FDA Proposes Rules Overhaul to Expand Availability of Experimental Drugs

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P06-194

December 11, 2006

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FDA Proposes Rules Overhaul to Expand Availability of Experimental Drugs

The Agency Also Clarifies Permissible Charges to Patients

The Food and Drug Administration (FDA) today 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 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.

" This proposed reform is carefully designed to balance several

objectives, " said Dr. 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. "

" FDA hopes this proposal will increase awareness in the healthcare

community of the range of options available for obtaining experimental

drugs for seriously ill patients, " added Dr. Janet Woodcock, FDA's

Deputy Commissioner for Operations. " By clarifying and streamlining the

processes, FDA also hopes to encourage companies to make such drugs

available, and reduce barriers for healthcare practitioners in

obtaining them. "

FDA has allowed many types of access to experimental therapies since the

1970's. Some of the larger programs, including those under the treatment

IND (Investigational New Drug) regulations, were successful in enabling

tens of thousands of patients with HIV/AIDS, cancer and cardiovascular

diseases to receive promising therapies before the products were

approved for marketing. However, the existing regulations did not

adequately describe the full range of programs available, explicitly

recognizing only emergency use for individual patients and widespread

treatment use access for large groups of patients. FDA believes it is

important that its regulations clearly reflect the full range of

treatment use programs available to ensure broad and equitable access

to experimental drugs for treatment use. 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 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) The most

significant proposals would:

(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.

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: .

* Mail/Hand delivery/Courier [For paper, disk, or CD-ROM

submissions]: Division of Dockets Management, 5630 Fishers Lane, Rm.

1061, Rockville, MD 20852.

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