Rare disease patients in UK wait average of two years for medications

A new report published by Irish biotechnology company Shire has shown that rare disease patients in England, UK, have to wait an average of two years for life-changing treatments to be funded on the National Health Service (NHS).

The finding also highlights the fact that more than half (52%) of the life-changing medications for rare diseases approved over the past 15 years in England are not funded for routine use.

Titled ‘Equity and Access: Making the UK a Rare Disease Leader’, the report has been funded and initiated by Shire and highlights the findings, along with a recommendation for collaboration with the government, to develop new, tailored process to examine rare disease medicines.

Based on the analysis of the availability of rare disease medicines across five countries in Europe, the report reveals that patient access in England falls behind that in France, Germany, Spain and Italy.

"We need to shine a light on these issues and put together an action plan to ensure that people living with rare diseases in England have fair and equal access to the treatment they need."

The company has brought together leaders from several medical communities, policy-makers, patient advocacy and healthcare to review information analysed by UK’s Office of Health Economics (OHE).

Shire UK and Ireland Country Cluster head and general manager Sebastian Stachowiak said: "Significant strides have been made in innovating and developing new medicines for people living with rare diseases, and yet patients in this country are still facing uncertain waits for treatments.

"We need to shine a light on these issues and put together an action plan to ensure that people living with rare diseases in England have fair and equal access to the treatment they need."

Out of the 143 medicines approved by the European Medicines Agency (EMA), 48% (68) were funded by the NHS in England, while 93% (133) were funded in Germany and 81% (116) in France.