OBJECTIVES: Explore payer knowledge, perception, and unmet needs related to Gorlin Syndrome (GS) and assess their expectations and evaluation criteria for novel topical therapies.

METHODS: A qualitative analysis was conducted from semi-structured interviews with 14 payers from Germany (3), the UK (3), Spain (3), and the USA (5), focused on understanding their perspectives on novel therapies for GS. Previously, a comprehensive analysis of the current GS treatment landscape was conducted, including the evaluation of existing therapies, their efficacy, and the associated economic burden. Secondary resources such as payer decisions, case studies, and professional journals were also reviewed.

RESULTS: Payers recognized the high clinical burden and negative impact on patient quality of life (QoL) associated with GS. In their view, systemic therapies present a poor tolerability and the available topical treatment options have limited efficacy and are cumbersome to use. Mohs surgery is the cornerstone of treatment, with associated challenges such as the risk of disfigurement and difficulties in sensitive lesions close to the eyes, lips or nose, as well as economic implications. Payers expressed a need for a treatment that could reduce the number and frequency of surgeries, while improving patient QoL and convenience. They saw the potential to target sensitive lesions difficult to remove surgically with neo-adjuvant or stabilizing topical therapies, thereby reducing the risk of progression to malignant or more complex lesions.

CONCLUSIONS: GS poses significant challenges in terms of clinical burden, impact on QoL and economic implications. From a payer perspective, there is a clear unmet need to explore alternative treatment approaches that can reduce the number and frequency of surgeries to protect the physical integrity of patients while improving their QoL.