OBJECTIVES: We have identified a recent trend for increasing recommendations with restrictions at the expense of full recommendations in health technology assessments (HTAs) by NICE. The objective of this study was to identify trends and the reasons for restricted recommendations for orphan drugs.

METHODS: We analysed NICE guidance for orphan drugs between 2014 and 2018, focussing on those that received restricted recommendations. Using MAP’s proprietary HTA database, we examined trends in the frequency and reasons for these recommendations. Cancer products routed to the Cancer Drugs Fund (CDF) were considered another form of restricted recommendation.

RESULTS: Orphan drugs received restricted recommendations in 10-15% of HTAs in 2014-2016. Unrestricted recommendations were granted to 50-60% of products over the same period. From 2017 onwards the proportions had reversed: restricted recommendations accounted for around 50% of products, and 10% were recommended without restrictions. This trend was also seen in non-orphan drugs with restricted recommendations at around 12% in 2016, which rose in 2017 and 2018 to over 50%.

2018 saw an increase in the proportion of NICE orphan medicines submissions (46% in 2016, 36% in 2017 and 64% in 2018), the majority being in oncology. An increasing proportion of these products lacked certainty in their underpinning data and were assigned to the CDF (4% in 2016, 12% in 2017 and 29% in 2018).

CONCLUSIONS: The increase in restricted recommendations may be due to NICE’s appraisal methodology becoming stricter for both orphan and non-orphan drugs. Companies may be increasingly willing to take the risk of submitting before sufficient data has been accumulated: a challenge for orphan medicines. We may speculate that some companies could have attempted to submit before the introduction of fees for NICE HTA in 2019. Further research is in progress to ascertain the reasons for these trends.