1. City University notes AJ Fischer October 2004

2. Nancy’s Questions Q1Different people present material with different cost perspectives? AWith all such conundrums, one hopes the different ways are not material to the decision to be made. If not, NICE muddles through. Wouldn’t you? Proper answer: NICE subjects data to Bayesian analysis, contributions being weighted by beliefs about the relative merits of each component. (Wish it were true!)

3. Nancy’s Questions Q2 What does NICE do when faced with CE ratios using different output measures? AIn terms of cost per QALY, the CE ratio should contain all relevant output measures. If benefits are not in terms of QALYs, the decision becomes difficult. Answers will be made on a case-by-case basis. Depends how clear-cut things are: if very high or very low cost per QALY, the correct decision will not be in doubt. If the case is near the threshold, then there will not be a great efficiency loss if the wrong decision is made.

4. Nancy’s Questions Q3What does NICE do when faced with QALYs measured in two different ways in a particular appraisal? AAgain, one hopes firstly that the decision to be made does not depend on the measurement method. If the method matters, then probably even God doesn’t know the right answer.

5. Nancy’s Naughty Question Q4How would NICE assess the costs and benefits of IVF? AThe costs do not pose much of a problem. The benefits, as ever, are more difficult. How many life years are to be given to a live embryo compared with a dead one or the absence of one? At 39 weeks? At 30 weeks? At 12 weeks? At 3 weeks? Who knows the answer? The Committee will make a decision, but will not really like doing so. They do so in the absence of any scientific or research guidance about what the answer should be. Further, is this about health or about lifestyle? Who should judge? Should the taxpayer pay for lifestyle improvements?

6. Nancy’s Questions Q5How does NICE weigh up “scientific” evidence with patient evidence? AIf patient evidence is “scientific”, it will become part of the scientific evidence. Therefore, what is left is not “scientific” and should be disregarded. Fortunately, that is not the end of the question: (1) we are all humans (2) X-inefficiency

7. Nancy’s Questions Q6How does NICE weigh up RCT evidence with evidence of poorer quality? AIf sufficient RCT evidence is available, then there will be no need to go to other, potentially biased, sources of evidence, such as case series or case studies. If no RCT evidence is available, or is sparse or of poor quality, then it makes sense to look towards other evidence.

8. Nancy’s Questions Q7NICE provides guidance at national level which must be followed locally. What if some local projects have lower cost per QALY? AThis is in part an equity-efficiency trade-off. It is not equitable to have postcode prescribing, so it has been made mandatory to follow NICE guidance. But there are limits. Currently, we just don’t know the worth of projects forgone.

9. Questions Nancy could have asked Q: Does uncertainty about a cost per QALY estimate matter to NICE? A: In a perfectly flexible world, no, it shouldn’t matter. Unlike clinical decisions, which require 95% confidence or more (so that uncertainty about an estimate will be considered), cost effectiveness decisions involve only money, so the risks across many projects can be pooled. In that case, uncertainty plays almost no role, and only the ICER mean matters. However, in a world where a YES decision, once made, cannot easily be reversed, this breaks down. If a YES answer of say £20,000 per QALY is uncertain, and turns out 3 years later to be £50,000 per QALY with near certainty, then the rational response would be to lower the ICER threshold in the first instance.

10. Questions Nancy could have asked Q: Suppose a cancer drug stops the cancer from progressing for 12 months. The trial then stops. It does not go on long enough to show any survival benefit, but there would probably be one. When this is modelled, the drug is not cost effective if zero survival benefit is assumed, but very cost effective if the survival benefit is assumed to be 12 months. What would NICE do? A: Your turn.