The objective of the Outcomes Research Guideline Index is to improve state-of-the-art methods in outcomes research and gain acceptance of their use in research and decision-making. The ‘GENERATING HEALTH CARE EVIDENCE » SYNTHESIZING EVIDENCE » USING EVIDENCE IN HEALTH CARE DECISIONS: OUTCOMES RESEARCH GUIDELINE INDEX was developed to: 1) identify current health care evidence methodology guidelines* (see guideline definition below) as well as useful textbooks and review papers; 2) identify health care evidence methodology guideline gaps; and 3) to provide a useful tool to prioritize and develop contentious evidence generation, synthesis and use methods. The ‘Outcomes Research Guideline Index’ is a web-based compendium of methods guidelines (as well as key textbooks and review papers) used in generating health care research evidence, synthesizing evidence, and using evidence in health care decision making. These methods are applicable to all diseases and health disorders and health care interventions.

This compendium is organized as follows:

Generating Health Care Evidence
  1. Quantitative Clinical Outcomes Assessment & Reporting Methods
  2. Qualitative Clinical Outcomes Assessment Methods
  3. Patient Preference Methods
  4. Health Economic Evaluation Methods
  5. Health Care Research Statistical Methods
  6. Social and Ethical Aspects of Health Technologies Research Methods
Synthesizing Health Care Evidence
  1. Evidence Synthesis Research Methods
  2. Modeling/Decision Analysis Methods
  3. Health Economic Evaluation Framework and Methods
  4. Social and Ethical Aspects of Health Technologies Research Methods
Using Evidence in Health Care Decisions
  1. Payer (population-based) Health Care Decision
  2. Individual Health Care Decisions by Clinicians & Patients

What Is HEOR?

In today's world, healthcare decision makers across the globe are often faced with the need to select therapeutic "interventions" from multiple treatment options, including biopharmaceuticals, medical devices, and healthcare services. However, the benefits and costs of these interventions can range dramatically and the benefits can be economic, clinical, both, or may include hard to measure costs or benefits the patient experiences directly. Health economics and outcomes research (HEOR) can help healthcare decision makers—including clinicians, governments, payers, health ministries, patients, and more—to adequately compare and choose among the available options.

ISPOR has defined the following HEOR topics in its taxonomy that are the key areas of focus for the organization and its membership:

*Health Care Evidence Methodology Guideline Definition

A health care evidence methodology guideline is a set of recommendations to improve the consistency and quality of evidence generation, evidence synthesis, and the use of evidence to inform individual and population-based decision making. A guideline is a statement by which to guide a course of action, developed on the basis of current available understanding of the field. Guidelines should be consensus developed by experts in the field; stakeholders are consulted in the guideline development; and a systematic process is used to generate the guideline containing clear, specific and unambiguous recommendations. The guideline is peer-reviewed, published in a reputable journal and available to the public for free. Guidelines provide some level of assurance to users about appropriate actions. By definition, following a guideline is never mandatory. Guidelines are not binding and are not enforced.

Comments:
Your assistance is needed in updating this ‘Outcomes Research Guideline Index’, please email us your comments, suggested guidelines, key textbooks or review articles (full citation and website link). Indicate the method for which these documents are provided.

Generating Health Care Evidence


I. Quantitative Clinical Outcomes Assessment & Reporting Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Randomized Clinical Research & Reporting Methods

Title: CONSORT 2010 Statement: Updated Guidelines for Reporting Parallel Group Randomized Trials
Source: CONSORT 2010 Statement: Updated Guidelines for Reporting Parallel Group Randomized Trials
Comments:

Title: Principles and Practice of Clinical Research by Gallin, J.I.,
Source: San Diego, CA: Academic Press, ed. 2002
Comments:

 

1. Adaptive clinical trials

Title: Standards for Patient-Centered Outcomes Research
Source: http://pcori.org/assets/Methodology-Report-072312.pdf

  
 

Title: PCORI Methodology Standards
Source: http://pcori.org/assets/PCORI-Methodology-Standards1.pdf

  

2. Pragmatic clinical trial/large simple clinical trials

Title: Guidelines for reporting experimental studies
Source: Guidelines for reporting experimental studies
Comments:

  
 

Title: Improving the reporting of pragmatic trials: an extension of the CONSORT statement.
Source: Improving the reporting of pragmatic trials: an extension of the CONSORT statement.
Comments:

  

3. Generalizability of clinical trial information-RCT vs. adaptive/pragmatic trial; geographic generalization

   

4. Validation of surrogate and intermediate endpoints

   

B. Prospective Observational Research & Reporting Methods

Title: Guidelines for reporting observational studies
Source: Guidelines for reporting observational studies
Comments:

Title: Risk Adjustment for Measuring Healthcare Outcomes by Iezzoni, L.I.
Source: 3rd ed. Chicago, IL: Health Administration Press, ed. 2003
Comments:

 

1. Safety/Pharmacovigilance methods

Title: Assessing Harms when Comparing Medical Interventions
Source: Assessing Harms when Comparing Medical Interventions
Comments:

  
 

Title: International Ethical Guidelines for Biomedical Research Involving Human Subjects
Source: Council for International Organizations of Medical Sciences (CIOMS)
Comments:

  

2. Pharmacoepidemiology methods

Title: European Network of Centes for Pharmcoepidemiology and Pharmacovigilance (ENCePP) ENCePP Guide on Methodological Standards in Pharmacoepdiemiolgy
Source: Standards and Guidances
Comments:

