FDA announces new 'streamlined' application for investigative drugs

The FDA has released a new application for investigative drugs that it says will cut the amount of time physicians spend filling out such requests, as part of a larger effort to “streamline” the process of providing medications still undergoing regulatory review to their patients with severe illness.

The final Individual Patient Expanded Access Investigational New Drug Application can be used by physicians to request expanded access to investigational drugs for patients who suffer from serious or immediately life-threatening diseases, in the absence of any comparable or satisfactory alternative treatment. According to a statement released by FDA Commissioner Robert Califf, MD, the new form is one part of a larger effort to improve the process by which physicians request “compassionate use” of investigative drugs.

Robert Califf, MD

“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,” Califf said in a press release. “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.”

According to Califf, the new form is “much shorter” than the previous application, and should take approximately 45 minutes to complete.

The FDA also released two additional final guidance documents on expanded access to investigative drugs. The first, in a question-and-answer format, explains what expanded access is, when and how to make a request, and the information that should be included in the request. The second document outlines the regulations governing how patients may be charged for investigational drugs.

“The FDA has a long history of supporting patient access to investigational treatment 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,” Califf said.

Additional reading:

http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM432717.pdf

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM351261.pdf

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm351264.pdf