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FDA Clarifies Investigational Drug Issues

Health Law Update Sarah E. Coyne

If your facility sponsors clinical research into investigational drugs, conducts research on drugs OR uses experimental drugs on occasion to treat patients, the FDA has provided some long-awaited clarification for you.

Two new Food and Drug Administration (FDA) rules related to investigational drugs take effect on October 13, 2009. The first rule addresses expanded access to investigational drugs (i.e., access outside of a clinical trial), while the second addresses when sponsors of clinical investigations may charge patients for investigational drugs.

The rules do not mark a radical policy shift for the FDA, but they do provide much-needed guidance to sponsors, investigators and physicians. Here is an overview of the FDA rules.

Rule on Expanded Access

FDA rules have long recognized that not every patient who may benefit from an investigational drug is enrolled in a clinical trial. Previous FDA rules recognized the concept of "expanded access" (also known as "treatment use" or sometimes "compassionate use"), but the rules did not clearly set forth the requirements/procedures for allowing patients outside of clinical trials to have this kind of access.

The new rule sets core criteria and procedures for expanded access, and establishes additional criteria and procedures depending on the category of patients accessing the drug. The rule formally creates three categories of access including a category for individual patients. The two other categories are for intermediate sized populations, generally up to 100 patients with similar treatment needs, and larger populations under a treatment protocol or treatment IND (investigational new drug application).

The FDA may approve an investigational drug to be used for the treatment of an individual patient by a licensed physician so long as all of the following are true:

  • The patient has a serious or immediately life-threatening disease or condition;

  • There is no comparable or satisfactory alternative therapy available to diagnose, monitor or treat the disease;
  • The potential patient benefit justifies the potential risks of the treatment use;
  • The potential risks are not unreasonable in the context of the disease or condition to be treated; and,
  • Providing the investigational drug for the requested use will not interfere with the initiation, conduct or completion of clinical investigations that could support marketing approval of the expanded access use, or otherwise compromise the potential development of the expanded access use. The patient cannot obtain the drug under another IND or protocol.

To initiate the FDA approval process, a physician or the drug sponsor must normally send an "expanded access submission" to the FDA. The new rule details what must be in the submission, and how an individual physician might work with a sponsor to fulfill the submission requirements. In emergency situations, the FDA may authorize expanded access use to begin without a written submission. However, the licensed physician or sponsor must explain how the expanded access use will meet the criteria for individual patient use and agrees to submit an expanded access submission within 15 working days of FDA's authorization of the use. (Under existing regulations that are unchanged by this rule the sponsor/physician may also be able to treat the patient without prior Institutional Review Board ("IRB") approval and/or informed consent, depending on the circumstances. Even if prior IRB approval is not required, existing regulations require the IRB to be notified within five working days of an emergency use.)

After expanded access use, the licensed physician or sponsor who requested the use must provide the FDA with a written summary of results. The report must include all adverse effects of the drug. When a significant number of similar individual patient expanded access requests have been submitted, the FDA may ask the sponsor to submit an IND or protocol for treatment use with an intermediate or larger population.

Rule on Charging for Investigational Drugs

Existing FDA rules did not reflect the fact that sponsors may wish to recoup costs of approved drugs manufactured by a third party. The new rule is clear - prior FDA approval is not required for a sponsor to charge patients for an approved drug manufactured by a third party for an approved indication in a clinical trial (as an active control, for example). For investigational drugs, whether in a clinical trial or for expanded access, the sponsor must have prior written authorization from the FDA to charge patients. Recoverable costs are limited, as set forth in the rule.

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For more details, or if you have any questions, please contact Sarah Coyne at 608-283-2435 / sarah.coyne@quarles.com or your Quarles & Brady attorney.