Cost-effectiveness analyses and utility weights
Published cost-effectiveness analyses and health-state utility weights that a model of this product could cite, adapt, or compare against, grouped by how each article relates to the product and its indication. Every value is quoted from the article it cites, and section 3 lists the searches that found the articles.
4 cost-effectiveness analyses and 3 studies reporting utility weights · 5 sources
Overview
| Item | Detail |
|---|---|
| Literature searched | Tufts Medical Center Cost-Effectiveness Analysis Registry, searched October 21, 2026; the queries are listed in section 3. |
| Cost-effectiveness analyses | 4 |
| Studies reporting utility weights | 3 |
Cost-effectiveness analyses
Analyses of SL1009 (sodium dichloroacetate)
No evidence found.
Treatments for the same indication
No evidence found.
The same disease in other populations or settings
Early exome sequencing versus typical care for newborns with suspected severe mitochondrial disorders, United States: Crawford et al. (2021)
Relevance: Evaluates early exome sequencing against typical diagnostic care for newborns with suspected mitochondrial disorders in the United States, the disorder group that includes pyruvate dehydrogenase complex deficiency, whose molecular genetic diagnosis section 2.2.1.5 describes.
Access: Free full text
| Arm | Discounted total QALYs | Discounted total costs per patient | ICER |
|---|---|---|---|
| “Trio eES” | “1.09” | “$282,281” | “Trio eES dominates” |
| “Singleton eES” | “1.10” | “$288,308” | “Singleton eES dominates” |
| “Trio TC” | “1.12” | “$334,170” | “$93,569” |
| “Singleton TC” | “1.08” | “$330,249” | “Ref.” |
Related diseases and analogous treatments
Ketogenic diet, vagus nerve stimulation, and resective surgery versus a third antiseizure drug in children with drug-resistant epilepsy and tuberous sclerosis complex, United States: Fallah et al. (2016)
Relevance: Evaluates the ketogenic diet, vagus nerve stimulation, and resective surgery against a third antiseizure drug in children with drug-resistant epilepsy in tuberous sclerosis complex in the United States, a U.S. analysis that costs ketogenic dietary therapy, the standard of care for pyruvate dehydrogenase complex deficiency listed in section 2.2.2.1.1.
Access: Not open access
Not quoted: The results are reported in the full text, which is closed access.
“A cost-utility analysis, from a third-party payer perspective, was performed for children with drug-resistant epilepsy that had failed to improve with 2 antiseizure drugs (ASDs) and that was amenable to resective epilepsy surgery, across a time-horizon of 5years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Four strategies were included: (1) resective epilepsy surgery, (2) vagus nerve stimulator (VNS) implantation, (3) ketogenic diet, and (4) addition of a third ASD (specifically, carbamazepine).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Given a willingness-to-pay (WTP) of $100,000 per QALY, the addition of a third ASD ($6600 for a gain of 4.14 QALYs) was the most cost-effective treatment strategy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In a secondary analysis, if the child whose epilepsy had failed to improve with 3 ASDs, ketogenic diet, addition of a fourth ASD, and resective epilepsy surgery were incrementally cost-effective treatment strategies.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Vagus nerve stimulator implantation was more expensive yet less effective than alternative strategies and should not be prioritized.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Ketogenic diet versus care as usual in children and adolescents with intractable epilepsy, Netherlands: Wijnen et al. (2017)
Relevance: Evaluates the ketogenic diet against care as usual in children and adolescents with intractable epilepsy in the Netherlands, for the dietary therapy that section 2.2.2.1.1 lists as the standard of care for pyruvate dehydrogenase complex deficiency.
