On February 4, 2015, FDA released draft guidance around its “Expanded Access” program, also known as “compassionate use.” The guidance proposes a greatly streamlined application form that physicians may use on behalf of individual patients to request access to investigational drugs that have not yet been approved by the FDA as safe and effective.
Generally, drugs that are not yet approved by FDA are not available to patients outside a clinical trial. However, in certain circumstances under the Expanded Access program, FDA allows Investigational New Drug (IND) sponsors to provide their drug products to individual patients or broader patient populations outside of a clinical investigation for treatment purposes.
Previously, a physician requesting expanded access on behalf of an individual patient was required to submit an extensive IND form, used by all sponsors, including drug manufacturers. FDA’s proposed guidance would change this process by providing a new, simplified application form for physicians. Furthermore, FDA proposes that, in emergency situations that require patient treatment before a form can be completed, a treating physician may call an FDA review division, certify that all regulatory requirements are met, and agree to submit the form within 15 days of FDA’s authorization of expanded access use.
In a blog post about the draft guidance, Associate FDA Commissioner Peter Lurie, M.D., M.P.H., estimated that physicians can complete the proposed Form FDA 3926 in approximately 45 minutes, as compared to the 100 hoursrequired under the previous IND form. Lurie notes that the previous form was originally designed for drug manufacturers seeking to begin human testing, not individual physicians and their individual patients.
The simplified process, however, will not change the otherwise applicable FDA requirements for authorization of Expanded Access for individual patients, namely: (1) that the patient have a serious disease or otherwise immediately life-threatening condition, (2) there is no other comparable alternative treatment available, (3) the potential patient benefits justify the potential risks, (4) those risks are not unreasonable in the context of the disease to be treated, (5) providing the investigational drug outside a clinical trial will not otherwise interfere with ongoing clinical investigations, and (6) the patient cannot obtain the investigational drug through another IND sponsor or protocol.
The guidance is expected to be published in the Federal Register in the near future. Comments and suggestions regarding the draft document are to be submitted within 60 days of publication in the Federal Register.