Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the potential impact of proposed changes to NICE’s Methods and Process on patients with very rare diseases.

A number of changes adopted by the National Institute for Health and Care Excellence (NICE) following its updated methods and processes manual for health technology assessments, will support access to innovative medicines for patients with rare diseases. NICE has introduced a new modifier to consider the severity of the disease or condition under consideration when making recommendations. As many rare conditions are also severe, its committees can give additional weight to the treatment benefits for these conditions.

Where there is uncertain evidence in relation to a medicine, which can affect patients with rare diseases where the population is small, NICE will now adopt a more flexible and proportional attitude towards evidence uncertainty within its decision making.