Title: Clinical Epidemiology: How to Do Clinical Practice Research by Haynes, R.B.
Source: 3rd ed. Philadelphia, PA: Lippincott Williams & Wilkins, 2006
Comments:

 
 

Title: Guidelines for Good Pharmacoepidemiology Practices (GPP)
Source: Guidelines for Good Pharmacoepidemiology Practices (GPP)
Comments:

Title: Clinical Trials: a Methodologic Perspective by Piantadosi, S.
Source: 2nd ed. New York, NY: John Wiley and Sons, Inc, 2005
Comments:

 
  

Title: Epidemiology by Gordis, L.
Source: 3rd ed. Philadelphia, PA: W.B. Saunders, 2004
Comments:

 
  

Title: Modern Epidemiology by Rothman J.K., Greenland S.
Source: 2nd edition. Philadelphia, PA: Lippincott Williams & Wilkins., 1998
Comments:

 
  

Title: Population Health: Concepts and Methods by Young T.K.
Source: Second edition. New York: Oxford University Press 2005
Comments:

 

3. Disease prevalence / incidence methods

Title: Strobe-Statement
Source: Strobe-Statement
Comments:

  

4. Patient registry methods

Title: Registries for Evaluating Patient Outcomes: A User’s Guide: 2nd Edition
Source: Registries for Evaluating Patient Outcomes: A User’s Guide: 2nd Edition
Comments:

  

5. Surveys

 

Title: Designing and Conducting Health Surveys: A Comprehensive Guide by Aday, L.A., Cornelius, L.J.
Source: 3rd ed. San Francisco, CA: Jossey-Bass Publishers, 2006
Comments:

 
  

Title: Doing Survey Research: a Guide to Quantitative Methods by Nardi, P.M.
Source: 2nd ed. Boston, MA: Pearson/Allyn & Bacon, 2006
Comments:

 
  

Title: Focus Groups: A Practical Guide for Applied Research by Krueger, R.A., Kasey, M.A.
Source: 3rd ed. Thousand Oaks, CA: Sage Publications, 2000
Comments:

 
  

Title: Mail and Internet Surveys - The Tailored Design Method by Dillman, D. A.
Source: 2nd edition. New York: John Wiley & Sons, Inc., 2007
Comments:

 
  

Title: Survey Methodology by Groves, R.M.
Source: New York, NY: John Wiley and Sons, Inc, et al. 2004
Comments:

 

6. Reporting of observational research

Title: STROBE Initiative. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies.
Source: The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies
Comments:

Title: Community Based Participatory Research for Health by Minkler, M., Wallerstein, N.
Source: San Francisco, CA: Jossey-Bass Publishers, eds. 2002
Comments:

 
 

Title: Observational Studies in Epidemiology (STROBE):explanation and elaboration.
Source: Strengthening the Reporting of Observational Studies in Epidemiology (STROBE):explanation and elaboration; Strobe-Statement
Comments:

Title: Cross-cultural Survey Methods by Harkness, J.A., van de Vijver, F.J.R., Mohler, P.P.
Source: Hoboken, NJ: John Wiley and Sons, Inc, eds. 2003
Comments:

 
  

Title: Experimental and Quasi-experimental Designs for Research by Campbell, D.T., Stanley, J.C.
Source: Chicago, IL: Rand McNally, 1969
Comments:

 
  

Title: Handbook of Health Research Methods, Investigation Measurement and Analysis by Bowling, A., Ebrahim, S.
Source: Washington, DC: National Academies Press, 2005
Comments:

 
  

Title: Methods in Community-based Participatory Research for Health by Israel, B.A., Eng, E., Schulz, A.J., Parker, E.A.
Source: San Francisco, CA: Jossey-Bass Publishers, eds. 2005
Comments:

 
  

Title: Social Science Approaches to Health Services Research by Choi, T., Greenberg, J.N.
Source: Ann Arbor, MI: Health Administration Press, eds. 1983
Comments:

 

C. Observational - Secondary Database Research Methods

   

1. Health care data – assessment of types, database design, and management

   

2. Health care data - medical record extraction and abstracting methods

   

3. Health care claims data analysis

Title: Guidelines for Good Database Selection and use in Pharmacoepidemiology Research
Source: Guidelines for Good Database Selection and use in Pharmacoepidemiology Research
Comments:

  

4. Retrospective (secondary) database analysis

Title: Observational Study Methods-Database Methods/A Checklist For Retrospective Database Studies
Source: Observational Study Methods-Database Methods/A Checklist For Retrospective Database Studies
Comments:

  

5. Retrospective (secondary) database analysis-confounding, bias, causal inference

Title: Retrospective Database Analysis – Bias & Confounding in the Design
Source: Retrospective Database Analysis – Bias & Confounding in the Design
Comments:

  
 

Title: Retrospective Database Analysis – Causal Inference
Source: Retrospective Database Analysis – Causal Inference
Comments:

  

6. Retrospective (secondary) database analysis-reporting

Title: Retrospective Database Analysis – Defining, Reporting & Interpreting
Source: Retrospective Database Analysis – Defining, Reporting & Interpreting
Comments:

  
 

Title: The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies
Source: The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies
Comments:

  

7. Generalizability of secondary database research methods and/or results

   

8. Observational comparative effectiveness research

Title: Developing a Protocol for Observational Comparative Effectiveness Research: A User's Guide
Source:
http://effectivehealthcare.ahrq.gov/ehc/products
/440/1166/User-Guide-Observational-CER-130113.pdf

Comments:

  

