Journal article
PATIENT-REPORTED OUTCOMES IN RARE LYSOSOMAL STORAGE DISEASES: KEY INFORMANT INTERVIEWS AND A SYSTEMATIC REVIEW PROTOCOL
Abstract
OBJECTIVES: To investigate the use, challenges and opportunities associated with using patient-reported outcomes (PROs) in studies with patients with rare lysosomal storage diseases (LSDs), we conducted interviews with researchers and health technology assessment (HTA) experts, and developed the methods for a systematic review of the literature. The purpose of the review is to identify the psychometrically sound generic and disease-specific …
Authors
Miller PA; Mulla SM; Adams-Webber T; Sivji Y; Guyatt GH; Johnston BC
Journal
International Journal of Technology Assessment in Health Care, Vol. 32, No. 6, pp. 393–399
Publisher
Cambridge University Press (CUP)
Publication Date
2016
DOI
10.1017/s0266462316000568
ISSN
0266-4623