How Restrictive are NICE ‘Optimised’ Decisions?

The National Institute for Health and Care Excellence (NICE) makes recommendations for the use of medicines in the National Health Service (NHS) in England based on their clinical and cost-effectiveness through their Technology Appraisal (TA) programme. The main decision outcomes are to ‘recommend’ the medicine (in line with marketing authorisation), ‘not recommend’ the medicine, or something in between: ‘optimised’. Optimised recommendations support the use of the medicine for a smaller group of patients than originally stated by the marketing authorisation, based on the findings of the appraisal relating to clinical or cost-effectiveness in different patient sub-groups.

The option for NICE to ‘optimise’ recommendations offers a route to treatment access for sub-groups of patients in whom a medicine offers a clinically- and cost-effective alternative to current practice in cases where this was not the case for the full patient population (determined by the licensed use and the scope of NICE’s appraisal). However, the level of patient access associated with optimised recommendations is unclear.

In this blog, we update OHE research into the proportion of NICE recommendations that are “optimised” and the extent to which “optimised” decisions restrict the eligible patient population compared to the scope of the NICE appraisal for 2020 to 2022. The method to estimate the level of patient access was developed by OHE researchers (O’Neill and Devlin, 2010). It was further utilised in our 2020 report looking at NICE decisions from 2015 to 2019 (Bulut et al., 2020).

Between 2010 and 2019, 31% of NICE’s “positive” decisions (recommended or optimised) were optimised, excluding those included in the Cancer Drugs Fund. Over the last two years (Jan 2020 to Dec 2022), this figure rose to 39%.

As part of our 2020-22 update, 16 out of 78 (21%) single technology appraisals (STAs) which had “optimised” recommendations included sufficient information to allow for the estimation of patient access. For comparison, between 2015 and 2019, 40 out of 112 (36%) appraisals included sufficient information, suggesting a decrease in the reporting of relevant information (primarily resource impact templates and statements) by NICE.

Just over half (56%) of optimised decisions recommended treatment in less than half of eligible patients, and a quarter (25%) recommended use in less than a quarter of patients within NICE’s appraisal scope. Between 2010 and 2019, these figures were 65% and 35%, respectively. On average, 45% of the patient population potentially eligible for the treatment under review was recommended for treatment in NICE’s ‘optimised’ recommendation. This is a slight increase from 39% for the period between 2015 to 2019. The overall level of access for optimised decisions from 2010 to 2022 is 36%.

NICE states that 84% of their recommendations are positive. However, a significant proportion of these are optimised recommendations, and we have demonstrated that the recommended level of patient access associated with these can be low. While the justification for these restrictions may be well-founded, more granular reporting of recommendations would help paint a more accurate picture of recommended levels of patient access associated with NICE decision outcomes.

Citations

Bulut, M., O’Neill, P. and Cole, A. (2020) NICE ‘Optimised’ Decisions: What is the Recommended Level of Patient Access?. OHE Contract Research. Available from https://www.ohe.org/publications/nice-optimised-decisions-what-recommended-level-patient-access/

O’Neill P, Devlin NJ. (2010). An analysis of NICE’s ‘restricted’ (or ‘optimized’) decisions. Pharmacoeconomics 28(11):987-93. doi: 10.2165/11536970-000000000-00000. PMID: 20936882.