Value-based decision-making for orphan drugs with multiple criteria decision analysis: burosumab for the treatment of X-linked hypophosphatemia

Abstract

Objective: Burosumab is an orphan medicinal product (OMP) approved in Europe for the treatment of X-linked hypophosphatemia (XLH). The aim of this study was to assess the value of burosumab versus conventional therapy for the treatment of paediatric XLH, through a multi-criteria decision analysis (MCDA) framework for health technology assessment (HTA) of OMPs in Portugal. Methods: The MCDA framework considered 14 criteria related to disease burden, therapeutic value and economic burden. A multidisciplinary panel of national stakeholders participated in a two-phase exercise. In the first phase, relative weights and part-worth utilities for the criteria and their levels were elicited and a reimbursement likelihood function was calibrated through adaptive conjoint analysis. In the second phase, burosumab and conventional therapy were assessed against the criteria, providing a global value score (0-100) and reimbursement likelihood (0-100%) for both. Results: Of the 14 criteria, disease burden, therapeutic value and economic burden criteria represented 27.29%, 57.17% and 15.53% of the total weight in the decision, respectively. All disease burden and some therapeutic value criteria, typically not included in traditional HTA, represented 47.88% of the total weight. Burosumab was unanimously considered superior to conventional therapy, with an average (range) global value score of 84.96 (82.48-86.54) against 48.06 (43.37-57.68), and reimbursement likelihood of 97.50% (96.78%-98.32%) against 43.66% (31.48%-68.73%), respectively. Conclusions: MCDA represents a powerful tool in HTA decision-making for OMPs. The results of this MCDA acknowledge burosumab as a disease-modifying drug, deemed superior to conventional therapy for the treatment of paediatric XLH.

Health-related quality of life of X-linked hypophosphatemia in Spain

“…Another, and relevant, reason to choose the EQ-5D instruments was that they are recommended for studies on the use of resources with adults and children for the effects of comparability of the results [22]. Likewise, this type of instruments to quantify the quality of life of patients with XLH are gaining more and more importance in recent years, so they should be valued accordingly [26] for this type of research. Moreover, in a recent meta-analysis of the health impact of rare diseases, the lowest quality of life across EQ-5D scores, VAS scores and Barthel index corresponded to patients with musculoskeletal diseases [27].…”

Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X-Linked Hypophosphatemia: Results From the Phase 3, Single-Arm International Trial

“…Burosumab is a fully human monoclonal FGF23 antibody recently approved for XLH treatment in children and adults. (10,11,(19)(20)(21)(22)(23) In phase 3 studies, burosumab significantly improved fracture healing and pain, increased serum phosphorus levels and circulating markers of bone turnover, and markedly reduced osteomalacia in adults with XLH. (24)(25)(26)(27) Here, we present the effect of burosumab on the mineralized bone volume and the degree of mineralization, the properties of the organic matrix, and the size of the periosteocytic lesions.…”