This document analyzes trends in patient access to new medicines based on NICE technology appraisal decisions between 2007-2013. It develops a measure "M" to represent the level of patient access, where M=100 indicates full access and M=0 no access. The analysis finds that for medicines assessed, the level of access was between 42-54% of potential patients. There was wide variation between therapy areas, with cancer having the lowest M score of 36. While decisions have declined to recommending use in around 2/3 of cases, the analysis estimates actual patient access was closer to 1/2 of potential patients.

1. Measuring Access Associated with NICE Technology Appraisal decisions
Phill O’Neill, Nancy Devlin. Office of Health Economics
For further information, contact poneill@ohe.org
1. BACKGROUND
NICE report that, between its establishment in 2000 and 30th June 2014, 79% of decisions have been either to recommend or optimise the use of the technology1. There is an implication that optimised decisions are positive and represent an appropriate use of resources based on clinical and cost effectiveness evidence. In a previously published paper we developed a measure, M, to summarise access associated with NICE technology optimised appraisal decisions2. This was defined as M=(p/P)X100, where M is a measure of the level of patient access (0 equals no access, 100 full access), P is the set of patients considered in the guidance as potential candidates for treatment (given the scope of appraisal and license), and p is the number of patients for whom NICE did recommend. By extending our previous analysis it is possible to assess trends in access associated with NICE decisions.
Acknowledgements This research was funded by Pfizer Ltd.
References 1. NICE (2014) Technology appraisal decisions. NICE Statistics. Available at: http://www.nice.org.uk/News/NICE-statistics [Accessed 30 July 2014]. 2. O’Neill P, Devlin N (2010) “An analysis of NICE’s restricted (or ‘optimised’) decisions” Pharmacoeconomics. 28(11), 987-993
2. AIMS
•To assess the level of patient access associated with NICE technology appraisal decisions for medicines published between 2007 and 2013. Since the establishment of the STA process.
•The primary aim is to provide a better way of describing NICE decisions and of analysing trends in decisions; and of measuring the implications of decisions for patient access to new medicines.
3. METHODS
Applying measure M to NICE HTA decisions for medicines between January 2007 and December 2013 we examine trends by therapeutic area and over time. In this paper, to understand trends, we build on our earlier work, which focussed on 'optimised' decisions, by extending the analysis to include recommended decisions (M=100) and not recommended decisions (M=0). For optimised decisions, where is was not possible to ascertain M, a score of 25, 50, 75 or excluded has been applied to test sensitivity of results.
4. RESULTS
Depending on assumptions made regarding the treatment of optimised decisions, where available data did not allow a score to be calculated, the M score ranged from 42 and 54 out of a 100 for the period 2007-2013 Therefore conclusions about overall trends are not sensitive to this assumption. The chart below plots M scores per year by TA type.
5. DISCUSSION
6. CONCLUSIONS
Overall, for medicines subject to NICE technology appraisal between 2007 and 2013, the results suggest that, relative to the maximum number of potentially relevant patients (as determined by medicine license), NICE’s decisions result in recommended access in the range of 42 to 54 out of 100 patients. There is wide variation among therapy areas. There are important classes with lower scores which have attracted many NICE appraisals, notably cancer and immunomodulation. It is the case that there are classes that have been invariably recommended close to or full access, Hepatitis C being the clearest example.
Assessing trends by therapy area for the period 2007 to 2013 M scores ranged from 36 for cancer to 100 for hepatitis C (note for optimised medicines where M cannot be determined assumed to be 50).
Figure 2: Measure M scores for the sample of medicines by therapy area 2007-2013
This analysis provides context for NICE reported trends in technology appraisal decisions. Although NICE report that they reach a positive decision in around 8 out of 10 cases the share of positive decisions has been declining and for the period matching our analysis the rate is around 2 out of 3 cases. By measuring patient access rather than decisions the number of patients for whom a medicine is recommended relative to patients could have been recommended is 1 in 2. Although trends over time are stable there is wide variation by therapy area. For example, all 7 cancer medicines assessed in 2013 were not recommended for use which helps to explain the relatively low M score for this therapy area. The results of this analysis show the NICE recommended level of access for the medicines in the sample. For the NHS to deliver this level of access, there would need to be 100% uptake of NICE guidance. In practice, uptake in the health care system is a by-product of the clinical and commissioning decisions made in the NHS, and it is beyond the scope of the analysis to assess whether this has been achieved. The NICE HTA processes which have produced these decision outcomes are a product of the clinical and cost effectiveness evidence and other factors which have been considered in the TA committees’ deliberations. Our report describes the decisions; the defensibility or otherwise of these decisions is beyond the remit of this report.
Figure 1: Measure M scores for the sample of medicines by TA type 2007-2013