Presentation (CTI)

OBJECTIVES: In 2022, NICE introduced the severity modifier in place of the end-of-life modifier with the aim of broadening the range of severe conditions that qualify for an additional weighting, effectively raising the cost-effectiveness threshold, while managing budget. However, NICE’s ‘opportunity cost-neutral’ approach may inadvertently limit patient access to late-stage treatments for severe diseases. This study aimed to determine the impact of the severity modifier on NICE HTA outcomes of orphan drugs and whether there is variation in patient access to oncology vs. non-oncology orphan drugs.
METHODS: NICE technology appraisals for drugs with EMA or MHRA orphan designation published between January 2022-December 2024 were identified. Terminated appraisals were excluded. Pre-defined topics, including disease area, NICE recommendation, and application of a severity modifier, were extracted.
RESULTS: Of the 62 orphan drug HTAs published between 2022-2024, 39% were oncology (24/62) and 61% were non-oncology (38/62). Of the oncology orphan drugs, most were recommended (83%; 20/24), but only 30% of which received a severity modifier weighting (6/20). 65% of oncology drug recommendations were dependent on funding via the Cancer Drug’s Fund or access through a Managed Access Agreement (MAA;13/20). Although a higher proportion of non-oncology orphan drugs were recommended (97% vs. 83% for oncology drugs), fewer recommendations included the application of a severity modifier (24%; 9/37). Only 14% of non-oncology drug positive recommendations were dependent on access through a MAA (5/37).
CONCLUSIONS: The lower rate of severity modifier application in non-oncology indications suggests a higher proportion of severe disease in oncology. The higher dependence on access agreements for oncology drugs may suggest challenges in value demonstration in oncology orphan indications. Non-oncology orphan drugs received a higher proportion of positive recommendations; however, the limited application of severity modifiers indicates that positive recommendations in rare diseases are not solely reliant on the severity modifier.