OBJECTIVES:

There is often a paucity of data in rare diseases to support the development of cost-effectiveness models that fully capture the impact of disease and the benefit of new treatments. Cost-effectiveness models can oversimplify the natural history of disease or rely on assumptions. Project HERCULES is a collaboration between Duchenne UK and eight pharmaceutical companies. One objective of this project was to develop a single, robust cost-effectiveness model for treatments in Duchenne Muscular Dystrophy (DMD).

METHODS

A core multi-state cohort model was developed to facilitate the economic evaluation of new treatments in DMD. Clinically and economically important health states were first determined with input from clinicians, patients and carers. Data were then collected and synthesised to provide a core data set. This data set included transition probabilities for standard of care, health-related quality of life utilities, resource use and cost. Transition probabilities were obtained from a natural history model developed using real-world data. Utilities, resource use and cost were obtained via a burden-of-illness study. A de novo patient reported outcome was also developed to facilitate future estimation of utilities in people with DMD. The model methods and data sources underwent review and critique via EMA early scientific advice, and input from clinicians and experts in health economics and health technology assessment (HTA).

RESULTS

Project HERCULES has provided a core model for the economic evaluation of new treatments in DMD that captures the impact of disease, the benefits of treatment, and is based on a robust data set.

CONCLUSIONS

Project HERCULES illustrates the potential for a paradigm shift in the development of cost-effectiveness models in rare diseases. One in which manufacturers collaborate to generate a robust core economic model and data set, and a standardised methodology is used across HTA submissions.