2023 IPPF Externally-Led Patient-Focused Drug Development Meeting:
Understanding the Unmet Needs of the Pemphigus and Pemphigoid Community
Externally-led patient-focused drug development (EL-PFDD) meetings bring together patients and care partners, U.S. Food and Drug Administration (FDA) representatives, pharmaceutical companies, doctors, and researchers who are experts in the particular disease. For the meeting on pemphigus and pemphigoid (P/P), the goal was to hear from patients about what it’s like to live with rare, autoimmune bullous skin diseases, so the FDA and pharmaceutical companies can understand the patient experience. This patient input can help the FDA make informed decisions on approvals of potential medicines for P/P, and help pharmaceutical companies to design therapies and clinical trials that are meaningful for patients. The EL-PFDD meeting on pemphigus and pemphigoid was held on January 25, 2023. The meeting was recorded in four separate sections and is available on the IPPF’s YouTube channel and below.
Specific goals of the meeting were to help to identify areas of unmet need of patients, identify the need to develop tools to assess the benefits of patient therapies, raise disease awareness, and engage the patient community in sharing their disease experience. The IPPF is focused on the next step of the meeting, which is developing the report: “IPPF Patient-Focused Drug Development Meeting, Understanding the Unmet Needs of the Pemphigus and Pemphigoid Community Voice of the Patient.” The report will be sent to the FDA and will provide a reference point for their decisions concerning the approval of potential medicines for pemphigus and pemphigoid. The report will be available for the public to read on the IPPF website.
- Welcome and Opening Remarks
- Patient Focused Drug Development at the FDA (Dr. Shari Targum)
- Current Treatment and Limitations (Dr. Aimee Payne)
- Background: Pemphigus and Pemphigoid (Dr. Pascal Joly)
- Patient Story Videos Session 1: Disease Symptoms and Treatments- How They Impact the Daily Lives of Patients
- Session 1 Continued: Disease Symptoms and Treatments- How They Impact the Daily Lives of Patients (patient, caregiver, and clinician panel)
- Lack of Healthcare Treatments, Awareness, and Education (Dr. Deedee Murrell)
- Clinical Trial Experience and Meaningful Benefit (Dr. Victoria Werth)
- Patient Story Videos Session II: Patients’ Perspective on Available Treatments for the Disease(s), Side Effects, and How to Improve Them
- Session II Continued: Patients’ Perspective on Available Treatments for the Disease(s), Side Effects, and How to Improve Them (patient, caregiver, and clinician panel)
- Public Questions/Comments by Patients
- Closing Remarks
The meeting agenda consisted of presentations on pemphigus and pemphigoid by disease experts, a presentation by the FDA, patient panels, and extensive audience discussion sessions.
- Welcome (Patrick Dunn, IPPF Executive Director)
- Opening Remarks (Marc Yale, IPPF Advocacy and Research Coordinator)
- Patient-Focused Drug Development at the FDA (Dr. Shari Targum)
- Current Treatments and Limitations (Dr. Aimee Payne, Philadelphia, USA)
- Background: Pemphigus & Pemphigoid (Dr. Pascal Joly, Rouen, France)
- Patient Story Videos
- Session 1: Disease Symptoms and Treatments: How They Impact the Daily Lives of Patients
- Session 1: Panel of patients, caregivers, clinicians will provide comments concurrent with live polling of the audience
- Session 1: Facilitated Group Discussion by Patients (patient representatives from the audience will be invited to contribute to the discussion)
- Welcome Back Remarks
- Lack of Healthcare Treatments, Awareness, and Education (Dr. Dedee Murrell, Sydney, Australia)
- Clinical Trial Experience and Meaningful Benefit (Dr. Victoria Werth, Philadelphia, USA)
- Patient Story Videos
- Session 2: Patients’ Perspective on Available Treatments for the Disease(s), Side Effects and How to Improve Them
- Session 2: Panel of patients, caregivers, clinicians will provide comments concurrent with live polling of the audience
- Session 2: Facilitated Group Discussion by Patients (Patient representatives from the audience will be invited to contribute to the discussion
- Public Questions/Comments by Patients (Pre-submitted)
- Closing Remarks (Patrick Dunn, IPPF Executive Director)
Shari L. Targum, MD, MPH
- Shari Targum, MD, MPH earned her medical degree from the NYU Grossman School of Medicine and Masters degree in Public Health from the Johns Hopkins Bloomberg School of Public Health. She is Deputy Director, Division of Dermatology and Dentistry, Office of Immunology and Inflammation, Office of New Drugs, in the Center for Drug Evaluation and Research (CDER) at the FDA. She brings over 30 years of regulatory, industry and clinical experience.
