The ISPOR BOOK:
Reliability and Validity of Data Sources for Disease and Health Management:

Section 3:  Patient-Reported Outcomes (PROs) & Other Self-Reported Data
Section Co-Editors: Robin Turpin PhD and Lisa Mucha PhD

Chapter 1: Overview (Robin Turpin, Heidi Waters & Larry Goldberg)

The overview chapter will discuss common data issues of PROs as they relate to health and disease management as well as provide a brief description of each section chapter and an introduction to chapter structure. 

1. Defining PROs; historical context and current uses
2. Impact of response rates on representativeness and quality improvement efforts
3. Recall, social desirability and fear
4. Confidentiality (e.g., HIPPA and other country-specific requirements)
5. Impact of incentives on data quality
6. Statistical checks and balances
7. Introduction to the structure of the section (i.e., criteria – instrument ‘grids’) and a brief description of each chapter in the section. 

Chapter 2: Instrument Design and Validation (Lisa Mucha, Anandi Law and Gergana Zlateva)

This chapter addresses the availability of validated tools, the pros and cons of using validation vs. non-validated tools, and limitations of using PROs – both economic and non-economic.

1. Terminology (PRO: QOL, functional assessments, disease assessments, unstructured interviews, etc.)
2. State of the field (systematic review of published and unpublished literature)
1. Evolution of PROs and nomenclature  - from clinician perspective to patient perspective
2. Framework used to design instruments measuring PROs
   Qualitative initiation
   Focus groups and item generation
   Reduction of items into domains/constructs
   Pilot testing
3. Validation of tools – primary psychometric properties measured – reliability, different types of validity, responsiveness and issues with each
4. Relationship of PRO with clinical and economic outcomes
5. Use of validated and non-validated tools
   Strengths related to historical perspective, i.e. including the patient in feedback, outcomes, explaining noncompliance with therapy
   Limitations of using PROs – validation, cost, length of tools &patient fatigue, survey issues (acquiescence, social desirability), response rate
6. Methods of administration:
   Self-administered vs. interviewer-administered; Pros and cons for both methods
   Paper vs. electronic vs. IVRS vs. telephone interview
   Validation issues

Chapter 3: Psychometric methods and health-related quality of life measurement

(I-Chan Huang and Jane Speight) 
 
The purpose of this chapter is to help researchers to be familiar with the standard psychometric methods, including reliability and validity, in the validation and selection of health-related quality of life (HRQOL) instruments.  Psychometric methods included in the chapter are test-retest reliability, internal consistency, construct validity, convergent/discriminant validity, criterion validity, known-group validity, and responsiveness.  Limitations of the standard psychometric methods in HRQOL researches will be discussed and the use of modern test theory, especially item response theory, to strengthen the standard methods will also be introduced.   

I. Overview

1. Importance of psychometric evidence in instrument validation and selection
2. Basic concept of psychometric methods (classic test theory)
3. Psychometric methods proposed by professional organizations (e.g., FDA, EMEA, EORTC, etc) in the instrument evaluation 
4. Introduce chapter arrangement

II. Reliability

1. Test-retest reliability
2. Internal consistency

III. Validity

1. Construct validity
2. Convergent/discriminant validity
3. Criterion validity 
4. Known-group validity
5. Responsiveness (longitudinal validity)

6. Clinical significance

1. Anchor-based approach
2. Distribution-based approach

IV. Modern test theory

1. Limitations of classic psychometric methods and solutions
2. The use of item response theory (IRT) to develop and validate HRQOL instruments 
3. Typical examples of using modern test theory to strength psychometric properties of HRQOL instruments

V. Conclusions and future directions

Chapter 4: Health-Related Quality of life/Functional Status (I-Chan Huang, Anandi Law, Jayashri Sankaranarayanen)

