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.
0 cost-effectiveness analyses and 2 studies reporting utility weights · 2 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 | 0 |
| Studies reporting utility weights | 2 |
Cost-effectiveness analyses
Analyses of Emcitate (tiratricol)
No evidence found.
Treatments for the same indication
No evidence found.
The same disease in other populations or settings
No evidence found.
Related diseases and analogous treatments
No evidence found.
Utility weights
Utility weights from analyses of Emcitate (tiratricol)
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
No evidence found.
Utility weights for related diseases and analogous treatments
Utility weights by Gross Motor Function Classification level with severe cognitive impairment, and caregiver disutilities, early juvenile metachromatic leukodystrophy: Bean et al. (2024)
Relevance: Utilities by Gross Motor Function Classification level with severe cognitive impairment, and caregiver disutilities by level, from a UK general-population vignette study used in a model of early juvenile metachromatic leukodystrophy, a genetic childhood neurological disorder whose motor function states correspond to the absent head control, sitting, and walking reported for MCT8 deficiency in section 2.2.1.8.1.
“Utility values to calculate QALYs associated with the long-term benefits and disease progression arms were generated from a vignette study conducted in the UK general population (n = 201).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The UK general population evaluated the quality of life of patients in the later stages of disease to be worse than death, generating negative utility values” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In addition, a caregiver disutility was included for patients in GMFC-MLD 3 to 6 to capture the negative effect on health-related quality of life that caring for an MLD patient has on their families.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights by intellectual outcome category, congenital hypothyroidism: Appelberg et al. (2023)
Relevance: Utilities by intellectual outcome category from a decision-analytic model of newborn screening for phenylketonuria and congenital hypothyroidism, a disorder of thyroid hormone supply to the developing brain, an outcome dimension that corresponds to the severe psychomotor impairment described for MCT8 deficiency in section 2.2.1.8.1.
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 | tiratricol OR "Triac" OR Emcitate | 0 |
| Methods | tiratricol OR Emcitate | 0 |
| Ratios | "MCT8" OR "Allan-Herndon-Dudley" OR "monocarboxylate transporter" | 0 |
| Weights | "MCT8" OR "Allan-Herndon-Dudley" OR "thyroid hormone transport" | 0 |
| Ratios | "thyroid hormone" AND (levothyroxine OR propylthiouracil) | 2 |
| Ratios | "congenital hypothyroidism" | 17 |
| Ratios | "Rett syndrome" OR trofinetide OR "CDKL5" OR ganaxolone | 0 |
| Weights | "Rett syndrome" OR "CDKL5" OR "Angelman" OR "Dravet" | 9 |
| Ratios | "metachromatic leukodystrophy" OR "adrenoleukodystrophy" OR "Krabbe" OR "Canavan" OR "Pelizaeus" | 12 |
| Weights | "metachromatic leukodystrophy" OR "adrenoleukodystrophy" OR "leukodystrophy" | 39 |
| Weights | "cerebral palsy" OR "Gross Motor Function Classification" | 86 |
| Weights | "hyperthyroidism" OR "thyrotoxicosis" | 39 |
| Weights | "intellectual disability" OR "developmental delay" OR "psychomotor" | 22 |
| Ratios | levothyroxine OR propylthiouracil | 9 |
| Methods | DITPA OR diiodothyropropionic OR "thyroid hormone analogue" OR "thyroid hormone analog" | 0 |