The Food and Drug Administration has taken an important step to expanding access to the antipsychotic clozapine, the only drug approved for treatment-resistant schizophrenia, among the most devastating mental illnesses .
The agency announced Monday that it was eliminating the requirement that patients submit blood tests before their prescriptions are met.
Clozapine, approved in 1989, is considered by many physicians as the most effective treatment for schizophrenia, and studies have shown that the drug significantly reduces suicidal behavior. Clozapine is also associated with a rare side effect called neutropenia, and a decrease in white blood cell count can be life-threatening in its most severe form.
In 2015, federal regulators imposed a regimen known as risk assessment and mitigation strategies, or REMS, where patients submit to weekly, biweekly, and monthly blood tests, upload to a database, and verify by a pharmacist. It didn't.
Doctors have long complained that clozapine is ineffective as a result.
Dr. Frederick C. nucifora, director of the Johns Hopkins School of Medicine's Adult Schizophrenia Clinic, believed that about 30% of patients with schizophrenia would benefit from clozapine.
“I was doing it badly, I had a lot of patients who struggled to function outside the hospital and were circulating a lot of medication,” he said. “When they go to clozapine, they really tend not to be hospitalized again. I've had people finish college and finish work. That's pretty amazing.”
The FDA's shift came after years of lobbying by pharmacists, clinicians and relatives for people with schizophrenia who catalogued cases of patients who deteriorated after losing access to clozapine.
“We had patients who had recurring psychosis, patients who were hospitalized, and patients who became violent,” said Raymond C. Love, professor emeritus at the University of Maryland Pharmacy.
When the FDA's advisory committee considered the requirements at its November meeting, he said agents must expand their public dockets to accommodate the number of people they want to talk to.
“That was how much of the protest,” he said. “There was someone crying.”
In a statement posted on its website on Monday, the FDA said it determined that the test regimen was “no longer needed to ensure that the medication benefits outweigh the weight.” Its prevalence is estimated to be around 0.9% worldwide, and decreases after the first six months of treatment.
However, Dr. John M. Kane, a leading schizophrenia researcher, said he does not expect substantial changes in prescriptions as a result of the changes. Even without a testing regimen, he said many doctors are hesitant to prescribe clozapine because they require close monitoring for the early months of treatment.
“Perhaps one in eight people in the United States who might benefit from clozapine get it,” said Dr. Kane, a professor of psychiatry at Zucker School of Medicine in Hofstra/Northwell. For some of them, he said drugs are “life-changing.”
Psychotic disorders like schizophrenia affect 1% to 3% of the adult population, but this disease is an overwhelming burden for society. Illness usually appears in late adolescence and is characterized by social withdrawal, cognitive impairment, and lack of motivation, such as hallucinations and delusions.
Research suggests that the risk of suicide in people with schizophrenia is at least 10 times more risky than the general population.
Many people manage their symptoms with the help of their families, while others cycle through imprisonment, homelessness and short hospitalization. A 2022 analysis of disease-related financial burdens published in the Journal of Clinical Psychiatry estimated annual costs of $343.2 billion, primarily from caregiving, premature birth and unemployment.
The first antipsychotic drug, chlorpromazine, became available 70 years ago, followed by two new therapies designed to alter the brain's dopamine pathways. However, the pace of discovery slowed down afterwards. This is because vast amount of research funds have been dedicated to investigating the genetics of diseases. Last September, the FDA approved Cobenfy, the first new antipsychotic treatment in decades.
