Pharmaceutical firms may soon be able to make experimental drugs even more widely available for a fee to seriously ill patients who have no other treatment options, under proposed rule changes from the US Food and Drug Administration.
The agency said that, under the proposals, expanded access for experimental drugs would be made available to individual patients and larger groups “when there is no satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.”
The FDA also proposed revisions to the regulations about how drugmakers recover costs for experimental drugs, and said charges are allowed in a clinical trial to facilitate development of new treatments.
There is nothing new in the FDA allowing access to experimental therapies. Since the 1970s, tens of thousands of patients with HIV/AIDS, cancer and cardiovascular diseases received promising therapies before the products were approved for marketing.
However, the changes are being made because the FDA’s current rules do not “adequately describe the full range of programmes available” and “have proven difficult to interpret in practice.” Janet Woodcock, the FDA's deputy commissioner for operations, said: “by clarifying and streamlining the processes, [the] FDA also hopes to encourage companies to make such drugs available, and reduce barriers for healthcare practitioners in obtaining them.”
The public has 90 days to comment on the proposed changes.