FDA Listening Session Gives Patients a Chance to Voice Their Concerns

Getting diagnosed is a significant challenge for pulmonary sarcoidosis patients

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by Steve Bryson, PhD |

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The Foundation for Sarcoidosis Research (FSR) has hosted a virtual session attended by people with pulmonary sarcoidosis and representatives from the U.S. Food and Drug Administration (FDA).

FDA Patient Listening Sessions are small, informal, non-public meetings that discuss medical conditions from a patient’s perspective. The sessions seek to improve the agency’s understanding of a condition, the effect of a disease on patients, and the barriers to diagnosis, treatment, and therapeutic development.

“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,” Mary McGowan, the foundation’s CEO, said in a press release. “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.”

A summary of the listening session was published in the FSR white paper “FDA Patient Listening Session on Pulmonary Sarcoidosis: Stories for Progress.”

Sarcoidosis is caused by the abnormal formation of granulomas — clumps of inflammatory cells — in one or more organs. When they form in the lungs, which occurs in about 90% of cases, the condition is called pulmonary sarcoidosis, with symptoms such as shortness of breath and a persistent cough.

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What’s the purpose of FDA Listening Sessions?

Patient Listening Sessions are one way that the patient community can share their perspectives by talking directly with FDA staff. To identify the highest priority topics and challenges of living with sarcoidosis before the sessions, the FSR surveyed the patient community, clinicians and researchers, biotech and pharmaceutical companies, and stakeholders.

Based on this feedback, McGowan, alongside Lisa Maier, MD, a pulmonologist at National Jewish Health in Colorado, and an active FSR scientific advisory board member, provided background on the highest barriers to diagnosis, treatment, and the advancement of clinical trials.

The listening session was attended by 50 FDA representatives from 16 offices and five divisions. This included the Office of the Commissioner, Office of Regulatory Affairs, Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health.

Six patients and one caregiver shared their stories of the challenges of misdiagnosis and the lengthy diagnostic process.

“I have been from doctor to doctor. I have spent most of the last ten years in absolute agony,” said an anonymous patient living with the condition.

The patient survey showed that 32% waited at least four years for a sarcoidosis diagnosis despite advanced technology such as X-rays, CT scans, MRIs, and biopsies. Up to 12% waited eight years or longer to be diagnosed, and 35% of respondents received cancer treatment before sarcoidosis was confirmed.

The attending caregiver also described the difficulties of caring for someone with sarcoidosis.

“We have had to give up on so many of our dreams over the last 8 years as I have watched sarcoidosis chip away at my husband bit by bit,” said Jean.

Nearly all surveyed patients (95%) indicated that their daily activities were limited due to the condition, with 75% noting financial impacts, and 56% reported leaving a job due to the diagnosis.

The patients strongly urged the FDA to see the need for therapeutic development as anti-inflammatory steroids are often prescribed as the first-line treatment for sarcoidosis. However, steroids have common side effects, including a rounded face, or so-called “moon face,” insomnia, irritability, and extreme weight gain.

“Steroids are the devil,” an anonymous patient said. “Make more medications available that will reduce the number of steroids and let me feel better.”

In addition, patients encouraged the FDA to make clinical trials and approval pathways more flexible, and find ways to enhance the incentive for manufacturers to seek new indications for existing therapies.

The FDA was urged to take a broader approach to acceptable data, clinical trial requirements, and outcome selection.

One patient, Erica, highlighted the need for decentralized trial options to better accommodate underserved populations. She encouraged the FDA to allow more responsive outcomes to make trials more accessible and accommodating to patients.

“Clinical trials [should consistently] check-in with patients to discuss not only their reaction to the drug but their mental health status…are imperative to improving representation in trials,” Erica added.

Maier noted that sarcoidosis is underfunded and its research needs more federal support. As a result, clinicians and researchers lack an understanding of the disease’s cause, symptoms, and natural progression.