The purpose of this Patient-Focused Drug Development meeting is to obtain input on the symptoms and other impacts that matte most to patients with AATD. FDA also intends to seek patients’ perspectives on current approaches to treating this disorder. FDA expects that this information will come directly from patients, caregivers, and patient advocates.
The questions that will be asked of patients and patient caregivers at the meeting are as follows. The meeting will be divided into two main topics:
1. The effects of Alpha-1 Antitrypsin Deficiency that matter most to you
2. Perspectives on the approaches to treatment
For each topic, a brief patient panel discussion will begin the dialogue. This will be followed by a facilitated discussion inviting comments from other patient and patient caregiver participants. In addition to input generated through this public meeting, FDA is interested in receiving patient input addressing these questions through electronic or written comments, which can be submitted to the Division of Dockets Management. For context, please indicate if you are commenting as a patient with AATD or on behalf of a child or loved one.
Registration to attend must be received by September 15, 2015
Registration from those individuals interested in presenting comments as part of the panel discussions should be received by July 31, 2015