1. Introduction to Outcomes Research
Laura T. Pizzi, PharmD
Office of
Health Policy
and Clinical
Outcomes 1

2. Overview Economics of the Healthcare Marketplace (Evolution of OR)
Definitions
When OR is Conducted
Types and Examples of Outcomes
Study Perspectives
Study Designs 3

3. Economics of the Healthcare Marketplace
Perfect Market:
Many Buyers
Many Sellers
Healthcare Market:
Many Buyers
Few Sellers
“Gatekeepers”
Discounted Fee-for-Service
Capitation
With-holds
Fee-for-Service
Fee-for-Product 5

5. Why is Outcomes Research Important?
Healthcare market is imperfect
Result is need to determine value of healthcare resources
Pharmaceutical Manufacturers
Managed Care Organizations
Pharmacy Benefit Managers (PBMs)
Hospitals/Health Systems
Government Providers (Medicare, Medicaid)
Society

6. Definition
Outcomes Research evaluations are concerned with evaluating the effects of medical intervention on clinical, economic and humanistic measures.

7. When is Outcomes Research Conducted?

8. Study Perspectives Patient Provider Payer Societal Physician
Hospital/Clinic
Payer
Health plan
Employer
Government
Societal

9. Types of Outcomes
Clinical
Economic
Humanistic

10. Clinical Outcomes
Definition: Medical events that occur as a result of disease or treatment*
Examples
Pain Relief
Cure of Infection
Myocardial Infarction
Death
*Bungay KM and Sanchez LA. Types of Economic and Humanistic Outcomes Assessments. In: Pharmacoeconomics and Outcomes: Applications for Patient Care (module 1). Kansas City: American College of Clinical Pharmacy, 1996.

11. Humanistic Outcomes
Definition: Consequences of the disease or treatment on patient functional status or quality of life*
Examples
Health Related Quality of Life (HR-QoL)
Patient Satisfaction
Functional Status
Patient Preferences
*Bungay KM and Sanchez LA. Types of Economic and Humanistic Outcomes Assessments. In: Pharmacoeconomics and Outcomes: Applications for Patient Care (module 1). Kansas City: American College of Clinical Pharmacy, 1996.

12. Patient Satisfaction
Definition: A consumer’s evaluation of the care received that indicates the extent to which their needs and wants are met*
Influenced by patient attitudes, values, and expectations
Typically measured by process and structure variables
Examples of Measurement Instruments
Patient Satisfaction Questionnaire (PSQ)
Press-Gainey Survey
*Johnson JA. Patient Satisfaction. In: Pharmacoeconomics and Outcomes: Applications for Patient Care (module 3). Kansas City: American College of Clinical Pharmacy, 1996.

13. Health-Related Quality of Life
Definition: Those aspects of life that are dominated or significantly influenced by personal health or activities performed to maintain or improve health*
Domains:Physical, mental, social, and general health perceptions
Measurement Instruments
General: l: MOS SF-36, SF-12, SIP, Nottingham Health Profile
Disease-Specific: Diabetes Impact Measurement Scale, Asthma TyPE Specification, WOMAC Osteoarthritis Index
*Bungay KM and Sanchez LA. Types of Economic and Humanistic Outcomes Assessments. In: Pharmacoeconomics and Outcomes: Applications for Patient Care (module 1). Kansas City: American College of Clinical Pharmacy, 1996.

14. Economic Outcomes
Definition: Direct, indirect, and intangible costs compared with the consequences of medical treatment alternatives
Examples
Drug Costs
Office Visits
ER Admissions
Inpatient Length of Stay
Productivity
*Bungay KM and Sanchez LA. Types of Economic and Humanistic Outcomes Assessments. In: Pharmacoeconomics and Outcomes: Applications for Patient Care (module 1). Kansas City: American College of Clinical Pharmacy, 1996.

