Evidence-Based Decision Making for Vaccines:

The Need for Ethical Grounding

Robert I. Field, JD, MPH, PhDDrexel University Schools of Law and Public Health

Arthur L. Caplan, PhDUniversity of Pennsylvania Center for Bioethics

David R. Curry, MSCenter 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

• 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:

• 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

Counselingand 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 EBDMMedical Interventions

• Compare interventions• Eliminate waste,

redundancy, ineffective treatments

• Limit utilization• Coverage

determinations• Health services

research – regional variations

• Clinical practice guidelines

Vaccines

• Compare vaccines to interventions

• Compare vaccination of different groups

• Encourage utilization• No comparisons of

– 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