Evidence-Based Decision Making for Vaccines: The Need for Ethical Grounding

1. 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

2. 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

3. 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

4. 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

5. 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

6. 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

7. 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

8. 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

9. Are vaccines different?Yes http://themostimportantnews.com/archives/the-fda-formally-approves-the-swine-flu-vaccine

10. 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).

11. Uses of EBDM Medical Interventions Vaccines Compare vaccines to interventions Compare vaccination of different groups Encourage utilization No comparisons of Vaccines to alternative prevention Vaccine to vaccine • Compare interventions • Eliminate waste, redundancy, ineffective treatments • Limit utilization • Coverage determinations • Health services research – regional variations • Clinical practice guidelines

12. 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?

13. 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

14. 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

15. 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

16. Vaccines and EBDM: ethical considerations • Autonomy • Vaccine declinations • Social norms • Autonomy – vaccine declinations • Pandemic availability • Communitarianism/personal responsibility - Infection control

17. 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

18. 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

19. Traditional CEA

20. Multiple Dimensions

21. 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

22. Starting points • Transparency • Communication Next step: call to action