The Food and Drug Administration said it has proposed regulatory changes to make experimental drugs more easily available to seriously ill patients with no other treatment options, and to clarify the circumstances and the costs for which a manufacturer can charge for an experimental drug.
Under the proposed rule, expanded access for experimental drugs would be available to individual patients, small patient groups, and larger populations under a treatment plan when there is no satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.
The FDA said it hopes to encourage companies to make these drugs available, and reduce barriers for healthcare professionals in obtaining them.
"This proposed reform is carefully designed to balance several objectives," said Dr. Andrew C. von Eschenbach, Acting FDA Commissioner. "One goal is to enable many more patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need for safeguarding the individual patient. Another equally important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market."
The regulations covering when it is appropriate to charge for an experimental drug need revisions because they fail to account for the full range of circumstances in which charging should be permissible and because they have proven difficult to interpret in practice, resulting in confusion over what costs could be recovered, the FDA said.
Among the proposals is revising current regulation regarding manufacturers' recovery of the costs of an experimental drug to clarify that the charges are permissible in a clinical trial only to facilitate development of drugs that promise significant advantages over existing therapies, and might not otherwise be developed because of their high cost.
The proposed rules, which are open for comment for 90 days are described in detail at the following CDER web address: http://www.fda.gov/cder/regulatory/applications/IND_PR.htm.