Cost Analysis
Models of DMD disease progression and the associated costs are used to evaluate the impact of new treatments. A recent example assessed the value of Elevidys prior to FDA approval. These analyses attempt to motivate payers to cover a therapy that significantly decreases lifetime medical and caregiver costs. To make these models more accessible, the model from Landfeldt et al. (2017) is reproduced below. The ability to produce custom models is in progress.
Lifetime Cost Models
Landfeldt et al. (2017) presented three different models for their cost-effective analysis. Model 1 is based on the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT). Model II is based on ambulatory status. Model III is based on ventilation status.
All models assume the patients progress from the first state to the last and do not go back to an earlier state. The transition from one state to the next is set by the age at which 50% of patients are before the transition and 50% are after the transition. For example, in Model III they assume the average age that patients require night-time ventilation is 21 years, and both day- and night-time ventilation is required at 28 years on average. Similarly, the ages in Model II were set to make 14 years as the average age of transition from ambulatory to non-ambulatory. If you hover over Age 14, roughly 51% are in the Early or Late Ambulatory group and 49% are in the Early or Late Non-Ambulatory groups.
Note: All costs are in 2015 Great British Pounds (GBP) to be easily compared to the published values.
Cost Effectiveness Analysis
Cost effectiveness analysis (CEA) is a method to compare the relative costs and outcomes of different treatments. For this execise, two models are created. One identical to the lifetime cost models above. The second model increases the average age for each state as a response to the treatment. In the example below, the theoretical treatment increased the transition age by 25%.
The assumption is that an effective treatment will delay disease progression, offset medical costs, and improve quality of life. These benefits should be financially greater than the treatment costs.
The data populating this page and the method for summarizing that data is discussed in the About section. Please contact us if you notice any errors are want clarication on what is presented here.