Updates to NICE Methods and Processes: Implications for the Future of the Highly Specialised Technology Programme

In February of this year, we saw the publication of new manuals for technology evaluations by the National Institute for Health and Care Excellence (NICE). Broadly, the updates to the NICE methods and processes aim to provide faster patient access to novel treatment options, ‘by allowing greater flexibility over decisions about value for money and consideration of a broader evidence base’.1

Historically, orphan medicines have been subject to significantly longer appraisals by NICE than non-orphan medicines.2 Greater deliberation is typically required, due to the uncertainty that is inherent in evidence collected from small and heterogenous patient populations. The commitment from NICE within the updated manuals to accept a greater level of uncertainty, in circumstances where evidence generation is shown to be difficult, signals a positive move towards more rapid access to rare disease treatments.3 By definition rare disease populations are small, and so the associated budget impact of orphan products is usually smaller and more predictable than non-orphan products – NICE have indicated willingness to accept more decision uncertainty where budget impact is minimal, in the interest of more rapid patient access to novel rare disease treatments.3

Interestingly, the updates to the NICE methods and processes have introduced greater alignment across the single technology appraisal (STA) and highly specialised technology (HST) programmes. There is now one unified manual outlining appraisal methods and processes for all programmes.3 In addition, whilst the STA and HST evidence submission templates used to follow a very different format, the new templates follow the same structure. This alignment should make it easier for manufacturers and any other stakeholders to navigate the NICE appraisal process. It is also encouraging to see successful features from the HST programme being carried across to the STA programme, such as structured consideration of ‘wider benefits beyond QALYs’ in the new STA template.4 However, this alignment between the programmes raises the question as to whether NICE are seeking to gradually bring all appraisals under one programme. Is there any benefit in retaining a separate pathway for evaluating treatments for ultra-rare and severely disabling or life shortening conditions? What is the added value of the HST programme, when the STA programme will employ methods designed to facilitate appraisals of rare disease products?

Recent research by the IMPACT-HTA project examined the influence of different health technology appraisal processes on the evaluation of the products nusinersen and voretigene neparvovec.5 The authors concluded that supplemental processes for rare disease treatments may facilitate greater consistency in decision making, and better management of rare disease specificities, such as patient and clinician input.5 However, supplemental methods and processes for the evaluation of rare disease treatments by NICE, such as acceptance of greater levels of uncertainty, should now be applied consistently across both the STA and HTA programmes.

The key remaining area where the HST programme offers a clear additional benefit to manufacturers over the STA programme is in the implications for product pricing. NICE have clarified in the updated topic selection manual that the HST programme is a deliberate departure from the STA process, in order to support access to innovative treatments for very rare conditions, considering the need to manage the impact of more costly rare disease treatments in the wider NHS budget.6 This position is reflected in the large difference that still remains between the STA and HST programmes in the maximum willingness to pay thresholds (HST programme: £100,000/QALY; STA programme: £20,000-30,000/QALY [excluding modifiers]).3 Manufacturers of more costly technologies such as gene therapies therefore still face significant barriers in demonstrating cost-effectiveness outside of the HST programme.

It will be interesting to see whether the NICE methods and processes updates lead to a consistent approach to decision making across the STA and HST programmes. If so, the HST programme will solely function to support the higher costs associated with innovative technologies for ultra-rare conditions.

A Microsoft PowerPoint summary of the updates to the NICE methods and processes, and their impacts for rare disease treatments can be accessed below.

Download PowerPoint


  1. National Institute for Health and Care Excellence. NICE publishes new combined methods and processes manual and topic selection manual for its health technology evaluation programmes. Available here. Last Accessed: March 2022.
  2. Clarke S, Ellis M, Brownrigg J. The impact of rarity in NICE’s health technology appraisals. Orphanet Journal of Rare Diseases 2021;16:218.
  3. National Institute for Health and Care Excellence. NICE health technology evaluations: the manual. Available here. Last Accessed: March 2022
  4. National Institute for Health and Care Excellence. Technology appraisal guidance: Submission template (Word). Available here. Last Accessed: March 2022.
  5. Whittal A, Nicod E, Drummond M, et al. Examining the impact of different country processes for appraising rare disease treatments: a case study analysis. International Journal of Technology Assessment in Health Care 2021;37:e65.
  6. National Institute for Health and Care Excellence. NICE health technology evaluation topic selection: the manual. Available here. Last Accessed: March 2022

Isabelle is an employee at Costello Medical. The views/opinions expressed are Isabelle’s own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners. If you would like any further information on the themes presented above, please do not hesitate to contact Isabelle Newell, (LinkedIn).