{"id":110249,"date":"2023-03-02T10:27:12","date_gmt":"2023-03-02T15:27:12","guid":{"rendered":"https:\/\/rarediseases.org\/?p=110249"},"modified":"2023-03-02T10:27:12","modified_gmt":"2023-03-02T15:27:12","slug":"nord-commemorates-five-years-of-fda-listening-sessions","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-commemorates-five-years-of-fda-listening-sessions\/","title":{"rendered":"NORD Commemorates Five Years of FDA Listening Sessions"},"content":{"rendered":"

By Debbie Drell\u00a0<\/strong><\/p>\n

I\u2019ve been a part of thousands of conference calls and virtual meetings during the course of my career. But while the details of many of those conversations get blurred over time, I can still clearly remember the voices and stories from a 60-minute conference call on October 23, 2018.<\/span>\u00a0<\/span><\/p>\n

On that seemingly nondescript day nearly five years ago, a small team of staff from the U.S. Food and Drug Administration (FDA) and National Organization for Rare Disorders (NORD) opened a conference call line to meet with seven people who had been diagnosed with hemophilia or were caring for children with this condition.<\/span>\u00a0<\/span><\/p>\n

What we heard was truly powerful.<\/span>\u00a0<\/span><\/p>\n

During that hour, patients and caregivers shared their fears and hopes for the future of gene therapy as a treatment for hemophilia \u2014 a rare, genetic bleeding disorder that is well-known for its cause of difficulty in blood clotting.<\/span>\u00a0<\/span><\/p>\n

The call was one of the first NORD\/FDA Patient Listening sessions, and patients shared quite vulnerably \u2014 sometimes through tears \u2014 their concerns and hopes for clinical trials involving gene therapy.<\/span>\u00a0<\/span><\/p>\n

The call gave the FDA a chance to hear directly from patients and caregivers about their experiences. The FDA\u2019s team wanted to learn many things, including whether someone living with hemophilia would be willing to participate in a clinical trial in gene therapy if there were risks involved. Specifically, they wanted to learn what level of benefit would outweigh the risks.<\/span>\u00a0<\/span><\/p>\n

It was during this call that I saw firsthand the power of these patient listening sessions.<\/span>\u00a0<\/span><\/p>\n

The FDA heard clearly from patients and caregivers that enrolling in clinical trials is a family decision \u2013 and that children must be involved as well:<\/span>\u00a0<\/span><\/p>\n

\u201c…the decision to enroll in a clinical trial affects many people beyond the patient. This could include their spouse, children, parents\/siblings, and other dependents, all of whom should be involved in the decision to enroll. If a clinical trial were available for children, the child should be old enough to be involved in the decision and understand the commitment of the trial.\u201d (citation: FDA-Requested Patient Listening Session Summaries<\/a>, https:\/\/www.fda.gov\/media\/124436\/download<\/a>)<\/em><\/p><\/blockquote>\n

Since then, the FDA and NORD have hosted a total of 14 listening sessions that bring the rare disease patient and caregiver voice to FDA scientists and researchers.\u202f<\/span>\u00a0<\/span><\/p>\n

This partnership offers people living with an array of rare diseases access to an important avenue for helping advance medical research and new product development and gives FDA professionals the chance to hear directly from rare disease patients and caregivers as they make decisions about new treatments and products.<\/span>\u00a0<\/span><\/p>\n

Creating Connections<\/span><\/b>\u00a0<\/span><\/p>\n

Any time a person with a rare disease has a chance to share their story with the FDA, it\u2019s a significant moment.\u202f<\/span>\u00a0<\/span><\/p>\n

But these opportunities do not happen in a vacuum. The FDA needs to be sure it is hearing from a diverse cross-section of patient voices and experiences in order to gain a deeper understanding of what matters most to those who are living with rare conditions. And the patients and caregivers who participate must feel safe and comfortable before they can provide personal and sometimes intimate accounts of their rare disease journey.<\/span>\u00a0<\/span><\/p>\n

That\u2019s why NORD is such an important partner in the Patient Listening Sessions program.<\/span>\u00a0<\/span><\/p>\n

As a result of our 40-year history of serving the rare disease community, we have built deep relationships with rare disease patients and patient organizations.<\/span>\u00a0<\/span><\/p>\n

In working with the FDA to arrange and facilitate these sessions, NORD engages diverse rare disease patient communities \u2014 ensuring the FDA hears perspectives that reflect a cross-section of those who are impacted by rare diseases.<\/span>\u00a0<\/span><\/p>\n

In addition to helping find patients and caregivers, NORD also helps prepare patients and caregivers participating in FDA listening sessions to ensure that they feel comfortable, safe, and confident about their participation.<\/span>\u00a0<\/span><\/p>\n

For example, when we approached one of NORD\u2019s 340 patient organization partners \u2014 the Proteus Syndrome Foundation \u2014 to take part in a 2022 listening session, we took the time to fully explain the process and help participants prepare for the opportunity.<\/span><\/p>\n

Our process empowered participants in the listening session to feel safe and comfortable when they took part in their listening session.<\/span>\u00a0<\/span><\/p>\n

\u201cOur community was unfamiliar with FDA Listening Sessions, but when NORD came to our Family Conference in Bethesda, MD, to speak about it, they became excited for the chance to inform the FDA of their experiences with proteus syndrome,\u201d said Kim Green, Executive Director, Proteus Syndrome Foundation.<\/span>\u00a0<\/span><\/p>\n

Advancing Research and Therapies<\/span><\/b>\u00a0<\/span><\/p>\n

Since the start of this partnership, NORD has helped coordinate listening sessions involving 100 patients and caregivers across a diverse range of more than 11 rare diseases ranging from Childhood Cerebral Adrenal Leukodystrophy (CCALD) to Fabry Disease and Mucopolysaccharidosis Type II (MPS II), or Hunter syndrome.<\/span>\u00a0<\/span><\/p>\n

Patients and caregivers have answered quite honestly some tough, and real-life questions, such as:<\/span><\/p>\n