The U.S. Food and Drug Administration (FDA) just made it easier for physicians to get critically needed, but not yet approved in the U.S., treatments to some of the nation’s most critically ill patients. FDA Commissioner Robert Califf announced recently the release of a new form designed to help expedite the process for physicians to obtain “expanded access” or “compassionate use” approval for investigational new drugs (INDs) and biologics for individual patients. Under the Federal Food, Drug and Cosmetic Act (FDCA), a physician may request expanded access to investigation new drugs and biologics, and the request must meet certain criteria for FDA to authorize expanded access (FDA says that it approves 99 percent of requests). Individual patient expanded access submissions made by private physicians are submitted as new INDs. The new Form FDA 3926, Individual Patient Expanded Access Investigational New Drug, was created to ease the paperwork burden on physicians seeking approval for individual patients who suffer from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative is available.

FDA concurrently issued a guidance in a question and answer format that explains the procedures to be followed when completing Form 3926. In the guidance, FDA clarified that in this context, the expanded access is not primarily intended to obtain information about the safety and effectiveness of a drug (as would be the case in a clinical trial), but rather, is intended to treat the patient when no satisfactory therapeutic alternative exists. Under the FDCA, expanded use is generally permitted under the following conditions:

• The patient and his or her licensed physician are both willing to participate and the person’s physician determines that there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the person’s disease or condition, and that the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition;
• FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
• FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval;
• The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient’s physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with applicable statute and regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product; and
  The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.

Until the issuance of Form 3926, physicians seeking approval for use of investigational new drugs outside of an approved clinical trial had to pursue a time-consuming and arduous approval process using forms 1571 and 1572, which were designed more for expanded use under an existing IND and for use by patient groups. Form 3956 is intended for single patient access, and FDA estimates that it should take about 45 minutes to complete. In emergency situations, approval can be obtained as quickly as a telephone call (or other rapid means of communication), as long as the physician making the request satisfies all of the information requirements contained in 21 CFR 312.305 and agrees to notify an investigational review board (IRB) of the emergency expanded access use with 5 business days of submission.

Commissioner Califf said in his statement that FDA is committed to reducing the procedural burdens on physicians and patients whenever possible, and wants to make the expanded access process as transparent as possible. The Agency has provided extensive information on its website and released two final guidance documents to assist in the preparation of Form 3926: Individual Patient Expanded Access Applications: Form FDA 3926 (Guidance for Industry) and Charging for Investigational Drugs Under an IND - Questions and Answers (Guidance for Industry). Instructions for completing the form are available here.

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