Research & Treatments
ADDRESS Study for Adults Living with Pemphigus A CALL FOR THE PATIENT COMMUNITY TO LEARN MORE ABOUT PARTICIPATING IN THE DRUG DEVELOPMENT PROCESS
John Holtz and Kathy Perez
Patient-focused awareness
At argenx we believe that working with patients in the early stages of drug development is critical. Patients who are living with rare autoimmune conditions are best able to share the needs of their community. All aspects of life can be affected by a rare disease diagnosis, and researchers must keep these needs at the forefront of clinical studies. One trial that is working directly with the pemphigus community is the ADDRESS study. The team at argenx is working toward including pemphigus patient feedback into trial designs in an effort to ensure that the community's needs are being heard. In 2019, the economic burden of 379 rare diseases in the US economy was found to be nearly $1 trillion
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Quarterly | Summer 2021
by the EveryLife Foundation for Rare Diseases. The report surveyed 1,399 members from the rare disease community, including the pemphigus community, and identified direct medical costs and indirect costs experienced by patients and families living with a rare disease. The first of its kind, this survey not only identified the economic burden of rare diseases, but also highlighted the need for additional patient-centered research and therapy development.1,2 The Food and Drug Administration (FDA) has realized the importance of listening to the patient community as well. This was highlighted in the February 2021 FDA Patient Listening Session on pemphigus and pemphigoid (P/P). During the listening session, five people living with P/P shared their journeys and
www.pemphigus.org
