The Fascioscapulohumeral Muscular Dystrophy Health Index (FSHDI) is a disease-specific, patient reported outcome measure for FSHDI. Each question in the FSHDI was selected based on its relevance to a large muscular dystrophy population, its ability to be consistently understood by patients, its reliability during test-retest evaluation, its content validity, its face validity, its construct validity, and its responsiveness in detecting therapeutic change. FDA guidelines were implemented during instrument development in order to allow the FSHDI to be utilized to support drug labeling claims. The instrument is capable of measuring the total health of a muscular dystrophy patient. Subscales of the FSHDI measure a patient’s perception of their health as it relates to disease specific areas (such as fatigue, pain, ambulation). Overall, the FSHDI is optimally suited to detect small but clinically relevant changes in several key areas of muscular dystrophy health over the course of a treatment trial.
This FSHDI is designed for use as a primary or secondary outcome measure during muscular dystrophy clinical trials. Alternatively, the FSHDI may be used as a marker of disease severity for patients with muscular dystrophy.
This FSHDI is a validated instrument capable of measuring clinically meaningful changes in patient health during clinical trials. As a disease-specific instrument, this outcome measure focuses on the symptoms that are most important to muscular dystrophy patients while excluding questions that are less relevant. The content validity, face validity, construct validity, convergent validity, test retest reliability, and responsiveness of this instrument has been previously evaluated in preparation for the FSHDI’s use in drug labeling applications.