Bipartisan legislation introduced on May 11 by U.S. Sen. Bill Cassidy (R-LA) would set limitations on exclusivity for orphan drugs, which are developed to treat medical conditions that, because they are so rare, would not be profitable to produce without government assistance.
“Vague laws can be exploited, resulting in fewer patients getting the treatments they need to survive,” Sen. Cassidy said in a May 13 statement. “This bill clarifies the law to ensure incentives to innovate continue to produce lifesaving cures for our rarest diseases.”
Sen. Cassidy cosponsored the Retaining Access and Restoring Exclusivity (RARE) Act, S. 4185, with bill sponsor U.S. Sen. Tammy Baldwin (D-WI) to give the U.S. Food and Drug Administration (FDA) the necessary authority to approve the same drug from different manufacturers if they aim to serve different patient populations, according to a bill summary provided by the lawmakers.
Additionally, S. 4185 would help combat manufacturers’ efforts to take advantage of the Sept. 30, 2021, U.S. Court of Appeals for the 11th Circuit’s decision in Catalyst Pharms., Inc. v. Becerra. In that case, the court ruled in favor of Catalyst Pharmaceuticals Inc., forcing the FDA to withdraw marketing approval for a drug that provides the only approved treatment option for children with a rare disease called Lambert-Eaton myasthenic (LEMS) syndrome, the summary says.
Because of the court’s decision, drug companies are currently incentivized to seek the broadest orphan drug designation as possible and then focus clinical studies only on the narrowest patient populations that would support approval, according to the summary, which says this results in companies being able to rely on the broader designated orphan disease and block approval for any different uses or other patient populations.
If enacted, S. 4185 would fix this issue by codifying the FDA’s long-standing interpretation of the Orphan Drug Act of 1983 to ensure that the scope of the orphan drug exclusivity is clarified to apply only to the same approved use or indication within such rare disease or condition instead of the same disease or condition, the summary states.
The National Organization for Rare Disorders and Children’s Wisconsin support S. 4185.