Independent assessment finds positive perceptions of expanded access program but calls on agency to do more to correct manufacturer misperceptions about adverse events and clarify when data from expanded access would support labeling extensions.

FDA must still determine internal procedures for receiving annual reports and handling adverse events through the controversial pathway that encourages access to unapproved therapies.

Two CEOs explain why their rare disease companies will not grant preapproval access to investigational drugs under new federal law; Alnylam sees no need for framework beyond FDA’s expanded access pathway, while Sarepta is focused on the ‘grand goal’ of getting drugs studied and approved for the patient community.