Voice of Patient Report

On Friday, October 8, 2021 the Gorlin Syndrome Alliance convened our community’s Externally Led Patient-Focused Drug Development (EL-PFDD) meeting to educate the FDA and other stakeholders on how Gorlin syndrome impacts the lives of those affected and their loved ones. In the six months leading up to the meeting, we made every effort to encourage everyone—patients and family members, at every stage of disease—to be involved. More than 261 individuals participated in the live, webcast event, and patients’ and family members’ testimony have been captured in our Voice of the Patient (VOP) report.

This landmark report documents the severe disease burden and unmet medical needs in patients’ own voices. It includes responses to live interactive polling as well as written statements and transcripts which are summarized in the report, and shared in full in the appendices. The VOP has been submitted to the FDA for inclusion in the framework used to evaluate future therapies for the treatment of the many possible manifestations of Gorlin syndrome. Patients’ views carry great weight with the agency when it considers whether to approve a new therapy.

The Voice of the Patient report, video of the live webcast, medical expert Dr. Joyce Teng’s slides, and transcripts are posted on the GSA website below. The photo gallery in Appendix 5 is also available HERE. Please know that this contains images that some may find disturbing.

Our community owes a deep debt of gratitude to the patients and family members who courageously shared their stories.