Recent research informs future improvement in the treatment for people with haemophilia, to inform the introduction of a more targeted approach to inform factor levels required to avoid a bleeding episode associated with physical activity

Antony Martin and co-authors from HCD Economics have recently published results of research intended to inform clinical understanding of minimum and ideal factor levels for participation in physical activities by people with haemophilia (PWH). The paper is available now in the Journal Haemophilia

The research was undertaken in recognition that the factor VIII level required to avoid a bleeding episode associated with physical activity is unknown. The authors believe that a study to address this knowledge gap is an important contribution to as PWH can derive benefits from physical activities, including improvements in joint, bone and muscle health.

This was an expert elicitation study which used the Sheffield Elicitation Framework (SHELF) method. The paper describes the procedure followed, including gathering of expert opinion and quantifying uncertainty around estimates, using multiple scenarios. The method is fully explained in the paper.

The external validity of any expert elicitation process depends on the identification of the parameters to be obtained (and assumptions), the selection of experts, and the elicitation process itself. SHELF is an alternative expert judgement elicitation method which adopts a structured approach for capturing quantitative expert estimates where evidence may be lacking.

The authors established that clinical expert estimates for minimum and ideal factor levels were found to increase with higher risk physical activity. These data may be used in exploratory analysis in health economic models to examine the possible impact of alternative treatment strategies. Further, these findings may be used to highlight existing unmet need in haemophilia.

These findings may be used to inform the value of alternative treatment regimens and approaches.

For further information on the study and the recent publication, please contact Anthony Martin,

Haemophilia is published by John Wiley & Sons Ltd.