9. Medication adherence / compliance / persistence

Title: A Checklist for Medication Compliance and Persistence Studies Using Retrospective Databases
Source: A Checklist for Medication Compliance and Persistence Studies Using Retrospective Databases
Comments:

  
 

Title: Medication Compliance and Persistence: Terminology and Definitions
Source: A Checklist for Medication Compliance and Persistence Studies Using Retrospective Databases
Comments:

  
 

Title: Methods for Integrating Medication Compliance and Persistence in Pharmacoeconomic Evaluations
Source: Methods for Integrating Medication Compliance and Persistence in Pharmacoeconomic Evaluations
Comments:

  

10. Real time analyses (rapid learning healthcare system)

   

D. Dealing with heterogeneity of treatment effect

   

1. Classical subgroup analysis

   

2. Personalized medicine methods – biomarkers; stratification of patients methods for clinical studies

   

E. Patient-Centered Outcomes Research

Title: Standards for Patient-Centered Outcomes Research
Source: http://pcori.org/assets/Methodology-Report-072312.pdf

  
 

Title: PCORI Methodology Standards
Source: http://pcori.org/assets/PCORI-Methodology-Standards1.pdf

  

II. Qualitative Clinical Outcomes Assessment Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Qualitative research methods

 

Title: Basics of Qualitative Research: Techniques and Procedures for Developing Grounded Theory by Strauss, A., Corbin, J. M. 
Source: Thousand Oaks, CA: Sage Publications, 1998 
Comments:

 
  

Title: Measuring Health: a Guide to Rating Scales and Questionnaires by McDowell, I., Newell, C.
Source: 3rd ed. New York, NY: Oxford University Press, 2006
Comments:

 
  

Title: Qualitative Data Analysis: An Expanded Sourcebook by Miles, M. B., Huberman, A. M. 
Source: 2nd ed. Thousand Oaks, CA: Sage Publications, 1994 
Comments:

 
  

Title: Qualitative Research and Evaluation Methods by Patton, M.Q. 
Source: 3rd ed. Thousand Oaks, CA: Sage Publications, 2002
Comments:

 
  

Title: The Sage Handbook of Qualitative Research by Denzin, N.K., Lincoln, Y.S.
Source: Thousand Oaks, CA: Sage Publications, eds. 2005 
Comments:

 

B. Patient reported outcomes assessment

Title: Incorporating the Patient's Perspective into Drug Development and Communication
Source:  Incorporating the Patient's Perspective into Drug Development and Communication
Comments:

Title: Fundamentals of Item Response Theory by Hambleton, R. K., Swaminathan, H., Rogers, J. 
Source: Newbury Park, CA: Sage Publications, 1991 
Comments:

 

1. Health related quality of life-translation

Title: Multinational Trials—Recommendations on the Translations Required,Approaches to Using the Same Language in Different Countries, and the Approaches to Support Pooling the Data:The ISPOR Patient-Reported Outcomes Translation and Linguistic Validation Good R
Source:  Multinational Trials—Recommendations on the Translations Required,Approaches to Using the Same Language in Different Countries, and the Approaches to Support Pooling the Data:The ISPOR Patient-Reported Outcomes Translation and Linguistic Validation Good R
Comments:

  
 

Title: Principles of Good Practice for the Translation and Cultural Adaptation Process for Patient-Reported Outcomes (PRO) Measures: Report of the ISPOR Task Force for Translation and Cultural Adaptation
Source:  Principles of Good Practice for the Translation and Cultural Adaptation Process for Patient-Reported Outcomes (PRO) Measures: Report of the ISPOR Task Force for Translation and Cultural Adaptation
Comments:

  

2. Patient Reported Outcomes Assessment-content validity, new and existing instruments

Title: Good Research Practices on New PRO Instruments – Assessing Respondent Understanding 
Source:  Good Research Practices on New PRO Instruments – Assessing Respondent Understanding 
Comments:

  
 

Title: Good Research Practices on New PRO Instruments – Eliciting Concepts 
Source:  Good Research Practices on New PRO Instruments – Eliciting Concepts
Comments:

  
 

Title: Use of Existing Patient-Reported Outcome (PRO) Instruments and Their Modification: The ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PROTask Force Report
Source:  Use of Existing Patient-Reported Outcome (PRO) Instruments and Their Modification:The ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modification PROTask Force Report
Comments:

  

3. PRO in children and adolescents methods

Title: Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR Good Research Practices for the Assessment of Patient-Reported Outcomes in Children and Adolescents Task Force
Source: https://staging.ispor.org/docs/default-source/resources/outcomes-research-guidelines-index/matza_et_al_2013_ispor_task_force_pros_in_children.pdf?sfvrsn=be193614_2
Comments:

  

4. ePRO systems validation methods

Title: Validation of Electronic Systems to Collect Patient-Reported Outcome (PRO) Data - Recommendations for Clinical Trial Teams: Report of the ISPOR ePRO Systems Validation Good Research Practices Task Force 
Source:  https://staging.ispor.org/docs/default-source/resources/outcomes-research-guidelines-index/piis109830151301797x.pdf?sfvrsn=f5aa091e_2
Comments:

  

5. Mixed modes to collecting PRO data methods

Title: PRO Task Force: Good Research Practices for Mixed Modes to Collect PRO Data in Clinical Trials
Source: PRO Task Force: GOOD RESEARCH PRACTICES FOR Mixed Modes to Collect PRO Data in Clinical Trials
Comments:

  

6. Patient and treatment Satisfaction

   

7. Work performance

   

C. Clinician-reported outcomes

   

D. Observer-reported outcomes

   

