BIA calls for new pathway for access to innovative rare disease medicines

The UK’s BioIndustry Association (BIA) today released its report, From innovation to impact: unlocking patient access to innovative rare disease medicines, calling for a new pathway to give UK patients access to innovative rare disease medicines.

The BIA finds that despite the UK’s rare disease ecosystem having a solid foundation, more work is needed to build a connected, flexible and risk-proportionate access pathway to turn discovery into delivery for patients facing high unmet need.

This report, developed by the BIA’s Rare Disease Industry Group (RDIG), lands at a critical time as the UK considers how innovative and cutting-edge medicines are valued following the landmark UK-US pharmaceuticals deal.