A report by MAP BioPharma (MAP) claims that NICE’s appraisal of rare disease medicines does not deliver equal access for patients, leading the company to urge for reform.
The findings show that routine availability of new medicines was "delayed or prevented entirely due to an inflexible, inappropriate assessment processes".
According to the report, of the 24 completed STA reviews of rare disease medicines between 2013 and 2017, as little as 13% were recommended for the full eligible population - in line with marketing authorisation. This compares with a full recommendation for more than two thirds of other medicines.
Furthermore, in the same period, 50% of rare disease medicines were given a “restricted recommendation”, compared to just 21% of non-rare disease medicines.
The report also found that of the non-cancer focused rare disease medicines reviewed, none were recommended within the full marketing authorisation.
It also points out that more rare cancer treatments benefit from the Cancer Drugs Fund (CDF) than other cancer medicines, "which demonstrates how rare disease medicines are more likely to require special considerations in their appraisals".
MAP argues that this is an issue "that is already having a direct impact on patients, many of whom have to wait longer to access new treatments or miss out due to process issues that could be addressed by making adjustments to existing systems".
Christian Hill, the firm's chief executive, stressed that this is one of the most important challenges that is holding back access for patients.
“MAP’s research illustrates that the current processes are not fit for purpose and we urge NICE, NHS England and the Government to work with us and the rare disease community to review how they can improve patient access to rare disease medicines, and give careful consideration to the case for change and recommendations set out in the report”.
An estimated 3.5 million people are affected by rare diseases in the UK. How quickly these patients can access the most recent treatments significantly affects not only the patients but also their families.