Section 5 of 6

Economic information and modeling report

7 evidence topics · 6 sources

Modeling overview

Summary

The identified economic evidence includes UK Markov models of a hypothetical intervention, the Project HERCULES natural-history framework, and a Japanese registry-based model using data from 730 participants. These are general DMD modeling resources, not a z-rostudirsen budget impact model. A separate systematic review identifies heterogeneity in utilities, healthcare-resource use, and costs as a source of modeling uncertainty. Product-specific price, annual treatment cost, cost-effectiveness, and budget impact estimates are not available in the evidence reproduced here.

Anticipated annual treatment cost

No evidence found.

Product-specific cost-effectiveness estimate

No evidence found.

Z-rostudirsen-specific HTA

No evidence found.

Z-rostudirsen-specific economic model

No evidence found.

U.S. payer distribution

“~ 55% Medicaid, 40% commercial, 5% Medicare/other” (opens the source at this quote in a new tab)

Utilities, costs, and healthcare-resource use

“Future economic modeling of DMD must accommodate the inherent uncertainties due to the heterogeneity of reported utilities, HCRU and costs.” (opens the source at this quote in a new tab)

DMD model frameworks

“three Markov cohort state-transition models” (opens the source at this quote in a new tab)
“a hypothetical intervention for DMD versus standard of care in a UK setting.” (opens the source at this quote in a new tab)

Project HERCULES natural-history model

“The model includes a detailed disease pathway for DMD, including the clinically and economically important transfer state.” (opens the source at this quote in a new tab)

Project HERCULES natural-history model: published correction

“In the originally published article, many of the supplementary materials were missing from the Supplementary Information file.” (opens the source at this quote in a new tab)

Registry-based Japanese economic model

“Age-specific transition probabilities were estimated by fitting a Weibull distribution to Remudy data of 730 patients.” (opens the source at this quote in a new tab)

Budget impact model

Approach and framework

No evidence found.

Perspective and time frame

No evidence found.

Epidemiology and eligible population inputs

No evidence found.

Cost assumptions

No evidence found.

Model outcomes

No evidence found.

Results

Base case

No evidence found.

Scenario analyses

No evidence found.

Budget impact model discussion

No evidence found.