Pharmacoeconomics 2008; 26 (9): 725-727EDITORIAL 1170-7690/08/0099-0725/$48.00/0

© 2008 Adis Data Information BV. All rights reserved.

NICE Guide to the Methods ofTechnology AppraisalPharmaceutical Industry Perspective

Julia Earnshaw1 and Gavin Lewis2

1 GlaxoSmithKline, Uxbridge, Middlesex, UK2 Roche Products, Welwyn Garden City, Hertfordshire, UK

With rising demands on healthcare expenditure, The briefing papers (as reproduced in this is-the UK NHS is increasingly looking to consider sue[2-7]) for the original workshops were preparedvalue when deciding which medicines to use. The and presented by leading academics and were thenNational Institute for Health and Clinical Excellence subject to challenge and debate via the workshops(NICE) is the key body in England responsible for with their broader representation. The write-up ofthe appraisal of medical interventions on behalf of the workshops by NICE technical teams were help-the NHS and its patients. Thus, when considering ful to enable the Steering Group to discuss thepatients’ access to new medicines, the principles and balance between methodological excellence andapplication of NICE’s Guide to the Methods of practical decision making.Technology Appraisal[1] remain highly influential. Two major elements of the guide were not sub-One of the challenges and key considerations in ject to the same process of comprehensive stake-updating this guide was producing guidance that holder discussion in advance of the Steering Groupbalanced quality and robustness with both the need consideration. While a briefing paper was producedof the NHS for guidance as early as possible and the concerning the level and use of a cost-effectivenessneed of patients for access to new technologies. threshold, it was not discussed in a workshop. Con-Ensuring that the recommended methods were ap- sensus was reached during the Steering Group dis-propriate in terms of reflecting the value of new cussion not to change the threshold. However, themedicines to patients was also important to ensure industry remains concerned that an area withthat the NHS obtains value for money while meeting profound implications for patients’ access to medi-the clinical needs of patients and that the industry cines in the UK was subject to relatively limitedreceives a reasonable return on its investment. debate as a part of changes to a methods guide. We

believe any future consideration on the level of the1. Reflections on the Review Process threshold should be as part of an independent review

and not be addressed in the review of NICE meth-The importance of the review was reflected in theods. A different approach was also taken on NICE’sthorough process of briefing documents,[2-7] work-Social Value Judgements document,[8] which wasshops, consultation and a dedicated Steering Groupsubject to separate consultation. The Steeringto generate the updated guide, and was welcomed byGroup’s remit in this case was limited to consideringthe industry. The authors of this editorial were in-how this proposed document impacted on the meth-volved as the industry member of the Steeringods rather than the document itself.Group (J. Earnshaw) and as a deputy for the Steering

Group and attendee at topic-specific workshops Finally, by definition, the methods review did not(G. Lewis). deal with any issues arising from the processes that

726 Earnshaw & Lewis

NICE uses for their appraisals. These are to be cal issues. While recognizing these challenges, wereviewed in a separate consultation during 2008–9 believe it is critical to develop this approach furtherand so process issues are not discussed further in this to ensure that the NHS continues to prioritize thoseeditorial. activities that are valued most highly by patients and

the public.2. Reflections on the Revised Methods The updated guide provides greater clarity in the

approach to the appropriate consideration of uncer-It is difficult to draw a balance between providing

tainty in terms of structure, data and parameters.clear principles on appropriate methods and the need

While recognizing that exploring uncertainty is anfor pragmatism and flexibility. We believe that the

important aspect of economic modelling, the rangeupdated guide generally achieves this balance, pro-

of analyses proposed may prove problematic –viding a vision of the necessary standard of methods

particularly in the context of the single technologyvia the reference case but retaining some degree ofappraisal timeline. It is also critical that an appropri-flexibility, for example, allowing alternative sourcesate and consistent approach is taken to consideringfor costing where application of NHS referenceuncertainty in decision making. The update guide[1]

costs does not result in clear face validity.explicitly states that “… the Committee will be more

The QALY still represents the primary measure-cautious about recommending a technology when

ment of health gain, and therefore the approachthey are less certain about the [incremental cost-

taken to generate QALY estimates forms a criticaleffectiveness ratios] ICERs presented.” Analysis of

aspect of the guide. A significant modification inuncertainty should therefore be focussed on the

approach relates to the values attributed to differentmore plausible circumstances, rather than identify-

states of health-related quality of life. Previously,ing the best or worst cases, which may in turn create

both time trade-off and standard gamble techniquesan exaggerated impression of uncertainty that does

were permitted as part of the reference case. Thenot reflect the most likely outcomes.

