Recommendations to Facilitate Expanded Access to Investigational Therapies for Seriously Ill Patients.

When clinical trial enrollment is not an option for seriously ill patients whose illnesses have not responded to approved treatment options, those patients and their physicians may consider gaining access to investigational therapies through a pathway established by the Food and Drug Administration (FDA) called expanded access. However, recent events have highlighted the challenging dynamics involved in accessing investigational therapies through expanded access that include a complex interplay of factors involving the patient, physician, drug company, FDA, and, increasingly, social media. The authors offer several potential strategies to streamline what is otherwise an arduous process for all involved.