F.D.A. Expands Access to Clozapine, a Key Treatment for Schizophrenia

The Food and Drug Administration has taken a crucial step toward expanding access to the antipsychotic medication clozapine, the only drug approved for treatment-resistant schizophrenia, among the most devastating of mental illnesses.

The agency announced on Monday that it was eliminating a requirement that patients submit blood tests before their prescriptions can be filled.

Clozapine, which was approved in 1989, is regarded by many physicians as the most effective available treatment for schizophrenia, and research shows that the drug significantly reduces suicidal behavior. Clozapine is also associated with a rare side effect called neutropenia, a drop in white blood cell counts that, in its most severe form, can be life-threatening.

In 2015, federal regulators imposed a regimen known as risk evaluation and mitigation strategies, or REMS, that required patients to submit to weekly, biweekly and monthly blood tests that had to be uploaded onto a database and verified by pharmacists.

Physicians have long complained that, as a result, clozapine is grossly underutilized.

Dr. Frederick C. Nucifora, director of the Adult Schizophrenia Clinic at the Johns Hopkins School of Medicine, said he believed that around 30 percent of patients with schizophrenia would benefit from clozapine — far more than the 4 percent who currently take it.

“I have had many patients who were doing terribly, who struggled to function outside the hospital, and cycled through many medications,” he said. “If they go on clozapine, they really tend to not be hospitalized again. I’ve had people go on to finish college and work. It’s quite remarkable.”

The F.D.A.’s shift came after years of lobbying by pharmacists, clinicians and relatives of people with schizophrenia, who cataloged the cases of patients who deteriorated after losing access to clozapine.

“There were patients who ended up relapsing into psychosis, patients who ended up hospitalized, patients who became violent,” said Raymond C. Love, a professor emeritus at the University of Maryland School of Pharmacy, who helped organize the effort.

When an advisory committee to the F.D.A. reviewed the requirement at a meeting in November, he said, the agency had to expand the public docket to accommodate the number of people who wanted to speak.

“That was how much the outcry was,” he said. “There were people in tears.”

In a statement published on its website on Monday, the F.D.A. said it had determined that the testing regimen was “no longer necessary to ensure that the benefits of the medicine outweigh” the risk of severe neutropenia. Its prevalence is estimated at around 0.9 percent worldwide and decreases after the first six months of treatment.

But Dr. John M. Kane, a leading schizophrenia researcher, said he did not expect a substantial shift in prescribing as a result of the change. Even without the testing regimen, he said, many physicians are hesitant to prescribe clozapine because it requires close monitoring during the early months of treatment.

“Probably 1 in 8 patients in the U.S. who might benefit from clozapine are getting it,” said Dr. Kane, a professor of psychiatry at the Zucker School of Medicine at Hofstra/Northwell. For a portion of them, he said, the medication is “life-changing.”

Psychotic disorders like schizophrenia affect 1 percent to 3 percent of the adult population, but the disease is a crushing burden to society. The illness typically emerges in late adolescence and is characterized by psychotic symptoms, like hallucinations or delusions, as well as by social withdrawal, cognitive impairment and a lack of motivation.

Studies suggest that the risk of suicide for people with schizophrenia is at least 10 times as high as that for the general population.

Though many people manage their symptoms with the help of their families, others cycle through incarcerations, homelessness and brief hospitalizations. A 2022 analysis of the economic burden associated with the disease published in The Journal of Clinical Psychiatry estimated the yearly cost at $343.2 billion, largely from caregiving, premature mortality and unemployment.

The first antipsychotic medication, chlorpromazine, became available 70 years ago, and was followed by two waves of new treatments designed to alter dopamine pathways in the brain. But the pace of discovery slowed after that, as vast sums of research funding were devoted to investigating the genetics of the disease. Last September, the F.D.A. approved Cobenfy, the first novel antipsychotic treatment in decades.