Question to the Department of Health and Social Care:

To ask the Secretary of State for Health, what assessment his Department has made of the potential effect on patients with (a) rare and (b) ultra-rare diseases of NICE and NHS England’s proposals for changes to the arrangements for evaluating and funding drugs and other health technologies appraised through NICE’s technology appraisal and highly specialised technologies programme, published in October 2016.

We are committed to ensuring that patients with rare and ultra-rare diseases in England are able to access effective treatments.

The changes to the National Institute for Health and Care Excellence’s Highly Specialised Technologies methods are intended to introduce a fairer, more transparent framework for the evaluation of technologies for very rare diseases that will enable truly transformative new drugs for patients with rare diseases to be made available to patients where companies are willing to set prices that fairly reflect the added benefit they bring.