15. Brainstorm Exercise: What Outcomes Are Important?
Asthma
Diabetes
Influenza

16. Outcomes Research Study Designs

17. Introduction to Pharmacoeconomics
Laura T. Pizzi, PharmD
Office of
Health Policy
and Clinical
Outcomes 1

18. Overview “Who” and “When” of Pharmacoeconomics Key PE Concepts
Forms of Cost
Types and Examples of Pharmaceconomic Studies
Assignment of Article Reviews 3

19. Definition of Pharmacoeconomics
“The description and analysis of the costs of drug therapy to health care systems and society” - Townsend, 1987
Consists of the identification, measurement, and comparison of costs and consequences of pharmaceutical products and services
A micro-science related to Health Economics

20. Questions That May Be Answered Through Pharmacoeconomics
What therapeutic areas should be targeted for drug development?
Should [new drug] be developed by our pharmaceutical company?
Should [new drug] be added to our formulary?
What is the drug’s cost in relation to clinical benefit when QoL is considered?
Should [new drug] be covered by a Medicaid plan?

21. When are Pharmacoeconomic Studies Conducted?

22. Key Pharmacoeconomic Concepts
Discount Rate
Utility
Sensitivity Analysis
Study Perspective
Quality-Adjusted Life Years (QALYs)
Charges vs. Reimbursement Rates vs. Costs
Units (Natural vs. Monetary)

23. Definition of Cost
“The [monetary] value of all goods, services, and other resources that are consumed in the provision of an intervention or related consequences”*
*Luce et al. Estimating costs in cost-effectiveness analysis. In: Gold, et al. Cost-Effectiveness in Health and Medicine

24. Rehab/SNFs/Nursing Homes
Types of Costs
Costs
Direct
Indirect
Non-Medical
Medical
Outpatient
Office Visits
Procedures
Drugs
Laboratory Tests
Inpatient
Hospitalizations
Procedures
Drugs
Laboratory Tests
Rehab/SNFs/Nursing Homes

25. *Earl-Slater, et al. Dis Manage Health Outcomes. 1997;2(2):65-76.
Forms of Cost*
Fixed costs
Variable costs
Opportunity costs
Average costs
Marginal costs
Incremental costs
*Earl-Slater, et al. Dis Manage Health Outcomes. 1997;2(2):65-76.

26. Sources of Data for Direct Medical Costs
Local Costs
Patient bills
Provider-specific claims data
Activity Based Costing
National or Regional Costs
National Hospital Discharge Survey
HCFA
MEPS (NMES)
HCUP

27. Types of Pharmacoeconomic Studies
Cost-of-Illness
Cost-Consequence
Cost-Minimization
Cost-Effectiveness
Decision Analysis
Cost-Benefit
Cost-Utility

28. Cost of Illness
Evaluation of all economic and humanistic resources associated with a given illness/disease
Examples
Cost of asthma
Cost of strokes caused by phenylpropanolamine (PPA) 15

29. Cost-Consequence Analysis
Evaluation of all clinical, economic, and humanistic outcomes associated with an intervention or group of interventions
Example
Review of a new migraine drug for addition to a hospital formulary
Clinical outcomes (HA relieved)
Economic outcomes (direct & indirect costs)
Humanistic outcomes (productivity loss) 16

30. Cost-Minimization Analysis
Used when two or more interventions are considered to have equivalent outcomes
Examples
Flouroquinolone versus SMX/TMP for the treatment of urinary tract infection
Prilosec versus Prevacid for the treatment of duodenal ulcers 17

31. Cost-Effectiveness Analysis
Evaluation of the costs of an intervention in relation to the outcome, where the outcome is expressed in natural units
Examples
Cost of inhaled steroid ($)
Asthma Exacerbation
Prevented
Cost of levofloxacin ($)
Infection Cured 18

32. Decision Analysis
A modeling technique where results of prior research studies and expert consensus are used to project the effects of a medical intervention to the population of use
Also called “Decision Trees”