1. Terminology/Taxonomy
   Health, Quality of Life, HRQOL, functional status, Generic, Disease-specific
   
   
2. Significance of HRQOL/functional status
1. what is the unique contribution of HRQOL/FS alongside clinical measures in clinical and disease management?
2. State of the field (systematic review of published and unpublished literature)
3. Brief historical perspective
4. Conceptual underpinnings of HRQOL
5. A conceptual framework: what are the relationships between functional status, HRQOL, overall QOL, and their associations with biological markers, symptoms, social demographic variables and environmental factors?  
6. Measurement of HRQOL
7. Specific domains of HRQOL and functional status measures and other issues of dimensionality
8. Generic and Dx specific tools
9. Validation of tools - primary psychometric properties measured, change measurement (MCID)
10. Type of HRQOL/functional status measures: health utility (e.g., EQ-5D) vs. health profile (e.g., SF-36) and the associations between both measures
11. Reference to numerous tools available and their applications
    Section on SF-tools
    
    
3. Approaches to standardization
   Cross cultural validation, etc
   
   
4. Other issues unique to HRQoL/FS
   Subjectivity in measurement; needs corroboration/correlation with objective clinical measurement; issues between standardization and customization (per patient); factors might bias HRQOL/FS reports (such as perceived social constraints, social support, social desirability, personality [optimism], etc); phenomena of response shift (or disability paradox); special populations (issues of HRQOL measures in pediatric and elderly populations); mode of administrations (does it make a difference using different modes such as face-to-face, pen-and-pencil, etc); proxy issue (congruence in proxy and patients in what specific domains)
   
   
5. Prescription for future development
   Gaps identified from ii, iii, iv
   
   
6. Practical applications - what's out there in terms of tools
   Will be a LONG list… of generic and Dx specific (I have a template of such an outline in hard copy), which has the name of tool, origin, validation results, strengths and weaknesses; by therapeutic area.

Chapter 5: Work Outcomes - Absenteeism, Presenteeism and Productivity (Lisa Mucha, Jayashri Sankaranarayanan, Sue Jennings)

1. Introduction and background
1. Terminology
1. Difference between absenteeism and losses in presenteeism
2. Overview of emerging importance of absenteeism, and losses in presenteeism (productivity)
3. Stakeholders in improved absenteeism and productivity
4. Brief literature review of absenteeism and productivity
2. Measurement of productivity
5. Data sources
6. Methodologies
7. Measurement challenges
1. Linking absence to disease
2. Assigning the optimal replacement wage
• The role of disease management in facilitating productivity
8. How disease management can help an employee remain working
1. Benefits to employers
2. Benefits to employees
3. Benefits to payers
9. Strengths and areas for improvement
10. Examples of disease management effecting productivity or absenteeism
11. Potential for disease management to impact presenteeism
• Summary
• Appendix – List of instruments and recommendation matrix

Chapter 6: Patient/caregiver/family satisfaction (Ljubica Besker-Ivasovic, Anandi Law, Lisa Mucha)

1. Terminology
   Satisfaction, Caregiver/family
   
   
2. State of the field (systematic review of published and unpublished literature);
1. Brief historical perspective – origins of Patient/Caregiver/Family Satisfaction
2. Emerging importance of satisfaction and its significance in current managed care environment
3. Conceptual underpinnings – Disconfirmation of expectations (marketing)
4. Framework used in health care – Ware et al
5. Psychometric validation – instruments developed, properties tested and results
6. Applications of tools
   
   
3. Prescription for future development;
   Gaps identified from ii.
   
   
4. Approaches to standardization;
   Generic satisfaction (core elements) versus specific to program
   
   
5. Other issues that are unique to satisfaction.
1. Subjectivity in measurement, confounders to satisfaction (treatment satisfaction, quality of care).
2. How are patient/C/F return to clinic or motivation to comply or other parameters related to satisfaction?
3. Relationship between Satisfaction and HRQOL
   
   
6. Guideline on evaluation on when you use a proxy
   
   
7. Available tools