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Bill Aims to Increase Enrollment for Rare-Disease Clinical Trials

Tuesday, June 23, 2009

A new bill introduced in the House of Representatives last week seeks to increase patient participation in clinical trials for rare diseases.

The “Improve Access to Clinical Trials Act,” sponsored by Reps. Edward Markey (D-Mass.) and Cliff Sterns (R-Fla.), would enable patients with rare diseases (defined as those with fewer than 200,000 patients) to participate in clinical trials without losing their eligibility for government healthcare benefits.

The current law, which prevents people who receive Supplemental Security Income (SSI) from accepting research compensation, limits participation in trials for rare diseases like cystic fibrosis, said Robert Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation.

“We’re really on the threshold of being able to do some great things for rare and orphan diseases. Companies are not going to invest in the development of drugs if there’s the slightest chance that the drug can’t be evaluated. Anything that could exclude 5% or 10% of the patients could be a real barrier,” Beall said.

The Cystic Fibrosis Foundation spent two years working on the new legislation and gaining support for the bill after a number of patients with cystic fibrosis said they would not participate in clinical trials because they risk losing their SSI and possibly Medicaid benefits.

“We went to the Social Security Administration and said, ‘Hey guys, does this make sense?’ We talked and we talked until we got blue in the face. They said basically the only way we can do this is a legislative deal,” Beall said.

The proposed bill would permit patients to be reimbursed up to $2,000 a year for participating in clinical trials, Beall said.

Increasing patient participation in clinical trials is particularly important to the Cystic Fibrosis Foundation because the organization has a pipeline of nearly 30 drugs waiting to be tested. The Foundation needs to double the number of patients participating in clinical trials over the next two years if they have any hope of getting those drugs to market, Beall said. For this reason, the Cystic Fibrosis Foundation pushed forward with the bill without taking the time to get other rare-disease foundations on board.

 “We face one of the most critical problems of all the orphan diseases and the rare diseases because of our pipeline. We have to get more clinical trials done, and we’ve got a timeline. We’re not waiting to construct consortiums or anything like that. We just had to push forward,” Beall said.

Now that the bill has been introduced, Beall said he has received numerous calls of support from other rare-disease organizations that didn’t know about the proposed legislation. The Cystic Fibrosis Foundation hopes to get the bill passed by year’s end.

“We’re optimistic about the bill’s chances,” Beall said. “Thirty members of Congress sponsored the bill before it was even introduced, and we have very senior members on as co-sponsors. A lot of leadership has been supportive of this, and no one has indicated anything but ample support.”

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