Question to the Department of Health and Social Care:

To ask His Majesty's Government how many times the National Institute for Health and Care Excellence has exercised its discretionary power to exercise flexibility in its application of routing criterion two of its Highly Specialised Technologies programme in circumstances where there is uncertainty on the incidence and prevalence of the disease and high unmet clinical need, to ensure fair and equitable access to innovative new treatments.

Since the introduction of the current criteria used for routing topics to the National Institute for Health and Care Excellence’s (NICE) Highly Specialised Technologies (HST) programme, the NICE has identified one topic routed to the HST programme where flexibility was exercised in relation to the eligible patient population. This topic was pegzilarginase for treating arginase-1 deficiency, for which the estimated eligible patient population was 320.