The National Institute for Health and Care Excellence (NICE) in England has a separate appraisal process for drugs for very rare conditions, i.e. Highly Specialised Therapies (HST). In April 2017, the HST process has been changed to incorporate a quantitative approach: automatically fund treatments with incremental cost-effectiveness ratio (ICERs) up to GBP 100,000 (EUR 113,008 based on the 2018 average GBP / EUR exchange rate) per quality-adjusted life year (QALY). For treatments with an ICER above GBP 100,000 per QALY, NICE will consider treatments that offer a substantial magnitude of improvement, with additional QALY weighting. We investigated the impact of this more quantitative approach on the likelihood of a HST receiving a positive recommendation.

All HST appraisals and draft guidance documents were reviewed (up to November 2018) and data were extracted on ICERs, incremental QALY gain, budget impact, and recommendations. The extracted data from each HST were assessed based on the interim HST guidance.

Eighteen products have been or are currently going through the NICE HST process. Of these, 8/18 (44%) have received a positive recommendation, while 5/18 (28%) have received a draft negative guidance, and for 5/18 (28%) products, no recommendations have been published. For the products with a positive outcome, 5/8 (63%) had incremental QALY gain of at least 10, qualifying these products for additional QALY weighting. For the products that received a draft negative recommendation, the negative decision was related to the cost-effectiveness estimates being higher than GBP 100,000 per QALY (5/5 reported) in all cases, while none of these products were eligible to receive a ‘QALY modifier’.

It has become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 per QALY for many products.

In April 2017, the National Institute for Health and Care Excellence (NICE) updated its guidance for highly specialized technology (HST) appraisals, whereby it would automatically fund technologies for very rare diseases that fall below a threshold of an incremental cost-effectiveness ratio (ICER) of GBP 100,000 (USD 133,000) per quality-adjusted life year (QALY). In addition, NICE proposed to introduce a ‘QALY modifier’, weighting QALYs gained by the size of gain, which will advantage treatments that offer greater QALY gains.

We reviewed all technologies reviewed through the NICE HST process until November 2017 and assessed whether additional QALYs may be awarded, and subsequently result in ICERs below the new NICE threshold.

Six products (eculizumab, elosulfase alfa, ataluren, migalistat, eliglustat, and asfotase alfa) have been through HST process. Within the appraisal documents, most analyses were cost consequence analyses with no ICERs reported. The estimated cost per patient per year ranged from approximately GBP 100,000 (USD 133,000) to GBP 400,000 (USD 532,000; listed prices). Of the six technologies, three resulted in at least ten incremental QALYs (eclizumab, elosulfase alfa and asfotase alfa). From the information in the public domain, it is unclear whether this would result in ICERs below GBP 100,000 (USD 133,000) per QALY.

It may become more difficult for HSTs to get recommended by NICE under the new guidance, which requires cost-effectiveness analyses, whereas previously there was no official ICER threshold. The additional weighting of QALYs may be insufficient to meet an ICER threshold of GBP 100,000 (USD 133,000) per QALY.