Drug Shortage Leaves Patients Without Immune-Disorder Treatment
A shortage of immune globulin has forced hospitals to ration treatments.
By Peter Loftus, The Wall Street Journal, August 9, 2019
A shortage of a versatile medicine used to treat immune disorders and other diseases has forced U.S. hospitals and infusion clinics to suspend treatment for many patients.
The medicine, immune globulin, contains antibodies harvested from plasma, a component of blood. The injected product helps people with compromised immune systems fight off infections, and treats certain muscle and nerve disorders. The drug’s shortage increases the risk of infection for patients and the amount of pain they are suffering, doctors say.
Recently, many hospitals and infusion clinics have received less immune globulin, or IG, than they need. Some have started to ration it, prioritizing it for patients who need it to stay alive and canceling infusions for patients deemed to have non-life-threatening conditions. The shortage has gotten to an acute status, said Michelle Vogel, vice president of patient advocacy and provider relations at CSI Pharmacy, a specialty pharmacy.
IG manufacturers, including Takeda Pharmaceuticals Co. and CSL Ltd., say there has been increased demand for IG. They cite higher rates of diagnosis of conditions that IG has long treated, plus new uses for the drug. The global market for these drugs was $11 billion in 2018, projected to rise to $17 billion in 2023, according to a recent Takeda presentation to investors.
The companies say they are boosting production. Still, supply snags have emerged. Takeda executives on a recent earnings call noted delays in shipments of certain IG products.
Pfizer Inc. notified customers in June they would receive minimal or no supply of the immune globulin Octagam through July, which Pfizer attributed to third-party manufacturing delays. Pfizer referred questions to the manufacturer, Octapharma, whose spokesman declined to comment.
Shortages of medicines including cancer drugs, saline and the EpiPen injection for allergic reactions have plagued the U.S. health system in recent years. Insufficient production capacity, manufacturing-quality problems and quirks in the reimbursement system for some drugs are among the causes.
As of June 30, there were 282 active U.S. drug shortages, up from 224 a year earlier, according to the University of Utah Drug Information Service.
Cincinnati Children’s Hospital in late June sent letters to about 150 families of patients who receive regular IG infusions informing them the hospital would suspend or reduce treatment for some patients.
The hospital said it was necessary to conserve supply for patients with life-threatening conditions, including people born with primary immunodeficiency, a group of rare diseases that impair the immune system, leaving patients at significant risk of infection if not treated, said Derek Wheeler, chief of staff at the hospital.
The hospital isn’t providing IG to some patients who can safely forgo it. There is an alternative treatment, for instance, for those with Guillain-Barre Syndrome, in which the immune system attacks nerves, Dr. Wheeler said.
“We’re essentially rationing to the patients with the greatest need,” he said.
Massachusetts General Hospital has canceled many infusion appointments since June, said Dr. Paul Biddinger, director of the hospital’s center for disaster medicine. Initially, the hospital was down to about 10% of its normal IG supply. It has received more, but in the coming months it doesn’t expect to get more than half of its normal supply, he said. The hospital expects the shortage to last into 2020.
“For the patients affected, it’s been very significant,” Dr. Biddinger said. “It puts them at elevated risk of infection. It puts them with increased severity of symptoms.”
Zachariah Otto of Aurora, Colo., started getting IG infusions in April to treat autonomic neuropathy, a condition that causes nerve damage and limits mobility. The 23-year-old said the treatment helped with muscle control and relieved pain. He was scheduled to get another dose in late July, but a week before the appointment the infusion clinic canceled because of the shortage. He is now on a waiting list for treatment.
“I understand the situation, but I’m definitely not looking forward to going back to having more pain,” he said.
Angela Swan’s 3-year-old daughter, Farrah, has been taking IG to treat juvenile dermatomyositis, a disease that causes skin rashes and muscle weakness. But after the last infusion in July, her rheumatologist said treatment would have to be suspended due to the shortage. Her daughter takes other medications, but “my concern is the risk of her getting an infection now and it causing a flare because she doesn’t have IVIG [intravenous immune globulin] to keep her symptoms at bay,” said Ms. Swan, who lives in Belfair, Wash.
Takeda said increased demand “combined with the length of time it takes to produce plasma-derived products…has led to very tight supply and, in some cases, supply interruptions for certain products across the U.S. market.”
In December, Takeda advised doctors not to start new patients on its Cuvitru and Hyqvia IG products because inventories were low. In June, inventory returned to levels that could support new patients, but Takeda said it is monitoring inventory.
Takeda also is experiencing supply interruptions of its IG Gammagard Liquid, which may persist throughout 2019.
Takeda said plasma-derived drugs take seven to 12 months to produce and supply to customers, so it is tough to make quick production shifts based on demand trends. It can take more than 1,000 plasma donations to make a one-year supply for one patient, Takeda said.
Plasma collection in the U.S. continues to rise, said Amy Efantis, chief executive of the Plasma Protein Therapeutics Association, which represents manufacturers of plasma-derived therapies. Several manufacturers operate collection centers that pay people to provide plasma.
CSL said it hasn’t experienced manufacturing issues and has consistently supplied its IG products Privigen and Hizentra “at rates well ahead of the market volume growth.”
But the company said it has seen increased demand to treat primary immune deficiencies and a condition called chronic inflammatory demyelinating polyneuropathy. There also has been increased use for so-called secondary immune deficiencies, which occur when the immune system is compromised because of a factor like HIV infection or cancer treatment.