Evidence-Based Decision Making for Vaccines:
The Need for Ethical Grounding
Robert I. Field, JD, MPH, PhD
Drexel University Schools of Law and Public Health
Arthur L. Caplan, PhD
University of Pennsylvania Center for Bioethics
David R. Curry, MS
Center for Vaccine Ethics and Policy
Interest in EBDM and vaccines
• Health reform
• Medical advances in vaccines
• Prospect of disease eradication/herd immunity
• Seeming abundance of vaccines
• Gates Foundation support
• Rising price of vaccines
Ethical/policy grounding for EBDM: purpose
• Rationalize medical priorities
– Systematize trade-offs
• Focus on spending
– Budgets must have limits
• Utilitarianism implied
– Make health care $$ stretch the furthest
– Make limited budgets do the most good
– Reduce wasteful spending on treatments with limited value
Ethical grounding for EBDM: concerns
• Conflict with societal norms
– Beneficence
• Rescue imperative - No cost is too great to save a human life
– Autonomy
• Patients and families should decide what is worth the cost
• Patients lead in demanding treatment
– Moral judgments
• Who is being treated? – children, elderly, prisoners
• What causes the disease
– Random – genetics
– Individual choice – lifestyle, disapproved behavior, lack of caution
– Fear of rationing
Ethical grounding for EBDM: concerns
• Other values left out of the equation
– Justice
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Fair allocation
Optimizing health outcomes vs. treating all patients equally
Small gains for many vs. large gains for a few
Bias toward younger, healthier people
– Communitarianism/personal responsibility
– Nonmaleficence – does EBDM get it right?
• Lurking question
– What is a life worth?
– Who’s perspective – experts, general public, patients
Criticisms of EBDM: logistics
• Is evidence base neutral?
– Conflicts of interest
• Influence of commercial and other interests
– Drug, biotech, device companies
– Private insurers
– Patient advocacy groups
• Biases in interpretation of results
• Biases in study methods
• Arbitrary outcomes
– Surrogate measures
– Survival only
– No quality of life considerations
Criticisms of EBDM
• Is evidence base reliable?
– New treatments – incomplete evidence
– Small number of studies
• No room for values/norms
– Rescue imperative
– Placing a value on human life
– Threshold for cost/QALY
• No account of suffering caused by the condition
• Subtext of cost containment
EBDM does not take account of:
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Risk of the intervention
Economic productivity of patients
Societal consequences of intervention
Pain and suffering caused by the disease
Preventability of the disease by other means
Individual risk of getting the disease
Logistical issues in implementing the intervention
Public perceptions of disease and patient characteristics
Chance of achieving eradication or herd immunity
Equity/justice in allocation
Are vaccines different?
Yes
http://themostimportantnews.com/archives/the-fda-formally-approves-the-swine-flu-vaccine
Public Health Pyramid
Counseling
and Education
Clinical Intervention
Long-Lasting Protective
Interventions
Making Individual Default Decisions Healthy
Socioeconomic Factors
Based on Frieden, T.R., Framework for Public Health Action: The Health Impact Pyramid, AJPH, 100(4):590-95 (2010).
Uses of EBDM
Medical Interventions
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Compare interventions
Eliminate waste,
redundancy, ineffective
treatments
Limit utilization
Coverage
determinations
Health services
research – regional
variations
Clinical practice
guidelines
Vaccines
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Compare vaccines to
interventions
Compare vaccination of
different groups
Encourage utilization
No comparisons of
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Vaccines to alternative
prevention
Vaccine to vaccine
How are vaccines different?
• Intervention is mostly utilitarian
– Focus is on population benefits
– Herd immunity
• Uncertain disease incidence without vaccine
– Undermines EBDM conclusions
– Who would have gotten the disease
• Undermines calculation of QALYs lost
• Beneficiaries are unknown, so beneficence is less direct
– More political pushback
– No rescue imperative
• Trade-offs
– Are there alternative prevention strategies?
– Cost of treatment in $$ and suffering
– How many would have gotten the disease without the vaccine?
How are vaccines different?
• Risk
– Free rider problem
– Disease risk is low in a vaccinated population, so less tolerance for vaccine risk
• Societal norms
– Fear of vaccines
– Autonomy
• Issue is declination, not access
• vaccine declinations
• Physicians lead in recommending treatment
– Resistance to mandates
– Communitarianism/personal responsibility
• Logistics
– Distribution
– Patient compliance
How are vaccines different?
• QALY threshold
– Consensus?
– Is it the same for statistical lives?
– Average value disguises wide individual variation
• Different evidence base
– Epidemiology of condition
– Vaccine effectiveness
• Different comparisons
– Alternative prevention techniques
– Cost/risks of treatment
Vaccines and EBDM: ethical considerations
• Utilitarian concerns
– Population protection/herd immunity
– Cost to the health system
– QALYs
• Justice concerns
– Allocation
– Access
• Beneficence concerns
– Target population – moral dimensions, vulnerability
• Nonmaleficence concerns
– Evidence base
Vaccines and EBDM: ethical considerations
• Autonomy
– Vaccine declinations
• Social norms
– Autonomy – vaccine declinations
– Pandemic availability
– Communitarianism/personal responsibility - Infection control
Vaccines and EBDM: implementation
considerations
• Kinds of evidence needed
• Principles of analysis
• Conflict policy for evidence and analysis
• Criteria for determining sufficiency of evidence
• Accountability for outcomes
Need to Consider Multiple Dimensions
• For example
– Provenge – therapeutic vaccine
• High cost, limited effectiveness, low risk, strong rescue imperative
– Mammograms under 50 – non-vaccine prevention
• Low cost, questionable effectiveness, some risk, strong autonomy concern
– HPV vaccine – vaccine with alternative prevention
• High cost for a vaccine, probable effectiveness, low risk, srong utilitarian value
– Polio vaccine – vaccine with no alternative prevention
• Low cost, high effectiveness, minimal risk, strong utilitarian and beneficence value
– Mosquito Nets – nonmedical prevention
• Very low cost, high effectiveness, minimal risk, strong utilitarian value
Traditional CEA
Multiple Dimensions
Recommendations
• Catalog and consider all relevant dimensions
• Develop technique for systematic comparison
• Avoid collapsing analysis into a single number (like QALYs)
• Clarify value assumptions
• Identify evidence needed
Starting points
• Transparency
• Communication
Next step: call to action