On February 4, 2015, the Food and Drug Administration (FDA) issued a new Draft Guidance titled Individual Patient Expanded Access Applications: Form FDA 3926. The Draft Guidance introduces draft Form FDA 3926, which – when finalized – may be submitted by licensed physicians to FDA for expanded access (or “compassionate use”) requests for individual patient Investigational New Drug Applications (INDs). When finalized, this new form is intended to significantly reduce the amount of time and effort it will take physicians to request expanded access for their patients. As a result, we think the availability of this new form may also significantly increase the number of compassionate use requests received by pharmaceutical and biotechnology companies. In 2014, FDA received 1,882 expanded access requests. (See Expanded Access Submission Receipts Report (Oct. 1, 2013 – Sept. 30, 2014)).
Individual patient expanded access permits physicians to use an investigational drug outside of a clinical investigation for an individual patient. Several criteria must be satisfied, as determined by FDA, including that the patient has a “serious or immediately life-threatening disease or condition and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.” (21 CFR 312.305(a)(1).) In addition to other criteria, FDA must conclude that the patient cannot obtain the drug under another IND or protocol, and the physician must decide that the investigational drug’s probable risk is not greater than the disease or condition’s probable risk. (21 CFR 312.310(a); 21 CFR 312.305(b)(2).)
FDA explained in the Draft Guidance that it issued Form 3926 because the agency is concerned that physicians may be encountering difficulty in requesting expanded access of investigational drugs for individual patients. Specifically, physicians are currently required to submit a cover sheet, Form FDA 1571 (used by sponsors for all types of IND submissions) and all associated IND documentation. The current form requires the filling in of 26 pieces of information and requires 8 attachments. According to FDA, the existing process is not “tailored” to requests for individual patient expanded access and can take physicians as long as 100 hours to complete. (See Draft Guidance, at 3; Peter Lurie, M.D, FDA Voice, A Big Step to Help the Patients Most in Need (FDA Blog).) In contrast, FDA estimates Form 3926 will take physicians 45 minutes to complete and will “minimize unnecessary red tape.” (FDA Blog.) The new draft form has 8 boxes that physicians have to fill in with information and only one attachment is required.
According to the agency, submission of finalized Form 3926 will comply with all IND and individual patient expanded access submission requirements and serve as a request to waive other applicable IND requirements. In other words, other information required in the IND regulations – including the Form 1571 – will not need to be included with the Form 3926. After FDA receives the form, in a non-emergency situation, FDA will either allow the treatment use to proceed, or will put the application on clinical hold and notify the physician. Treatment with the investigational drug may proceed once FDA notifies the physician, or if no notification occurs, 30 days after the agency receives the finalized form. Treatment in the case of an emergency situation follows an expedited process and timeline.
In addition, the Draft Guidance explains that when a licensed physician wants to obtain an investigational drug for an individual patient, before submitting the application to FDA, the physician should first ensure that the manufacturer of the investigational drug is willing to provide the drug. If the manufacturer agrees to provide the drug, the manufacturer should provide the physician with a letter of authorization that permits FDA to refer to information the manufacturer has submitted to FDA (e.g., in a commercial IND), if applicable. (See Draft Guidance, at 4).