In rare diseases, the patient voice must be optimised.

Supporting the patient community to provide them with a platform for reciprocal relations with payer and government bodies and the medical community is the central approach of HCD Economics. Our work in rare diseases including haemophilia and von Willebrand’s disease are a case in point.

Study governance provided by an expert group of patient advocates, clinicians, academics and health economists working at the highest level of involvement as equal partners, provides insights including critical pathways of disease, impact of disease, and patient preferences. The resultant shared-decision evidence base supplements community efforts in lobbying for maintenance and improvement of treatment, service provision reviews, political messaging, and public health actions.