A Patient-Relevant Measurement Strategy to Assess Clinical Benefit of Novel Therapies for Non-metastatic Cutaneous Squamous Cell Carcinoma.

Introduction: Although cutaneous squamous cell carcinoma (CSCC) is the second most common type of skin cancer, research describing the patient experience is limited. This study sought to create a conceptual model of non-metastatic disease, to assess patient-reported outcome (PRO) instruments commonly used in CSCC against this model, and to develop a patient-relevant measurement strategy for evaluating the benefit of new therapies.
Methods: Researchers conducted a literature review, a review of patient blogs, and interviews with dermatologists to draft the conceptual model. A total of 22 patients with CSCC participated in 60-min phone interviews, which were subsequently transcribed, coded, and analyzed; the conceptual model was then updated. PRO instruments used in CSCC were assessed for content validity on the basis of this.
Results: The CSCC patient experience includes physical symptoms, psychological impacts, and behavior changes. Existing PRO instruments were assessed against the conceptual model using targeted subdomains considered to be relevant for assessing clinical benefit. Four modules of the FACE-Q ^® Skin Cancer instrument, plus de novo items developed for concepts not assessed by the FACE-Q ^® [lesion symptoms, negative treatment effects (including symptomatic), and experience of care], provide the best coverage for the concepts of interest hypothesized to show the benefit of novel treatments.
Conclusions: This research provides a comprehensive understanding of the experience of patients with non-metastatic CSCC, and the effects of its treatment. It also identifies unmet needs in a subgroup of patients reporting negative treatment experiences. Further cognitive debriefing and psychometric analysis of de novo items are warranted for applications in clinical research.
Competing Interests: Declarations Conflict of Interest Diana Rofail, Matthew G. Fury, and Chieh-I Chen are employees and shareholders of Regeneron Pharmaceuticals, Inc. Anna Ciesluk, Teya Lovell, and Patrick Marquis are researchers who received payment from Regeneron Pharmaceuticals, Inc., for the study. Ethical Approval Study documents, including the protocol, demographic and health information form, interview guide, screener, and informed consent forms received ethical approval from WCG IRB (ref. number 20202867). All participants provided informed consent to participate and were compensated for their time in line with fair market value. The study was performed in accordance with the ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.
(© 2024. The Author(s).)