Dear Dr. Joffe and Dr. Stein,
We will share your June 17 letter with the NPC community today. However, we feel the FDA decision to allow only the early-infantile children and children currently in the EAP program to have access to Adrabetadex is not sufficient. All children and adults should have access to this program in the spirit of transparency and fairness, unless there is data that FDA possesses to confirm that the intervention exceeds its potential for effectiveness from a patient-centered perspective.
As you are aware, for the past few years our community has been presented clinical trial and real world data that demonstrates the effectiveness of Adrabetadex for NPC patients. Much of this data is published in peer-reviewed journals and some is currently being collected and unpublished. Additionally, there exists real world data from patients and families (representing all ages) who have told and shown us how they have seen a clinical benefit from Adrabetadex. We feel strongly that you should speak to every possible NPC patient/family directly and learn about their experience while taking this therapy.
Also, the NPC community has carefully captured its benefit-risk perspectives to support regulatory review of potential NPC therapies. This information has been shared with the FDA as part of our coordinated patient-focused drug development initiative. It is clear to us that the totality of data shows Adrabetadex has positive effects and NPC patients and families understand the risks. Each of them deserves the opportunity to seek access under an Expanded Access Program.
Again, we feel if you talk to these patients and families and review all the data that is available, you will agree with our urgent appeal. Please consider this request. We would be happy to respond to any questions, and to put you in touch with these families and appreciate your reconsideration of this important matter.
Cindy Parseghian
Sean Kassen
cc: Dr. Katie Donahue
Dr. Julie Beitz
Dr. Patrizia Cavazzoni
Sean Kassen, PhD
Director, Ara Parseghian Medical Research Fund, University of Notre Dame
215 Jordan Hall of Science
Notre Dame, Indiana 46556-4618
574-631-4689 (office)
574-302-7568 (cell)
web: Parseghianfund.nd.edu
