jth: John starts

1. jth: John starts Health and Cost Data InputsAdvanced Training in Clinical Research DCEA Lecture 4January 27, 2004UCSF Department of Epidemiology and BiostatisticsJohn Hsu, MD, MBA, MSCEKFRI/DOR - jth@dor.kaiser.org

2. Lectures Assessing health outcomes Assessing costs Data inputs

3. Cost-Effectiveness Ratio  Costs (Numerator) _________________________________ • Health (Denominator) The Incremental Cost of Obtaining an Additional Unit of Health From One Decision Compared With Another Decision (How much additional health do we receive for each additional dollar spent?)

4. Today’s Objectives • To Understand the General Issues in Gathering and Presenting Health and Cost Data Inputs • To Understand Data Sources and Synthesis Methods for Health and Cost Inputs • To Understand Common Criticisms Surrounding Health and Cost Data Inputs

5. Structure • General Approach • Health Inputs • Cost Inputs • Presentation • Review / References

6. 1. General Approach • Research Question & Conceptual Model • Model Inputs: • Measures of Health States & Preferences • Measures of Resource Use • Tradeoffs - Refine Model Given Data Availability • Best Estimates and Plausible Ranges - Base Case and Range

7. 1. Goals / Criticisms • Credibility • Comparability • Comparability of Inputs • Comparability of Assumptions • Presentation of Information • Transparency

8. 1. Model - To Clip Or Not To Clip

9. 1. Data Inputs

10. 2. Health Inputs • Health State Outcomes • Relevant Outcome States • Probability Estimates • Health Preferences Weights • Preference Weights for Outcomes • Utilities, QALYs • Population Characteristics • Relevant Population • Disease Prevalence in Population

11. 2. Health Inputs - Steps • List Potential Health Outcome States and Relevant Population • Find Data on States & Probabilities - Start With Comprehensive Literature Search • Find Data on Utilities • Find Data on Population Characteristics

12. 2. Health States Key Questions: • What Are the Potential Clinical Outcome Differences Across the Study Groups? • What Are the Relevant Health States Over Time for the Disease Under Study? • When Do These States Occur, and How Long Do They Last? • What Are the Likely Side Effects or Other Unintended Consequences for Each Group? • What Are Key Outcomes of Interest for Relevant Stakeholders, e.g. Patients, Clinicians, Payers, Employers, Policy Makers, Society As a Whole? • For Which Health States, Are There Credible Estimates? • Are These Estimates Appropriate for Your RQ and for Your Population?

13. 2. Example - Aneurysm Analysis For the aneurysm analysis, health outcomes were estimated from multiple sources: • Aneurysm rupture rates large cohort study • SAH case fatality meta-analysis • SAH disability medium cohort study, meta-analysis • RR mortality with disability small cohort study • Surgical mortality, disability meta-analysis • RR rupture (= 0) expert opinion (informal)

14. 2. Preference Weights - Utilities • Disease-specific Utilities • Generic Utilities Key Questions: • Do Credible Estimates Exist for Your RQ? • Are the Data Appropriate for Your RQ and Your Model? • Whose Perspective Are You Taking? • Disease Specific Ratings • Community Ratings V. Patient Ratings

15. 2. Utilities - Aneurysm Analysis For the aneurysm analysis, utilities were estimated by extrapolation: • Disability studies of similar types of neurological disability • Worry formula calibrated to utility for similar anxiety • Mild symptoms studies of similar type of pain

16. 2. Population Characteristics • Prevalence of the Disease • Competing Risks Key Questions: • What is the Relevant Disease Prevalence? • National, Representative Samples • Disease Surveillance Databases • Integrated Delivery System Databases • Claims Databases • What Competing Risks Exist (Unrelated to RQ)? • Do Credible Estimates Exist? • Are These Estimates Appropriate for Your RQ?

17. 2. Population Characteristics - Aneurysm Analysis • Prevalence of disease - not needed, not a study about screening or an estimate of total societal costs • All-cause mortality - very important because of the low risk of aneurysm rupture and hence the high risk of dying before rupture occurs • Estimated by age and sex from a data base maintained at CDC, available on the internet.

18. 2. Health: How to Find Inputs • Comprehensive Literature Review • Primary Data Collection

19. 2. Literature Review • Availability and Quality of Data • Definitive Random Control Trials (RCTs) • Small RCTs and Quasi-experimental Studies • Large Cohort Studies • Small Cohort Studies • Expert Opinion • Relevance of Data to RQ and to Population • Synthesis of Data • Meta-analysis • Consensus

20. 2. Health - Primary Data • Clinical Trials • Direct Health State Utilities / Preference Weights Assessments • Expert Opinion

21. 2. Commonly Used Health Data Sources • Clinical Trials • CMS/VA Databases • IDS Databases (KP) • Disease Registries • Quality of Well-Being Index (QWB), Health Utilities Index (HUI) - www.healthutilities.com/overview.htm • Disability/Distress Index, EuroQol Instrument

