WASHINGTON (KXAN) — The Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act, a bill aimed at improving access to potentially lifesaving drugs, was introduced Monday in Washington.
The bill was presented by U.S. Representative Michael McCaul, a Central Texas congressman. McCaul was also a friend of Austin attorney Andrea Sloan, 45, who waged a brave, public battle for compassionate use in 2013 while in the final stages of ovarian cancer. After months of requests, an anonymous drug company ultimately granted Sloan compassionate use of a drug, but her disease had progressed quickly and she passed away on Jan. 1, 2014.
McCaul introduced the new legislation in Sloan’s honor, hoping to keep other patients from having to wait so long for their requests to be granted—time they do not have.
The congressman says the CURE Act would make important reforms to the Food and Drug Administration’s compassionate use program, “by ensuring seriously ill patients have the right to know the compassionate use policies of pharmaceutical companies.” If companies refuse to grant expanded access of a drug, under the proposed legislation, they would also be required to explain to patients the reason why their request was denied.
Under the current system, the decision to grant compassionate use is at the sole discretion of the pharmaceutical company. If granted, it must be approved by the FDA on a case by case basis. A 2013 KXAN investigation found the overwhelming majority of cases granted by drug companies are approved by the FDA.
“Patients with life-threatening conditions are desperate for treatments that hold the potential to save and prolong their lives,” McCaul said in a statement. “In some extreme cases, experimental drugs or devices may be a patient’s only hope when FDA-approved treatments are not available and when patients are unable to enroll in a clinical trial. The Andrea Sloan CURE Act will give patients and doctors up front information about how to access experimental treatments and provide drug companies more certainty about the compassionate use program.”
The Andrea Sloan CURE Act would:
- Ensure that pharmaceutical companies have publicly accessible compassionate use policies for drugs treating serious or life-threatening conditions;
- Dignify patients with an explanation if their request for compassionate request is denied;
- Require the Government Accountability Office to conduct a thorough analysis of the current compassionate use program, including the number of patient denials and reasons why companies reject requests;
- Establish a compassionate use Task Force to provide recommendations to further improve the compassionate use program; and
- Require the FDA to finalize its May 2013 draft compassionate use guidance for industry and clarify how it interprets and uses adverse drug event data in compassionate use cases.