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

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

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

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

When is Outcomes Research Conducted?

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

Types of Outcomes Clinical Economic Humanistic

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.

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.

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.

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.

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.

Brainstorm Exercise: What Outcomes Are Important? Asthma Diabetes Influenza

Outcomes Research Study Designs

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

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

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

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?

When are Pharmacoeconomic Studies Conducted?

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)

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. 1996.

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

*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.

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

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

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

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

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

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

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”

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

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

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

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

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”

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

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

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

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

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

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

Key Measures of Survey Quality Reliability Validity

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

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)

Advantages & Disadvantages of Available Survey Methods

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)

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

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