Supporting
access for all

Receiving a diagnosis of a rare disease is often life-altering for the patient and their family, and we have a responsibility to support patients and their families as they explore available treatment options. In developing new medicines for children and adults with rare and ultrarare diseases, we want to ensure that our investigational therapies—those that have not yet been approved by regulatory authorities such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in Europe—are available to patients through approved mechanisms. The preferred way to gain access to our investigational therapies is by participating in a clinical trial. However, some patients with a serious or immediately life-threatening disease may not be eligible to participate in clinical studies and may not have other treatment options. In those cases, we make our investigational therapies available to qualified patients under certain circumstances of critical and compelling need through our early access program. We appreciate that our patients’ lives may be in crisis and the critical obligation we share to engage and identify potential options.