Professor Ron Akehurst
Dean and Professor of Health Economics, School of Health and Related Research

Professor Akehurst began by explaining that the National Institute for Clinical Excellence (NICE) has three main functions:

• Appraisal of therapeutic technologies;
• Releasing guidelines for their use; and
• Carrying out audits on its effectiveness.

The NICE appraisal process is conducted to assess both the effectiveness and cost-effectiveness of a technology, i.e. a drug, surgery or a form of care, bearing in mind the Secretary of State for Health's policy priorities and taking into account input from patients, healthcare professionals and clinical experts.

The Appraisal Committee does not look for cost savings equal to the cost of a new drug and the Treasury monitors the decisions of NICE for their impact on the total expenditure on health care. Reports often highlight how money may be saved by a new technology and that although new technologies may cost more they are likely to deliver greater health gains. The appraisal guidelines will usually indicate:

• Whether a technology is supported and in which patient groups.
• When treatment should be started.
• When treatment should stop (e.g. when there is no prospect of further benefit).

Decisions about whether to go ahead with a technology are based on the apparent strength of effectiveness, the quality of the evidence supporting that view, and its cost-effectiveness, often measured in cost per quality adjusted life year (QALY). Greater generosity may be afforded in case where no alternative treatments exist, if the proportion of the population over which overheads will be spread is small and if the technology is innovative and has the potential to be very useful.

The appraisal process is highly evidence based and includes systematic reviews of randomised controlled trials and uncontrolled studies and often formal (mathematical) modelling, measuring the impact of technology in terms of other diseases and mortality.

Fundamentally, the appraisal assesses what benefits a technology delivers to patients relative to its cost and ultimately aims to establish which technologies will have the greatest benefit by comparing one state of ill health with another. It is therefore important for patients to communicate how their ill health affects their quality of life. Generic measures of quality of life are most useful, such as the Short-Form 36 (SF-36). This is the most widely used measure of general health and involves the self-completion of a 36-item questionnaire, which creates a profile of an individual across eight health dimensions. Various activities are graded on a scale of 1 to 3, where 1 indicates an individual is limited a lot and 3 not limited at all. Alternative scoring procedures include dimension and summary scores, involving physical and mental component summaries or a preference-based single index.

Comparisons between SF-36 data from the general public and those with rheumatoid arthritis revealed that in nearly all eight health categories the general population felt in better health. SF-36 data also showed the positive effects over time on the health of rheumatoid arthritis patients who had responded to therapy compared to non-responders.

A second generic measure of patients' quality of life is the Euroqol (EQ5D) - a measure developed to produce a single dimensional score using preference weights on health states. This questionnaire is limited to five health dimensions, meaning that it takes only minutes to complete but that it can be less sensitive when used for some conditions. This measure is used to give an idea of the benefits of a therapy, i.e. the number of quality adjusted life years can be determined, and if patients live longer whilst on a therapy their quality of life during those years can be measured.

Professor Akehurst concluded that the implications of the appraisal process for pituitary research were as follows:

• Demonstrating effectiveness of new treatments is essential but evidence of an improved quality of life must also be presented.
• Cost-effectiveness must be demonstrated in ways that allow comparisons to other therapies.
• Modern studies and clinical trials are expected to record evidence on generic quality of life, and without systematic implementation of measures to collect this it will be difficult to achieve funding for new treatments.