Externally-Led Patient Focused Drug Development (EL-PFDD)

Voice of the Patient Report

The comments collected may be used in the final PFDD Voice of the Patient report (with identifying information removed).

The Wiskott-Aldrich Foundation is holding an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting on Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia (XLT). This virtual meeting is an important opportunity for patients and caregivers to inform FDA representatives, academic and scientific researchers, medical professionals, and pharmaceutical companies about personal experiences regarding the symptoms and daily impact of WAS/XLT, as well as thoughts on current and future approaches to therapies. 

Agenda

About PFDD Meetings

• The PFDD Program was created by the FDA several years ago as a way to systematically gather information from patients and caregivers about their conditions, especially symptoms and daily impact, as well as thoughts on current and future approaches to therapies. This information helps inform FDA's drug development decision making process.

• The Wiskott-Aldrich Foundation is thankful for FDA’s approval of our proposal, recognizing the importance of learning about Classic Wiskott-Aldrich Syndrome (WAS) and X-Linked Thrombocytopenia.

• Externally-Led PFDD’s are hosted by an organization other than the FDA, in this case the Wiskott-Aldrich Foundation.

Learn more about the PFDD 

Meeting Objectives

• Give patients and caregivers a platform to share with key stakeholders and the public what it means to live with or be a caregiver for someone who has WAS/XLT, including symptoms, daily impact, and overall quality of life

• Provoke discussions around existing treatments for WAS/XLT and thoughts on future approaches to treatments.

• Publish and share a WAS/XLT Voice of the Patient report following the meeting in the summer of 2023.

Watch January 12, 2023 WAF EL-PFDD Community Webinar