Obama signs improved compensation bill for rare-disease clinical trial participants
Two hurdles were cleared for a relatively small number of clinical trial participants when President Barack Obama signed the Ensuring Access to Clinical Trials Act of 2015. The legislation enables individuals with rare diseases to receive up to $2,000 for participating in clinical trials without having that compensation counted as income when calculating eligibility.
The two-page bill, which allows that exclusion, also makes permanent the original law, known as the Improving Access to Clinical Trials Act of 2009, which was set to expire in October. The House of Representatives approved the change in September after the Senate voted on it in July. The president signed it earlier this month.
“Although the $2,000 is at the upper end, the average compensation has been between $500 and $1,500, based on the length of time and number of site visits, as the money is not for travel and lodging but more for the patient’s time taken off from work or school needed to participate in the trial,” said Paul Melmeyer, associate director of public policy at the National Organization for Rare Disorders (NORD), adding that the funds come from clinical trial sponsors. “As far as the cost goes, there was no congressional opposition.”
A key part of the enabling legislation, which was supported by 75 patient, industry, academic and medical organizations, is that an application for those funds will no longer require scrutiny for Supplemental Security Income (SSI) and Medicaid as part of their eligibility. The original law required a government-benefits review to meet income requirements—a concern that some supporters of the revisions said could hold back people with rare diseases from trial participation.
“Patients and families shouldn’t have to choose between participating in clinical trials where they can gain access to potentially life-saving treatments or social safety net programs,” Rep. Jim McGovern (D-Mass.), one of the co-sponsors of the bipartisan legislation, said in a prepared statement.
Prior to the passage of the 2009 law, some people with rare diseases chose not to participate in clinical research because, according to NORD, they feared that the compensation from the trials would make them ineligible to receive government benefits. Meanwhile, those receiving SSI and Medicaid who already were participating in such research were afraid of losing those same benefits.
Rare diseases’ limited patient population and the precise requirements for participating in clinical trials made it difficult for researchers to recruit the number of participants needed for their trials, according to the Cystic Fibrosis Foundation, which along with the Muscular Dystrophy Association worked with NORD to get congressional passage of the legislation.
“The legislation will have a real impact on whether individuals can continue to participate in clinical trials for muscular dystrophy, ALS (amyotrophic lateral sclerosis) and other neuromuscular and rare diseases,” said Kristin Stephenson, vice president of policy and advocacy at the Muscular Dystrophy Association. “These diseases are expensive to live with, and Medicaid is essential for many to afford healthcare. If participating in a clinical trial could mean potentially losing Medicaid or SSI, it could have a chilling effect on the development of treatments and cures.”
Of the estimated 7,000 rare diseases, only 450 of them have identified treatments and 75% of the patients affected are children. A rare disease population in the U.S. is defined as having up to 200,000 people with the illness, although most rare diseases are significantly smaller.
“Today, 30 million people and their families have added hope that their tremendous unmet medical needs are one step closer to being addressed,” NORD President and CEO Peter Saltonstall said in a prepared statement. “Studying one rare disease can often lead to understanding of other rare diseases, as well as understanding of more common diseases.”
Ronald Rosenberg is a former business and science reporter for The Boston Globe. He has written features for New Scientist and Inc. magazine. His lengthy journalism career includes editing an award-winning weekly newspaper in Cornwall, N.Y. Ron also was a media relations specialist for the science faculty at Boston University, and a Knight Science Journalism Fellow at the Massachusetts Institute of Technology.
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