OBJECTIVES: The NICE Highly Specialised Technologies (HST) assessment methods for very rare conditions were published in May 2013. The objective of this study was to characterize the published guidances and to identify and explore themes to help sponsors prepare for an HST evaluation.

METHODS: We accessed all of NICE’s published HST guidances and supporting evidence papers since January 2015, and excluded guidances in consultation, development, or proposed due to lack of final recommendations. To discern trends, data was systematically collected on: patient population size; final list price; time from final scope to publication; differences between Evidence Review Group (ERG) and sponsor cost-effectiveness model attributes; and NICE recommendations.

RESULTS: From the initial set of 21 guidances, CONCLUSIONS: NICE HST recommendations have been published predominately for drugs with very low patient populations, high expected list prices, and most with high QALY gains. Sponsors may be able to use this information to predict whether a product is likely to receive HST review. In many cases the recommendation required negotiation longer than one year and a discount and/or MAA to address uncertainty, thus, sponsors should collaborate with authorities early on submissions to help speed negotiations.