Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment his Department has made of the adequacy of appraisal processes for (a) Freidrich’s Ataxia, (b) Neiman Pick’s disease and (c) other rare diseases for exceptional conditions.

The National Institute for Health and Care Excellence’s (NICE) methods have been proven to be suitable for the evaluation of rare disease medicines, where companies are willing to price their medicines fairly. NICE’s approval rate for medicines for rare diseases is in line with its overall approval rate for new medicines, with 91% of medicines licensed for rare diseases recommended for some or all of the eligible patient population since April 2024.

NICE initiated an appraisal of Skyclarys (omaveloxolone) for the treatment of Friedrich’s Ataxia for people aged 16 years old and over but has had to terminate its appraisal as the company has withdrawn its evidence submission. NICE will review its decision if the company decides to make a new submission. Olipudase alfa for treating Niemann-Pick disease types B and AB was evaluated through NICE’s highly specialised technologies (HST) programme, which is reserved for a small number of medicines for very rare and very severe diseases. The HST programme uses a much higher cost-effectiveness threshold than a standard NICE appraisal that recognises the challenges of developing medicines for very small patient populations. NICE’s independent evaluation committee recognised that the evidence showed that the treatment improved lung function but found that there were uncertainties in the economic model. Even when taking into account the substantial clinical benefits of treatment and the higher threshold, it was unable to recommend it as a cost-effective use of National Health Service resources.