Rare diseases (according to the European definition includes diseases with a prevalence less than 5 per 10 000 inhabitants) pose some specific challenges: data availability at the launch of a new product is typically quite scarce due to low patient numbers and the cost of treatment is typically higher due to a smaller market in which companies can recoup investments. This introduces greater uncertainty in the decision making process.
We have conducted several projects in this area:
- Development of cost-effectiveness models for use in submissions
- Preference studies to understand the impact of rare conditions
- Studying disease burden
- Development of innovative payment models