“To examine long-term retention rate, clinical outcomes, cost-utility and cost-effectiveness of the Ketogenic Diet (KD) compared with care as usual (CAU) in children and adolescents with intractable epilepsy from a societal perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Participants were randomized into a KD or CAU group.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Seizure frequency, quality adjusted life years (QALYs), side-effects, seizure severity, health care costs, production losses, patient and family costs were assessed at baseline and during 16-months of follow-up.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“48 children were included in the analyses of this study (26 from KD group).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Mean costs per patient in the CAU group were €53,367 (extrapolated) compared to €61,019 per patient in the KD group, resulting in an ICER of €46,564 per responder.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Cost per QALY rose well above any acceptable ceiling ratio.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“However, as only a minimal difference in QALYs was found between the KD group and the CAU group, the resulting cost per QALY ratios were inconclusive.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
mRNA therapy versus dietary management and organ transplantation in methylmalonic and propionic acidemia, United Kingdom: Bretos-Azcona et al. (2024)
Relevance: Evaluates novel mRNA therapies against a standard of care of dietary management and organ transplantation in newborns with methylmalonic and propionic acidemia in an early cost-utility model from a United Kingdom payer perspective, a comparator that includes dietary management as the ketogenic diet in section 2.2.2.1.1 does for pyruvate dehydrogenase complex deficiency.
“The objective of this research was to synthesize available evidence on the costs and clinical consequences associated with MMA and PA for the purpose of exploratory economic evaluation of novel mRNA-based therapies using an early cost-utility model from the United Kingdom payer perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A Markov model was constructed to simulate the costs and outcomes associated with novel mRNA therapies, compared with a combination of dietary management and organ transplantation (standard of care) among hypothetical cohorts of new-born patients with MMA and PA.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“mRNA therapy was associated with an additional 5.7 and 1.3 quality-adjusted life-years (QALYs) gained per patient lifetime among patients with MMA and PA, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Assuming a willingness to pay range of £100,000-£300,000 per QALY gained, the model demonstrated mRNA therapy to be cost-effective in MMA and PA at an annual treatment cost of £70,452-£94,575 and £31,313-£36,695, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights
Utility weights from analyses of SL1009 (sodium dichloroacetate)
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
Utility weights for a severe mitochondrial disorder during and after neonatal intensive care: Crawford et al. (2021)
Relevance: Utilities for a severe mitochondrial disorder during and after neonatal intensive care, taken from published literature, expert opinion, and the Pediatric Health Information System database, for the disorder group that includes pyruvate dehydrogenase complex deficiency.
Access: Free full text
| Health state | Utility |
|---|---|
| “NICU” | “0.87” |
| “Post-NICU” | “0.60” |
Utility weights for related diseases and analogous treatments
Utility weights for methylmalonic and propionic acidemia on dietary management, with and without developmental delay: Bretos-Azcona et al. (2024)
Relevance: Utilities for methylmalonic and propionic acidemia on dietary management, with and without developmental delay, from an early model of mRNA therapies.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Utility weights around mild, moderate, and severe stavudine-related peripheral neuropathy, HIV infection: Rosen et al. (2008)
Relevance: Utilities around mild, moderate, and severe stavudine-related peripheral neuropathy in HIV infection, an adverse event of the same type as the sensory and motor neuropathy that section 2.1.2.7.1 reports with chronic dichloroacetate exposure.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which this page does not quote.
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Ratios | dichloroacetate OR "SL1009" OR "pyruvate dehydrogenase" | 0 |
| Methods | dichloroacetate OR "pyruvate dehydrogenase" OR "lactic acidosis" | 0 |
| Ratios | "mitochondrial disease" OR "mitochondrial disorder" OR "Leigh syndrome" OR "MELAS" OR "mitochondrial myopathy" | 3 |
| Weights | "mitochondrial" OR "lactic acidosis" OR "Leigh" | 55 |
| Ratios | "ketogenic diet" OR "ketogenic dietary" | 8 |
| Ratios | "inborn error" OR "methylmalonic" OR "propionic acidemia" OR "urea cycle" OR "organic acidemia" | 4 |
| Ratios | idebenone OR "Leber hereditary optic neuropathy" OR "Friedreich ataxia" OR omaveloxolone OR elamipretide OR "Barth syndrome" | 0 |
| Weights | "Friedreich" OR "Leber" OR "GLUT1" OR "glucose transporter type 1" OR "inborn error" | 11 |
| Weights | "peripheral neuropathy" | 83 |