NICE introduced the highly specialised technology (HST) programme in 2015 to consider drugs for very rare conditions. Willingness-to-pay thresholds in HST are higher and evidence requirements generally lower than in standard technology appraisals (TAs). Topics are selected based on seven criteria, all of which must apply for the drug to be assessed via the HST programme.

This study compared the number of criteria met by orphan designation drugs assessed through the HST programme versus those assessed by the standard TA process.

Drugs receiving orphan designation from the European Medicines Agency between January 2014 and May 2019 were extracted and grouped according to their eventual NICE appraisal pathway. Five TA and five HST drugs were randomly selected and checked against HST criteria to assess how many they met.

Drugs assessed through HST processes met 5-7 of the 7 criteria, whilst drugs assessed through TA processes met 2-5 criteria. Two of 5 HST drugs, and three of 5 TA drugs were judged to meet 5/7 HST criteria. Of the 2 HST drugs meeting only 5 criteria, neither met the life-long use criteria, one was not expected to be used in the context of a highly specialised service, and one did not target a clinically distinct patient group. Of TA-assessed drugs, use in a highly specialised service was the criteria most commonly not met (4 of 5 drugs). All TA and HST drugs were judged to meet the criteria relating to need for national commissioning.

In general, drugs assessed through HST met most, but not all of the criteria. Orphan drugs in the TA process on average met less criteria, but many met the same number as those assessed via HST. Consequently, some orphan drugs are being assessed via more challenging routes for reasons that are unpredictable and unclear.