Posting Preapproval Access Policies Could Help Prevent ‘Blame And Shame’ Under Right To Try
Many biopharmas have not publicly posted their policies for evaluating and responding to expanded access requests as required under the 21st Century Cures Act. Putting this information out will help set patient community expectations, but will not viewed by US FDA as off-label promotion, experts say at BIO annual meeting.
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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.