Name: Emcitate (tiratricol) in MCT8 deficiency: Economic burden and healthcare resource utilization
Published: 2026-09-14T15:39:06-04:00

Product information and disease description Place of product in therapy Disease description Burden of MCT8 deficiency

Economic burden and healthcare resource utilization

4 evidence topics · 1 source

United States real-world cost study: objective, data sources, and population

“Evaluate the healthcare resource utilization and cost of care.” (opens the source at this quote in a new tab)
“The lack of a dedicated ICD-10 code for MCT8 deficiency contributes to the challenge of conducting analysis utilizing real-world data.” (opens the source at this quote in a new tab)
“In total, 44 unique patients with a confirmed MCT8 deficiency diagnosis were analyzed.” (opens the source at this quote in a new tab)
“The mean (SD) age of the patients was 5.9 (6.9) years, with a mean follow-up of 72.4 (20.8) months.” (opens the source at this quote in a new tab)
“This research was conducted in collaboration with Inizio Ignite Putnam and was funded by Egetis Therapeutics.” (opens the source at this quote in a new tab)

United States real-world cost study: all-cause costs and utilization

Measure	Value
All-cause cost per patient per year	“$245,393 PPPY”

“Patients experienced nearly 7 inpatient visits per year, corresponding to approximately $327,000 PPPY in hospital-based costs, indicating substantial acute care utilization.” (opens the source at this quote in a new tab)
“Outpatient visits occurred more than twice per month, highlighting the high burden of care.” (opens the source at this quote in a new tab)
“Inpatient care was the largest contributor to healthcare costs, despite outpatient visits representing the highest-utilization setting on a PPPY basis (>2 visits per month).” (opens the source at this quote in a new tab)

United States real-world cost study: clinical drivers of cost

“Cardiovascular-related and infection-related encounters were key cost drivers; cardiovascular events contributed substantially to costs despite representing fewer visits, suggesting that each cardiovascular-related episode is associated with high costs.” (opens the source at this quote in a new tab)
“Nearly all patients had weight-related claims, primarily characterized by underweight/malnutrition and failure to thrive.” (opens the source at this quote in a new tab)
“Cardiovascular claims affected approximately 64% of patients, with over 33% of patients experiencing tachycardia.” (opens the source at this quote in a new tab)
“Infection-related events were common, with 16% of patients experiencing sepsis.” (opens the source at this quote in a new tab)

United States real-world cost study: limitations stated by the authors

“Consistent with rare diseases, the relatively smaller sample size, may make the generalizability of the findings limited.” (opens the source at this quote in a new tab)
“Claims and EHR data were combined for this study; however, this integration may have introduced the potential for underestimation of cost due to differences in data capture across data sources.” (opens the source at this quote in a new tab)
“Cost estimates for infection-related, sepsis-related, cardiac-related, and weight-related categories may be subject to overestimation, as these categories were not mutually exclusive, potentially resulting in overlapping attribution of costs.” (opens the source at this quote in a new tab)