McCaul Language to Explore 'Compassionate Use' Passes Appropriations Committee

McCaul Language to Explore 'Compassionate Use' Passes Appropriations Committee
Ensures Review to Make Experimental Life-Saving Cancer Treatments More Widely Available

WASHINGTON, DC – The U.S. House Appropriations Committee approved language proposed by Congressman Michael McCaul (R-TX) that would ensure a Government Accountability Office (GAO) review of Expanded Access (aka: Compassionate Use) of experimental drugs that hold lifesaving potential for terminally ill cancer patients who have exhausted all other treatment options.

The following language was included in the FY'15 Agriculture Appropriations Bill, which passed the Appropriations Committee today:

“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.”

Congressman McCaul: “I applaud the Appropriations Committee for including this language.  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.

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. 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."

Congressman McCaul is founder and co-chairman of the non-partisan Congressional Childhood Cancer Caucus.

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