requests for comment.

Mangum called vincristine an "an essential component to a lot of the backbone of a lot of our therapies." He added that his hospital has enough of the drug to last through the rest of the year.

Other drugs, like the Erwinaze needed for Eliot Hayes' treatment, have been in short supply for years, Mangum said.

At the request of Congress, the FDA recently studied 163 drugs that had been in short supply. Its report said drug shortages have been increasing and lasting longer, in some cases more than 8 years.

One reason is older drugs on the market, with generics that are less valuable to drug makers. Another is problems with manufacturing or product quality, which led to 62% of shortages in the drugs the FDA studied.

The "product quality problems" that have contributed to the current Erwinaze shortage have also been blamed for past shortages.

In a scathing January 19, 2017, letter to Porton Biopharma -- the Salisbury, England-based company that makes the Erwinaze distributed by Jazz Pharmaceuticals -- the FDA excoriated the company for repeatedly failing to act on violations that led to contaminations in its manufacturing facility.

"These repeated failures demonstrate that management oversight and control over the manufacture of drugs at your facility is inadequate, and that your previous corrective actions did not address persistent contamination hazards and drug quality issues," the FDA's letter read.

In a statement, a Porton spokesperson said the company has since changed some suppliers and introduced "new components" to ensure quality. The spokesperson said Porton has resolved the "historical issues" that the FDA identified and that "subsequent inspections have satisfied the regulatory authorities."

However, the spokesperson did not provide an answer about what led to the latest contamination of one of its Erwinaze batches. 

For its part, Jazz knows "it's critical to improve reliability of supply" and is working to help Porton Biopharma "overcome its manufacturing issues," said Andrew Civers-Davis, Jazz's director of global corporate communications. Civers-Davis added that Jazz is working to make Erwinaze more available and is developing other drugs to address the worldwide shortage.

He acknowledged, however, that "Until the manufacturing issues are resolved by PBL, we do expect intermittent shortage and stock out of supply through the end of 2019 and into 2020."

Rationing Life-Saving Drugs

Shortages can have an especially devastating effect on childhood cancer patients.

The FDA report noted that about 90% of children diagnosed with acute lymphoblastic leukemia, the most common type of pediatric cancer, are curable. However, the agency added, most of the drugs used to treat this form of leukemia are older. Between 2009 to 2019, nine out of 11 of those drugs were "in and out of shortage."

When shortages occur, they can create a host of problems for patients and hospitals, which are forced to compete with one another. In these instances, competition comes in the form not necessarily of who can pay the most money, but "who can get in line quickest and who can grab that stock," Mangum said.

"It becomes like a rat race for who is going to have stocks to actually protect the kids," Sophie Hayes said.

This reality creates a dilemma because hospitals must order drugs ahead of time based on the need for existing patients and estimates of future patients. These medical institutions, then, have to make sure they have enough of a drug to provide care to their own patients while trying to ensure they don't overstock knowing that other hospitals and patients are also in need, according to Mangum.

Worse yet is the effect on patients like Hayes.

"Unfortunately, there are times that we have to resort to rationing of chemotherapy medicine," Mangum said.

"That's a really difficult decision to make, and it's heart-wrenching to have to have those types of discussions," he added.

The fear is that "patients with curable cancer might not be cured simply because the drug wasn't available," he said.

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Frédérique Pottier
Eliot Hayes rests on his mother, Sophie, during treatment.

In Hayes' case, he had to wait about three weeks between when he found out he needed Erwinaze and when he actually got "lucky" and received his dose, his mother said. The only reason he finally got the medicine was because another patient had an allergic reaction.

"The unhappiness of somebody is what becomes your happiness," Sophie Hayes said.

But by this point, so much time had elapsed that Eliot Hayes had to be administered two treatments of Erwinaze back-to-back, meaning thrice-weekly doses for four weeks instead of the normal two.

After each dose, ammonia in his body continued to accumulate, a side effect of the treatment. He continued to feel worse. But because he had waited so long, he had to skip the usual month-long break between treatments and trudge ahead.

By the time the third treatment began, the ammonia running through his veins landed him in the intensive care unit for three days, where he was put on a regimen of laxatives and fluids. "The whole point was to try and flush the ammonia out of my blood," Hayes said.

He recovered, but the ups and downs of his treatment have taken their toll.

He's different than he was when treatment began earlier this year. In the first month of treatment, Hayes recalled, he was eating about 7,000 calories a day and still lost about 40 pounds, the muscles fading from the soccer player's legs. His once-slim face became swollen and his full head of hair disappeared.

"I started to not be able to recognize myself in the mirror at one point," he said. "I've had moments where I've felt like I literally can't do anything. I just want to sleep the whole time."

In a cruel twist of fate, the Erwinaze shortage almost forced him to go back to the first drug that almost killed him. He would have had to be administered the medicine in small doses over six hours in the intensive care unit, medical staff at the ready, hoping that he didn't go into shock once more.

He and his mother wrestled for days with the decision, but in another stroke of luck, Nemours procured more Erwinaze.

Other patients, however, aren't so lucky. According to the Jazz Pharmaceuticals website, its Erwinaze is "currently out of stock," and the FDA is grappling with how to prevent such shortages.

The Food and Drug Administration Safety and Innovation Act, passed in 2012, requires drug manufacturers to notify the FDA whenever they make moves that might cause a shortage. However, the law only does so much and the FDA, in its recent report, acknowledges that more needs to be done.

Among the proposed solutions include the incentivizing of companies to make less profitable drugs, creating a system that rates drug manufacturers on their quality management to incentivize them to make improvements and lengthening expiration dates on medicines to ensure shortages aren't exacerbated by the tossing out of drugs "because they exceed a labeled shelf life based on unnecessarily short expiration dates."

However, it remains unclear how, if, or when those proposals may be put into practice.

In the meantime, patients like Hayes remain trapped in the current system.

The teen's life has been upended by the cancer, but he hopes to bounce back. This year, he had to forgo his acceptance to Durham University in Newcastle, England, in order to receive treatment. Given the circumstances, the university agreed to defer his acceptance.

By this time next year, he hopes to be studying science at Durham. His mother hopes to move with him "at least at the beginning." 

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Hayes Family
Eliot Hayes taking a break from treatment to play with a dog.

Whether Sophie Hayes is out there or not, the family is heartened. They've heard the hospital in Newcastle has a good oncology department, and Hayes' treatments will continue.

But he's conscious of another grim reality: he's struggled through the Erwinaze shortage and he'll possibly have to struggle again. For the next two years, treatment will require vincristine -- if he can get it.

"Not knowing that it might not be available is quite worrying, and it's frustrating as well," Hayes said.

Meanwhile, his mother is already thinking of how her son and family will traverse the road ahead.

"We tend to go through life planning, hoping for the best, and then adjusting," Sophie Hayes said.

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