Inclusion of the Patient Voice in Developing Holistic Treatment Goals for Rare Skin Diseases

Treatment goals for generalized pustular psoriasis (GPP) are poorly defined, based mostly on data from plaque psoriasis, and are not reflective of the patient experience. Shared decision-making between patients and physicians around treatment goals can lead to improved outcomes and patient satisfaction. Furthermore, health regulatory bodies have issued guidance on the integration of patient experience data, along with other insights from patients and caregivers into the development of new treatments. Patient perspectives have been incorporated into consensus-shaping exercises to better understand treatment goals for plaque psoriasis, though a similar approach has been lacking in GPP. Here, we report insights from the first consensus-shaping exercise in GPP to incorporate the patient perspective. An expert panel was assembled comprising patient representatives and dermatologists with recent or current experience in treating GPP. Through the use of a targeted questionnaire (based on a systematic literature review), the objective was to achieve consensus on key principles of GPP treatment, including short-term and long-term goals. Patients and physicians rated their level of agreement on 26 different statements and gave additional comments in a free-text field. Consensus per statement was defined as ≥80% agreement across the panel. The panel comprised 30 dermatologists and 3 patient representatives. Each of the 3 patients (Asia, n=2; USA, n=1) had >10 years’ lived experience of GPP; one patient was a member of a GPP patient advocacy group. Consensus between physicians and patients was reached on all statements relating to effective disease management, including the need for tailored treatment plans. Overall, physicians considered the treatment of certain symptoms (e.g. skin pustules) as the most important clinical goal and metric for treatment success. However, patients considered alleviation of other symptoms (alongside skin and systemic symptoms) to be equally important due to the psychological and emotional impact of this in their daily lives. Developing treatment goals for rare skin diseases such as GPP is challenging due to limited published evidence on symptom burden and a relative lack of approved treatment options. The inclusion of patient representatives in this consensus-shaping exercise for GPP provided personal insights regarding the wider impact of the disease on patients’ lived experience. Patients should be empowered as active, shared decision-makers to ensure that treatment goals for chronic diseases are holistic, reflecting not only clinical outcomes, but also symptoms that have the greatest impact on quality of life. Funding This study was supported and funded by Boehringer Ingelheim.