FDA Proposes Wider Availability of Experimental Drugs

PharmaManufacturing.com
12/12/2006

On Dec. 11, the Food and Drug Administration (FDA) proposed significant regulatory changes to make experimental drugs more widely and easily available to seriously ill patients with no other treatment options, and to clarify the circumstances and 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.

"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 important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market."

The proposal will increase awareness of the range of options available for obtaining experimental drugs for seriously ill patients, and by clarifying and streamlining the processes, the FDA hopes to encourage companies to make such drugs available and reduce the barriers in obtaining them.

Although the FDA has allowed many types of access to experimental therapies since the 1970s, the existing regulations do not adequately describe the full range of programs available, explicitly recognizing only emergency use for individual patients and widespread treatment access for large patient groups.

The proposed rules are open for comment for 90 days and are described in detail at the following CDER Web address:
http://www.fda.gov/cder/regulatory/applications/IND_PR.htm
Written comments, identified by Docket No. 2006N-0062 and RIN 0910-AF14 (for expanded access proposals) and Docket No. 2006N-0061 and/or RIN 0910-AF13 (for cost recovery proposals) may be submitted by any of the following methods:

Federal eRulemaking Portal: http://www.regulations.gov
Follow the instructions for submitting comments.
Agency Web site: http://www.fda.gov/dockets/ecomments
Follow the instructions for submitting comments on the agency Web site.
FAX: (301) 827-6870
For paper, disk, or CD-ROM submissions via mail, courier or hand delivery:
Division of Dockets Management, 5630 Fishers Lane, Rm. 1061, Rockville, Md. 20852.