CAF Speaks at FDA Pediatric Advisory Committee Meeting

September 17, 2015 – The FDA (Food and Drug Administration) Pediatric Advisory Committee (PAC) advises and makes recommendations to the Commissioner of Food and Drugs regarding a range of issues related to prescription drugs intended for use in the pediatric population. A panel of doctors and other medical specialists sit on this panel, with appropriate FDA staff providing support. CAF’s National Vice President Amy Celento currently serves as its patient/family advocate, a role that is vital in providing the point of view of parents and family members and for which she has expert skills. As a mandate for when the FDA expanded the indication of Exjade for use among non-transfusion-dependent thalassemia patients (including those that fall within the pediatric population), the FDA reviewed all Exjade-related Adverse Events reported through the FDA Adverse Events Reporting System (FAERS) from January 23, 2013 through February 28, 2015. (Information on the case reports can be found at…/PediatricAdvisoryCommitt…/UCM461193.pdf.)

The FDA PAC met on September 16 to consider the review submitted by the FDA. This meeting opened an opportunity for CAF to ask the FDA its opinion on whether data available supported the need to amend the Exjade label and advise users to temporarily stop its use in the case of febrile illness. With this in mind, CAF testified during the public presentation portion of the meeting, as did Sarah Baqueri-Connolly, who detailed for the committee circumstances surrounding her daughter’s tragic death. Due to CAF and Sarah’s testimony, the Committee requested that the FDA further review case histories and all other information available through manufacturer’s reports and provide more data so that the committee can make a determination if a label change should be added clarifying whether Exjade use should be halted temporarily when fever is present. (CAF’s testimony is available at

We thank Sarah for her stirring testimony and the Committee for listening to our concerns and taking a huge step toward making sure that patients and doctors have information that can help ensure the safety of patients.

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