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Economic Evaluation Journal Club:
PINT TrialDmitry Dukhovny, MD MPH
Instructor in Pediatrics, Harvard Medical SchoolNeonatologist, Beth Israel Deaconess Medical Center
Journal ClubFebruary 19, 2013
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Conflicts of Interest
I have no conflicts of interest
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Agenda
Why Economic Evaluations?
Brief overview of PINT Trial
PINT EE by Kamholz et al.
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Objectives
1. To understand the key components of an economic evaluation
2. To identify sources to assist with critical appraisal of economic evaluations
3. To be able to critically assess an economic evaluation
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Why do an economic evaluation?
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SPR Workshop: Dollars and SPR Workshop: Dollars and Sense Sense
Framing: Type of Analysis
Costing Cost-minimization Cost-effectiveness Cost-utility Cost-benefit
“Incomplete” Economic Evaluations
“Complete” Economic Evaluations
© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic
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Framing: Study DesignFraming: Study Design
Frankenstein’s Monster?
Vampire of Trials?
O’Brien B. Med Care. 1996;34(12 Suppl):DS99-108
Decision Analysis Randomized Trial
Slide used with permission from J AF Zupancic
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SPR Workshop: Dollars and SPR Workshop: Dollars and Sense Sense
Cost-Effectiveness Study
Cost-Effectiveness = Costs of Treatment A – Costs of Treatment B
Effects of Treatment A – Effects of Treatment B
© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic
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Health Expenditures as a Percentage of Gross Domestic Product (GDP) in Selected OECD Countries, 1960–2009.
Fineberg HV. N Engl J Med 2012;366:1020-1027.
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How can we can Neonatology more efficient?
1. Decrease cost
2. Improve or not change quality
3. Don’t push anything off to someone else
David Cutler, BIDMC Epi confernece 2/7/2013
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Overview of thePINT Trial
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PICOT P ELBW (BW<1,000 g), GA<31 wks, <48 hrs old at enrollment
I transfusion algorithm
C higher vs. lower threshold (depends on DOL, respiratory support and type of sample)
O – Primary: Death before d/c home or survival with BPD, severe ROP (3-5), Brain
injury (PVL, ventriculomegally)– Secondary: Death or CP, Cognitive Delay (MDI<70), severe visual (<20/200 in 1
eye) or hearing impairment (amplification or cochlear implant)
T – Primary: Discharge Home– Secondary: 18 to 21 months’ corrected age
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Figure 1 from Kirpalani et al. J Pediatr 2006;149:301-7.
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Table 2 from Kirpalani et al. J Pediatr 2006;149:301-7.
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Table 1 from Kirpalani et al. J Pediatr 2006;149:301-7.
How were these thresholds determined?
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Figure 2 from Kirpalani et al. J Pediatr 2006;149:301-7.
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Table 5 from Kirpalani et al. J Pediatr 2006;149:301-7.
Primary Outcome
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Table 6 from Kirpalani et al. J Pediatr 2006;149:301-7.
Secondary Outcomes
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Table 3 from Whyte RK, et al. Pediatrics 2009;123:207-13.
Follow Up Trial: n=430/451
If cut off is MDI<85, cognitive delay favors high threshold group: Adjusted OR 1.81 [1.12, 2.93] (p=0.016)
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Conclusions Higher Hgb level resulted in more
transfusions, but little evidence of benefit at:– First discharge home– 18 to 21 months’ corrected age
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If the outcome is equivalent, then how do you decide?
