McCaul Introduces Bill to Improve Access to Potentially Lifesaving Drugs for Sick Patients

McCaul Introduces Bill to Improve Access to Potentially Lifesaving Drugs for Sick Patients
The Andrea Sloan Compassionate Use Reform and Enhancement Act

WASHINGTON, DC –Today, U.S. Representative Michael McCaul (R-TX) introduced H.R. 909, the Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act, which would make important reforms to the Food and Drug Administration's (FDA) expanded access ("compassionate use") process, by ensuring seriously ill patients have the right to know the compassionate use policies of pharmaceutical companies developing the most innovative treatments. He was joined by Representative G.K. Butterfield (D-NC), Rep. Michael Burgess (R-TX), Rep. Doris Matsui (D-CA), Representative Morgan Griffith (R-VA), and Representative Leonard Lance (R-NJ). The bill is named after Andrea Sloan, an Austin resident who died on New Year's Day 2014 after being denied access to a potentially lifesaving drugs to treat her seven-year battle with ovarian cancer.

The FDA permits patients, on a case-by-case basis, to access treatments still in the development process and outside of the clinical trial setting when certain criteria are met. Recent high profile cases, such as Andrea Sloan and seven-year-old pediatric cancer patient Josh Hardy's requests for access to experimental drugs, and the recent passage of "Right to Try" laws in several states, cry out for a federal response to reform and enhance the compassionate use program. In 2014, the FDA saw a 92% increase in compassionate use requests.

Upon introduction of the bill, Representative McCaul said, "Across the United States, patients with life-threatening conditions are desperate for treatments that hold the potential to save and prolong their lives. 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 will:

• 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.

McCaul has been a leader on compassionate use since the passing of Andrea Sloan early 2014. He released a white paper on compassionate use in October shortly after the annual Childhood Cancer Summit that he hosted in Washington where he brought together leaders from the pharmaceutical industry and government to discuss patient access to experimental drugs. A section-by section of the bill can be found here.

The Andrea Sloan CURE Act has been included in the 21st Century Cures Act Discussion Document that the Energy and Commerce Committee unveiled in January.

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