Johns Hopkins University Hospital physician and Cystic Fibrosis Foundation Vice President of Therapeutics Development Michael Boyle, M.D., testified on Friday, September 18th before the House Energy and Commerce Committee’s on Health, urging Subcommittee members to support Bill H.R. 209, which would make a permanent the Ensuring Access to Clinical Trials Act (EACT) of 2009, currently set to expire on October 5, 2015. EACT allows people with rare diseases such as cystic fibrosis to participate in clinical trials and receive up to $2,000 annually in compensation for participation in clinical trials, without it counting towards their Supplemental Security Income (SSI) and Medicaid eligibility.
“There are more clinical trials underway this year for new CF treatments than ever before. We must ensure that nothing puts this research at risk and that no one with CF is excluded from participating in clinical trials for potentially life-saving treatments,” Dr. Boyle contends in a CF Foundation release. “The Cystic Fibrosis Foundation urges Congress to pass the Ensuring Access to Clinical Trials Act without delay.”
Cystic Fibrosis (CF) is a rare, progressive, life-threatening autosomal recessive genetic disorder that affects the lungs and digestive systems. The defective gene and its protein product that causes CF disrupt the body’s ability to hydrate and effectively clear mucus in the lungs and/or digestive tract In persons with CF. affects the normal functioning of the lungs and digestive system, and causes the body to produce unusually thick, sticky mucus that attracts foreign pathogens, such as viruses and bacteria, making patients more susceptible to infectious diseases (i.e. pneumonia). The high microbial burden causes inflammation of the lung tissue and a high likelihood of tissue destruction due to the frequency of infections that most often lead to pulmonary exacerbation in these patients including loss of lung function, restricted breathing, obstruction of the pancreas which stops natural enzymes from helping the body break down and absorb food and results in digestive problems.
According to the Centers for Disease Control (CDC), approximately 1,000 new CF cases are diagnosed in the US each year, with more than 75 percent of those patients under the age of two at the time of diagnosis. An estimated 30,000 children and adults in the US and 70,000 worldwide have the disease.
Patients diagnosed with CF typically have lifespans of approximately 30 years, but that metric has been improving with greater understanding of the disease and development of better treatments resulting in many CF patients now living into their forties, and a few into their fifties.
In his testimony to the Subcommittee, Dr. Boyle spoke from experience as a physician at the Johns Hopkins University Hospital Cystic Fibrosis Center where he has witnessed first-hand the devastating effects cystic fibrosis has on patients, as well as the life-altering impact of clinical trials in developing new treatments. In his remarks, the doctor cited the poignant case of a young man with CF who declined to participate in a clinical trial for a drug later determined to be the most effective treatment for his type because the $750 compensation he would have received would put his much-needed Medicaid and SSI support in jeopardy, since forgoing compensation is not allowed by most hospital review boards. The young man passed away from complications of CF approximately four months later.
In addition to highlighting how this bureaucratic barrier can inhibit people with CF from accessing potentially life-saving treatments, Dr. Boyle described to the Subcommittee how it makes the challenges already confronting researchers in recruiting participants from rare disease communities with very small populations even more difficult.
The EACT passed a vote by the Senate on July 16, 2015 — introduced by Senators Ron Wyden (D-OR), Orrin G. Hatch (R-UT), Edward J. Markey (D-MA), and Sherrod Brown (D-OH) in the Senate and Representatives Lloyd Doggett (D-TX), Tom Marino (R-PA) and Jim McGovern (D-MA) in the House. The CF Foundation championed the original IACT legislation and has actively supported the Ensuring Access to Clinical Trials Act in 2015.
Recognized by the National Institutes of Health as a model of care for a chronic disease, the Bethesda, Maryland based Cystic Fibrosis Foundation is a donor-supported nonprofit organization. For more information, visit
Cystic Fibrosis Foundation
Johns Hopkins University Hospital
The U.S. Food and Drug Administration (FDA)
National Institutes of Health
Johns Hopkins University Hospital
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