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# AATD Patients Want Better Treatment Options
- URL: https://www.fdaweb.com/aatd-patients-want-better-treatment-options/
- Published: 2016-09-15T12:00:00.000Z
- Updated: 2026-09-14T21:32:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5136819

According to a just-released FDA summary, patients with Alpha-1 Antitrypsin Deficiency (AATD) said at a 9/29/15 FDA public meeting that while significant treatment advances have occurred, much remains to be done and they would like to see a cure, through gene therapy or a lung or liver transplant, with no accompanying side effects or need for continuing maintenance of therapy. FDA held the meeting to solicit patient input on drug development, as part of its Patient-Focused Drug Development initiative under PDUFA 5\. The agency has committed to more systematically gather patients’ perspectives on their conditions and on therapies to treat their conditions by holding at least 20 meetings over five years, each focused on a specific disease area.

The meeting [summary](http://www.fda.gov/downloads/BiologicsBloodVaccines/NewsEvents/WorkshopsMeetingsConferences/UCM520518.pdf?ref=fdaweb.com) says that about 850 AATD patients or patient representatives actively participated in the session that was also attended by FDA representatives, staff from other federal agencies, industry, healthcare professionals, and patient organizations. Discussion focused on **(1)** disease symptoms and daily impacts that matter most to patients, and **(2)** patients’ perspectives on current approaches to treating AATD.

Patients cited advancements such as augmentation therapy, improved oxygen delivery systems, and transplantation. But they expressed a desire for better and more innovative treatment methods. Many patients indicated that options for treatment are not ideal, and are often accompanied by discomfort or risk. And they said that one of the biggest challenges to effective therapy was the cost of augmentation therapy and insurance limitations.

If a cure is not possible, patients said, they want safe, effective, potent, inexpensive very long acting and easy to administer products. Many participants said they have participated or would be willing to participate in a clinical trial, although a substantial number said they would join a trial only if they could also remain on their current therapy.