Pharmacoeconomics & Health Outcomes

Guidelines for Assessing

Pharmacoeconomic
Studies

Leon E. Cosler, R.Ph., Ph.D.

Associate Professor of Pharmacoeconomics
Albany College of Pharmacy
Road Map

1. Why do we need guidelines?

2. Discuss guidelines for P-eco literature

3. Summarize what’s important

• Where’s he getting this? Text Ch. 16
Why are “guidelines” necessary?

1. Pharmacoeconomics is ‘new’
- guidelines can help build confidence

2. Having “no rules” invites problems

- desired results achieved by poor methods

3. No “regulatory” organization (yet…)

- FDA (most likely, but not yet…)
Why are “guidelines” necessary?

4. Without guidelines, no basis to evaluate

- studies often have opposing conclusions

5. “Plug in your own numbers”

- bad design yields bad results

6. There are too many choices

- methodologies, alternatives, assumptions
- some (industry) folks worried about this
Other Demands for Guidelines:

• Dramatic rise in the number of studies

• Increasing importance of $$$ decisions

• Study quality (to date) has been poor:

- many terms are misused
- some studies biased / misleading
- important criteria lacking
Evaluation important for:

• Government(s)
- maximize use of limited resources
• Pharmaceutical Industry
- prove products value
• MCOs
- manage costs in a competitive market
• Providers
- obligated to maximize patient outcomes
An APPLIED Example:

Prevalence of Smoking Abstinence

Economic Aspects to evaluate

• Measuring costs and outcomes

- ‘bang for the buck’ is new
• Types of PE analyses
- advantages / disadvantages not well known
• Perspective
- so important… so poorly understood
• A fair comparator
- test new treatment versus gold standard
Aspects to evaluate (continued)

• Sensitivity Analyses
- important to include, bias is still possible
• Costs
- what types? what’s included and why?
• Outcomes
- must be appropriate & measurable
• Simulation modeling
- fast, but potential for bias
- assumptions based on published literature
Aspects to evaluate (continued)

• Time Horizon & Discounting

- appropriate?

• Disclosure
- investigators & funding sources

• Generalizability
- requires transparency
‘Checklists’ for PE Literature Evaluation

• BMJ first to create guidelines for publishing

• Checklist created

• Manuscripts evaluated in 3 general areas:

• study design
• data collection
• analysis & interpretation of results

- 35 items described & score

Guidelines for C/E Studies

• 1993 Panel on Cost Effectiveness:

• resulting in 1996 JAMA manuscript(s)
1. limits of C/E and the ‘reference case’
2. components in the C/E ratio
3. measuring costs
4. valuing consequences in the denominator
5. estimating effectiveness of the intervention
6. time preference and discounting
7. handling uncertainty
8. reporting guidelines
Guidelines for Pharmacoeconomic Research

• “Task Force on Principles for Economic

Analysis of Health Care Technology”
• conducted by Leonard Davis Institute
• members from industry, gov’t, & academia

- 2 sets of guidelines:
• researcher independence
• reporting economic outcomes research

- published in 1995 (Annals Internal Medicine)

PhRMA guidelines (1995)

• 7 key areas defined:

• research design
• protocol objectives
• report contents
• costs / resources
• effectiveness / benefits
• data sources
• extrapolation of results
Common Criteria Themes:

1. specific, well-defined problem statement

2. comprehensive list of alternatives considered
3. study perspective clearly stated
4. comprehensive list of costs & benefits stated
5. discounting if and when appropriate
6. appropriate sensitivity analysis
7. marginal / incremental ratios
8. comprehensive presentation of results
Using Guidelines to Evaluate Literature

• Why so many “proposed” guidelines?

- no standards
- no government regulatory authority (yet)
- organizations have different goals in mind…
- survey reveal several “controversial” areas:
• role of simulation models
• inclusion of indirect costs and benefits
• choice of discount rate
• method of measuring “utility”
How well are we doing?

• Key findings
- Weakest areas:
• stating assumptions
• perspective
• discounting
• ethical considerations
• sensitivity analysis

- Rx Journals weaker than medical

How well are we doing?
Criterion Percent of Studies
Meeting This
Criterion

Incremental ratios 2%
Appropriate discounting 3%
Sensitvity Analysis 6%
All issues of concern addressed 20%
All important costs & outcomes 31%
Well defined question 48%
Evidence of Effectiveness 49%
Costs and Outcomes valued credibly 54%
Comprehensive list of alternatives 71%
Costs and Outcomes valued accurately 85%

Total N = 65 (1985 - 1990)

2002 – we still have a long way to go

• Rx submissions to Australian gov’t

- 67% had major methodological problems
• 28.5% of these problems related to model or clinical assumptions
- 9 cases had serious calculation errors
That’s all for today!