CDH International is proud to be one of 85 advocacy organizations who came together and urged the Senate to include the S. 4185, the RARE Act, in the FDASALA. This wee, the Senate FDASALA manager’s amendment was released and as expected, and as we reported in our update last week, there were no RARE Act provisions included in it. The HELP Committee’s markup is on the calendar for today at 10:00 am.
The RARE Act/Baldwin Amendment #1 clarifies the intent of the Orphan Drug Act, ensuring orphan drug exclusivity is tied to that approved use or indication and is applied retroactively and prospectively.
Under the Cassidy amendment, for some of the 500 orphan drugs already on the market on the date of enactment, the exclusivity could be changed to the broader designated use, leaving little incentive for drug sponsors to conduct additional clinical trials in more challenging populations, like children. It also creates a great deal of confusion and is likely to lead to many years of litigation and court interpretation of this overly complicated and unnecessary text. Ultimately, this could delay patient access to products that have been determined to be safe and effective for their specific condition and circumstances.
Senator Baldwin and Senator Cassidy’s amendments are different, and therefore NORD (and all participating organizations) will be urging Senators to vote YES on Senator Baldwin’s amendment #1 and vote NO on Senator Cassidy’s amendment #1 should he request a roll call vote on his amendment.