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FDA Announces Proposed Regulations to Overhaul and Expand the Availability of Experimental Drugs and to Clarify Permissible Charges to Patients
December 12, 2006

Yesterday, FDA announced that two proposed rules will be published in the Federal Register on December 14, 2006, that (if implemented) will "make experimental drugs more widely and easily available to seriously ill patients with no other treatment options and [will] clarify the circumstances and the costs for which a manufacturer can charge for an experimental drug." The first proposal would amend FDA's IND regulations at part 312 to clarify existing "expanded access" regulations and to add new types of expanded access for treatment use. The second proposal would amend FDA's current charging regulations at 21 C.F.R. § 312.7 to clarify when charging is appropriate and what costs can be recovered, and sets forth charging criteria for the different types of "expanded access" options discussed in the first proposal.

FDA's press release announcing the proposed rules discusses the most significant aspects of these proposals:
(1) Modernize applicable regulations to include all circumstances under which access to experimental drugs is permitted, including:
  • single patients in non-emergency and emergency settings;
  • small groups of patients; and
  • larger groups of patients under a treatment IND. To authorize these expanded access treatment uses, FDA generally must be satisfied that the patient's serious or immediately life-threatening disease or condition has no satisfactory approved therapy; that the potential benefit for the patient justifies the potential risks; and that providing the therapy will not interfere with the drug's development.
(2) Make experimental drugs more widely available in appropriate situations by establishing criteria that link the level of evidence needed to support the use of an experimental drug to the seriousness of the disease and the number of patients likely to be treated with the drug;

(3) Revise the current regulation regarding manufacturers' recovery of the costs of an experimental drug to:
  • clarify that such 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;
  • clarify that allowing charging for treatment use of an experimental drug is intended to facilitate and encourage access to drugs that might not be made available for treatment use unless a manufacturer is able to recover its costs.
The proposal also would simplify the cost recovery calculation by making clear that charges for an experimental drug used in a clinical trial may include only direct costs associated with the drug's development, and that charges for experimental drugs for treatment use may also include administrative costs of making the drug available for intermediate patient populations and under large scale treatment INDs.

Click here for FDA's press release and advance copies of the proposed rules.

If you have any questions regarding the above information, please contact:
Josephine M. Torrente (202) 737-7554 jtorrente@hpm.com
Kurt R. Karst (202) 737-7544 kkarst@hpm.com