NICE’s Highly-Specialised Technologies (HST) process is a method for evaluating medicines and treatments specifically indicated for ultra-orphan diseases (affecting <1 in 50,000 people) that are unlikely to meet the typical HTA criteria.
HST guidance is uncommon – only 14 guidance’s have been published since the program’s inception in 2015. In the last two years, this has included Metreleptin for treating lipodystrophy (2.5 per million people) and Volanesorsen for treating familial chylomicronaemia (1 to 2 per million people).
So, how does NICE determine if a product should be considered within the HST process? They take a number of factors, outlined below, into account when determining if a product should be assessed under the standard STA process or within the HST process:
- The target patient group for the technology in its licensed indication is so small that treatment will usually be concentrated in very few centres in the NHS
- The target patient group is distinct for clinical reasons
- The condition is chronic and severely disabling
- The technology is expected to be used exclusively in the context of a highly specialised service
- The technology is likely to have a very high acquisition cost
- The technology has the potential for life long use
- The need for national commissioning of the technology is significant.
This criteria for the HST are currently undergoing review and it is likely that ‘lifelong’ and ‘chronic’ diseases are currently key principles for the HST, are going to be removed to reflect the rise of one-off gene therapies.
What are the key differences between the HST and the STA?
Additionally, in the HST, there is a greater consideration of the impact beyond the direct health benefit. This includes more focus on the significance of non-health benefits, whether a substantial proportion of the costs or benefits are incurred outside of the NHS and personal/social services, and the potential for long-term benefits to the NHS in terms of research and innovation.
In the HST, if the most plausible ICER is over £100,000 per QALY, but the treatment is considered to offer considerable QALY gains (>10), then the HST Evaluation Committee will consider a QALY modifier. This adds extra weighting to the QALY benefits, allowing a therapy to still fall within the HST £100,000 per QALY limit. Therefore, in theory, a therapy delivering up to 30 QALY’s could have a £300,000 per QALY threshold.
What should manufacturers do to prepare for the HST?
- Critically evaluate the product to determine if it meets HST criteria
- Have early engagement with NICE to confirm pathway and implications for UK access
- Determine evidence gaps for HST submission
- Considering limited evidence base associated with rare diseases it is likely that you will have to rely on clinical expert opinion to address the evidence gaps
- Consider the need for commercial agreements to gather additional evidence to support UK access and reduce NICE uncertainty regarding the value of the product
- Engage with NHS clinical and commercial teams to determine how the product will be utilised and commissioned within NHS England
- Closely monitor upcoming changes to the HST, especially around the qualifying criteria