NICE urged to reform assessments for rare disease drugs

Cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) has been encouraged to change the way it assesses applications to gain reimbursement for rare disease medicines in England.

Consultancy MAP BioPharma makes the recommendations to the NICE, as well as to the National Health Service and the UK government, in a report claiming that the current system does not deliver equal access for patients, with routine availability of new medicines delayed or prevented entirely due to inflexible, inappropriate assessment processes.

According to the report, of the 24 completed single technology appraisal (STA) reviews of rare disease drugs between 2013 and 2017, very few were recommended for the full eligible population, in line with marketing authorization). This compares with a full recommendation for more than two thirds of other medicines.