PRO Less transfusions
– Blood product exposure– Medical Errors– Less IVs– COST
CON Trends towards slightly
worse outcomes– NEC/Bowel Perforation– Length of Stay– Death or Impairment at 18-
21 months’ corrected
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Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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Big Picture
RCT: – Generalizability– In what directions do the issues in the trial
bias the result? EE:
– Generalizability– Stakeholder– Policy
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Framing of EE
Type of Study
DesignPerspectiveTime Horizon
Cost-Effectiveness Analysis
Alongside RCTThird Party Payer18-21 months’
CGA
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Costs 2008 Canadian Dollars Discount Rate 3% (a dollar in your hand
right now is worth more then the same dollar in 1 hour or 1 month)
Used case report forms from RCT– Per diem costs (based on respiratory support)
Adjusted for nurse to patient ratio
– Transfusion Costs– Surgery– Physician fees– Re-hospitalization post discharge home
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Table 1 from Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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SPR Workshop: Dollars and SPR Workshop: Dollars and Sense Sense
Types of Resource CostsHealth Care Related Costs
– Direct Medical Costs Variable: Drugs, personnel, tests Fixed: Land, equipment
Non-Health Care Related Costs– Direct Non-Medical Costs
Child care, parking, meals, gym membership
Productivity Costs – “Absenteesim”
Work absence of family or patient due to illness
– “Presenteeism” Decreased productivity of family or patient due to illness
– Employment choices due to condition (eg CP)© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic
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Effectiveness
Survivor without BPD (at 1st discharge home)
Survivor without NDI (at 18-21 months’)
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Trial Author Year
Perspective
Time Horizon
Measure of Effectiveness
Surfactant Rescue
Backhouse
1994
3rd party payer
1 year Survivor w/o impairment
iNO for PPHN
Lorch 2004
Societal Discharge home
Per life year gained; QALY
ECMO Petrou 2006
3rd Party Payer
7 years Per life year gained
TIPP Zupancic 2006
3rd party payer
18 months CGA
Survivor w/o impairment
NO CLD Zupancic 2009
3rd party payer
D/C home Survivor w/o BPD
SOD McBride 2009
3rd party payer
D/C home;1 year of age
Survivor w/o BPD; Chronic Respiratory morbidity averted
ET ROP Kamholz 2009
3rd party payer
9 months Cost per eye with severe visual impairment averted
PINT Kamholz Draft
3rd party payer
18 to 21 months
Survivor w/o impairment
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Analysis
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SPR Workshop: Dollars and SPR Workshop: Dollars and Sense Sense
Cost-Effectiveness Study
Cost-Effectiveness = Costs of Treatment A – Costs of Treatment B
Effects of Treatment A – Effects of Treatment B
© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic© 2012 Dukhovny, Lorch, Profit, Kamholz, Zupancic
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Table 2 from Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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Uncertainty/Sensitivity Analyses
Deterministic Probabilistic
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Supplement Table from Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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iCER Plot
Figure 1 from Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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Cost-Effectiveness Acceptability Curve
Figure 2 from Kamholz et al. Archives of disease in childhood Fetal and neonatal edition 2012;97:F93-8
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Limitations
International Trial, but only used Canadian Costs
Time horizon at 2 years Quality of Life Data Societal Perspective (would taking family
expenses move the estimate in one direction or another?)
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Summary
PINT trial showed similar outcomes at discharge and 18-21 months’ corrected age for different transfusion thresholds– Trends for better in higher threshold group
The cost estimate appears favorable towards the higher threshold group, but a wide confidence interval around it
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Take Home Points
If clinical equivalence between 2 treatment options, must consider a “risk-benefits” calculus
Systematically look at Economic Evaluations just like you do at RCTs
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References for Critical Appraisal of Economic Evaluations
Drummond MF, Jefferson TO. Guidelines for authors and peer reviewers of economic submissions to the BMJ. The BMJ Economic Evaluation Working Party. BMJ 1996;313(7052):275-83.
– British Medical Journal has a checklist that is required to be filled out along with the paper submission:
http://resources.bmj.com/bmj/authors/checklists-forms/health-economics
Ungar WJ, Santos MT. The Pediatric Quality Appraisal Questionnaire: an instrument for evaluation of the pediatric health economics literature. Value Health 2003;6(5):584-94.