Treatment goals in hypertrophic cardiomyopathy (HCM) focus on the relief of symptoms; however, there is no HCM-specific measure to evaluate patient symptoms for consideration during drug development and in clinical practice.

This study describes the development and evaluation of the HCM Symptom Questionnaire version 1.0 (HCMSQv1.0), a new patient-reported outcome (PRO) measure, using qualitative and quantitative methods in line with guidance for industry on PRO measures from the US Food and Drug Administration.

The results from this study demonstrate that the HCMSQ is a fit-for-purpose PRO instrument for the assessment of treatment benefit in patients with HCM following the removal of items 4 and 5, making a 9-item scale (the HCMSQv2.0). This is of key relevance for future studies in such patient populations.