The Journey from Compassion to Affirmation - The Use of Expanded Access Trial Data in Drug Approvals

Expanded access to investigational drugs is an option for patients with a serious disease or immediately life-threatening condition to gain access for treatment with an investigational medical product . Recently, development programs have successfully created new paradigms for totality of data to accommodate the difficulties of studying extremely heterogeneous ultra-rare diseases. Utilizing expanded access data in US and EU registration dossiers can help to fulfill the promise that the scientific advances translates into availability for patients. This session will highlight successful applications for rare diseases that relied upon data from EAPs presented as case studies. The presentation will uncover the challenges and key considerations in creating global regulatory strategies that incorporate such nontraditional data. In addition, the issues that arise when the product is not approved will be addressed.