Dr. Pascal Joly, Rouen University, France
- Pascal Joly is a Professor of Dermatology and Head of the Department of Dermatology at Centre Hospitalier Universitaire Rouen in Rouen, France. He is also Head of the French Reference Centre for autoimmune blistering diseases. He gained his medical degree from Paris V University in Paris, France in 1990, his PhD in immunodermatology from Rouen University Hospital in 1994, and qualified in oncodermatology in 1995. Professor Joly’s main areas of interest are immunodermatology and oncodermatology, including basic research and clinical and therapeutic trials on autoimmune bullous skin diseases and drug-induced reactions. He was President of the French Study Group on autoimmune blistering diseases and is currently director of the French Reference Centre for autoimmune diseases.Professor Joly has published over 500 articles in peer-reviewed journals including The New England Journal of Medicine, Nature, The Lancet, and Journal of Investigative Dermatology. He is an editorial board member of British Journal of Dermatology and a reviewer for numerous journals, including The New England Journal of Medicine, BMJ, Journal of the American Academy of Dermatology, and European Journal of Dermatology. He was the recipient of a Scientific Achievement Award from the European Academy of Dermatology and Venereology (EADV) in 2016, an International Leadership Award from the International League of Dermatological Societies (ILDS) in 2017, and a Presidential Citation Award from the American Academy of Dermatology (AAD) in 2017.
Dr. Dedee Murrell, University of NSW, Sydney, Australia
- Professor Dedee Murrell is Chair, Department of Dermatology, St George Hospital, University of NSW, in Sydney, Australia. She completed medical training at Cambridge and Oxford Universities, 3 years of internal medicine in the UK and USA, dermatology training at UNC-Chapel Hill, a fellowship in dermatopharmacology at Duke, blistering diseases and cell biology at New York University, and then became a clinical scholar at Rockefeller University in New York. She holds a doctorate on the pathogenesis of blistering disorders, her main subspecialty interest, and her current research focuses on the development and validation of clinical outcome measures for the Autoimmune blistering diseases to enable clinical trials to proceed in these orphan diseases. She was a member of the Medical Advisory Board of the IPPF 1997-2017 and founder of the Australasian Blistering Diseases Foundation in 2007.
- Dedee F. Murrell MA(Cambridge) BMBCh (Oxford) FAAD (USA) MD (UNSW) FACD FRCP (Edin) DSc (Oxford)
Professor, Faculty of Medicine, UNSW Medical School, Sydney, Australia
Head, Dept of Dermatology, St George Hospital Campus, Kogarah, Sydney, AustraliaProfessorial Fellow (Honorary), The George Institute for Global Health, SydneyChair, Communications Committee, EADVPresident, Australasian Blistering Diseases FoundationPresident, world’s first dermathon – the International Congress of Dermatology 2021 Melbourne Nov 10-13, 2021Past PositionsFirst International Board Member, EADV 2018-2021President, Australasian Society for Dermatological Research 2018-2020Executive Board, International Society of Pediatric Dermatology 2016-19Founding Co-Editor, International Journal of Women’s Dermatology
Board of Directors, Women’s Dermatologic Society 2012-16
Chair, International Sections, WDS 2009-2019Chair, World Congress Task Fund, American Academy of Dermatology 2013-16
Chair of Communications, International Society of Dermatology 2013-2017Executive Vice President, Intl Soc Derm 2010-2013Chair & Vice Chair, Maria Duran Committee, Intl Soc Derm 2007-2011Vice President, World Congress of Dermatology 2023 Sydney Bid
Associate Editor, British Journal of Dermatology
Dr. Aimee Payne, University of Pennsylvania, Philadelphia, PA
- Dr. Payne is a Professor of Dermatology and Director of the Clinical Autoimmunity Center of Excellence at the University of Pennsylvania. She received her BS in Biology from Stanford University and her MD/PhD from Washington University School of Medicine, followed by dermatology residency at the University of Pennsylvania. Her research has investigated how autoimmunity occurs in order to develop better targeted therapies for disease, which has led to a novel precision cellular immunotherapy for mucosal pemphigus vulgaris that is currently being evaluated in a phase 1 clinical trial. Dr. Payne also serves as Associate Director of the Penn Medical Scientist Training Program, chair of the NIAMS Board of Scientific Counselors, and co-founder of Cabaletta Bio, focused on engineering targeted cellular immunotherapies for B cell-mediated diseases.
