Idiopathic Thrombocytopenia Purpura Development of a PRO Strategy
Research type
Research Study
Full title
Idiopathic Thrombocytopenia Purpura Development of a Patient Reported Outcome (PRO) Strategy
IRAS ID
186806
Contact name
Angus Gunn
Contact email
Duration of Study in the UK
0 years, 3 months, 1 days
Research summary
UCB Biopharma Sprl, a pharmaceutical company is considering drug development for several rare autoimmune diseases, including idiopathic thrombocytopenia purpura (ITP). UCB wishes to explore ITP symptoms and daily impact from the patient’s perspective, and to use this information in the assessment of new treatments. UCB has commissioned Modus Outcomes, an independent outcomes research and consulting firm to conduct a research study and address this goal. The proposed study has been designed to investigate the range of symptoms experienced by adult ITP patients and the spectrum of daily impact of ITP with the aim of conceptualising “treatment benefit” in ITP.
Trained Modus Outcomes interviewer (Dr Sophie Cleanthous) will conduct two rounds of telephone interviews with a total of 60 people living with ITP. Interviews will last approximately 60 minutes and will be audio-taped. NO personal information about participants will be shared with the pharmaceutical company.
The first round of interviews will be exploratory, aiming to elicit the concepts important to patients in relation to their condition. Qualitative analysis will be used to map out aspects of treatment benefit in ITP (including symptoms and general impact and function) and generate items which can be used at a later stage for the assessment of treatment benefit in ITP (i.e. items that can later be used in a questionnaire). The second round of interviews will be semi-structured aiming to assess the comprehensiveness of the ”treatment benefit” conceptual framework and to cognitively debrief the newly generated items produced by the analysis of the first round interviews.
This study will enhance knowledge of ITP, highlighting concepts which are most important to patients, and will further propose a treatment benefit conceptual framework and draft item stems which could form the basis for the assessment of treatments in ITP.
REC name
Wales REC 6
REC reference
15/WA/0290
Date of REC Opinion
10 Aug 2015
REC opinion
Further Information Favourable Opinion