Section Terms and abbreviations 1 Executive summary 2 Product information and disease description 3 Clinical evidence 4 Economic information and modeling report 5 References Terms and abbreviations
1 Executive summary1.1.1 Burden of Pyruvate dehydrogenase complex deficiency1.1.2 Sodium dichloroacetate efficacy and safety1.1.4 Conclusions
2 Product information and disease description2.1 Product description2.2 Place of product in therapy
3 Clinical evidence3.1.1 SL1009-013.1.2 SL1009-023.1.3 Controlled clinical trial of dichloroacetate for congenital lactic acidosis in children3.1.4 Dichloroacetate randomized controlled trial in MELAS3.1.5 Long-term safety of dichloroacetate in congenital lactic acidosis3.1.6 Chronic treatment of mitochondrial disease with dichloroacetate3.1.7 PDHX-related neonatal case report3.1.8 PDCD case monitored by localized proton magnetic resonance spectroscopy3.1.9 Partial pyruvate decarboxylase deficiency treated with dichloroacetate and benzoate3.1.10 Oral dichloroacetate in chronic congenital lactic acidosis3.1.11 Neonatal pyruvate dehydrogenase deficiency with lipoate-responsive lactic acidaemia3.1.12 Transient response to dichloroacetate in an infant with pyruvate decarboxylase deficiency3.1.13 Dichloroacetate as treatment for congenital lactic acidosis3.1.14 Dichloroacetate in the treatment of congenital lactic acidosis3.1.15 PHEMI3.1.16 Effect of sodium dichloroacetate on human pyruvate metabolism
4 Economic information and modeling report4.1 Modeling overview
5 References
Section 5 of 6
Economic information and modeling report 1 evidence topic · 2 sources
Summary The economic evidence in this report concerns broader mitochondrial disease populations rather than a budget impact model for SL1009. A U.S. claims study reported per member per month costs of $4,829 for pediatric participants and $3,100 for adults. An Australian study estimated annual average household costs of AU$112,721, with 92% attributable to societal costs. These estimates describe disease burden, not SL1009 acquisition costs, treatment-related cost offsets, or incremental budget impact. The report contains no SL1009-specific budget impact model.
4.2.1 Approach and frameworkNo evidence found.
4.2.2 Perspective and time frameNo evidence found.
4.2.3 Epidemiology and eligible population inputsNo evidence found.
4.2.7 Budget impact model discussionNo evidence found.