Cooley’s Anemia Advocacy Forum Update: CAF Meets with Government Agencies

October 2, 2014The Cooley’s Anemia Advocacy Forum (CAAF) is a Cooley’s Anemia Foundation project aimed at helping thalassemia patients and families learn how to make their voices heard on federal issues of importance to the thalassemia community. (A brochure about CAAF can be downloaded by clicking here.)

The following report comes from Lyle Dennis and Katie Schubert of the Cooley’s Anemia Advocacy Forum:

On Wednesday, September 24, the Cooley’s Anemia Foundation hosted a government-wide stakeholder meeting at the National Institutes of Health (NIH) main campus in Bethesda, MD. Foundation President Tony Viola, Executive Director Gina Cioffi and Medical Advisory Board member Dr. Alexis Thompson opened the meeting and presented to the group of 20 the challenges faced by thalassemia patients, and the work of the Foundation to address those challenges.

Representatives from the National Heart, Lung and Blood Institute, National Institute on Diabetes, Digestive and Kidney Diseases, Centers for Disease Control and Prevention, Health Resources and Services Administration, National Center on Advancing Translational Sciences and the US Food and Drug Administration all participated in this important dialogue. The objectives of the seven-hour long meeting were to understand the major thalassemia-related initiatives in the federal government and to identify ways in which the Foundation and federal government can work more closely together for the benefit of patients and their families.

Following presentations from Gina and Dr. Thompson, the NHLBI, NIDDK, CDC, HRSA, and NCATS officials each presented information related to their ongoing thalassemia-related projects, while FDA was able to offer general observation and comments on the issues that had been discussed.

The very interesting and “meaty” part of the day occurred during open discussion in the afternoon. There was a great deal of conversation about some of major themes that emerged throughout the day including current management strategies (e.g. transfusion/chelation and stem cell transplant) versus evolving management strategies (e.g. matched unrelated transplants, gene therapy) versus experimental management strategies (e.g. novel chelators, gene editing). There was also a lot of talk about the relationship between thalassemia and other hemoglobinopathies moving forward. A significant discussion portion of the discussion revolved around the Affordable Care Act and challenges faced by thalassemia patients.

A full summary of the meeting is being worked up by CRD. We will be making that available to all of you, along with copies of the slides that were presented by the participants in the meeting.

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