Qualitative evaluation of meaningful change in Dravet syndrome as measured by the Vineland-3: Caregiver and clinician perspectives.

The Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) is used to assess non-seizure outcomes in developmental and epileptic encephalopathies, including Dravet syndrome (DS); meaningful change thresholds (MCTs) have not been established. This qualitative study examined MCTs for the Vineland-3 and associated life impacts. Semi-structured interviews with 18 caregivers of children/adolescents with DS, including concept elicitation then completion and qualitative exploration of meaningful change for eight Vineland-3 subdomains considered most relevant to DS were conducted.

Qualitative In-trial Interviews: Methods, Challenges, and Best Practice.

Qualitative in-trial interviews with clinical trial participants are a means of providing rich, in-depth patient experience data to supplement and complement data captured by clinical outcome assessments and other clinical trial efficacy endpoints. Such in-trial interview data can be used to build understanding of disease and treatment experiences, evaluate content validity of clinical outcome assessments, aid interpretation of scores and meaningful changes, inform trial design feasibility and operational considerations, and provide supportive evidence regarding safety, efficacy, and effectiveness. Despite the rapid growth of in-trial interviews as part of clinical development programs in the pharmaceutical industry in recent years, published guidelines regarding the methods, conduct, and implementation of in-trial interviews are scarce.

Qualitative concept elicitation and cognitive debriefing interviews of symptoms, impacts and selected customized PROMIS Short Forms: a study in patients with axial spondyloarthritis.

Background: Sleep disturbance, pain, and fatigue are key symptoms/impacts of axial spondyloarthritis (axSpA). Three customized Patient-Reported Outcomes Measurement Information System (PROMIS) Short Forms (Sleep Disturbance, Pain Interference, and Fatigue) have been proposed for use in axSpA to assess these key disease concepts. This study was designed to further understand the patient experience of axSpA and evaluate the content validity of the three customized PROMIS Short Forms to support their use in axSpA clinical trials.