Comparative valuation of longstanding and novel treatments for sickle cell disease: a multinational MCDA study

In Europe, care for sickle cell disease (SCD) is more extensive compared to low-income countries. However, individuals with SCD still experience a relatively poor quality of life, with life expectancy lagging about two decades behind the general population. This study employs multi-criteria decision analysis (MCDA) to assess the value of established (hydroxyurea and hematopoietic stem cell transplantation) and innovative treatments (voxelotor, crizanlizumab, and CTX001 gene-edited therapy) for SCD. A diverse group of stakeholders from France, Belgium, and England evaluated the treatments using a mixed-methods approach which involved online surveys and focus group discussions. The assessment considered multiple criteria suggested in literature for valuing orphan drugs, including disease severity, rarity, level of research/uncertainty, innovation/unique indication, follow-up measures, unmet medical need, treatment efficacy, treatment cost, treatment safety, treatment convenience, and broader economic consequences. In the initial phase, stakeholders unanimously emphasized the significance of treatment safety, efficacy, and disease severity in reimbursement decisions for orphan drugs. Disease rarity and broader economic consequences were considered of lesser importance by the French-Belgian and English groups, respectively. In the subsequent phase, hydroxyurea was ranked highest in overall value, while CTX001 gene-edited therapy was rated lowest by both groups. Amongst French-Belgian stakeholders, voxelotor, crizanlizumab, and HSCT were the second, third, and fourth most valued treatments. In the English group, HSCT, voxelotor, and crizanlizumab held the second, third, and fourth positions. Emerging drugs for SCD offer hope for increased treatment options. Though hydroxyurea is currently favored, stakeholders emphasized the lack of optimal therapies for SCD and need for advancements tailored to this population's healthcare needs. This study underscores the importance of involving diverse stakeholders and considering various criteria beyond traditional cost-effectiveness and budget impact in valuing orphan drugs.