NEJM Interview: Yaara Zisman-Ilani on navigating the uncertainty surrounding medication management in autism care.

Progress toward developing medications for autism has been limited. Autistic people, their families, and clinicians are often caught between the imperative to address symptoms and the reality that available options are associated with uncertain benefits and potential harms. I'm Stephen Morris, the managing editor of the New England Journal of Medicine, and I'm talking with Yaara Zisman-Elani, an associate professor in the Department of Social and Behavioral Sciences at the Barnett College of Public Health and in the Department of Psychiatry and Behavioral Science at the Lewis Katz School of Medicine, both at Temple University. Dr. Zisman-Elani has co-authored a perspective article about shared decision-making in autism care. Dr. Zisman-Elani, you write in your perspective article that only two medications have been approved by the FDA for use in autism. What symptoms do those medications target, and how effective are they?

There are two medications that are currently approved for treating co-occurring symptoms that are associated with autism. Medication are risperidone and aripiprazole, and the target is really to address irritability, aggression, sleep, anxiety, attention. We are not targeting the core condition of autism itself.

So why, in fact, has it been difficult to develop medications that could modify that core neurodevelopmental set of characteristics that are associated with autism?

So autism is really an extraordinary heterogeneous neurodevelopmental condition. The biology that affects social communication differences, repetitive behavior, sensory processing, and other characterization of autism varies substantially across different individuals, and this makes, in my opinion, drug development particularly challenging. So while we have made tremendous progress in understanding what is autism, it is very challenging to translate that knowledge into medication that can modify or help or change the core characteristic. And so far we see it is more difficult than many anticipated.

And then what about the psychoeducational interventions? How effective are those approaches, and are they widely available?

For some people, they are effective; for others, less. They are more effective to address behavioral aspect related to autism. They are less about the symptoms themselves. But as you mentioned, the main challenge with those behavioral and educational intervention is their accessibility. They are not always available. They are not always covered by insurance. And a lot of time, if they are available, the wait list is very, very long. So we are finding ourself in a situation that even if those psychological and educational and behavioral intervention are helpful, sometimes because the wait list is very long, families and autistic individuals are looking for more immediate solution and also clinician, which bring us to medications that are more available and covered by insurance.

And then how common is it for autistic people to receive off-label or alternative medications?

It is very common because situation as we view it is that clinicians, family, autistic person, policymakers, everyone are geared with good intention, with really wanting to help