Enzymatic replacement therapy for lysosomal storage disorders: Drug evaluations review in Spain

Abstract

Introduction: In the European Union companies only need to demonstrate that the risk-benefit balance of the new drug is favourable to obtain the authorization to sell new drugs. Hence a comparison with available treatments, a cost-effectiveness analysis and the place in the therapy of the new drug are not required. Therefore, it is necessary to carry out these analyses in an additional study. In Spain there is no national government agency, which conducts a centralized evaluation and makes decisions on funding and are the Pharmacy and Therapeutics committees that take mandatory decisions for the entire region.Objectives: To identify all drug assessments and health technology assessment reports of the enzymatic replacement treatments for the four LSD considered in this study, including Mucopolysaccharidosis (MPS), Gaucher, Fabry and Pompe disease at the national, regional and hospital level; and to summarize the efficacy in terms of outcome measures, adverse events, economic impact and final recommendations and indicate potential improvements.Methods: 19 reviews at the regional and hospital level and one therapeutic positioning report at national level for MPS were considered for this. 2Results: In general, the drugs evaluated in LSD have demonstrated to be effective in the most frequent clinical forms of disease. However, there are clinical phenotypes that still do not have available an effective treatment.Conclusions: It is expected that new therapies, such as intrathecal therapies or gene therapy could be evaluated in the different types of LSD demonstrating positive effects, especially in the previous phenotypes.

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“…This issue of Health Policy and Technology contains an article by Darba et al that describes what they found in Spanish reports of the efficacy and safety of treatments for rare lysosomal storage diseases (LSDs) like mucopolysaccharidosis (MPS), Gaucher disease, Fabry disease and Pompe disease. [1] Based on their findings, it seems that the treatments for LSDs work to differing degrees in different subgroups and that the evidence of their effectiveness varies substantially. The authors note that new methods to assess effectiveness are needed since the current methods will not be very feasible in cases where just one subgroup of patients with a rare disease represents the entire target population.…”