Senators Bill Cassidy, M.D (R-LA) https://www.cassidy.senate.gov and Tammy Baldwin (D-WI) https://www.baldwin.senate.gov have introduced the Retaining Access and Restoring Exclusivity (RARE) Act which would help preserve access to treatments for rare diseases especially for children.
According to Senator Cassidy, “Vague laws can be exploited, resulting in fewer patients getting the treatments they need to survive. This bill clarifies the law to ensure incentives are in place to innovate and will continue to produce lifesaving cures for our rarest diseases.”
On September 30, 2021, the U.S. Court of Appeals for the 11th Circuit issued their decision on Catalyst Pharms, Inc. v. Becerra and ruled in favor of Catalyst Pharmaceuticals, Inc. which forced FDA to withdraw marketing approval for a drug. In doing so, that provides the only approved treatment option for children with a rare disease called Lambert-Eaton Myasthenic Syndrome (LEMS).
Because of the 2021 court decision, drug companies are currently incentivized to seek the broadest orphan drug designation as possible and focus clinical studies only on the narrowest patient populations that would support approval. In doing so, Companies would then rely on the broader designated orphan disease and block approval for any different uses or approval for other patient populations.
Peter L. Saltonstall, President and CEO National Organization for Rare Disorders (NORD), applauds Senator Baldwin and Senator Cassidy for recognizing the urgent need to safeguard the Orphan Drug Act (ODA) and preserve the original intent of the law.
He further explained, “More than 90% of the 7,000 known rare diseases still have no treatment or cure, so it is critical the incentives as envisioned by ODA, continue to exist so that new orphan drugs are developed into the future.”