PATIENT-REPORTED OUTCOMES IN RARE LYSOSOMAL STORAGE DISEASES: KEY INFORMANT INTERVIEWS AND A SYSTEMATIC REVIEW PROTOCOL.
Patricia A MillerSohail M MullaThomasin Adams-WebberYasmin SivjiGordon H GuyattBradley C JohnstonPublished in: International journal of technology assessment in health care (2016)
The interviews highlight current challenges and opportunities experienced by researchers and HTA experts involved in work with rare LSDs. The ongoing systematic review will highlight the experience, opportunities, and limitations of PROs in LSDs and provide suggestions for future research.