updated guide restricts the choice of health valuationAn area in which the guide appears to have takento time trade-off only. While this may improve

a more pragmatic approach is the identification ofconsistency of judgement, it may exclude a signif-relevant comparators. It recommends using theicant proportion of the available literature on utilityscoping meeting to identify the most likely compa-scores. In contrast, there is welcome flexibility inrators, with consideration given to routine and bestthe methods of eliciting utility values, often one ofNHS practice (including existing NICE guidance).the largest practical challenges for UK manufactur-We welcome this, as it will enable the evidenceers to address as part of evidence generation. Asynthesis to focus on the most relevant comparators,reference case based on the EQ-5D may not beand not complicate the analysis by including anpossible to generate within the relevant phase IIIexhaustive list that may be rarely used.randomized controlled trials, and the guide also

recognizes that it may not be “an appropriate mea- A final area that provided a major area of debatesure in all circumstances.”[1] The guide helpfully was the use of mixed treatment comparisons. Al-allows for alternative health-state elicitation meth- though these techniques have the potential to beods, although a high level of evidence is required to useful in decision making, they remain controver-support such alternatives (see section 5.4.9 of the sial, and the level and extent of the capability withinguide[1]). the UK health technology appraisal community to

perform and interpret such analyses may restrict theUncertainty still exists for manufacturers as topracticality of this approach, particularly in the shorthow utilities will be interpreted by the Appraisalterm. The current guide recommends targeting theseCommittee. The current guide also does not permitto particular circumstances outside the referenceweighting of QALYs according to characteristics ofcase, which we believe to be a sensible approach.the individual because of unresolved methodologi-

© 2008 Adis Data Information BV. All rights reserved. Pharmacoeconomics 2008; 26 (9)

Editorial 727

Chapter 6 of the updated guide outlines NICE’s 3. Conclusionsapproach to appraisal and decision making, and iscritical in clarifying how the Appraisal Committee The updated guide has achieved a reasonableinterprets the evidence presented and how the judge- balance between new approaches and what can bements are made on what constitutes value for the practically achieved and utilized to inform decisionNHS in England and Wales. The guide states that making. The need to ensure appropriate expertiseNICE does not make decisions purely on the basis of and capacity across all stakeholders, including thecost per QALY gained and outlines the range of Appraisal Committee, is vital. Certain issues need tofactors to be taken into account. They state that there be considered beyond the methods review. Theseis no formal threshold, but highlight the judgements include consideration of society’s (and hence theto be made by the Appraisal Committee based on the NHS’s) willingness to pay for health and beingmost plausible ICER – only recommending a tech- clearer on broader definitions of value, includingnology with an ICER above £30 000 when an “in- how innovation is rewarded, to ensure that patientscreasingly strong” case is made regarding the confi- continue to benefit from new medicines within thedence in the estimate of cost effectiveness, the ex- NHS.tent to which the benefits are not included in theQALY measurement, and the innovative nature ofthe technology. A range of other factors such as the References

1. National Institute for Health and Clinical Excellence. Guide tosocial value judgements[8] need also to be taken intothe methods of technology appraisal. London: NICE, 2008consideration. We welcome the recognition that the [online]. Available from URL: http://www.nice.org.uk/media/

guiding principles of the NHS are not limited to B52/A7/TAMethodsGuideUpdatedJune2008.pdf [Accessed2008 Jul 30]QALY maximization and that other important fac-

2. Brazier J. Valuing health states for use in cost-effectivenesstors such as equity and process of delivery must also analysis. Pharmacoeconomics 2008; 26 (9): 769-79be considered. However, given the range of factors 3. Claxton K. Exploring uncertainty in cost-effectiveness analysis.

Pharmacoeconomics 2008; 26 (9): 781-98to be taken into account, the industry remains con-4. McCabe C, Claxton K, Culyer AJ. The NICE cost-effectivenesscerned whether these wide range of factors will be

threshold: what it is and what that means. Pharmacoeconomicsadequately considered. It is critical that these factors 2008; 26 (9): 733-44are not marginalized in the decision-making pro- 5. Miners A. Estimating ‘costs’ for cost-effectiveness analysis.

Pharmacoeconomics 2008; 26 (9): 745-51cess, and that they are formally considered and6. Sculpher M. Subgroups and heterogeneity in cost-effectivenessclearly documented in the final guidance.

analysis. Pharmacoeconomics 2008; 26 (9): 799-806Finally, consistency of interpretation and appli- 7. Sutton A, Ades AE, Cooper N, et al, on behalf of the NICE

Decision Support Unit. Use of indirect and mixed treatmentcation of the principles in the guide may become ancomparisons for technology assessment. Pharmacoeconomicsincreasing challenge as the available methods be- 2008; 26 (9): 753-67

come more complex. With the increasing level of 8. National Institute for Health and Clinical Excellence. Socialvalue judgements: principles for the development of NICEsophistication of health technology appraisal meth-guidance. 2nd ed [online]. Available from URL: http://www.ods, the need for all Appraisal Committee members nice.org.uk/media/998/50/SVJ2forpublicconsultation.pdf [Ac-

and other stakeholders to be adequately trained is cessed 2008 Jul 30]

ever more important so that they are not disen-franchised by the highly technical focus of the evi- Correspondence: Julia Earnshaw, GlaxoSmithKline, Stock-dence base. ley Park West, Uxbridge, Middlesex, UB11 1BT, UK.

© 2008 Adis Data Information BV. All rights reserved. Pharmacoeconomics 2008; 26 (9)