Introduction to Outcomes Research

Laura T. Pizzi, PharmD

Office ofHealth Policyand ClinicalOutcomes

Overview

• Economics of the Healthcare Marketplace (Evolution of OR)

• Definitions• When OR is Conducted• Types and Examples of Outcomes• Study Perspectives • Study Designs

Perfect Market:Perfect Market:

Many BuyersMany Buyers

Many SellersMany Sellers

Perfect Market:Perfect Market:

Many BuyersMany Buyers

Many SellersMany Sellers

Fee-for-Service

Fee-for-Product

Economics of the Healthcare Marketplace

Healthcare Market:Healthcare Market:

Many BuyersMany Buyers

Few SellersFew Sellers

““Gatekeepers”Gatekeepers”

Healthcare Market:Healthcare Market:

Many BuyersMany Buyers

Few SellersFew Sellers

““Gatekeepers”Gatekeepers”

Discounted Fee-for-Service

Capitation

With-holds

1.9

0.8

-1.8

5.6

-0.9

-0.1

1.5

3

-1.6

3.2 3.1

2.1

-3.5

0.5

2.3

3.5

-4

-3

-2

-1

0

1

2

3

4

5

6

1995 1996 1997 1998

IndemnityPPOPOSHMO

U.S. Healthcare Cost Increases (%)*1995-1998

*William M. Mercer, Inc. National Survey of Employer-Sponsored Health Plans. 1998.

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.

IDENTIFY KEYDEVELOPME NTOPPORTUNITIES

IDENTIFY KEYDEVELOPME NTOPPORTUNITIES

PROOF OF CONCEPTPROOF OF CONCEPT DETERMINE SAFETY,EFFICACY , VALUE

DETERMINE SAFETY,EFFICACY , VALUE

OBTAINREGULATORY

APPROV AL

OBTAINREGULATORY

APPROV AL

MARKETTHE DRUGMARKET

THE DRUG

MAXIMIZECOMM ERCIALOPPORTUNITY

MAXIMIZECOMM ERCIALOPPORTUNITYPREPARE THE MARKETPREPARE THE MARKET

Market & MedicalNeeds AssessmentMarket & Medical

Needs Assessment

Disease ModelingDisease Modeling Outcomes Research Planning and Implementation/P ricingOutcomes Research Planning and Implementation/P ricing

Advisory Boards (Opinion Leaders, Providers, Payers)Advisory Boards (Opinion Leaders, Providers, Payers) Sales/Field Force TrainingSales/Field Force Training

NP Strategy Development and ImplementationNP Strategy Development and Implementation Phase IV Studies (Clinical & Outcomes)Phase IV Studies (Clinical & Outcomes)

Advertising and Communication Planning and ImplementationAdvertising and Communication Planning and Implementation

Training (S peaker, MSLs)Training (S peaker, MSLs)

Publ ication StrategyPubl ication Strategy

Knowledge ManagementKnowledge Management

Team Structure, Behavior, TrainingTeam Structure, Behavior, Training

Pre-Phase I Phase I Phase IIA Phase IIB Phase IIIA Phase IIIB Phase IV End ofOpportunity

Competi tor StrategiesCompeti tor Strategies

Message MappingMessage Mapping

Disease Management P lanning and ImplementationDisease Management P lanning and Implementation

When is Outcomes Research Conducted?

Study Perspectives

• Patient• Provider

– 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

Past Present Future

Retrospective Studies

Database Analysis

Chart Review

Prospective Studies

Cohort Studies

Randomized, Controlled

Models

Decision Analysis

Cost-Effectiveness

Cost of Illness

Cost-Consequence

Outcomes Research Study Designs

Introduction to Pharmacoeconomics

Laura T. Pizzi, PharmD

Office ofHealth Policyand ClinicalOutcomes

Overview

• “Who” and “When” of Pharmacoeconomics• Key PE Concepts• Forms of Cost• Types and Examples of Pharmaceconomic

Studies• Assignment of Article Reviews

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?

TargetTherapeutic

Areas

ClinicalResearch:

Phase I

Phase II

Phase III

Postmarketing:

Phase IV

Adapted from: Bootman LJ, Townsend RJ, and McGhan WF. Principles ofPharmacoeconomics. Cincinnati, OH: Harvey Whitney Books Company, 1996

Investigational

Seller (Rx Company)

Approved

Buyers (HC Providers)

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.

DirecDirectt

IndirectIndirect

MedicalMedicalNon-MedicalNon-Medical

InpatientInpatient

HospitalizationsHospitalizations

ProceduresProcedures

DrugsDrugs

Laboratory TestsLaboratory Tests

Rehab/SNFs/Nursing Rehab/SNFs/Nursing HomesHomes

OutpatientOutpatient

Office VisitsOffice Visits

ProceduresProcedures

DrugsDrugs

Laboratory TestsLaboratory Tests

CostsCosts

Types of Costs

• Fixed costs

• Variable costs

• Opportunity costs

• Average costs

• Marginal costs

• Incremental costs

Forms of Cost*

*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)

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)

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

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

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 ($)

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

Laura T. Pizzi, PharmD

Office ofHealth Policyand ClinicalOutcomes

Use of Surveys in Health Services

Research

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– 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

Enter Data

Code Data

Analyze Data

Report ResultsReport Results

Write Report

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 MethodsCost Response

RateAmount ofData

Data EntryTime

Mail Low Mod Mod High unlessOCR

Web Low Depends Small Low

Telephone Mod (if IVR);High (if LiveOperator)

High Mod-High High unlessCATI

Live High High High High

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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