Externally Led Patient-Focused Drug Development Meeting

October 31, 2023

The Externally Led Patient-Focused Drug Development Meeting was an event that took place on October 31, 2023.

The purpose of this meeting was to provide a platform for Externally Led Patient-Focused Drug Development Meeting (PFDD) and for the general public to gain insight into the experiences of patients with Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD). This meeting will facilitate an understanding of the impact of these disorders on their lives and the current treatments, care, and research available. The meeting will culminate with the development of a Voice of the Patient report based on the information provided by patients and patient representatives during this meeting.

We have identified the following objectives:

• To discuss the extent of the need for therapies in the management of the complex presentation of hEDS and HSD.
• To describe the degree to which the untreated symptoms affect the daily life and quality of life of individuals with hEDS or HSD.
• Identify the difficulties patients have obtaining therapies or drugs for their condition.

The Patient-Focused Drug Development (PFDD) program is all about getting input from patients about their diseases and how they’re treated. This helps the FDA make better decisions when approving new drugs.

In PFDD meetings, the FDA, along with others like drug makers and doctors, listen to patients, their families, and advocates. They talk about what symptoms are most important to them, how the disease affects their daily lives, and their experiences with current treatments. This information helps the FDA make decisions about new drugs, from development to approval.