III. Patient Preference Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Stated preferences

   

1. Contingent valuation

 

Title: Contingent Valuation: A Comprehensive Bibliography and History by Carson R. 
Source: Cheltenham, UK: Edward Elgar, 2011
Comments:

 
  

Title: Economic Valuation with States Preferences Techniques: A Maual by Bateman I, et al.
Source: Cheltenham, UK: Edward Elgar, 2002
Comments:

 

i. Willingness to Pay

 

Title: Methods for the Economic Evaluation of Health Care Programmes (3rd edition, chapter 7) by Drummond, MF et al.
Source: Oxford, UK: Oxford University Press, 2005
Comments:

Title: Willingness to pay: a valid and reliable measure of health state preference? By O'Brien B, Viramontes JL.
Source: Medical Decision Making 1994;14:289-97
Comments:

   

Title: Theory versus practice: A review of “willingness-to-pay” in health and health care, by Olsen JA, Smith RD.
Source:  Health Economics 2001;10:39-52.
Comments:

ii. Probability threshold techniques

  

Title: Using a trade-off technique to assess patients' treatment preferences for benign prostatic hyperplasia by Llewellyn-Thomas H et al.
Source: Medical Decision Making 1995;16:262-272.
Comments:  

2. Conjoint Analysis

Title: ISPOR conjoint analysis good research practices checklist TF report 
Source:  Conjoint Analysis Applications in Health—a Checklist: A Report of the ISPOR Good Research Practices for Conjoint Analysis Task Force
Comments:

Title: Applied Choice Analysis: A Primer by Hensher DA, Rose JM, Greene WH.
Source: New York: Cambridge University Press 2005
Comments:

Title: Discrete-choice experiments in health economics: a review of the literature by de Bekker-Grob E, et al.
Source:  Health Economics 2012;21:145-72
Comments:

 

Title: Constructing Experimental Designs for Discrete-Choice Experiments: Report of the ISPOR Conjoint Analysis Experimental Design Task Force
Source:  https://staging.ispor.org/docs/default-source/resources/outcomes-research-guidelines-index/piis1098301512041629.pdf?sfvrsn=d9c2e90c_0
Comments:

 

Title: Stated Choice Methods by Louviere JJ, Hensher DA, Swait JD.
Source: New York: Cambridge University Press 2000
Comments:

Title: Conjoint analysis applications in health – how are studies being designed and reported? An update on current practice in the published literature between 2005 and 2008 by Marshall DA et al.
Source:  Patient  2010;3:249-56.
Comments:

 

Title: ISPOR conjoint analysis statistical methods TF report (in development)
Source: 
Comments:

 

Title: Things are looking up since we started listening to patients: trends in the application of conjoint analysis in health 1982-2007 by Bridges JFR, et al.
Source:  Patient 2008;1: 273-82

3. Health-state utility measurement

 

Title: Utility Assessment by Mauskopf JA
Source: Methods and Designs for Outcomes Research, American Society of Health-System Pharmacists, 2006 Comments:

 

i. Direct elicitation (time tradeoff, standard gamble)

  

Title: Standard gamble, time tradeoff, and rating scale – experimental results on the ranking properties of QALYs by Bleichrodt H, Johanneson M.
Source:  Journal of Health Economics; 1997:155-75.

ii. Multi- attribute utility

  

Title: Multi-attribute and single-attribute utility functions for the Health Utilities Index Mark 3 system by Feeny D et al.
Source:  Medical Care 2002;40:113-28

B. Revealed preferences (patient decision-making behavior measurement)

   

IV. Health Economic Evaluation Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Costing Method

   

1. Health care resource use (measuring costs)

Title: Good Research Practices for Measuring Drug Costs in Cost Effectiveness Analyses: Issues and Recommendations: The ISPOR Drug Cost Task Force Report—Part I
Source:  Good Research Practices for Measuring Drug Costs in Cost Effectiveness Analyses: Issues and Recommendations:The ISPOR Drug Cost Task Force Report—Part I
Comments:

  
 

Title: Good Research Practices for Measuring Drug Costs in Cost Effectiveness Analyses:An Industry Perspective: The ISPOR Drug Cost Task Force Report—Part V
Source:  Good Research Practices for Measuring Drug Costs in Cost Effectiveness Analyses:An Industry Perspective: The ISPOR Drug Cost Task Force Report—Part V
Comments:

  
 

Title: Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses: Medicare, Medicaid and Other US Government Payers Perspectives: The ISPOR Drug Cost Task Force Report—Part IV
Source:  Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses: Medicare, Medicaid and Other US Government Payers Perspectives: The ISPOR Drug Cost Task Force Report—Part IV
Comments:

  
 

Title: Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:A Managed Care Perspective: The ISPOR Drug Cost Task Force Report—Part III
Source:  Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:A Managed Care Perspective: The ISPOR Drug Cost Task Force Report—Part III
Comments:

  
 

Title: Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:A Societal Perspective: The ISPOR Drug Cost Task Force Report—Part II
Source:  Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:A Societal Perspective: The ISPOR Drug Cost Task Force Report—Part II
Comments:

  
 

Title: Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:An International Perspective:The ISPOR Drug Cost Task Force Report—Part VI
Source:  Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses:An International Perspective:The ISPOR Drug Cost Task Force Report—Part VI
Comments:

  

2. Cost of illness/burden of disease

   

3. Budget impact

Title: Principles of Good Practice for Budget Impact Analysis: Report of the ISPOR Task Force on Good Research Practices—Budget Impact Analysis 
Source:  Principles of Good Practice for Budget Impact Analysis: Report of the ISPOR Task Force on Good Research Practices—Budget Impact Analysis 
Comments:

