New Legislation Seeks to Boost Participation in Clinical Trials for Rare Diseases

June 15, 2009

New legislation introduced today would allow patients with rare diseases to participate in clinical drug studies without losing their eligibility for government healthcare coverage.

Current law prevents many people who receive Supplemental Security Income (SSI) from accepting research compensation because it would make them ineligible to continue receiving government medical benefits. This financial penalty prevents significant numbers of people with rare diseases from participating in clinical studies.

Researchers developing drugs to treat rare diseases struggle to recruit participants for clinical trials because of limited patient populations. This is particularly true for cystic fibrosis (CF). More than 30 promising CF drugs are in development, yet only about 30,000 people in the United States have this life-threatening, genetic disease.

“We are grateful to Representatives Edward Markey and Cliff Stearns for introducing this important bill that allows more people with rare diseases – including cystic fibrosis – to participate in clinical trials,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “Cystic fibrosis is a devastating disease and this new legislation clears the way for promising drugs to move more swiftly from the research phase into the hands of people who need them.”

The bill is co-sponsored by Representatives Edward J. Markey (D-MA) and Cliff Stearns (R-FL) and 30 members of the House of Representatives.

Read the full press release here.