Stakeholders
- Patients
- Healthcare providers: physicians, nurses, and other healthcare professionals
- Policymakers and government agencies
- Healthcare payers: public and private payers
- Health economists and researchers
- Pharmaceutical and medical device manufacturers
Cost component
- Direct Medical Costs
- The medicine itself, infusion supplies, services, etc
- Genotyping cost (e.g., APOE)??
- Monitoring: MRI scans to assess ARIA, PET scans to assess amyloid clearance
- Adverse event-associated costs (e.g., hospitalization), potential effects on cormorbidities?
- Indirect Medical Costs
- Costs in specialised patient care or nursing homes
- Costs of training
- Non-Medical Costs
- Lost productivity and wages due to dementia-related absenteeism or early retirement.
- Costs of informal caregiving, including the time and effort spent by family members or friends providing care.
- Transportation costs
Cost reduction
- Direct Medical Costs
- Potential effects on cormorbidities?
- Indirect Medical Costs
- Costs in specialised patient care or nursing homes
- Indirect Non-Medical Costs
- Lost productivity and wages due to dementia-related absenteeism or early retirement.
- Costs of informal caregiving, including the time and effort spent by family members or friends providing care.
Outcomes of interst in cost-effectiveness analyses
- Disease stage changes and survival
- Cognitive and functional abilities
- Quality of life
- Caregiver burden, stress and quality of life
- QALYs
- Changes in behavioral symptoms (e.g., agitation, aggression)
- Hospitalization rates and lengths of stay
- Utilization of healthcare services
- Changes in patients’ ability to engage in daily activities
Potential data sources for cost-effectiveness analyses
- Published data from clinical trials
- Published data on AD natural history, its associated costs
- Observational studies based on registers and cohorts, etc.
Statistical analyses and parameter specifications
- Multistate modeling: need real data
- Microsimulation: need to define the population and rules
- Disease stage and stage-specific health care cost, additional societal cost, utility (quality of life associated with different health states or interventions), mortality
- Time scale and unit
- Transition probability of outcomes and adverse event, their hazard ratios
- Effect-specific QALYs: e.g., 0.8 gains in QALYs per 20% treatment effects??
- Output
- Incremental cost-effectiveness ratios (ICER): difference in cost between two possible interventions (e.g., new intervention vs. standard care), divided by the difference in their effect, usually costs/QALYs
- Value-based prices=(WTP Threshold) or cost-effectiveness threshold \(\times\) (Incremental QALYs); willingess-to-pay (WTP),the maximum amount society is willing to pay for an additional QALY
- Incremental QALYs = QALYs with New Intervention - QALYs with Comparator or Standard Intervention
Challenges to consider in cost-effectiveness analyses
- Methodological challenges
- Extrapolation from RCT results
- Assumptions of treatment in populations not represented in the clinical trials and treatment adherence
- Limited data on factors that may influence physician prescribing behavior
- Account for benefits/harm in terms of comorbidities
- Rely on published data which may be subject to biases
- Quantify non-medical costs, e.g., informal care costs
- Other considerations
- Rapid changes in the field, biomarkers, treatments
- How to determine willingness to pay and cost-effectiveness threshold?
Reference:
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