AUTHORS: Amrit Kaliasethi, Tia Kwong, Mariam Bibi
OBJECTIVES
In 2013, England’s National Institute for Health and Care Excellence (NICE) introduced an alternative appraisal pathway for very rare disease treatments: the Highly Specialised Technology (HST) program. HST aims to enable quicker patient access to innovative treatments which would struggle to meet the evidentiary requirements of the single technology appraisal (STA) program. In March 2017, changes to the HST program were made to reduce delays in appraisals. NICE updated the HST routing criteria in February 2022. This study aims to determine whether NICE has improved efficiency in evaluating therapies through the HST pathway after the program changes.
METHODS
All completed evaluations published by NICE from the HST program’s inception to August 2024 were included. 28 evaluations were analysed to understand the process timing, from preparation of the scope to the publication of the final guidance, as well as the publication of Evaluation Consultation Documents. To determine improved efficiency in the HST program over time, the 28 evaluations were split between those with the final scope published before March 2017, between April 2017-January 2022, and after February 2022.
RESULTS
3, 18 and 7 HST guidance with final scope dates before March 2017, between April 2017-January 2022, and after February 2022, respectively, were identified.
Overall, the median days from final scope to final guidance was 414 days (range 275 – 2261 days). Median days were 398 days (393 – 1015 days) before March 2017, 421 days (275 – 2261 days) between April 2017-January 2022, and 408 days (range 306 – 1861 days) after February 2022.
CONCLUSION
This research has shown that NICE remains far off from their 168-day target for the highly specialised technologies evaluations, despite changes to the program in the past seven years. The median HST duration has slightly increased since the program’s inception. This may delay patients’ access to potentially life-changing medicines.
Read our full research below (available post-conference).
Plus, read our other research abstracts for WODC Europe 2024 here.