OBJECTIVES: Since May 2013, the assessment of Ultra Orphan Drugs (UODs) in England has been carried out through the Highly Specialised Technologies (HST) route. The objective of this study was to compare annual treatment prices of Orphan Drugs (ODs) before and after the implementation of HST and the difference in prices between ODs and UODs in England

METHODS: ODs approved in Europe were extracted from the European Medicines Agency (EMA) website and HST final evaluations reports published until 30/03/2018 were identified. Ex-factory prices were extracted from IHS POLI database and annual treatment prices per patient were calculated using the posology and treatment duration mentioned in the summary of products characteristics

RESULTS: Ten ODs were reviewed by National Institute for Health and Care Excellence before May 2013 and 24 ODs after May 2013. The OD annual prices ranged between £5,270 and £99,750 before May 2013 and between £3,909 and £146,574 after May 2013. The mean ODs annual prices were almost 2 times higher after the implementation of the HST: mean annual prices of £31,404 (median=£16,746) before May 2013 versus £54,082 after May 2013 (median=£39,390). Furthermore, since 2013, seven UODs have been reviewed through HST with a mean annual prices of £429,972 (median=£326,872), nearly 8 times higher than ODs mean annual prices (mean =£47,411; median=£32,720).

CONCLUSIONS: HST appraisals led to the approval of UODs, which are associated with very high prices, potentially due to the fact that HST focuses on the unmet needs without taking into consideration the UOD cost-effectiveness ratio. Therefore, NICE has proposed in April 2017 changes to the HST by introducing cost-effectiveness decision rules with an incremental cost-effectiveness ratio (ICER) for HST ranging from £100,000/QALY to £300,000/QALY; this threshold is 5 to 15 time higher than the one used for non-UODs.