Issues in the systematic review of drugs for rare diseases: A case study of Eculizumab for AHUS (2012)

Atypical haemolytic uraemic syndrome (aHUS) is a rare disease affecting approximately 170 people in the UK. The outcome is often poor, with 53% of familial cases and 37% of sporadic cases resulting in end stage renal failure or death. A systematic review was undertaken to assess the efficacy and safety of eculizumab for aHUS. We report key outcomes and highlight issues of applying standard systematic review methods where the evidence base is not well developed.