CHALLENGES IN RECRUITING PATIENTS FOR THE LINGUISTIC VALIDATION OF PRO INSTRUMENTS DEVELOPED FOR RARE DISEASES- A CASE STUDY WITH ALAGILLE SYNDROME
Author(s)
Kennedy C1, Abetz-Webb L2, Lambe J3
1Lumena, San Diego, CA, USA, 2Patient-Centred Outcomes Assessments LTD, Macclesfield, Cheshire, UK, 3Mapi, Lyon, France
OBJECTIVES Observer and Patient Itch-reported Outcome Instruments (ItchRO) are electronic morning and evening diaries developed in US English to assess itch-related symptom severity (rubbing, scratching, skin damage, sleep disturbances or irritability) in pediatric patients suffering from rare cholestatic liver diseases such as Alagille Syndrome (ALGS). Itching is a key symptom in ALGS and significantly impacts on a child’s daily life. This research presents the recruitment challenges faced during the linguistic validation of the ItchRO into French, German, Polish and Spanish in ALGS patients. METHODS Native translators performed two forward and two back translations of the English ItchRO. Cognitive interviews with five children with ALGS (patient report) and five parents (observer report) were to be performed. Key inclusion criteria were: children aged 5-9 years with cholestatic liver disease (ALGS) or progressive familial intrahepatic cholestasis (PFIC), who were also experiencing itching. RESULTS Recruitment was challenging and threatened to delay the clinical trial programme. Thus, recruitment was extended to include children who were 3-17 years of age, and in Spain and Germany, patients with alternative pathologies that manifested primarily with generalized pruritus were included. The final sample included: 15 with ALGS (France n=5; Poland n=5; Germany n=3; Spain n=2), 2 with PFIC (Spain), 2 with atopic dermatitis (Germany) and 1 with contact dermatitis (Spain). The ItchRO translations were well understood; there were no apparent differences in translation interpretations by disease state. CONCLUSIONS Recruitment in linguistic validation projects in rare diseases can be a hurdle, yet patient understanding, rather than those of ‘lay people,’ is preferable in paediatrics to ensure adequate interpretation, which can vary substantially across children. As a result patients with similar symptom and impact characteristics could be recruited if recruiting the primary population is likely to delay clinical trial research in rare diseases, where there is an urgent need for treatment.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PRM181
Topic
Methodological & Statistical Research
Topic Subcategory
PRO & Related Methods
Disease
Rare and Orphan Diseases