As Pediatric Rare Disease Patients Grow Up, Can We Keep Pace?

By Rami Levin

Companies frequently talk about being patient-centric. But what I don’t hear discussed as often is the role of the patient after a drug is approved. And I am convinced it is equally important. Why? Because patients are not static; they grow and change over time. The medicines and support our industry provides have to evolve to meet their changing needs.

I’ve seen the importance of this firsthand. One of the medicines my company provides to patients treats a rare disease that is almost exclusively diagnosed in infancy. Our first-generation product was a capsule that parents had to open and mix into formula or milk, but they had to adjust this process often to increase the dose as the infants gained weight. After hearing about the difficulty some people were experiencing with this, we decided to develop an oral suspension formulation.