FDA Listening Session on Pemphigus and Pemphigoid

listening session

Published April 13, 2021
On February 8, 2021, the US Food and Drug Administration (FDA) held a Listening Session with patients representing the International Pemphigus & Pemphigoid Foundation (IPPF). Patient Listening Sessions are intended to be an opportunity for the FDA’s medical product centers to engage with patients and their advocates. The IPPF session was patient-led, meaning that the IPPF requested and received the permission to share its members’ perspectives with the FDA.

Listening session objective

The objective for this listening session was to have a dialogue with the FDA to share the emotional experience of the patient journey as well as the burden these diseases have on all aspects of a person’s life. This includes the time it takes to get a diagnosis, the burdens of treatment options, and the undertreated areas of the diseases that affect the physical, emotional/psychological, and financial health of five pemphigus and pemphigoid patients.

Summary of topics discussed

Pemphigus and pemphigoid are rare, ultra-orphan, autoimmune, blistering diseases that result in potentially life-threatening destruction of the skin and mucosa. The patient’s immune system makes antibodies that attack healthy cells in the skin or mucous membranes. As a result, skin cells separate from each other, fluid collects between skin layers, and blisters form. These blisters may cover a large area of skin. This results in fragile, extremely tender lesions that do not go away without proper treatment. It takes the average pemphigus or pemphigoid patient five healthcare providers and ten months to obtain a correct diagnosis. Currently, no cure exists for pemphigus or pemphigoid, only treatments and remission.

According to recent literature in the British Journal of Dermatology, pemphigus is rarer than pemphigoid. The approximate incidence of pemphigus is .58 – .80:100,000 people, and the approximate incidence of pemphigoid is 7.3 – 7.93:100,000 people.

These diseases are known to affect people across gender, racial, and cultural lines. However, there are certain groups of people who have a higher incidence of the diseases, such as Eastern Europeans of Jewish descent and people of Mediterranean, Northern India, and Persian descent.

The FDA Listening Session included further discussion on:

  • Diagnostic delays
  • Treatment options
  • Medical burdens
  • Investigational research
  • Mental and social burdens

Read the entire summary of the FDA Listening Session on Pemphigus and Pemphigoid by downloading the PDF.