Clinical trial exit interviews to characterise patients’ experience and treatment benefit in patients with activated phosphoinositide 3-kinase delta syndrome (APDS)
Development of a COA strategy for Down’s syndrome in adults and children
Clinical meaningfulness of scores on the Vineland Adaptive Behaviour Scale (VABS-II), Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) and Clinician Global Impression (CGI) in Down’s syndrome
Strategic literature review to identify COA instruments to assess the concept of independence in Down’s syndrome
Qualitative interviews and development of a COA strategy for Down’s syndrome in adults and children
Subgroup analysis by chronological age of individuals with Down’s syndrome
Development of the evidence base regarding treatment patterns, cost and burden of disease for patients with hereditary angioedema to support the use of a new treatment
Communications package in hereditary angioedema
Clinical endpoint review, recommendations and update of an FDA submission package in hereditary angioedema
Hereditary angioedema clinical trial endpoint review, recommendations and FDA PRO submission package
Pooled clinical trial validation analysis of a PRO primary endpoint in hereditary angioedema to include in FDA submission package
FDA meeting support for a hereditary angioedema submission
Evaluation of market research reports in hereditary angioedema to support PRO submission package
Analysis of Visual Analogue Scale (VAS) summary scores with Last Observation Carried Forward (LOCF) in hereditary angioedema
Psychometric validation analysis of VAS summary scores in hereditary angioedema
Patient interviews for the face and content validation of the Patient Global Impression in hereditary angioedema
Psychometric validation of the 6-item and 8-item composite severity scores in hereditary angioedema
Consulting on the linguistic validation of a hereditary angioedema questionnaire into French, German and Portuguese
Cognitive debriefing of a questionnaire in hereditary angioedema patients
Validation and modifications to a questionnaire in hereditary angioedema patients
Consultancy services for linguistic validation of a hereditary angioedema diary
Publication strategy to present the results of studies in hereditary angioedema
Patient interviews to support a PRO submission package in hereditary angioedema
PRO briefing document in hereditary angioedema
Strategic consulting and analysis in hereditary angioedema
Re-analysis of patient composite score in hereditary angioedema
Training materials for a PRO questionnaire in hereditary angioedema
Exploration of IQ as a relevant endpoint in Hunter’s syndrome
Endpoint review in Hunter’s syndrome
PRO dossier on the Disease Activity Score (DAS), Scales of Independent Behaviour-Revised (SIB-R), Behaviour Rating Inventory of Executive Function (BRIEF) and Vineland Adaptive Behaviour Scales (VABS) for Hunter’s syndrome
Strategic PRO and ObsRO analysis of a Hunter’s Syndrome clinical trial database
Regulatory support for questionnaires in Hunter’s syndrome
Understanding the experience of the mucopolysaccharidosis I (MPS I) patient to improve outcome assessments: evidence from the literature and treatment experts
Cognitive debriefing interviews on the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ)
Evaluating the MPS-HAQ for use in patient registries to support EMA regulatory commitments
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