  

4. Practicalities of estimating productivity costs

   

5. Transferability of economic information

Title: Economic Evaluation Methods/Transferability of Economic Evaluations Across Jurisdictions
Source:  Economic Evaluation Methods/Transferability of Economic Evaluations Across Jurisdictions
Comments:

  

B. Health Economic Evaluation Reporting

Title:  Consolidated Health Economic Evaluation Reporting Standards (CHEERS)—Explanation and Elaboration: A Report of the ISPOR Health Economic Evaluation Publication Guidelines Good Reporting Practices Task Force 
Source:  https://www.ispor.org/ValueInHealth/ShowValueInHealth.aspx?issue=3D35FDBC-D569-431D-8C27-378B8F99EC67

  

V. Health Care Research Statistical Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Frequentist Methods

 

Title: Biostatistics: a Methodology for the Health Sciences by Van Belle, G., Fisher, L.D., Heagerty, P., Lumley, T. 
Source: 2nd ed. New York, NY: John Wiley and Sons, Inc., 2004
Comments:

 
  

Title: Data Analysis Using Stata by Kohler, U., Kreuter, F. 
Source: College Station, TX: Stata Press, 2005
Comments:

 
  

Title: Fundamentals of Biostatistics by Rosner, B. 
Source: 6th ed. Pacific Grove, CA: Duxbury Press, 2006
Comments:

 
  

Title: Nonparametric Statistics by Hollander, M., Wolfe D. 
Source: 2nd ed. New York, NY: Wiley-InterScience, 1999
Comments:

 
  

Title: Statistical Methods for Health Care Research by Munro, B.H. 
Source: Philadelphia, PA: Lippincott Williams & Wilkins, 2005
Comments:

 
  

Title: Statistical Methods in Medical Research by Armitage, P., Berry, G., Matthews, J.N.S. 
Source: 4th ed. Boston, MA, Oxford: Blackwell Scientific Publications, 2001
Comments:

 

B. Bayesian Analytical Methods

 

Title: A Primer on Bayesian Statistics in Health Economics and Outcomes Research by O'Hagan A, Luce B. 
Source: Bethesda, Maryland: Medtap International, 2003
Comments:

 

C. Specific estimation methods

   

1. Multivariable regression

 

Title: Applied Linear Regression Models by Netter J., Wasserman W., Kutner M.H. 
Source: 2nd ed. Boston, MA:Irwin, 1989 
Comments:

 
  

Title: Applied Logistic Regression by Hosmer D.L., Lemeshow S. 
Source: 2nd edition. New York, NY: John Wiley and Sons, 2001
Comments:

 
  

Title: Applied Regression Analysis and Other Multivariable Methods by Kleinbaum, L.
Source: 3rd Edition. Pacific Grove: Duxbury Press, et al. 1998 
Comments:

 
  

Title: Regression Models for Categorical and Limited Dependent Variables by Long, J.S. 
Source: Thousand Oaks, CA: Sage Publications, 1997 
Comments:

 
  

Title: Regression Models for Categorical Data Using Stata by Long, J.S., Freese, J. 
Source: 2nd ed. College Station, TX: Stata Press, 2006 
Comments:

 

2. Parametric and semi-parametric survival modeling

 

Title: Modeling Survival Data in Medical Research by Collett D 
Source: London: Chapman and Hall/CRC, 1994
Comments:

 

3. Longitudinal data analysis

   

4. Propensity score methods

   

5. Stratification methods

   

6. Mixture modeling methods

   

7. Instrumental variables methods

   

8. Structural equation modeling methods

   

9. Marginal structural modeling methods

   

D. Health econometric methods

 

Title: Econometric Analysis by Greene, L. 
Source: 5th ed. Upper Saddle River, NJ: Prentice Hall, 2003
Comments:

 
  

Title: Guide to Econometrics by Kennedy, P.
Source: 5th ed. Cambridge, MA: MIT Press, 2003 
Comments:

 

1. Econometric analysis of cost data

   

2. Econometric analysis of quality of life data

   

3. Econometric analysis for health care program evaluation (including selection modeling and cost-benefit analysis)

   

4. Econometric analysis of health care supply & demand

   

VI. Social and Ethical Aspects of Health Technologies Research Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS

A. Analysis of the delivery models of technology, and of resources, management and cultural issues within a variety of stakeholders in- and outside the organization including the health care system level

   

B. Analysis of social aspects (patients' and significant others' considerations, worries and experiences with the health technology

   

C. Ethical analysis of social and moral norms and values relevant for the technology in question

   

Synthesizing Health Care Evidence


I. Evidence Synthesis Research Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. Systematic reviewTitle: Guidelines for reporting systematic reviews and meta-analysis
Source: Stroup DF, Berlin JA, Morton SC, Olkin I, Williamson GD, Rennie D, Moher D, Becker BJ, Sipe TA, Thacker SB. Meta-analysis of observational studies in epidemiology: a proposal for reporting. Meta-analysis Of Observational Studies in Epidemiology (MOOSE) group. JAMA 2000; 283(15):2008-2012. 
Guidelines for reporting systematic reviews and meta-analysis
Comments:
Title: Meta-Analysis in Medicine and Health Policy by Stangl, D. and Berry, D.
Source: New York, eds. 2000 
Comments:
 
 Title: Standards for reporting literature searches
Source: 
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1629442/
pdf/i1536-5050-094-04-0421.pdf
  
