1. Quality of life and Cost-Effectiveness An Interactive Introduction Prof. Jan J. v. Busschbach, Ph.D. Erasmus MC Medical Psychology and Psychotherapy Viersprong Institute for studies on Personality Disorders

2. New cancer therapy SymptomsDrug XDrug Y Survival days 300 400 Days sick of chemotherapy 10 150 Days sick of disease 100 30 TWiST 190 220

3. Time Without Symptoms of disease and subjective Toxic effects of treatment: TWiST  Richard Gelber  statistician  Count …  Days not sick from treatment  Days not sick from disease 3

4. Fit new therapy in fixed budget  50 patients each year (per hospital)  Drug x: 50 x euro 1.750 = euro 87.500  Drug y: 50 x euro 2.000 = euro 100.000  Drug budget for x or y = euro 50.000  Number of patient Drug x: euro 50.000 / 1.750 = 28.5 patients Drug y: euro 50.000 / 2.000 = 25.0 patients  Survival in days Drug x: 28.5 patients x 300 days = 8.550 days Drug y: 25.0 patients x 400 days = 10.000 days  Survival in TWiST Drug x: 28.5 patients x 190 TWiST = 5.415 days Drug y: 25.0 patients x 220 TWiST = 5.500 days

5. TWiST: ignores differences in quality of life  TWiST  Healthy = 1  Sick (dead) = 0  Q-TWiST Quality of life adjusted TWiST  Make intermediate values 1.0; 0.75; 0.50; 0.25; 0.00  How to scale quality of life? 5 0.0 Quality of life 1.0

6. Visual Analogue Scale  Does the scale fit Q-TWIST?  Is 2 days 0.5 = 1 day 1.0? 6 Dead Normal health X ?=?=

7.  Example  Blindness  Time trade-off value is 0.5  Life span = 80 years  0.5 x 80 = 40 QALYs Quality Adjusted Life Years (QALY) 7 0.00 1.00 X Life years 40 80 0.5 x 80 = 40 QALYs

8. Time Trade-Off  Wheelchair  With a life expectancy: 50 years  How many years would you trade-off for a cure?  Max. trade-off: 10 years  QALY(wheel) = QALY(healthy)  Y * V(wheel) = Y * V(healthy)  50 V(wheel) = 40 * 1.00  V(wheel) = 0.80 8

9. QALY  Count life years  Value (V) quality of life (Q)  V(Q) = [0..1] 1 = Healthy 0 = Dead  One dimension  Adjusted life years (Y) for value quality of life  QALY = Y * V(Q) Y: numbers of life years Q: health state V(Q): the value of health state Q  Also called “utility analysis”

10. Q-TWiST = QALY  Several initiatives early seventies  Epidemiologist and health economists  Part of QALY concept  Quality Adjusted Life Years  QALY = Q-TWiST 10

11. Area under the curve

12.  A new wheelchair for elderly (iBOT)  Special post natal care Which health care program is the most cost-effective?

13. www.ibotnow.com 13 SegwayDean Kamen

14.  A new wheelchair for elderly (iBOT)  Increases quality of life = 0.1  10 years benefit  Extra costs: $ 3,000 per life year  QALY = Y x V(Q) = 10 x 0.1 = 1 QALY  Costs are 10 x $3,000 = $30,000  Cost/QALY = 30,000/QALY  Special post natal care  Quality of life = 0.8  35 year  Costs are $250,000  QALY = 35 x 0.8 = 28 QALY  Cost/QALY = 8,929/QALY Which health care program is the most cost-effective?

15. QALY league table

16. 6000 Citations in 2009 16

17. Orphan drugs  Pompe disease  Classical form: € 300.000 – 900.000 per QALY  Non classical form: up to € 15.000.000 per QALY  If maximum = € 80.000 Ration is almost 1:200  Low cost effectiveness but…  High burden  Low prevalence  Little own influence on disease  High consensus in the field Coalition patient, industry, doctors and media Low perceived incertainty 17

18. Light version cost effectiveness  Formal cost effectiveness is expensive  Is there a light version?

19. What do we have?  Costs  Patient count  Costs per Patient  DBC / DOT  Cost per DBC  TWiST  Costs per Time without psychosis  Costs per Time in normal health  Cost per Recovered patient  Routine Outcome Monitoring (ROM)  Could be of help here

20. Routine Outcome Monitoring  ROM has the potential of  Cost per ‘outcome’ ratio  Difficulties getting data at end of treatment 20

21. Cost effectiveness  Cost benefit  Benefit in monetary terms minus cost  Can seldom be done in health care What is the value of a life year  Cost per QALY  Cost utility analysis  Makes comparisons possible between diseases  Cost per effect  Cost effectiveness  Like: Cost per cure  Stays within one disease

22. Improve cost effectiveness  Other ways to improve cost effectiveness  Insight in costs  Stop rules 22

23. Costs often unknown…  Cost price therapy is mostly unknown in metal health  No insight in costs of components therapy  Typically salary + fixed overhead (for instance 37%)

24. Activity Based Costing can help 24

25. Insights in costs will allow for…  Informal cost effectiveness analysis  Which therapy is most cost effective?  Assumes that outcomes / patients are sufficient comparable  Effects  Cost per ‘cure’  Cost per increase on a specific scale  Cost per DBC 25

26. Weighting components  Which components of therapy contribute most to the cost price?  Does this ranking relates to the indented effects?  Benchmark 26

27. Stop rules  We seem to know when a therapy is needed  But do we know when to stop?  If all the ‘potential’ of the patient is reached?

28. Within social health insurance  Reasonable stop rules might be:  When no progress is made anymore  When the patient is comparable with the general population > 5 – 10% 28

29. Monitor the patient  ….frequently during therapy  Looks like Routine Outcome Measure  but with a high frequency 29

30. Position patients versus normal population 30

31. Monitoring reduces the number of treatments  Michael Lambert  N = 400  Kim de Jong et al in press  Erasmus MC

32. …and gives better results 32 Feed back Non feed back

33. Conclusion  Holy grail  Formal cost effectiveness analysis (CEA)  Costs per QALY  Holy grail might be too expensive  Formal cost effectiveness is indeed expensive  Informal CEA might already reveal much  Cost per treatment  Cost per successful treatment  There is a need for real cost prices  Especially price of components  To start bench mark procedure