- The document analyzes decisions made by the National Institute for Health and Care Excellence (NICE) in the UK to recommend health technologies "in line with clinical practice". - Between 2007-2014, NICE made 77 such recommendations, mostly for medicines. The implications for patient access are unclear. - The study develops a framework of six criteria used by NICE to make these recommendations. It also calculates a measure of patient access for the 64 medicine recommendations. - On average, the recommendations allowed access for only 35% of potentially eligible patients based on marketing authorization and appraisal scope, indicating they are more restrictive than a fully "recommended" decision.

1. AN ANALYSIS OF NICE ‘RECOMMENDED IN LINE WITH
CLINICAL PRACTICE’ HEALTH TECHNOLOGY
ASSESSMENT DECISIONS
Phill O’Neill1, Amanda Chapman1, Nancy Devlin1, Aurea Duran2
1. Office of Health Economics 2. Pfizer Ltd.
For further information, contact poneill@ohe.org
1. BACKGROUND
The outcome of technology appraisal (TA) decisions by the
National Institute of Health and Care Excellence (NICE) in the
UK are of 4 ‘types’: Recommended (either in line with clinical
practice or in line with marketing authorisation), Optimised,
Only in research and Not Recommended [1]. Between January
2007 and September 2014 NICE made 77 “recommended in
line with clinical practice” (RiLwCP) decisions (64 of which
were for medicines). These are counted as “recommended” in
NICE’s summary statistics of its decisions.
The implications for patient access of this categorisation are not
clear. In order to capture common features of these decisions
we develop a framework for the criteria used to reach decisions,
as reported in guidance documentation. To assess patient
access we use a previously developed method [2] - the ‘M’
score - to calculate the access associated with each RiLwCP
decision.
Acknowledgements
This research was funded and initiated by Pfizer Ltd.
References
[1] NICE (2014) Technology appraisal decisions. NICE Statistics.
Available at: http://www.nice.org.uk/News/NICE-statistics [Accessed 5
October 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 develop a framework to assess the criteria underlying
RiLwCP decisions.
• To assess the level of patient access associated with NICE TA
decisions for medicines that are categorised as RiLwCP.
3. METHODS
• By considering the final recommendations, summaries of
evidence and committee’s considerations, we identified six
emerging themes related to choice of recommendation.
• We calculate a measure of patient access (M: 0=no access,
100=full access) for each of the 64 RiLwCP decisions for
medicines made between January 2007 and September 2014.
M=(p/P)X100, where P is the set of potentially eligible patients
(given the TA scope and license), and p is the number of
patients for whom NICE did recommend the treatment.
4. RESULTS
For many of the RiLwCP decisions assessed we were able to attribute more than one criterion to the decision, and found there to be
some subjectivity in interpreting guidance documentation. The table below provides an example of each criterion.
5. DISCUSSION 6. CONCLUSIONS
• Our analysis provides some context for NICE reported outcomes
of TA decisions, specifically the nature of recommendations ‘in
line with clinical practice’.
• Whilst each appraisal and committee deliberation is different, the
terminology used to describe NICE decision outcomes is
important, as this acts as a reference point for many users of
NICE guidance.
• The use of RiLwCP as a decision outcome is not as transparent
as other classes of NICE decision. In particular, there is
ambiguity in some cases between the criteria for defining an
outcome as RiLwCP and ‘optimised’.
• It is often appropriate for HTA bodies to make recommendations
for products which do not extend to the full marketing
authorisation, for example due to evidence of clinical or cost-
effectiveness in different populations. These decisions involve
optimising treatment.
• According to NICE, a recommendation in line with clinical
practice reflects how the new treatment would fit with current
clinical practice. Whereas criteria 1 – 4 outlined above reflect
clinical practice (to varying degrees of formality), in many cases
we could identify only clinical/cost-effectiveness as the
motivation for limiting the patient population (with respect to
marketing authorisation). Along with the fact that many
decisions were associated with M scores below 50 (i.e.
recommended for less than half the patients for whom it is
licensed), this makes it difficult to distinguish between RiLwCP
and ‘optimised’ recommendations.
Criterion Example
1. Reference to previous NICE TA
Explicitly refers to a previous NICE TA of the same or a related product
TA294 Afilbercept for wet age-related macular degeneration. Afilbercet recommended for use in
patients according to same criteria as previous NICE TA for competitor product, until both can be
appraised in a multiple technology appraisal. This was despite suggestion by clinical experts that
Afilbercept may provide benefits to a wider patient population.
2. Reference to relevant clinical guideline
Recommendations are matched to existing clinical guidelines including
NICE guidelines
TA188 Somatropin for the treatment of growth failure in children. BTS/SIGN guideline is referred
to describe the step-wise approach to treatment that Somatropin use should conform with.
3. Fits within an established pathway of care
Recommendation aligned with a pre-existing patient management
pathway (with no particular ‘guideline’ for reference)
TA223 Treatment of intermittent claudication in people with peripheral arterial disease, where
Naftidrofuryl “represents one part of a wider programme of management”.
4. "Clinical opinion“
Guidance is explicitly influenced by input from clinical experts
TA315 Canagliflozin in combination therapy for treating type 2 diabetes. Only recommended
where sulfonylurea was not appropriate given extensive experience of clinicians with the current
treatment.
5. Clinical/Cost-effectiveness evidence matching
Guidance recommendation is shaped by the clinical and/or cost-
effectiveness evidence for the technology
TA181 Pemetrexed for the first-line treatment of non-small-cell lung cancer. Recommended for
specific histologically-determined subgroup based on clinical effectiveness evidence. Requires a
change in clinical practice to implement.
6. Non-pharmaceutical
All non-medicinal products in our sample that were recommended were done so ‘in line with
clinical practice’.
We were able to calculate the M score for 32 of the 64 RiLwCP decisions; the average M score for those recommendations was 35
(range: 3 – 100); this means that the medicine was recommended for on average 35% of potentially eligible patients (based on license
and TA scope). If we conservatively assume that M was 100 for all cases where M could not be calculated, then the average M would be
67 - still only two-thirds of potentially eligible patients.