Date post: | 12-Apr-2017 |
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DESIGNING A PEDIATRIC ECONOMIC EVALUATION
The Clinical Problem:Extremely Low Birth Weight (EBLW)Newborns <1000 g (0.4% of Canadian births, 9% of NICU
admissions)ELBW infants become anemic due to frequent blood sampling
and an immature hematopoietic system, leading to need for transfusion of allogeneic red blood cells (RBCs) in at least 94%
Previous RBC transfusion guidelines recommended the maintenance of hemoglobin of ELBW infants at “physiologic” levels, not well supported
Risks and benefits of RBC transfusions to ELBW infants: Limiting RBC transfusions may reduce transfusion-associated infection and iron overload, but the resulting low hemoglobin levels may result in morbidities associated with chronic anemic hypoxemia
Study in adults reported no benefit in survival when comparing liberal transfusions to maintain Hb between 100-120 g/L to a more restrictive strategy maintaining between 70-90 g/L
Could the same apply to newborns?
An RCT was performed to evaluate and compare the two management strategies
Premature Infants in Need of Transfusion (PINT) Trial ELBW infants <1000 g, gestational age <31 wks, <48 hrs
oldRandomized parallel 10-centre unblinded trial comparing
low or high hemoglobin thresholds for transfusion Actual threshold depended on need for respiratory
support and infant’s age (in days)Higher threshold results in more transfusions with less
risk of anemia, but potentially higher costs and more AEsFollow-up period 18-21 months corrected gestational ageLow threshold group n=223, high threshold group n =228
Kirpalani H, Whyte RK, Andersen C, et al. The Premature Infants in Need of Transfusion (PINT) Study: a randomized, controlled trial of a restrictive (low) versus liberal (high) transfusion threshold for extremely low birth weight infants. J Pediatr 2006;149:301–7.
How would you go about planning an economic evaluation alongside this clinical trial?
What sorts of things do you need to think about in the planning phases?
What about during execution?And after the trial?
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Study PurposeTarget audience
• Clinical practitioners• Health care institutions• Provincial ministries of health• Third party payers
Research question• Interventions being compared• Outcome (specify type of health consequence or benefit)• Analytic technique
PINT Economic Evaluation Research QuestionTo measure the incremental cost of high Hb threshold
transfusion vs. standard care (low Hb threshold) per unit of benefit gained
Primary effectiveness measure from RCT:• infants surviving without neurodevelopmental impairment (NDI)• NDI = composite measure defined as presence of at least one of:
• cognitive impairment defined as an MDI <70• Cerebral palsy• visual impairment with corrected visual acuity <20/200 in the better eye• hearing impairment requiring amplification or cochlear implant
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Study PerspectivesSOCIETY
• time losses from work, other• restricted days• premature death
PUBLIC HEALTH CARE SYSTEM
HOSPITAL• inpatient care• Procedures• overhead
DRUG FORMULARY• medications
PROVIDER SERVICES• outpatient visits• tests & procedures• allied/complementary
PRIVATE PAYER• medications• devices
PATIENT • travel• medications• co-payments• uninsured care
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
The Interventions
The Interventions
Kirpalani et al. The premature infants in need of transfusion (pint) study: a randomized, controlled trial of a restrictive (low) versus liberal (high) transfusion threshold for extremely low birth weight infants. J Pediatr 2006;149:301-7.
Cost-Effectiveness Plane
More EffectiveLess Effective
More Costly
Less Costly
Compared to standard care, the intervention is:
I
IIIII
IV
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Costing StepsItem identification
• payer perspective• time horizon
Item measurementCost valuation
Item Identification - Cost CategoriesDirect costs
• Intervention costs• Health care services and resources• Patient (family) costs
Productivity (indirect) costs• Patient• Other family members/informal caregivers
Direct - Health Care ResourcesWhat types of health care resources are related to ELBW blood transfusion?
• transfusion• inpatient care (ward costs, nursing services, physician services)• inpatient procedures (ventilation support, Hb tests, other lab tests)• surgical procedures, surgeon services• prescription medications• emergency room visits (tests, assessments)• ambulance services• readmissions• outpatient physician visits, scheduled and urgent• laboratory tests• home care (nursing, ventilation, oxygen)• devices
Direct - Patient CostsWhat types of health care costs do patients (families) have to pay related to ELBW blood transfusion?
• medication co-payments• pharmacy dispensing fees• uninsured services (special tests)• private nurses, home care• devices• complementary health professionals• complementary medicines• transportation to access care, accommodation
• How these are categorized depends on the payer perspective - some may be covered by public or private health plans
Productivity (Indirect) CostsTime Losses
• absence from paid labour• absence from unpaid labour• restricted days (presenteeism)• caregiver time• travel time to access care• waiting time• lost leisure time• long-term disability• premature death
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
OutcomesClinically meaningful
• To clinicians• To decision makers
Time horizon• How might this change the outcomes selected?
OutcomesPrimary effectiveness measure from RCT: infants
surviving without neurodevelopmental impairment (NDI), defined as presence of at least one of:• cognitive impairment defined as an MDI <70• Cerebral palsy• visual impairment with corrected visual acuity <20/200 in the better
eye• hearing impairment requiring amplification or cochlear implant
Immediate outcomes• survival without severe retinopathy, bronchopulmonary dysplasia,
or brain injury by head ultrasound
Time HorizonHow long an interval is needed to capture all relevant costs and consequences?
• Health care costs related to patient illness • short-term disability• long-term disability
• Health care costs related to treatment of adverse events and complications
• Disease fluctuation• Latent effects of disease or treatment• Trade-off between trial feasibility and cost, and need for results to
inform decision-making• Extension through modeling
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Analytic ApproachCost-Effectiveness Analysis
Cost-Utility Analysis
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Data sourcesClinical trial data case report formsChartsLinkage with administrative databases
Economic Evaluation Step-by-Step1. State the purpose2. Determine the study's perspective3. Describe the relevant alternative interventions4. Identify, and describe measurement and valuation of all
relevant costs5. Determine appropriate outcomes6. Choose analytic approach7. Identify data sources and methods for analysis8. Analytical considerations9. Interpretation and dissemination
Analytical ConsiderationsDiscounting future costs and benefitsTime horizon dependentSensitivity analysis plan
• What variables would you focus on?
Generalizability
ConclusionsChild health economic evaluation must:
• Be policy relevant• Respond to needs of health care providers making
decisions for individual patients and those of decision-makers allocating budgets
• Consider gaps in methods:• Availability and validity of outcome measures• Ability to model costs and outcomes over the lifetime• Integration of family preferences• Consideration of multi-sectoral costs and consequences
PEDE databasehttp://pede.ccb.sickkids.ca/pede
To learn more …
http://www.sickkids.ca/research/TASK/
Ontario Child Health Support Unitwww.ochsu.ca