For a rare disease, an epidemiological approach is the right way to go. Partly, as there is unlikely to be any historical sales data to support a sales-based approach.

Rare diseases affect more than five million people in Thailand, and roughly 80% are caused by changes to DNA. While standard pathways to rare disease diagnosis can take up to seven years ...

With over 40% of all medicines in the pipeline aimed at rare diseases, the future looks positive for increasing the number of treatments available. However, as Ben Hargreaves discovers ...