Efficient Early Drug Development for Ulcerative Colitis

Section: Discussionmentioning

confidence: 99%

Real‐world clinical, endoscopic and radiographic efficacy of vedolizumab for the treatment of inflammatory bowel disease

Kotze

Almutairdi

et al. 2018

“…Treatment options have improved over the past two decades with the introduction of several new classes of therapeutic agents; nevertheless, a substantial proportion of patients do not respond or lose response to available treatments. Consequently, multiple compounds are currently in early and late phase clinical trials 1,2 …”

Section: Introductionmentioning

confidence: 99%

“…According to the aforementioned FDA guidance, a valid and reliable PRO instrument is capable of measuring clinically meaningful aspects of disease activity that are most relevant to patients and therefore allows for assessment of the relative benefits of new treatments in a readily interpretable manner. PRO instrument creation and validation is a rigorous, resource‐intensive process that can take several years to complete 2,10 . In addition to the CD‐PRO/SS and UC‐PRO/SS measures, relevant examples in gastrointestinal research include the Experience Sampling Method Patient‐Reported Outcome (ESM‐PROM) 11 for the assessment of irritable bowel syndrome and the Eosinophilic Esophagitis Activity Index Patient‐Reported Outcome (EEsAI PRO) 12 …”

Section: Introductionmentioning

confidence: 99%

Development of the symptoms and impacts questionnaire for Crohn's disease and ulcerative colitis

Dulai

Jairath

Khanna

et al. 2020

Self Cite

Background: Patient-reported outcome (PRO) measures historically used in inflammatory bowel disease have been considered inadequate to support future drug labelling claims by regulatory agencies. Aims: To develop PRO tools for use in Crohn's disease (CD) and ulcerative colitis (UC) following guidance issued by the US FDA and the ISPOR (International Society for Pharmacoeconomics and Outcomes Research). Methods: Concept elicitation and cognitive interviews were conducted in adult patients (≥18 years) across the United States and Canada. Semi-structured interview guides were used to collect data, and interview transcripts were coded and analysed. Concept elicitation results were considered alongside existing literature and clinical expert opinion to identify candidate PRO items. Cognitive interviews evaluated concept relevance, interpretability and structure, and facilitated instrument refinement.Concept elicitation participants, except those with an ostomy, underwent centrally read endoscopy to assess inflammatory status. Results:In all, 54 participants (mean age: 46.2 years; 66.7% female) were included in the CD concept elicitation interviews. In total, 80 symptom concepts and 61 impact concepts were identified. After three waves of cognitive interviews, the 31-item Symptoms and Impacts Questionnaire for CD (SIQ-CD) was developed. In the UC concept elicitation phase, 53 participants were interviewed (mean age: 41.4 years; 49.1% female). In total, 79 symptoms concepts and 49 impact concepts were identified. Following two waves of cognitive interviews, the 29-item Symptoms and Impacts Questionnaire for UC (SIQ-UC) was developed. Both instruments include four symptom and six impact domains. Conclusions:We developed PROs to support CD and UC drug labelling claims.Psychometric validation studies to evaluate instrument reliability and responsiveness are ongoing.

“…Whilst this circumstance offers great promise to patients, many challenges remain in developing new drugs for UC. These include recruitment challenges driven by multiple competing programs, the increased availability of effective treatments in the clinic, increasing complexity of trial design and evolving regulatory endpoints …”

Section: Introductionmentioning

confidence: 99%

Systematic review with meta‐analysis: endoscopic and histologic placebo rates in induction and maintenance trials of ulcerative colitis

Guizzetti

Panaccione

et al. 2018