22. 2. Current Recommendations*:US Panel on Cost-effectiveness in Health and Medicine • Provide a Reference Case Analysis • Select Data Inputs From Highest Quality Sources That Are Relevant to the RQ and the Population • Expert Opinion Is Relevant Only When No Other Adequate Data Exists • Use QALYs to Incorporate Morbidity and Mortality Into a Single Measure • Use Community Preferences for Health States • Perform a Sensitivity Analysis on Inputs * See Gold MR et al. for Complete Set of Recommendations

23. 3. Costs Inputs • Direct Costs • Fixed v. Variable Costs • Time Costs • Cost-Benefit Analyses (CBA)

24. 3. Basic Definitions - Costs • Resources • Costs / Forgone Opportunities • Money

25. 3. Costs • Published Estimates • TC = Q * C [Resources * Unit Costs] • Quantity Consumed • Cost per Unit • Discounting • Time Value of Money • Time Preference • Perspectives

26. 3. Direct Costs “Resources that are consumed in the provision of an intervention or in dealing with the side effects or other current and future consequences linked to it.” • Direct Health Care Costs • Diagnostic Tests, Drugs, Durable Medical Equipment • Health Care Personnel Costs • Direct Non-health Care Costs • Transportation Costs • Care-taker Costs, Child Care • Fixed v. Variable Costs

27. 3. Time Costs • Productivity Costs • Morbidity Costs • Mortality Costs • Friction / Transaction Costs

28. 3. Costs: How to Find Inputs • Comprehensive Literature Review • Primary Data Collection

29. 3. Cost Literature Review • Availability and Quality of Data • Meta-analysis of RCTs • RCTs • Quasi-experimental Studies • Cohort Studies • Expert Opinion • Relevance of Data • Comparability of Data

30. 3. Cost Inputs - Primary Data Collection • Existing Data Sources • Charges • Cost-accounting Systems • Micro-cost Estimates / Time-motion Studies

31. 3. Aneurysm Analysis Cost input Value (range) Source Clipping $25,150 (18,000-35,000) Cohort study – cost accounting system Moderate/severe disability $20,000/yr (13,000-30,000) Published estimate SAH hospitalization $47,000 ($33,000-$67,000) Cohort study – cost accounting system Discount rate 3% (0-5) CEA guidelines Time Costs Not Included - Could be based on the time for surgery and recovery. Assuming one month of lost time, at 10 hours/day and $10/hour = $3,000.

32. 3. Commonly Used Cost Data Sources • Cost-accounting Systems • CMS DRG (Inpatient) • Cost-to-Charges: • Medicare Provider and Analysis Review File • Medicare Cost Report File • CMS Fee Schedule / RBRVS (Outpatient) • Average Wholesale Price / Red Book (Drugs)

33. 3. Current Recommendations*:US Panel on Cost-effectiveness in Health and Medicine • Use Societal Perspective for Calculating Resource Units and Costs (Reference/base Case) • Include All Relevant Direct Costs • Exclude Indirect/productivity Costs in General • Include Variable Costs, but Exclude Fixed Costs • Use Marginal / Incremental Costs Rather Than Average Costs * See Gold MR et al. for Complete Set of Recommendations

34. 3. Cost-Benefit Analyses • Economic Value of Health Outcomes • $ v. $ • Estimates • Human Capital Approach • Willingness to Pay

35. 4. Final Product - Aneurysm Analysis

36. 4. Presentation/Transparency* • * Societal Perspective, Funding R01-123456 • Clearly Stated Reference / Base Case • Clearly Stated Data Source, Perspective, and Potential Conflicts of Interest • Sensitivity Analysis Across Range of Values • Clearly Stated Discount Rate

37. 5. Common Criticisms • Input Estimation Not Credible • Analysis Not Comparable • Structure Not Transparent • Perspective • Modeling Assumptions • Discount Rates • Bias • Funding Sources

38. 5. Quality of Literature* • Increasing Number Over Time – 228 Articles Reviewed • 89% Listed Modeling Assumptions • 83% Described the Comparator Intervention • 89% Reported Sensitivity Analyses • 52% Stated Perspective • 54% Provided Model Diagram • 64% Listed Discount Rate • 66% Reported Funding Source • Methods for Reporting Health and Cost Inputs Varied Widely * Neumann PJ et al. The quality of reporting in published cost-utility analyses, 1976-1997. Ann of Intern Med. 2000; 132: 964 – 972.

39. 5. Additional References • US Preventative Services Task Force, Guide to Clinical Preventative Services, 2nd Edition, 1996 • Drummond MF et al. Methods for the economic evaluation of health care programmes. NY: Oxford University Press, 1997. • LeLorier J et al. Discrepancies between meta-analyses and subsequent large randomized, controlled trials. NEJM 1997; 337: 536 - 542. • Light RJ, Pillemer DB. Summing up: the science of reviewing research. Cambridge, MA: Harvard University Press, 1984. • Gold MR et al. Cost-effectiveness in Health and Medicine. NY: Oxford University Press, 1996. • Neumann PJ et al. The quality of reporting in published cost-utility analyses, 1976-1997. Ann of Intern Med. 2000; 132: 964 – 972.