   Title: Center for Reviews and Dissemination (CRD): Systematic Reviews
Source:  http://www.york.ac.uk/inst
/crd/index_guidance.htm
  Title: Cochrane Handbook for Systematic Reviews of Interventions
Source:  http://handbook.cochrane.org/
Comments:
 
B. Meta-analysisTitle: Guidelines for reporting systematic reviews and meta-analysis
Source: Stroup DF, Berlin JA, Morton SC, Olkin I, Williamson GD, Rennie D, Moher D, Becker BJ, Sipe TA, Thacker SB. Meta-analysis of observational studies in epidemiology: a proposal for reporting. Meta-analysis Of Observational Studies in Epidemiology (MOOSE) group. JAMA 2000; 283(15):2008-2012.
Guidelines for reporting systematic reviews and meta-analysis
Comments:
Title: Meta-Analysis in Medicine and Health Policy by Stangl, D. and Berry, D.
Source: New York, eds. 2000 
Comments:
 
   Title: Meta-analysis of individual participant data: rationale, conduct, and reporting by Riley RD, Lambert PC, bo-Zaid G. 
Source: BMJ 2010; 340:c221.
  Title: Methods for meta-analysis in medical research by Sutton AJ, Abrams KR, Jones DR, Sheldon TA, Song F. 
Source: London: Wiley, 2000
 
  Title: Systematic Reviews in Health Care: Meta-Analysis in Context, Second Edition by(editors): Matthias Egger, George Davey Smith, Douglas G Altman
Source: BMJ Publishing Group 2001
Comment:
 
C. Indirect treatment & network meta-analysis comparisonsTitle: Conducting Indirect-Treatment-Comparison and Network-Meta-Analysis Studies: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices—Part 2
Source:  Conducting Indirect-Treatment-Comparison and Network-Meta-Analysis Studies: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices—Part 2
Comments:
Title: Evidence Synthesis for Decision Making in Health Care by  NJ Welton, A.J Sutton, NJ Cooper, K.R. Abrams, AE Ades. 
Source: Wiley-Blackwell, 2012
 
 Title: EUnetHTA guideline “Comparators and comparisons – Direct and Indirect Comparisons”
Source: http://www.eunethta.eu/sites/5026.fedimbo.belgium.be
/files/Direct%20and%20indirect%20comparisons.pdf
  
D. Handling bias in evidence synthesis studies   
1. Publication bias   
2. Observational study confounding and selection bias   
3. Generalizability of RCTs and observational studies   
4. Assessing the quality of evidence Title: Medical Technologies Evaluation Programme
Source:  Medical Technologies Evaluation Programme
Comments:
 
E. Individual patient data synthesis   
F. Heterogeneity: measurement, meta regression, sub-group analysis Title: Systematic Reviews in Health Care: Meta-Analysis in Context, Second Edition
Editor(s): Matthias Egger, George Davey Smith, Douglas G Altman
Source: 2001 BMJ Publishing Group
 
G. Methods for integrating stakeholders (e.g., patients, clinicians) aspects into HTA Title: Health Technology Assessment Handbook
Source:  Chapter 7 in Health Technology Assessment Handbook
Comments:
 

II. Modeling/Decision Analysis Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. Conceptualizing a modelTitle:  Conceptualizing a Model: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-2
Source: Conceptual Modeling Working Group
Comments:
  
B. Model parameter estimation and uncertaintyTitle:  Model Parameter Estimation and Uncertainty Analysis: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-6
Source:  Model Parameter Estimation and Uncertainty Working Group
Comments:
Title: Decision Modeling for Health Economic Evaluation by Briggs A, Claxton K, Sculpher M. 
Source: Oxford: Oxford University Press, 2006.
Comments:
 
C. Model transparency and validationTitle:  Model Transparency and Validation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-7
Source:  Model Transparency and Validation Working Group
Comments:
  
D. State transition modelingTitle:  State-Transition Modeling: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-3
Source:  State-Transition Modeling Working Group
Comments:
  
E. Modeling using discrete event simulationTitle:  Modeling using Discrete Event Simulation: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-4
Source:  Discrete Event Simulation Working Group
Comments:
  
F. Dynamic Transition ModelingTitle:  Dynamic Transmission Modeling: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-5-5
Source:  Dynamic Transmission Modeling Working Group
Comments:
  
G. Health Systems Modeling Methods   
H. Methods to assess new technologies during product development   
I. Multi-criteria decision analysis   
1. Multi criteria decision analysis vs. deliberative methods for economic analysis - incorporating preferences and uncertainty  Title: Multi-criteria clinical decision support: A primer on the use of multiple criteria decision making methods to promote evidence-based, patient-centered healthcare by Dolan JG 
Source:  Patient 2010; 3(4): 229-248 
Comments: Provides a general introduction multi-criteria decision methods; methods include balance sheet, even swap method, ordinal ranking methods, direct weighting methods, multi-attribute decision analysis, and AHP; includes examples and glossary of terms
  Title: Trends in multiple criteria decision analysis by Greco S, Ehrgott M, Figueira JR (Eds). 
Source: Springer, 2010 edition
Comment:
Title: Multiple criteria decision analysis for health technology assessment: Report by the Decision Support Unit by Thokala P and Durenas A 
Source:  Value in Health 2012; 15(8): 1172-1181.
Comments: Includes introduction to most common types of MCDA models and critical review of state-of-the-art methods for incorporating multiple criteria in health technology assessment
  Title: Multiple criteria decision analysis: State of the art surveys by Greco S (Ed) 
Source: Springer, 2004
Comment:
 
