Bone Abstracts

Objectives: Burosumab is an anti-FGF23 fully human monoclonal antibody, recently approved for the treatment of X-linked hypophosphatemia (XLH), presenting a novel treatment approach compared to conventional therapy (CT), composed of oral phosphate and active vitamin D. The objective of this study is to perform a Multi-Criteria Decision Analysis (MCDA) to assess the value of burosumab for the treatment of paediatric patients with XLH in Portugal, in comparison to CT.

Methods: MCDA is a method that provides a comprehensive and systematic assessment of therapies’ value. A framework, developed specifically for the assessment of orphan drugs, consisting of 14 criteria related to the burden of disease (4), therapeutic value (7) and economic burden (3) was used. Eight national stakeholders, including four physicians, two patient representatives, one health economist and one health policy decision-maker, participated in a two-round process. In the first round, all participants established the relative weights (importance) of each criterion by the means of a preference elicitation adaptive questionnaire related to orphan drugs in general, while in the second round, both burosumab and CT were assessed by all physicians and one patient representative against the pre-established criteria. Results from the first and second rounds were then combined to provide a global score for each treatment alternative out of 100.

Results: In the first round, criteria related to the therapeutic value were weighted higher than disease burden and economic impact related criteria. Treatment clinical impact, treatment safety and tolerability, and disease severity were the most valued criteria, with relative weights of 9.80%, 9.72% and 8.71% of the total score, respectively. On the other end, rarity of the disease, direct medical costs and direct non-medical costs were the least valued criteria, contributing with 3.79%, 4.55% and 4.59% of the total score, respectively. Second round results and global scores for both treatment alternatives will be presented in the poster.

Conclusion: This MCDA’s first round results demonstrated that therapeutic value related criteria are the most valued criteria for the assessment of orphan drugs, based on different Portuguese stakeholders’ preferences.

Disclosure: This study was funded by Kyowa-Kirin Portugal.