"This bill provides potentially lifesaving treatments through clinical trials for those suffering with rare diseases, like cystic fibrosis (CF), a life-threatening genetic disease that affects about 30,000 people nationwide," Inhofe said. "This hits especially close to home for me because I have a staffer with CF who has participated in trials that seek better treatments for rare diseases. About half of these patients are on Medicare or Medicaid and are eligible for SSI benefits."
Inhofe continued, "This bill allows patients with a rare disease to disregard up to $2,000 of compensation received for participation in a clinical trial in their SSI and Medicaid income calculations. Though it will have a negligible impact on the federal budget, it will make a dramatic difference in the lives of those who will gain access to potentially life-saving treatments by enrolling in clinical trials as well as all those in the future whose lives will be improved by the medical advances that arise from this research.
"For many suffering from rare diseases, access to clinical trials are their best hope for treatment," Wyden said. "This legislation will make sure the small financial incentives these people receive will not be counted against them if they are on SSI or Medicaid. Patients suffering from rare diseases should not have to choose between their best hope for treatment or losing benefits, nor be denied the access more financially fortunate patients receive."
"Scientists and researchers across our nation continually produce new therapies that have the potential to save the lives of countless Americans who suffer from life-threatening rare illnesses," said Shelby. "These patients should not be forced to choose between the health benefits they desperately need and the opportunity to participate in a clinical trial that could improve their medical condition. I am confident that this legislation will open a pathway for more patients to receive life-saving treatments."
"For those living with a rare disease, clinical drug studies can offer a ray of hope: access to cutting-edge medical technologies that may help treat or even cure a serious illness," said Dodd. "Currently, individuals who receive public assistance often do not participate in compensated clinical trials for fear of losing their Medicaid or Supplemental Security Income. This legislation will remedy this inequity by ensuring that more Americans, including those who receive public assistance, have access to these potentially life-saving clinical drug studies."
Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation said, "We are grateful for the participation of so many key Senators on this important bill. This critical legislation will help speed the delivery of promising new drugs to the people who need them, improving health and prolonging lives."
Under current law, the small compensation provided to trial participants, which averages around $500, is included as additional income and could cause a person to lose their public assistance benefits, like Supplemental Security Income (SSI) and Medicaid. These benefits are crucial for patients living with rare diseases. For instance, nearly 50% of the CF population uses SSI or Medicaid. As a result, patients choose not to enroll in clinical trials that could dramatically improve their lives out of the fear that they may lose the benefits on which they rely.
I-ACT will protect those receiving these public benefits from having small financial incentives for clinical trials counted against them. The legislation also includes safeguards to ensure against those taking advantage of the system. The bill includes a study by the Government Accountability Office to make sure the program is working and remains fair to those with rare diseases. The legislation will also sunset in five years so that Congress can reexamine the program and the GAO report on the program.
Cystic fibrosis used to be primarily a childhood disease because people simply didn't live long enough to reach adulthood. But now, thanks to the many treatments discovered through clinical trials, the average life expectancy is 37 years old. Additionally, these advances in science allow CF patients to live more normal lives and not spend all their time in hospitals or attached to respiratory machines. The more CF patients can participate in clinical trials, the faster scientists can discover new treatments and eventually a cure. There are more than 30 promising drugs in the research pipeline right now that the CF Foundation is calling miracle drugs so it is imperative that patients have access to clinical trials so these drugs can get on the market.