The U.S. Food and Drug Administration (FDA) has just made it easier for patients to get clozapine, the only drug approved for treatment-resistant schizophrenia.
The agency has removed a long-standing requirement for patients to submit lab test results showing healthy levels of white blood cells known as neutrophils in order to receive prescriptions for clozapine.
The requirement was originally put in place to prevent a rare side effect known as severe neutropenia: a dangerously low white blood cell count that makes people prone to life-threatening infections. The FDA said lifting the lab test requirement could help expand access to schizophrenia treatment.
“This is a major advance, as it removes a significant barrier to clozapine use,” says Frederick Nucifora, DO, PhD, an associate professor and the director of the adult schizophrenia clinic at Johns Hopkins School of Medicine in Baltimore.
“This change ensures that patients will not be denied medication simply because they missed a blood draw due to illness, transportation issues, or other barriers,” Dr. Nucifora says. “As a result, more patients will be able to start and stay on clozapine without unnecessary disruptions, improving their stability and overall quality of life.”
Clozapine Plays an Important Role in Schizophrenia Treatment
Many people with schizophrenia can manage their condition with antipsychotics. But about 1 in 3 of them are considered treatment-resistant because they don’t respond enough — or at all — to these drugs.
Clozapine is the only approved option for these treatment-resistant patients.
“Clozapine treatment can enable patients to stay out of the hospital, complete their education, and even maintain employment,” Nucifora says. “Without clozapine, these individuals may experience relentless psychotic symptoms, leading to frequent hospitalizations, severe functional impairment, and an alarmingly high suicide risk — up to 10 percent.”
Serious Risks of Untreated Schizophrenia
The risks of untreated schizophrenia far outweigh the risk of severe neutropenia with clozapine, Nucifora adds. One recent study of 974 adults with schizophrenia found no cases of severe neutropenia among clozapine users.
“Clozapine is associated with a very low risk of severe neutropenia,” says Deanna Kelly, PharmD, a psychiatry professor at the University of Maryland School of Medicine in Baltimore.
Periodic Blood Testing Will Still Be Needed
People who take clozapine will need periodic blood testing to monitor for signs of neutropenia, but the FDA change to the prescribing rules removes a cumbersome paperwork trail that often resulted in delayed care, Dr. Kelly says.
Lab tests are most important during the initial weeks of clozapine treatment, when the risk of neutropenia is higher than it is after longer periods of treatment, says John Kane, MD, a psychiatry professor at the Donald and Barbara Zucker School of Medicine at Hofstra Northwell in Hempsted, New York.
“The risks in the first six months justify the weekly monitoring,” Dr. Kane says. “Many experts believe that monitoring could be discontinued after one or two years.”
Even if the new FDA approach to clozapine doesn’t immediately lead to a surge in prescriptions, the potential to access this drug more easily may be truly life-altering for patients, says David Goldsmith, MD, an associate professor of psychiatry at Emory University School of Medicine in Atlanta.
“Many patients I work with have remarkable responses to clozapine, including stopping frequent hospitalizations, overall symptomatic improvements, and true functional gains including getting back to work, school, and living independently,” Dr. Goldsmith says. “The number of patients who are able to get access to clozapine is woefully low, and I strongly believe that if we increased access to clozapine, we could make remarkable changes to people’s lives and the psychiatric system overall.”
EDITORIAL SOURCES
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Brandt AS et al. The Timing and Severity of Clozapine-Associated Neutropenia in the US: Is the Risk Overstated? Schizophrenia Research. August 29, 2024.
Emily Kay Votruba has copyedited and fact-checked for national magazines, websites, and books since 1997, including Self, GQ, Gourmet, Golf Magazine,Outside, Cornell University Press, Penguin Random House, and Harper's Magazine. Her projects have included cookbooks (Padma Lakshmi's Tangy Tart Hot & Sweet), self-help and advice titles (Mika Brzezinski's Know Your Value: Women, Money, and Getting What You're Worth), memoirs (Larry King's My Remarkable Journey), and science (Now You See It: How the Brain Science of Attention Will Transform How We Live, Work, and Learn, by Cathy Davidson). She started freelancing for Everyday Health in 2016.
Lisa Rapaport is a journalist with more than 20 years of experience on the health beat as a writer and editor. She holds a master’s degree from the UC Berkeley Graduate School of Journalism and spent a year as a Knight-Wallace journalism fellow at the University of Michigan. Her work has appeared in dozens of local and national media outlets, including Reuters, Bloomberg, WNYC, The Washington Post,Los Angeles Times, Scientific American, San Jose Mercury News, Oakland Tribune, Huffington Post, Yahoo! News, The Sacramento Bee, and The Buffalo News.
