Why The Right-To-Try Law Is Not Right For Some Biotech Companies
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.
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FDA must still determine internal procedures for receiving annual reports and handling adverse events through the controversial pathway that encourages access to unapproved therapies.
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.
Internal group headed by Principal Deputy Commissioner Rachel Sherman will develop implementation recommendations, which could include new guidance, regulations, a Q&A document or ‘nothing at all,’ Commissioner Gottlieb tells BIO annual meeting; he promises consultation with the law’s supporters and critics on Capitol Hill ‘to fully understand what the intent was with different provisions.’