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

New cancer therapy SymptomsDrug XDrug Y Survival days Days sick of chemotherapy Days sick of disease TWiST

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

Fit new therapy in fixed budget  50 patients each year (per hospital)  Drug x: 50 x euro = euro  Drug y: 50 x euro = euro  Drug budget for x or y = euro  Number of patient Drug x: euro / = 28.5 patients Drug y: euro / = 25.0 patients  Survival in days Drug x: 28.5 patients x 300 days = days Drug y: 25.0 patients x 400 days = days  Survival in TWiST Drug x: 28.5 patients x 190 TWiST = days Drug y: 25.0 patients x 220 TWiST = days

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? Quality of life 1.0

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

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

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) =

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”

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

Area under the curve

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

13 SegwayDean Kamen

 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?

QALY league table

6000 Citations in

Orphan drugs  Pompe disease  Classical form: € – per QALY  Non classical form: up to € per QALY  If maximum = € 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

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

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

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

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

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

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%)

Activity Based Costing can help 24

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

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

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?

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

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

Position patients versus normal population 30

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

…and gives better results 32 Feed back Non feed back

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