Question to the Department of Health and Social Care:

To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 11 March (HL15195), what assessment they have made of the suitability of conventional cost-effectiveness assessments for medicines where structural evidence constraints arising from small patient populations cannot be resolved.

The National Institute for Health and Care Excellence (NICE) is the independent body that makes recommendations for the National Health Service on whether new medicines should be routinely funded by the NHS based on an assessment of clinical and cost effectiveness. NICE’s methods have been proven to be suitable for evaluating rare disease medicines where companies engage with the process and set prices for medicines that fairly reflect their clinical benefits.

NICE can recommend promising new non-cancer medicines for use through the Innovative Medicines Fund (IMF) where there is clinical uncertainty that may be resolved through the collection of real-world evidence. The IMF provides time limited access to innovative non-cancer medicines while further evidence is generated to address uncertainties identified by the NICE appraisal committee. Following a managed access period of up to five years, NICE reviews the updated evidence to determine whether the medicine should be routinely commissioned by the NHS.

The NICE methods manual allows its independent committees to apply flexibilities in circumstances where evidence generation is particularly difficult, for example in rare disease, technologies for use predominantly in children, and innovative and complex technologies. In these specific circumstances, the committee may be able to make recommendations accepting a higher degree of uncertainty. The committee will consider how the nature of the condition or technology affects the ability to generate high-quality evidence before applying greater flexibility.