Patient advocacy foundation holds listening session with FDA

The Foundation for Sarcoidosis Research (FSR) recently hosted a virtual patient listening session on the disease with the US Food and Drug Administration (FDA). The agency’s listening sessions are informal, non-regulatory, invitation-only discussions in which patient perspectives are shared, with the goal of deepening the FDA’s understanding of a disease or health condition, as well as the burden on patients and loved ones, challenges in care, and barriers faced in product development, diagnoses, and treatment.

Sarcoidosis is a rare inflammatory disease characterized by the formation of granulomas—tiny clumps of inflammatory cells—in one or more organs of the body. Despite increasing advances in research, sarcoidosis remains difficult to diagnose with limited treatment options and no known cure. Approximately 175,000 people live with sarcoidosis in the US.

Prior to this listening session, FSR surveyed its stakeholders, patient community, clinicians, researchers, and pharmaceutical companies. The goal was to help to identify topics and challenges of pulmonary sarcoidosis with the highest priority.

Next, building upon the feedback culled from the survey, FSR's CEO Mary McGowan, and Lisa Maier from National Jewish Health (and an active member of FSR's scientific advisory board) provided background on the greatest barriers to diagnosis, treatment, and the advancement of clinical trials.

The FDA attended with 50 representatives from 16 offices and 5 divisions. Participants in the listening session included: The Office of the Commissioner (OC), Office of Regulatory Affairs (ORA), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH), and Center for Drug Evaluation and Research (CDER).

During the listening session, half a dozen patients and one caregiver impacted by sarcoidosis shared their personal experiences, highlighting the challenges of misdiagnosis and of long diagnosis journeys. The group recounted their struggles associated with living with sarcoidosis, and they encouraged the FDA leaders present to consider the critical need for drug development and the necessity for more flexible clinical trials and approval pathways.

"We are so thankful to our incredible patient speakers who provided the FDA with intimate and raw accounts of the difficulties associated with living with sarcoidosis," said McGowan. "Through this listening session and our continued engagement with the FDA, we hope to be able to carve the path to increased clinical trials, the approval of more therapies, and improved patient outcomes."

Next, on August 3 at 5 pm EST, FSR will host a webinar during which the audience can learn more about the session and next steps; read here for more information​. The summary whitepaper is available at www.fda.gov/PatientListeningSessionSummaries​.

FSR is an international organization dedicated to finding a cure for sarcoidosis and improving care for sarcoidosis patients through research, education, and support. Since its establishment in 2000, FSR has fostered over $6m USD in sarcoidosis-specific research efforts. Visit www.stopsarcoidosis.org​ to learn more.