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Development and validation of the TSC-PROM1416Especially now that disease-modifying, and often long-term and expensive therapies are increasingly available, there is an urgent need for a TSCspecific PROM to measure effects of clinical parameters and treatment on disease-specific functioning, in both clinical and research settings. The use of a TSC-specific PROM in clinical trials can provide valuable evidence of the risks and benefits of treatments from a patient perspective which can inform regulatory approvals, clinical guidelines, and health policy, as it captures information that is relevant to the individual with TSC.32 Therefore, the development of a reliable and valid instrument that measures domains and symptoms relevant to individuals with TSC is a top priority for patient organizations, researchers, and healthcare providers.5,33,34The aim of the current study was to develop and validate a TSC-specific PROM that captures the impact of TSC on physical and mental functions, activity and participation, and social support received by individuals with TSC, using the framework of the ICF (World Health Organization, 2001). The questionnaire is called TSC-PROM and consists of separate versions in English and Dutch for self-report and proxy-report, with the latter being the most suitable option to receive information about the possibly experienced issues for individuals who are unable to report on themselves.MethodsStandards from the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) were used to develop the questionnaire.33,34 Development and validation consisted of the following studies: 1) PROM development, 2) content validity, and 3) structural validity, internal consistency, and construct validity (Figure 1).Annelieke Muller sHL.indd 141 14-11-2023 09:07