OBJECTIVES: While many authors suggested using a multi-criteria approach for orphan drug assessment and proposed a list of determinants of orphan drug value, studies on social preferences regarding these determinants remain limited. The current study aimed at identifying preferences of the French general population regarding a number of determinants in a discrete choice experiment.

METHODS: The list of attributes was formed based on a literature search and was refined through expert interviews, a focus group, and a pilot study. The final list included nine attributes: disease disability and mortality, number of patients, availability of alternative treatments, treatment impact on disease disability and survival, treatment safety, uncertainty around therapeutic effect, and annual treatment cost per patient. Participants were presented with 12 questions containing two drugs profiles described according to the attributes and were asked to choose one (or none) treatment. The questionnaire was distributed using a web platform. A conditional logit model was used for statistical analyses and included all attributes and a dummy variable corresponding to a choice of none treatment. An interaction between the number of patients and per patient cost was tested.

RESULTS: A total of 958 persons participated in the study. The highest estimate weight was observed for treatment impact (p<0.0001) on survival and uncertainty around therapeutic effect (p<0.0001). Participants were also sensitive to the availability of alternative treatments (p=0.0014), treatment safety (p<0.0001) and impact on disability (p=0.0001), disease mortality (p<0.0001) . Participant preferred more prevalent diseases (p<0.0001). Although the interaction between the number of patients and per patient cost was significant (p<0.0001), the trend in preferences toward lower per patient costs was not observed.

CONCLUSIONS: The society does not seem to support drugs for less prevalent diseases and to be aware about drug pricing in general. Should special measures for orphan drugs be introduced, education on their necessity is needed.