Food and Drug Administration - August 12, 2009
Richard Klein & Kimberly Struble

On August 12, 2009, the Food and Drug Administration (FDA) published two new rules related to investigational drugs. 

One new rule, “Expanded Access to Investigational Drugs for Treatment Use,” seeks to clarify the methods available to seriously ill patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need to safeguard the individual patient and ensure the continued integrity of the scientific process that brings safe and effective drugs to the market.  The second, “Charging for Investigational Drugs Under an Investigational New Drug Application,” clarifies the specific circumstances and the types of costs for which a manufacturer can charge a patient for an investigational drug, either as part of a clinical trial, or for treatment use outside the scope of a clinical trial through expanded access.

Expanded Access
The changes are meant to consolidate and clarify the requirements and facilitate making investigational drugs and biologics more widely available to seriously ill patients, including those with HIV/AIDS, who have no other treatment options. The proposed changes also seek to clarify the specific circumstances and the types of costs for which a manufacturer can charge for an investigational drug made available for the purpose of treatment, or in clinical trials.

FDA hopes the new rules will increase awareness in the patient and healthcare communities of the range of options available for obtaining investigational drugs for seriously ill patients, encourage companies to make such drugs available, and reduce barriers to obtaining them.

The new expanded access rule defines three categories of patient populations to whom investigational drugs could be made available for the purpose of treatment outside of a clinical trial through expanded access, when there is no satisfactory alternative therapy, and defines requirements and safeguards for each. They are

• individual patients,
• groups of patients smaller than that typical of a treatment IND or treatment protocol (FDA may ask a sponsor to consolidate expanded access under this section when the agency has received a significant number of requests for individual patient expanded access to an investigational drug for the same use),
• and larger populations where widespread treatment use is appropriate (the Treatment IND).

FDA has allowed many types of access to investigational therapies since the 1970's. Some of the larger programs, including those under the Treatment IND (Investigational New Drug) regulations, enabled 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.  The new rule is meant to clearly reflect the full range of treatment use programs available, and ensure broad and equitable access to investigational drugs for treatment use.

Significant changes in the new rule include:

• Updating applicable regulations to include all circumstances under which access to investigational 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.

• Establishing criteria that link the level of evidence needed to support the use of an investigational drug to the seriousness of the disease and the number of patients likely to be treated with the drug in an expanded access program;

Charging for Investigational Drugs
FDA’s regulations describing when it is appropriate to charge for an investigational drug did not account for the full range of circumstances in which charging should be permissible, and because they had proven difficult to interpret in practice, resulted in confusion over exactly what costs could be recovered by sponsors making drug products available through expanded access programs.

The new rule revises the regulation regarding recovery of the costs of an investigational drug to:

• clarify that allowing charging for treatment use of an investigational 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
• 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.

The change simplifies the cost recovery calculation by making clear that charges for an investigational drug used in a clinical trial may include only direct costs associated with the drug's development, and that charges for investigational drugs for treatment use may also include administrative costs of making the drug available for intermediate patient populations and under large scale treatment INDs.

Indirect costs, including costs incurred primarily to produce the drug for commercial sale, cannot be recovered

For additional information, see Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs

Richard Klein
Office of Special Health Issues
Food and Drug Administration

Kimberly Struble
Division of Antiviral Drug Products
Food and Drug Administration

090812
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SOURCE: Food and Drug Administration (FDA).

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