OBJECTIVES: In May 2015, the German Institute for Quality and Efficiency in Health Care (IQWiG) published an assessment of treatment for haemophilia patients. The aims of the report were: 1) to map current evidence pertaining to long-term factor treatment of patients with severe haemophilia (SH); 2) to conduct a benefit assessment of prophylactic versus on-demand treatment strategy in the continuous treatment of patients with SH, with morbidity and health-related quality of life (HRQOL) as specified endpoints; and 3) to investigate the extent to which guidelines for the continuous treatment of patients with SH in Germany are based on current evidence.

METHODS: A critical review of the report scope and methodology, with an analysis of pre-existing data from an observational burden of illness study in Germany and other European countries (CHESS), in order to showcase the utility of ‘real world’ data in healthcare policy design.

RESULTS: The report and supporting research were comprehensive yet limiting in their exclusion of studies undertaken outside of a clinical trial setting. Several endpoints could not be evaluated due to insufficient evidence. Outcomes associated with primary (lifelong) and secondary prophylaxis (PPX/SPX) regimens were not differentiated, despite studied differences in outcomes between the two approaches. Neither patient factors (e.g. bleeding phenotype) nor treatment factors (e.g. trough levels) were considered in the assessment. In contrast to the report’s conclusions, patients receiving PPX within CHESS reported significantly higher HRQOL, lower bleed frequency, fewer areas of chronic synovitis, and reduced pain versus on-demand and SPX.

CONCLUSIONS: The IQWiG report methodology provides a limited approach, particularly in the evaluation of treatments for rare diseases, as reflected in the assessment of prophylaxis and on-demand treatment in SH. A more pragmatic approach to evidence collation, in which the idiosyncrasies of this rare disease may be effectively captured, is recommended for future evaluations.