33. Cost-Benefit Analysis
Evaluation of the costs of an intervention in relation to the outcome, where the outcome is expressed in dollars
Example
Cost of a Heart Drug($)
Reduced Hospital Days ($) 19

34. Cost-Utility Analysis
Evaluation of the cost of an intervention in relation to humanistic-adjusted outcome expressed in natural units
Examples
Cost of Stroke Treatment
QALY after Stroke
Cost of Headache Medication
Sick Days 20

35. Use of Surveys in Health Services Research
Laura T. Pizzi, PharmD
Office of
Health Policy
and Clinical
Outcomes 1

36. Overview Applications of surveys in health services research
Why survey?
Established instruments
Study design
Key measures of survey quality
Survey research process
Administration methods
Developing a quality survey
Potential limitations of surveys

37. Terminology “Survey” = “Instrument” = “Questionnaire”
“Index” or“Scale” usually refers to question(s) that require(s) rating
In clinical trials, investigator surveys may be called “Case Report Forms” or “Encounter Forms”

38. What are the applications of surveys in health services research?
Traditional clinical research
Outcomes research
Clinical
Economic
Humanistic
Satisfaction

39. Why survey? Limitations associated with other data sources
Can be cheaper than other research methods
Marketing objectives
Determine patient satisfaction
Improve medication persistency
Many established scales are available

40. Established Instruments: Some Examples
Quality of Life: SF-12, SF-36
Pain: Visual Analog Scale (VAS)
General Health: Clinical Global Impression (CGI)

41. Important Considerations Regarding Established Instruments
Always search the literature for established instruments before creating your own
Instruments shown to be reliable/valid in a previously-studied population may not be reliable/ valid in your population
Changing the method of administration may effect reliability/validity
“Cutting and Pasting” destroys reliability and validity of established instruments
Some established surveys may require you to obtain permission for use and/or a small usage fee

42. Study Design Time Frame Descriptive versus Analytical Longitudinal
Cross-sectional
Case-control (Group Comparison)
Descriptive versus Analytical

43. Descriptive versus Analytical Studies
Descriptive Studies
Describe characteristics of group(s)
Answer the question “What?”
Comparison group(s) not required
No hypothesis needed
Analytical Studies
Explain characteristic(s) of group(s)
Answer the question “Why?”
Require comparison group(s)
Hypothesis needed

44. Key Measures of Survey Quality
Reliability
Validity

45. Survey Research Process
Develop Questions
Format Survey
Pretest Survey
Administer Survey
Re-administer Survey
Code Data
Report Results
Enter Data
Write Report
Analyze Data

46. Survey Administration Methods
Self-administered
Mail
Web
Interview
Telephone
Traditional
Technology-Enhanced
Random Digit Dialing
Interactive Voice Response
Computer-Assisted Telephone Interview (CATI)
In-Person (Face-to-Face)

47. Advantages & Disadvantages of Available Survey Methods

48. Developing a Quality Survey: “The Devil is in the Details”
Formatting
Question and questionnaire length
Response options
Branching
Introduction / cover letter
Directions for completion
Incentives
Sample (should be representative of target population)

49. Potential Limitations of Surveys
Bias (What kinds?)
Sampling issues
Sample size
Sample characteristics
Response rate
Time (especially for mail surveys)
Uncertain reliability/validity
Not good for demonstrating causality

50. Disclaimer
“This workforce solution was funded by a grant awarded under the Workforce Innovation in Regional Development (WIRED) as implemented by the U.S. Department of Labor’s Employment and Training Administration.  The solution was created by the grantee and does not necessarily reflect the official position of the U.S. Department of Labor.  The Department of Labor makes no guarantees, warranties, or assurances of any kind, express or implied, with respect to such information, including any information on linked sites and including, but not limited to, accuracy of the information or its completeness, timeliness, usefulness, adequacy, continued availability, or ownership.  This solution is copyrighted by the institution that created it. Internal use by an organization and/or personal use by an individual for non-commercial purposes is permissible.  All other uses require the prior authorization of the copyright owner.”