FDA lobbies Congress over rare disease court ruling with wide implications
Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.
The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.
While seemingly straightforward, the FDA now says the decision could have catastrophic consequences because of the way the decision addresses the scope of orphan drug exclusivity, potentially allowing companies to target a tiny indication for a specific disease and then automatically receive 7 years of exclusivity for the entire disease.
For instance, if the FDA approves a drug for the “treatment of adult patients with a particular gene mutation with cystic fibrosis,” the approval of the application will block the FDA from approving – for seven years (orphan drug exclusivity) – another company’s application “for the same drug for any indication [bold is FDA’s] within cystic fibrosis, including for children and for patients without the particular mutation.”
In a rare statement on the court’s decision, which FDA Law Blog points out is the equivalent of the agency lobbying Congress, the agency makes clear that without congressional action:
In the coming months, the FDA will need to consider how the decision affects drugs with active terms of orphan drug exclusivity as well as currently marketed drugs, including generics. Going forward, the FDA expects that some drugs that are in late-stage development, or that have already been submitted for marketing application review, would be blocked from approval under the Catalyst decision’s interpretation of the [Orphan Drug] Act.
The agency also explains how this new court ruling incentivizes sponsors to seek approval and exclusivity for their drugs by focusing on the smallest, easiest-to-study populations.
“Under the interpretation in the Catalyst decision, such an approval would result in exclusivity for their drug for the entire disease, even though the sponsors did not invest in studying and developing their drug for all individuals with the disease,” the agency explains.
Responding to a question from Sen. Tammy Baldwin (D-WI) on the issue, CDER director Patrizia Cavazzoni also recently said she wants to work with Congress to find a solution as this decision “will send a chill into the development of rare diseases and it will disproportionately affect children with rare diseases. It’s essential to continue to generate the study of drugs in children, so this decision will really go counter to that.”
Baldwin and Sen. Bill Cassidy (R-LA) introduced bipartisan legislation last week, known as the Retaining Access and Restoring Exclusivity (RARE) Act, which would fix this court-related issue by codifying the FDA’s longstanding interpretation of the Orphan Drug Act of 1983 to ensure that the scope of the orphan 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 FDA Law Blog also notes that as the world waits to hear whether or not the Supreme Court will take up the Catalyst case, the user fee reauthorization bill making its way through Congress also “provides the perfect vehicle for the Agency to try and get this accomplished.”
And indeed, a section of the bill marked up in the House, titled “Clarifying application of exclusive approval, certification, or licensure for drugs designated for rare diseases or conditions,” would amend the statute to replace the phrase “same disease or condition” (and similar phrases) with “same approved indication or use within such rare disease or condition” (and similar phrases).