1. Comparative Effectiveness: Implications for the Pharmaceutical Sector Health Policy Audioconference February 23, 2009
Dr Marc Berger
Vice-President, Global Health Outcomes Eli Lilly and Company

2. It Comes Down to Basics Payers want value for money…
Pharmaceutical sector wants money for value!
Comparative effectiveness research is necessary but not sufficient to promote these objectives.
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3. The Pharmaceutical Sector can respond by linking Health Outcomes to Product Development Strategies
Develop drugs that deliver better patient outcomes that are valued by payers and providers
Better patient outcomes means embracing Comparative Effectiveness. New treatments will need to be superior to currently available therapy in some dimension of value important to payers, providers, and patients.
Valued by payers and providers means that better outcomes must translate into obtaining good pricing, reimbursement, and access; and more rapid uptake – but are the goalposts continuing to move?
Delivery: The pharmaceutical sector will need to choose either to provide the opportunities for improvements in patient outcomes or to share / take on responsibility for realizing patient outcomes
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4. Delivering Outcomes Adoption + Use  Outcomes
Early
Adopters
Late
Standard of Care?
Time
# of Adopters
Increase in
Clinical Trial
Process Outcomes
Endpoint Outcomes
Consensus
Guideline
Performance
Measure
Reimbursement & Coverage
Quality
Improvement
Adoption
Evidence of relative value over Standards of Care
EBM / HTA Analyses
Guidelines / Recommendations
Patient Access
Reimbursement and Formulary Design
Physician Rx Writing
Use
Patient Compliance & Adherence
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5. How are the goalposts set (Formulary Committee Staff,
for assessing value?
Economic Information
Framing
Key Questions
Rigor Required
Evidence Review
and Synthesis
(Formulary Committee Staff,
EPCs, DERP,
Academic Analysts)
Evidence-Based
Decision Making
(Formulary Committee
of PBM, Private Payer,
States, NICE, etc.)
Budget
Constraints
Studies 1 2
Equity
Evidence
Review
+/- HECON
Models
Precedent
Acceptability 3
Values &
Preferences
Decisions
Teutsch S, Berger M, Evidence Synthesis and Evidence-based Decision Making: Related But Distinct Processes.
Medical Decision Making Sep-Oct 2005, pp
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6. ISSUE: What constitutes adequate evidence?
Harbour, R. et al. BMJ 2001;323:
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7. ISSUE: Evidence Evaluation is an evolving discipline
Country
Who
Evaluates
Stakeholder Input
Transparency
Timeline
Flexible Appeals
Scope
Study Data
Australia - PBAC
Pharma +
Acad
Low but increasing
Moderate
<120 days
Yes Rx
$/QALY
RCTs
Canada - CDR
Low
<180 days No
Germany - IQWiG
Post Launch
Efficiency Frontier 
Scotland - SMC
CEA
Sweden
Adequate
RCTs/
OBS
UK - NICE
Acad +
Pharma
Med/ Rx $/QALY 
US - AHRQ
EPCs NA
Med/Rx
Comp Eff
Systematic Review
*Processes in Australia and Germany have undergone recent reforms that are not yet well tested (Australia) or implemented (Germany). Australia has instituted an independent review option rather than an appeals process. None of these agencies meet the full criteria of Accountability for Reasonableness.
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8. Issue: Medicine is evolving toward Tailored Therapeutics
Therapeutic Area Effectiveness Rate (%)
25%
+ Benefit
- Risk
─ Benefit
+ Risk
Spear et al. TRENDS in Molecular
Medicine Vol. 7 No. 5 May 2001
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9. ISSUE: Data Quality and Quantity
Who is responsible for generating the evidence?
Who is responsible for synthesizing the evidence?
IOM EBM Roundtable Pharma Sector Statement:
Shared public-private responsibility
Requires collaboration of all stakeholders
Requires establishment of methodological and evidence standards
What types of studies are necessary/sufficient for different decisions?
What are good research practices for observational studies?
Requires the development of standards/best practices for communicating evidence to consumers and patients
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10. Comparative Effectiveness Legislation
The mission of any entity conducting CE research should focus on improving the science and practice of medicine in a way that does not impede access to care and diminish patient outcomes for the sake of cost.
The scope of CE research should be broad including evaluations of benefit design. The scope of CE research must extend beyond analyses comparing pharmaceutical treatments. To enhance its credibility, CER requires an environment and processes that are transparent and inclusive of all stakeholders. Conducting CER in multiple settings will promote new, valid, and reliable methodologies and a broad body of research.
Studies limited to comparing drug therapies cannot be expected to solve all the health care cost and quality issues in the U.S. health care system. Because drugs comprise approximately 11% of overall health care spending, the potential payoff for health care reform is greater by also studying other areas of health care, including health delivery systems, benefit designs, and procedures.
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