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Appraising end of life medicines

Consultation 2008: End of life care criteria

1

Summary

1.1 The Institute intends to supplement the advice it gives to its Appraisal Committees when they are asked to appraise life-extending medicines licensed for terminal illnesses affecting small numbers of patients, which, following appraisal, are deemed to have a incremental cost effectiveness ratio in excess of the upper end of the range normally approved by the Appraisal Committees, but which nevertheless offer demonstrable survival benefits over current NHS practice. The Institute's Guide to the Methods of Technology Appraisal recognises that there will be circumstances in which it may be appropriate to recommend the use of treatments with higher incremental cost effectiveness ratios (ICERs). It states (with reference to the Institute's standard appraisal criteria) that:
Above a most plausible ICER of £30,000 per QALY gained, the Committee will need to identify an increasingly stronger case for supporting the technology as an effective use of NHS resources.
The purpose of this supplemental advice is to indicate to the Appraisal Committees the circumstances in which it may be appropriate to recommend the use of life-extending medicines licensed for small populations with terminal illnesses.
1.2 The Institute has always expected its Appraisal Committees to take account of circumstances in which a treatment is being offered and the nature of what it offers to those who might benefit from it. Although it has not explicitly directed the Committees to give special weight to life extending medicines, they have recognised, in the recommendations they have made, the importance of life to those with terminal illnesses. In considering this supplemental advice, the Institute has taken account of the approach the Appraisal Committees have taken in the past in these circumstances, together with the principles in its guide to the use of Social Value Judgements. It has also applied the consideration its Citizens Council has given to defining clinical need, the approach the NHS should take to funding rare conditions and to the way the Institute should take account of the severity of a patient's condition in developing their recommendations.
1.3 The population for which the medicine is indicated and licensed should not normally exceed 7,000, with the intention of including medicines for rarer cancers and other uncommon conditions, and small groups within larger populations. The reason for selecting a small population maximum figure is that it may sometimes be the case that the costs involved in developing medicines for small groups of patients need to be reflected in a higher price, at least for the first indication. This supplemental advice is not intended to cover medicines developed for what are commonly described as ultra orphan conditions involving very small groups of patients. Treatments for these conditions are not referred to NICE for appraisal and are generally dealt by the National Specialist Commissioning Advisory Group.
1.4 To be considered under this supplementary advice, a medicine will need to have been through a single or multiple technology appraisal by NICE where the most plausible point estimate or range for the ICER exceeds the upper end (£30,000) of the range normally considered by the Appraisal Committees to represent a cost effective use of NHS resources.
1.5 Each candidate medicine will need to meet a set of criteria (see section 2). The Appraisal Committee will apply these criteria as an addendum to section 6.2.25 of the Guide to the Methods of Technology Appraisal and will formulate recommendations accordingly.
1.6 At the conclusion of the appraisal process, NICE will issue its guidance, which will set out the Appraisal Committee's conclusions, including the fact that the medicine had been appraised using the criteria set out in section 2.1.
1.7 Subject to agreement with the Department of Health, medicines recommended for use on the basis of the criteria set out in section 2.1, will normally be subject to an appropriately designed programme of evidence development to ensure that the anticipated survival gains are evident when it is used in routine practice. The outcome of this exercise will be evaluated when the guidance is reviewed. The design of the studies will be determined by the Institute and will need to be funded centrally. NICE will be responsible for managing the data collection exercise and assessing and reporting the outcome. Manufacturers will be given access to anonymised data and a summary of the results published.
1.8 The Institute intends to apply the supplementary advice, following consultation, from January 2009. Consultation will run until 10 December.

