Posted 22 February 2016
By Zachary Brennan
The Senate Committee on Homeland Security and Governmental Affairs late last week sent a letter to US Food and Drug Administration (FDA) Acting Commissioner Stephen Ostroff with new questions on compassionate use, FDA’s approval timelines and helping patients with terminal illnesses.
The letter, part of the committee's oversight of the drug approval process, offers some background on the user fee process (as the latest iteration for the Prescription Drug User Fee Act (PDUFA) heads for clearance) and the progress FDA has made in approving new drugs, but says that despite the high number of approvals, patients and advocacy groups are concerned about the speed with which new drugs come to market, particularly for potentially life-saving treatments.
Sen. Ron Johnson (R-WI) mentions a draft guidance issued by FDA last February that would make it easier for patients to get access to investigational drugs on a compassionate use basis. Johnson also notes that the streamlined process has yet to be finalized even as FDA has said it expects the updated form included in the guidance to reduce the application process for compassionate use from 100 hours to 45 minutes.
Despite a lack of progress to the expedited version of compassionate use, FDA consistently approves such requests, and from 2010 through 2014, it rejected just 33 requests out of 5,995 received, approving 99.5% of all compassionate use requests. In 2014, FDA approved more than 1,800 compassionate use requests, which was about double the number approved in 2013.
And problems with the compassionate use program aren’t always the fault of FDA, which doesn’t have the last say in whether a patient can access the investigational drug. After receiving an FDA request for compassionate use, pharmaceutical companies must agree to provide the investigational drug, which can present difficult situations if a clinical trial is already in progress.
In addition to questioning Ostroff on why the draft guidance on the streamlined application has not been finalized yet, Johnson also questioned FDA on approval statistics, how the process for manufacturers dealing with compassionate use requests can be streamlined, how data received from expanded access requests is used, and the “most common cases and conditions for accelerated approval, fast track and compassionate uses.”
Increased patient interest in obtaining certain investigational products has also resulted in a series of new state laws that attempt to grant patients the “Right to Try” drugs, though none of the laws have yet offered any examples of successfully getting a patient access to an investigational drug.
Johnson and the Senate committee will meet Thursday to discuss the compassionate use program with witnesses including Darcy Olsen, president and CEO of the Goldwater Institute, which lobbied on behalf of the right-to-try legislation. Dr. Joseph Gulfo, who last week said it isn't part of FDA's mission to regulate patient outcomes, will also testify.
Chairman Johnson Letter to FDA