Government to review ‘compassionate use’ system

Rep. Michael McCaul, R-Texas. (AP Photo/Jacquelyn Martin)

AUSTIN (KXAN) — Access to experimental and potentially life saving cancer treatments could get easier thanks to a new government review of the process.

The U.S. House Appropriations Committee approved language proposed by Congressman Michael McCaul that would ensure a government accountability office review of expanded access, also known as compassionate use, of experimental drugs that hold lifesaving potential for terminally ill cancer patients who have exhausted all other treatment options.

KXAN started investigating this issue in August 2013, when Austin advocate Andrea Sloan was fighting to get an experimental ovarian cancer drug her doctors believed could prolong her life. The pharmaceutical company who makes the drug refused to provide it to Sloan, even though the Food and Drug Administration said she was a good candidate.

The language included in the Agriculture Appropriations Bill, which passed the Appropriations Committee Thursday, says:

“The Committee is concerned with a lack of useful data regarding the number of Expanded Access (sometimes called compassionate use) requests made on behalf of patients that are denied by sponsors of investigational products. In order to obtain an accurate understanding of the scope of this problem, the Committee requests that GAO conduct a review of how the FDA is working with all stakeholders to accelerate the approval of innovative, save and effective medicines, and how FDA takes into account safety and efficacy data from expanded access programs.”

“I applaud the Appropriations Committee for including this language,” McCaul said Thursday. “It is a significant first step in finding ways to make treatments more widely available. This language will guarantee a non-partisan Congressional review of the difficulties patients have obtaining life-saving treatments through Compassionate Use and by including this language in statute, it increases awareness of a great medical need that is currently unmet.”

In November, KXAN traveled to Washington D.C. to find out what Congress was doing to fix the system. McCaul said he was working to reform the compassionate use process in the United States.

“As the costs of drug development have increased and the time it takes to develop new treatments has grown, terminally ill patients are increasingly asking drug manufacturers for compassionate use to try potentially lifesaving experimental drugs when all other options have been exhausted,” McCaul said.

“With the increase in compassionate use requests, members of Congress are acknowledging we must have more information to help us identify barriers for both the patients that need the life saving drugs and the companies developing them.  I look forward to continuing to work with my colleagues in Congress and with stakeholders on all sides of this issue on ways to improve expanded access,” he concluded.

Through an open records request to the FDA, KXAN learned that between October 2009 and October 2012, the FDA granted 3,149 of 3,170 requests for compassionate use. Currently, the drug manufacturer must agree to grant compassionate use even before the FDA approves it.

Another drug company ultimately granted Andrea Sloan compassionate use of their drug, but her cancer had spread significantly and she died on January 1, 2014.

Congressman McCaul is founder and co-chairman of the non-partisan Congressional Childhood Cancer Caucus. provides commenting to allow for constructive discussion on the stories we cover. In order to comment here, you acknowledge you have read and agreed to our Terms of Service. Users who violate these terms, including use of vulgar language or racial slurs, will be banned. If you see an inappropriate comment, please flag it for our moderators to review.

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