2. Incorporating preferences and uncertainty in health care decisions - analytical hierarchy process   
3. Fuzzy logic decision support systems Title: Fuzzy multi-criteria decision making: theory and applications with recent developments by Kahraman C (Ed) 
Source: Springer, 2008
Comment:
 
J. Clinical decision analysis Title: Decision Making in Health and Medicine: Integrating Evidence and Values. 
Source: Cambridge University Press, 2001
 
  Title: Medical Tests-White Paper Series
Source: Agency for Healthcare Research and Quality (US); Rockville (MD): 2009-
 

III. Health Economic Evaluation Framework and Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. General methodsTitle: General Methods for the Assessment of the Relation of Benefits to Costs
Source:  General Methods for the Assessment of the Relation of Benefits to Costs
Comments:
Title: Methods for the Economic Evaluation of Health Care Programmes. 3rd ed. by Drummond MF, Sculpher MJ, Torrance GW, O'Brien B, Stoddart GL. 
Source: Oxford: Oxford University Press, 2005. 
Comments:
 
  Title: Cost-Effectiveness in Health Care by Gold, M.R., Siegel, J.E., Russell, L.B., Weinstein, W.C.
Source: New York, NY: Oxford University Press., eds. 1996 
Comments:
 
 Title: Guide to the Methods of Technology Appraisal (reference N0515)
Source:  Guide to the Methods of Technology Appraisal (reference N0515)
Comments:
  
 Title: Guidelines for the Economic Evaluation of Health Technologies: Canada
Source:  Guidelines for the Economic Evaluation of Health Technologies: Canada
Comments:
  
 Title: The AMCP Format for Formulary Submissions
Source:  The AMCP Format for Formulary Submissions
Comments:
  
   Title: When should decision analytic modeling be used in the economic evaluation of health care? 
Source: Eur J Health Econ. 2003;4:143–150.
B. General Methods - OutcomesTitle: EUnetHTA guideline “Endpoints used for relative effectiveness assessment of pharmaceuticals
Health-Related Quality Of Life And Utility Measures
Source:  https://5026.fedimbo.belgium.be/sites/
5026.fedimbo.belgium.be/files/
Health-related%20quality%20of%20life.pdf
 Title: A Different Approach to Health State Valuation
Source:  A Different Approach to Health State Valuation
Comments:
   Title: Editorial: Moving the QALY Forward or Just Stuck in Traffic?
Source:  Editorial: Moving the QALY Forward or Just Stuck in Traffic?
Comments:
   Title: Editorial: On the Benefits of Modeling Using QALYs for Societal Resource Allocation: The Model Is the Message
Source:  Editorial: On the Benefits of Modeling Using QALYs for Societal Resource Allocation: The Model Is the Message
Comments:
   Title: Moving the QALY Forward: Rationale for Change
Source:  Moving the QALY Forward: Rationale for Change
Comments:
   Title: QALYs: Some Challenges
Source:  QALYs: Some Challenges
Comments:
   Title: QALYs: The Basics
Source:  QALYs: The Basics
Comments:
   Title: Retaining, and Enhancing, the QALY
Source:  Retaining, and Enhancing, the QALY
Comments:
   Title: The Use of QALYs in Clinical and Patient Decision-Making:Issues and Prospects
Source:  The Use of QALYs in Clinical and Patient Decision-Making:Issues and Prospects
Comments:
   Title: Toward a Consensus on the QALY
Source:  Toward a Consensus on the QALY
C. General Methods - Costs   
1. Cost-benefit/net benefit analysis Title: Applied Methods of Cost-Benefit Analysis in Health Care (Handbooks in Health Economic Evaluation by McIntosh E, Clarke P, Frew E, Louviere J. 
Source: Oxford University Press, 2010 
Comments:
 
2. Cost-effectiveness (efficacy) analysisTitle: Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA Task Force Report
Source: Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA Task Force Report
Comments:
  
3. Economic study reportingTitle: Health Economic Evaluation Publication Guidelines Good Reporting Practices
Source:  Health Economic Evaluation Publication Guidelnes – CHEERS*: Good Reporting Practices Task Force
Comments:
  
4. Economic analysis: the role of clinical trials and modelsTitle: Economic evaluation alongside randomized controlled trials: design, conduct, analysis, and reporting
Source:  Economic evaluation alongside randomized controlled trials: design, conduct, analysis, and reporting
Comments:
  
 Title: Economic evaluation using decision analytical modeling: design, conduct, analysis, and reporting
Source:  Economic evaluation using decision analytical modeling: design, conduct, analysis, and reporting
Comments:
 Title: Whither trial-based economic evaluation for health care decision making? by Sculpher MJ, Claxton KP, Drummond MF, McCabe C.
Source:  Health Economics 2006;15:677-87.
   Title: When should decision analytic modeling be used in the economic evaluation of health care? 
Source: Eur J Health Econ. 2003;4:143–150.
D. Determining cost effectiveness thresholds  Title: The NICE cost-effectiveness threshold: what it is and what that means by McCabe C, Claxton K, Culyer AJ .
Source: Pharmacoeconomics 2008;26:733-44
Comments:
   Title: What Does the Value of Modern Medicine Say About the $50,000 per Quality-Adjusted Life-Year Decision Rule?
Source:  What Does the Value of Modern Medicine Say About the $50,000 per Quality-Adjusted Life-Year Decision Rule?
Comments:
E. How to deal with equity issues in economic evaluations   
F. Methods for research prioritization including value of information analysis Title: Decision Modeling for Health Economic Evaluation by Briggs A, Claxton K, Sculpher M. 
Source: Oxford: Oxford University Press, 2006.
Comments:
 
