We vehemently refute the notion that an expanded access program cannot be conducted in parallel with a clinical trial, and reject the notion that doing so poses an ethical issue. In fact, keeping a therapy that can only reach someone who doesn’t qualify for a trial through the provision of expanded access from them poses a far greater ethical concern.

As described by the FDA – expanded access is a “pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.” SLC6A1 clearly fits the definition. 

%d bloggers like this: