1. How to weight cost effectiveness in appraisal NVTAG / CVZ course: The appraisal process, work in progress 22th of April 2009 Jan van Busschbach

2. 1 Context investigation CVZ  Cost effectiveness is considered in all new reimbursement application  Cost effectiveness is an important aspect in the appraisal  How to implement cost effectiveness in appraisal?

3. Two research questions: 1)What is a “good” and what a “bad” cost effectiveness?  What is the threshold value?  In terms of costs per QALY 2)How does one weight cost effectiveness with other considerations? 2

4. What is the threshold value?  Should there be a threshold value?  If there was one: what is the point in weighting with other arguments?  A threshold provokes strategic behavior  Is there a normative paradigm (theory), that provides such threshold?  The need for a threshold is pragmatic  It helps to chose between good and bad  Its value is historical determined 3

5. Threshold most likely a range  A range like in England and Scotland  £ 20.000 - £ 30.000  But higher values are possible  As defined by RvZ maximum € 80.000 per QALY  In de media € 80.000 seem threshold  But much lower values also possible 4

6. Cost effectiveness in practice  Threshold might stand for average cost effectiveness in practice  Average cost per QALY  Meerding et al, 2007  Cardiovascular diseases: € 2.000 to € 5.000 per QALY  Oncology: €16.000 tot € 18.000 per QALY  In practice:  A range  Averages cost effectiveness is lower than used in most debates about the threshold 5

7. 6 Conclusion research question 1 1.What is a “good” and what a “bad” cost effectiveness?  What is the threshold value?  In terms of costs per QALY  There is no empirically or theoretically fixed value  More likely: a range (of thresholds)  Other variables determine good or bad cost effectiveness

8. A variable threshold  Research question 2  How does one weight cost effectiveness with other considerations?  Same question as:  Is the threshold variable?  If so: which variables have an influence?  For instance:  does disease burden interacts with threshold value?  CvZ models 2001, RvZ model 2006, 2007 7

9. 8 A variable threshold The RvZ model: interaction with burden Burden of Disease Costs per QALY

10. Interaction with Burden  Burden of disease most often discussed  As candidate to alter decision making  To weight cost effectiveness  Know as the equity debate 1) Maximize average population health…  Without looking at burden of disease 2) Focus on the worse of….  Without looking at the average population health  Interaction is intermediate position in debate 9

11. Interaction with burden often suggested 10

12. Methodology issues  How to measure burden?  What should be the form of the curve? 11 Cost per QALY Burden of disease

13. But we do know…  The function is continuously ascending  Burden can be measured  Next presentation: Elly Stolk  We can deduct the curve from research  Population preferences  The appraisal committee 12

14. Next to burden….  Other argument than burden might be also be relevant  Examples are rarity (orphan drugs), budget impact, live style etc.  Some might increase the threshold, some might lower it… 13

15. 14 Increasing or lowering the threshold  Increasing  Burden  Rarity (orphan drugs)  Relates to much informal care  Risks for others  Lowering  Limited relation to domain of health care  High budget impact  High future medical costs  Unsuitable for insurance because of high incidence  Unsuitable for insurance because of autonomy patient

16. Increasing threshold, and critics  Burden  But lower population health…  Rarity (orphan drugs)  Cause of disease becomes more important that burden and effectiveness…  Does not make much sense from epidemiology point of view  Relates to much informal care  Could be include in the CE-ratio…  Risks for others  Could be include in the CE-ratio…

17. Lowering the threshold, and critics  Limited relation to domain of health care  What is the domain of health care...?  High budget impact  Focus on costs, not on cost effectiveness  High future medical costs  Could be include in the CE-ratio…  Unsuitable for insurance because of high incidence  Might cause people to avoid health care  Unsuitable for insurance because of autonomy patient  Might cause people to avoid health care

18. Conclusions  There does not seem to be a fixed threshold  Many factors might alter threshold  Burden of disease is best described  Decisions of the appraisal committee will reveal trade-off between cost effectiveness and other arguments