OBJECTIVES : Project HERCULES is a collaboration between Duchenne UK and eight pharmaceutical companies; pooling resources and expert input to develop a core cost-effectiveness model for Duchenne Muscular Dystrophy (DMD). Whilst model users would utilise the same structure, flexibility was required to accommodate different interventions, treatment effects, comparators, patient populations and types of analysis. Due to the collaborative nature of the project, the challenge was to develop the model without disclosure of confidential information or breaching competition rules.

METHODS : A multi-state cohort model was developed with health states informed by clinicians, patients and carers. Data were collected and synthesised to provide a core data set. To avoid the need to share commercially sensitive information on pipeline products, contributing manufacturers and clinical experts discussed all possible treatment effects of future therapies in DMD. Input from the manufacturers, HTA experts and health economists also identified the characteristics of a therapy, decision problem and audience that would necessitate flexibility within the core model. In addition, manufacturers had the opportunity for one-to-one discussions with the developers to ensure the model met their individual needs.

RESULTS The cost-effectiveness model developed includes flexibility to evaluate different emerging treatments in DMD. The user can define their decision problem: patient population, intervention, comparator, type of analysis and perspective. They can define modelling assumptions, including cycle length, time horizon and the health states in which the intervention is received. Most importantly, the model includes functionality to accommodate different treatment effects. Interventions can slow or halt progression, improve function loss or cure, reduce or avoid the use of steroids, and/or avoid acute events.

CONCLUSIONS : This case study illustrates the potential for collaboration between manufacturers in rare diseases to develop a robust, core cost-effectiveness model which meets the needs of the different manufacturers without compromising confidentiality.