The pediatrician's role in prescribing trofinetide for Rett syndrome

Alan Percy, MD, Professor of Pediatrics, Neurology, Neurobiology, Genetics, and Psychology at the University of Alabama, Birmingham, and lead author of the LILAC-1 and LILAC-2 studies, recently discussed the role of physicians in prescribing trofinetide (Daybue; Acadia Pharmaceuticals) for Rett syndrome. His observations highlight important developments in the prescribing process, while also emphasizing the collaborative effort needed to optimize patient care.

"It's very interesting, and I can't give you an exact percentage, but maybe as high as 50% or so of the children that we have seen have had the medication prescribed by their private physician," Percy noted. "So the private physicians are being informed by the parents that this medication is available, and they are reaching out to the pharmacists to gain approval."

"I think that general physicians should be quite confident in being able to prescribe this. They should reach out to so-called centers of excellence," he explained. For physicians unfamiliar with the treatment, these centers can offer guidance and support, helping to bridge the knowledge gap.

Percy also provided practical advice on how to begin administering the medication, particularly for physicians who may be new to treating Rett syndrome. "They also should know that they can start at, say, 50% of the recommended weight-based dose and increase gradually. We have had individuals who have increased by 1 milliliter a week," Percy said. Although this approach might seem slow, he emphasized the importance of gradual increases to minimize side effects and achieve the optimal dose.

As the treatment becomes more widely prescribed, feedback from parents remains crucial. Percy expressed a desire for greater communication between physicians and families, stating, "The question always is, at the end of 6 months or a year, what are the parents seeing? And that is something that we would like to know."