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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

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

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

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

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

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

Analytic ApproachCost-Effectiveness Analysis

Cost-Utility Analysis

Data sourcesClinical trial data case report formsChartsLinkage with administrative databases

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

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