Dr. Victoria Werth, University of Pennsylvania, Philadelphia, PA
- Dr. Werth received her MD from Johns Hopkins School of Medicine, followed by residency and a postdoctoral research fellowship in Immunodermatology at New York University. Dr. Werth moved to Penn in 1989 as Chief of Dermatology at the Philadelphia VA Hospital. At Penn she directs the Autoimmune Skin Disease study unit, and performs clinical and translational research studies in autoimmune skin disease funded by NIH, autoimmune foundations, and industry. She has performed a number of industry trials for pemphigus and bullous pemphigoid. She has a basic research lab at the VA devoted to autoimmune skin disease and photobiology, funded by NIH and the VA. Dr. Werth’s clinical practice specializes in the diagnosis and treatment of patients with autoimmune skin diseases, including autoimmune blistering disease, lupus erythematosus, and dermatomyositis. She led an international effort to develop disease severity tools for several autoimmune blistering diseases, including pemphigus and bullous pemphigoid. These tools are now used in international trials that are developing new drugs for autoimmune blistering diseases, including the trial leading to the recent FDA approval of rituximab for pemphigus vulgaris.
Dr. Werth has also performed studies to determine the impact of bullous diseases on quality-of-life. Her laboratory has also performed translational research to better understand how novel therapies that modulate inflammatory cells work in autoimmune skin disease. She has been on the IPPF Medical Advisory Board since the beginning of the foundation. Dr. Werth has received numerous honors for her work, including the lifetime achievement award from the Medical Dermatology Society, the Rose Hirschler Award from the Women’s Dermatologic Society, and the American Skin Association’s Research Achievement Award in Autoimmune & Inflammatory Skin Disorders, the Lifetime Career Educator Award from the Dermatology Foundation, and the Naomi Kanof Clinical Investigator Award from the Society of Investigative Dermatology .
What are EL-PFDD Meetings?
William Llewellyn from the FDA shares more information about the role of EL-PFDD meetings in this video.
View the December 14th IPPF webinar that provided an overview of the EL-PFDD meeting
Ways to Participate
You’re invited to participate in a survey to help the IPPF better understand your disease experience. The survey information will be shared with IPPF stakeholders like the FDA, drug development companies, and researchers to help illustrate the need for patient-focused drug development. Your participation is completely voluntary. This survey will take approximately 6-8 minutes to complete. We will accept survey responses through March 3, 2023.
We are still accepting comments for Voice of the Patient Report:
- Your comments should address one of the following topics:
- Your disease symptoms and treatments and how they impact your daily life.
- Your perspective on available treatments for your disease, their side effects and how to improve them.
- Your written comment should be 400 words or less with a 12 point font-size, Times New Roman or Arial font.
- All comments in the “Voice of the Patient Report” will be posted in perpetuity of the IPPF and FDA websites. Please don’t include any identifying information in your comments.
- Please share your comments through the submission form below, or send us an email with your comment at email@example.com
EL-PFDD Comment Submission
The IPPF would like to give a special thanks to the EveryLife Foundation for
Rare Diseases for the Rare Giving Event Sponsorship