Opinion: The FDA removed the red tape around one of the best drugs for children with schizophrenia. But the fear remains

On Feb. 24, 2025, the Food and Drug Administration did something it arguably should have done long ago: It removed the elaborate monitoring program that has governed one of psychiatry’s most important drugs, clozapine, for more than 30 years. For anyone who treats serious mental illness, this is welcome news. But I want to say something more complicated about it, because I have spent a large part of my career prescribing this drug (or trying to) to the most vulnerable population there is: children with psychosis. The paperwork was never really the problem. Very few things in all of medicine have struck me as close to miraculous. Vaccines. Antibiotics. And, in children with childhood-onset schizophrenia, clozapine. That will not surprise anyone who has worked with it in schizophrenia. Clozapine is the most effective antipsychotic we have for the sickest patients: the roughly 1 in 3 people with schizophrenia whose illness does not yield to other medicines. It is the only drug approved for treatment-resistant schizophrenia, the only one shown to reduce suicide, and it is associated with lower death rates than the alternatives. When nothing else has worked, clozapine can, sometimes, give a person back to themselves. I have watched it happen in children, in the carefully controlled trials my colleagues and I ran, where young people who had been lost to relentless psychosis began, within days, to return to their families. And yet it is one of the most underused drugs in all of medicine. In the United States, only about 4% of people with schizophrenia receive clozapine, though perhaps a third could benefit, and the figure should probably be higher still in childhood-onset schizophrenia, which tends to be even more treatment-resistant. There is, in plain terms, a proven, lifesaving, sometimes life-restoring treatment that American medicine mostly declines to give. For years, simply to prescribe clozapine, a clinician had to enroll in an FDA-mandated registry. Patients submitted to regular blood draws, which are weekly at the start, with each result logged into a national database before a pharmacy could release the next supply. The goal was to catch the small number of patients in whom clozapine can sharply reduce the white cells that fight infection. Whatever caution it began in, there is good evidence it also kept the drug from people who needed it. That is the very program the FDA has now retired, perhaps influenced by advocacy efforts, including those by parents. But the deeper barrier was fear. Clozapine carries a fearsome reputation: the blood tests, the long list of side effects, the rare but genuine danger. This has scared clinicians; they just don’t want to take the risk. And the FDA can remove the registry, but cannot make the fear go away. This is especially true when dealing with children, in whom neutropenia tends to be more common than in adults. I have watched capable, caring clinicians steer around clozapine for years, reaching for a fourth or fifth less-effective medication rather than the one most likely to work, because avoiding it simply felt safer. Recently I could not find a single child psychiatrist across four academic centers willing to start clozapine in a child with childhood-onset schizophrenia. When I first started using clozapine, I was afraid as well. But it soon became clear that it was not a particularly difficult medicine to work with. Yes, you have to monitor the side effects carefully, but the benefits simply made the risk worth taking. And as I started seeing the benefits of clozapine, the fear quickly went away. After all, in other areas of medicine, we treat children with many risky medications (such as chemotherapeutic drugs) because we know they are lifelines for those illnesses. We need to approach clozapine the same way when it comes to schizophrenia. In my experience, parents were rarely the problem. They understood, they were desperate, had often done the work of getting over their fear, and were ahead of the curve. They typically had already heard and read about clozapine and were ready to be on board as soon as we were ready, partly because they had tried everything and nothing had worked. I used to say, after seeing what I saw at the National Institute of Mental Health, “If someone in my family was diagnosed with schizophrenia, I would want them on clozapine yesterday.” The cost of our avoidance of clozapine is invisible but enormous, measured in people who stay sick, and in lives that end early, for want of a drug already sitting on the shelf. I do not mean to minimize the side effects of clozapine. The risks are very real. My colleagues and I ran into them at the NIMH every now and then. We went on to study how often it happened in our children, and who was most at risk. The problem is concentrated in the first months of treatment, and it never quite reaches zero. But we found ways through: We showed, for instance, that adding lithium could lift the neutrophil count enough to keep a child safely on clozapine, and we published it. Its other side effects, too, can be managed, and there is ample literature, including from our own group, on how to do that in children. In fact, as we published more, we began to notice something new: Some of the children referred to our study arrived already on clozapine, something we had never seen in the study’s earlier years. Those of us who worked closely with clozapine had argued for years that its monitoring was stricter than the evidence justified; that a rigid, uniform schedule, largely unchanged for decades, treated every patient as though the danger were constant and equal, when in truth it was concentrated in the early months, foreseeable, and, in most cases, manageable. The FDA’s recent decision, in that sense, is less a reversal than an overdue acknowledgment of what the people closest to the drug had been saying all along. Notably, the FDA still recommends the blood tests; it has simply stopped forcing them. This is a welcome change, and it means the responsibility that used to live inside a federal registry now lives where it probably always should have: in the judgment of the clinician and the informed choice of the patient. That is the real opportunity in this decision, and the real test of it. Lifting the program is not permission to be careless; it is an invitation to be wise, and better informed. Ideally it will empower clinicians to reach for clozapine sooner, for the people most likely to be saved by it, including the young. The worst outcome would be to lose the bureaucratic barrier and go on avoiding the drug anyway, only now without even a rule to blame. And perhaps, and this is my real hope, it will move us to reach for clozapine earlier, rather than only as a last resort. To my colleagues who care for the youngest and sickest patients, I would put it as plainly as I can: Do not let fear make this decision for you. We have spent three decades treating clozapine as the door you open only after every other has failed. The science has never justified waiting that long. The government has now removed one of our excuses. Whether we use this moment to actually reach the patients who need it, or simply find a new reason to look away, is up to us. It always was. Nitin Gogtay, M.D., is a psychiatrist, physician-scientist, and author of “The Vanishing Children” (HarperCollins, January 2027). He spent almost two decades studying and conducting blinded clinical trials of clozapine on childhood-onset schizophrenia at the National Institute of Mental Health.