Abstract
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. 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. From the initial set of 21 guidances, 7 met the inclusion criteria. All conditions were for populations <180, (average was <100). The final average annual list price was £299K (excluding the one-time administration gene therapy). Sponsor models produced base case incremental QALYs of 12.6 on average (versus 7.5 QALYs in ERG models, with 4 drugs of 10+ QALYs). The final NICE recommendations each had stipulations, including: confidential discounts (N=4); Managed Access agreements (MAA, N=3, 2 of which were in combination with discounts), and suggested new or additional data collection (N=2). The negotiation timeline from the publication of the final scope to a published guidance took on average 550 days, with the fastest at 275. 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.
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