2

Criteria for appraisal of end of life medicines

2.1 The Institute will amend the advice it gives to its Appraisal Committees to ask them to consider recommending the use of medicines, with an incremental cost effectiveness ratio in excess of £30,000, where all the following criteria are met:
2.1.1 The medicine is indicated, in its licence, for a patient population normally not exceeding 7000 new patients per annum, and;
2.1.2 The medicine is indicated for the treatment of patients with a diagnosis of a terminal illness and who are not, on average, expected to live for more than 24 months, and;
2.1.3 There is sufficient evidence to indicate that the medicine offers a substantial extension to life, compared to current NHS treatment.
2.2 In order to recommend use of a treatment, the Appraisal Committee will need to be satisfied that:
2.2.1 The estimates of the extension to life are robust and can be shown or reasonably inferred from either progression free survival or overall survival (in trials in which cross-over has occurred and been accounted for in the effectiveness review). Life-extension inferred from modelled mortality gains, where the effects of the intervention are only on morbidity will not be sufficient, and;
2.2.2 The assumptions used in the economic modelling should be plausible objective and robust, and;
2.2.3 No alternative treatment with comparable benefits is available through the NHS.
2.3 Higher ICERs will need to be justified by demonstrable increases in extension to life, and a sound case for the impact of innovating for a small patient population.

3

Review of the resulting guidance

3.1 The guidance produced using these criteria will be subject to review in accordance with the Institute's current arrangements. It is proposed that the review should normally take place no later than 2 years after the publication of the original guidance. The review can be either brought forward or delayed, depending on the outcome of the data collection exercise or the availability of other new evidence.
3.2 Medicines approved following the application of the supplemental advice will not necessarily be regarded or accepted as standard comparators for future appraisals of new medicines introduced for the same condition. This will be to ensure that a high ICER does not automatically become the benchmark for the assessment of medicines subsequently introduced for the same indication. Second and subsequent licences for the same product will be considered on their individual merits. The Appraisal Committee will take into account the cumulative population for each product in considering the strength of any case, for justifying a higher ICER, which employs, in whole or part, a small population argument.

4

Implementation and evaluation

4.1 The reason for providing this supplemental advice and the wording of the criteria for appraising end of life medicines will be subject to consultation for 5 weeks. A final version of the supplemental advice will be made available to the Appraisal Committees in time for their January 2009 meetings.
4.2 The Institute wants to ensure that the new criteria achieve their purpose and will, therefore, put arrangements in place for an independent evaluation in due course. This may involve short term methodological research.
4.3 This evaluation will examine the application of the criteria. The Institute would also wish to assure itself that the proposed scheme is consistent with its responsibilities under equalities legislation and its equalities scheme.
4.4 The Institute will design and manage the evaluation, the results of which would be published and used to make modifications to the supplemental advice.

5

Costs

5.1 The principal cost consequences will be the purchase price of the medicines recommended where, under the current arrangements, a positive recommendation may not have been made. Although the Institute cannot predict the cases in which the application of these criteria will result in positive recommendations, based on its current forward work programme, it is likely to be a small number. The other major costs are likely to be any treatment-specific administration associated with the medicines recommended, the costs of the data collection and the proposed evaluation of these new arrangements.
5.2 Although the cost of each data collection exercise will vary from case to case, it would be reasonable to budget for around £100,000 for each medicine approved under the proposed scheme.
5.3 The Institute is unlikely to incur additional costs in applying the supplemental advice and will absorb those that it does. The Institute may, however, wish to seek help, from the Department of Health, with the costs of the formal evaluation referred to in section 4.2.

6

Consultation questions

6.1 The Institute would like to receive comments on the proposal to issue supplemental advice to its Appraisal Committees, in the form of an amendment to its Guide to the Methods of Technology Appraisal.
6.2 The particular matters on which the Institute is seeking a response are as follows:
6.2.1 Your view on the proposition that the Committee should be asked to place additional weight on proven survival benefits in patients with terminal illness and short life expectancy;
6.2.2 Whether the wording in section 2, in particular, is appropriate and will help the Appraisal Committees achieve the objective set out in section 1;
6.2.3 Whether there are valid alternative methods which might be used to achieve the same ends, in the short or the long term.
6.2.4 Other comments
6.3 Responses to consultation should be made by 5.00 pm on Wednesday 10 December 2008.

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.