VALIDITY OF CHANGE IN RECALL PERIOD FOR THE NORFOLK QOL PATIENT REPORTED OUTCOME (PRO) MEASURE
Author(s)
von Maltzahn R1, Benjamin K2, Murphy R2, Lipman K3, d la Cruz M2
1GSK, Uxbridge, UK, 2ICON, Gaithersburg, MD, USA, 3ICON plc, Dublin, Ireland
OBJECTIVES: Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare degenerative disease with a progression pattern similar to that observed in diabetic neuropathy (Vinik et al., 2014). The Norfolk QOL-DN was developed to capture patients’ perceptions of the effects of diabetic neuropathy and has been validated in TTR-FAP patients. Based on regulatory feedback the recall period of the measure was changed from 4- to 1-week. The objective of this research was to determine the validity and acceptability of the one-week recall period. METHODS: Individual qualitative telephone interviews were conducted with patients diagnosed with TTR-FAP. Interviews were conducted by trained interviewers between May 2016 and June 2016. At the start of the interview, subjects completed the Norfolk QOL-DN while “thinking aloud,” or talking about the questionnaire instructions and items and their response to them. Participants were also asked questions about the ease of completing the Norfolk QOL-DN, focusing on the relevance of the one-week recall period. Interviews were audio-recorded and transcribed and analysed using qualitative thematic and content analysis. RESULTS: Twenty participants were interviewed; 55% male; mean age 61.1 years. No participants had significant difficulties understanding how to answer the Norfolk QOL-DN questions using a one-week recall period. All participants had experienced at least one of the symptoms covered by the instrument in the past week. The most prevalent type of symptom was neuropathy-related; All participants experienced at least one neuropathy symptom within the one-week time period. Generic functioning and HRQoL items were somewhat less sensitive as participants had adjusted their lives to their disability, not because they had problems with the recall period.
CONCLUSIONS: Participants in this study were able to understand and respond to the Norfolk QOL-DN items using the one-week recall period. Additional work exploring the item relevance and performance within TTR FAP would add to the rigour of this measure within this population.
Conference/Value in Health Info
2017-11, ISPOR Europe 2017, Glasgow, Scotland
Value in Health, Vol. 20, No. 9 (October 2017)
Code
PSY93
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare and Orphan Diseases