Validation of disease burden and health economic model assumptions through expert elicitation

Our client wanted to conduct expert elicitation to achieve consensus on several key elements in the health economic evaluation of a rare disease, including the importance of disease burden in predicting long-term patient outcomes and the assumptions used in the health economic model.

We recruited more than 20 clinicians with experience treating the disease and conducted a validation session with real time voting and feedback to establish consensus across a range of areas, including anticipated place in therapy, the generalisability of clinical trial results to real world practice, and links between the outcomes of the clinical trials and long-term patient prognosis. The clinicians also provided validation of the client’s approach to health economic modelling.

Our client was able to leverage consensus in a rare disease area to reduce uncertainty in health economic modelling. They were able to effectively justify their approach to health economic modelling and methods for capturing overall disease burden: our study findings were used to underpin a successful submission to NICE.