Using a discrete choice experiments to explore societal preferences for valuing new drugs for rare diseases.

ABSTRACT:

Background

Rare diseases affect few people, but collectively they affect a substantial proportion of the population. Limited treatment options and the additional challenges of securing public funding make reimbursement decision making particularly complex, mainly due to the inherent evidence uncertainty, lack of understanding of these diseases and the greater impact on non-health outcomes. This study explored societal preferences regarding which factors should be valued when considering public subsidy of drugs to treat rare diseases.

Methods

A discrete choice experiment was developed, and respondents were asked to assume the role of a Government advisor and decide on funding between 2 drugs to treat a rare disease. Attributes were identified from the literature and focus gro

SUBMITTER: Vargas C 

PROVIDER: S-EPMC12751238 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature