Statement From FDA Commissioner Robert Califf, MD, on the Release of the Final Individual Patient Expanded Access Form

“Today, the FDA finalized its efforts to streamline the process used by physicians to request expanded access, often called ‘compassionate use,’ to investigational drugs and biologics for their patients. As a physician, I understand the importance of being able to access investigational treatments for a patient when there are no other options to treat their serious disease or condition.”

Reducing Procedural Burdens

“Access to investigational treatments requires the active cooperation of the FDA, industry, and health-care professionals in order to be successful. But we know that navigating that process can be challenging and time-consuming, and we are committed to reducing the procedural burdens on physicians and patients whenever possible.”

Robert Califf, MD

“One way we hope to make things simpler for physicians is to reduce the amount of time they spend filling out a request for access to an investigational drug. That’s why today we are releasing the final Individual Patient Expanded Access Investigational New Drug Application—Form FDA 3926.¹ The new form can be used by physicians to request expanded access to investigational drugs for individual patients who suffer from serious or immediately life-threatening diseases and for whom no comparable or satisfactory alternative therapy is available. It is much shorter than the form previously used for individual patient expanded access requests and is designed specifically for these requests. It should take about 45 minutes for a physician to complete the new form. Along with the new form, we are also releasing step-by-step instructions on how to complete it.”

Additional Documents

“We want the expanded access process to be as clear as possible. That is why the agency is also releasing two additional final guidance documents today. One is in a question-and-answer format² and explains what expand access is; when and how to request expanded access; and the type of information that should be included in requests. The other explains the regulations regarding how patients may be charged for investigational drugs.”³

“The FDA has a long history of supporting patient access to investigational treatments and we hope the information released today will help health-care professionals, patients, and industry to more easily navigate the expanded access process and help patients who qualify to gain access to potentially life-saving investigational treatments.” ■

References

1. Individual Patient Expanded Access Applications: Form FDA 3926. Available at fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM432717.pdf. Accessed June 2, 2016.

2. Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers. Available at fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM351261.pdf. Accessed June 2, 2016.

3. Charging for Investigational Drugs Under an IND—Questions and Answers. Available at fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm351264.pdf. Accessed June 2, 2016.