   Title: Calculating partial expected value of perfect information via Monte Carlo sampling algorithms by Brennan A, Kharroubi S, O'Hagan A, Chilcott J Medical 
Source: Medical Decision Making 2007;27:448-70
Comments:
G. Incorporating heterogeneity (personalization / individualization) in economic evaluations  Title: Subgroups and heterogeneity in cost-effectiveness analysis by Sculpher MJ. 
Source: Pharmacoeconomics 2008;26:799-806
Comments:
   Title: Value of information on preference heterogeneity and individualized care by Basu A, Meltzer D. 
Source: Medical Decision Making 2007;27:112-27
Comments:
   Title: Challenges in the development and reimbursement of personalized medicine-payer and manufacturer perspectives and implications for health economics and outcomes research: a report of the ISPOR personalized medicine special interest group.
Source: Value Health. 2012 Dec;15(8):1162-71.
  Title: Pharmacogenomics Bias - Systematic distortion of study results by genetic heterogeneity (DIMDI HTA Methods Report). 
Source: GMS Health Technol Assess. 2008
Comment:
 
H. HTA Agency Guidelines  Title: Key principles for the improved conduct of health technology assessments for resource allocation decisions.
Source: Int J Technol Assess Health Care. 2008

IV. Social and Ethical Aspects of Health Technologies Research Methods

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. Review of studies of delivery models of technology, and of resources, management and cultural issues within a variety of stakeholders in- and outside the organization including the health care system level Title: HTA Core Model® 
Source:  http://fio.stakes.fi/htacore/ViewHandbook.aspx
#domainguidance7

Comment:
 
  Title: Health Technology Assessment Handbook
Source:  Chapter 8 in Health Technology Assessment
Comments:
 
B. Review of studies of social aspects (patients' and significant others' considerations, worries and experiences with the health technology Title: HTA Core Model® 
Source: http://fio.stakes.fi/htacore/ViewHandbook.aspx
#domainguidance8

Comment:
 
  Title: Health Technology Assessment Handbook
Source: Chapter 7 in Health Technology Assessment
Comment:
 
C. Review of ethical analyses of social and moral norms and values relevant for the technology in question Title: HTA Core Model®
Source:
http://fio.stakes.fi/htacore/ViewHandbook.aspx
#domainguidance6

Comment:
 
  Title: Health Technology Assessment Handbook
Source:  Chapter 2 in Health Technology Assessment
Comments
 

Using Evidence in Health Care Decisions


I. Payer (population-based) Health Care Decision

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. HTA terminologyTitle: HTA Glossary – 
Source:  http://htaglossary.net/HomePage
Title: ISPOR Book of Terms – 
Source:  https://www.ispor.org/publications/books/healthcare-cost-quality-and-outcomes-ispor-book-of-terms
Title: What is Health Care Technology Assessment
Source:  http://www.medicine.ox.ac.uk/
bandolier/painres/download/whatis/
What_is_health_tech.pdf
  Title: Health Technology Assessment: Konzepte, Methoden, Praxis fur Wissenschaft und Entscheidungsfindung. 
Source:  http://books.google.ca/books/
about/Health_technology_Assessment.html
?id=hWQCkhGnkogC&redir_esc=y
 
  Title: Evaluating Investments in Health Care Systems: Health Technology Assessment
Source:  http://www.amazon.ca/
Evaluating-Investments-Health-Care-Systems/dp/3319025430
 
B. Framework/principles for HTA and benchmarking HTA organizationsTitle: AHRQ Series Paper 2: Principles for developing guidance: AHRQ and the Effective Health-Care Program Title: Key principles for the improved conduct of health technology assessments for resource allocation decisions.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/18601792
 Title: Understanding Health Technology Assessment (HTA) 
Source:  http://img.eurordis.org/newsletter/
pdf/nov-2010/58-1%20HEE
%20Guide%20To%20HTA%20
for%20Patients%20English.pdf
 Title: Developing Health Technology Assessment to address health care system needs.
Source:  http://www.ncbi.nlm.nih.gov
/pubmed/19889471
 Title: The HTA Core Model 2.0 – www.corehta.info [Internet]. EUnetHTA Work Package 8; 2013. 
Source:  http://www.corehta.info/model
/HTACoreModel2.0.pdf
 Title Health Technology Assessments by the National Institute for Health and Clinical Excellence A Qualitative Study
Source: http://www.springer.com/public+health/
book/978-0-387-71995-5
 Title: Danish Centre for Health Technology Assessment, Health technology assessment handbook 
Source:  http://www.dacehta.dk
  
C. Structure / governance / organizational aspects of HTA  Title: Health technology assessment agencies: an international overview of organizational aspects. 
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/17937828
1. Organization readinessTitle: An Approach to Organizational Self Assessment Developed for the IUCN M&E Team by Universalia 
Source(s):  http://cmsdata.iucn.org/
downloads/oa_overview_00.pdf
 

https://www.anzsog.edu.au/media/
upload/publication/131_Flynn-and-Ugyel- Diagnostic-Tool-ANZAM-2013.pdf
  
D. Priority setting for HTATitle: Priority Setting for Health Technology Assessment: Theoretical Considerations and Practical Approaches: A paper produced by the Priority Setting Subgroup of the EUR-ASSESS Project
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/9194351
Title: Setting Priorities for Health Technologies Assessment: A Model Process 
Source:  http://www.nap.edu/catalog/2011.html
Title: Priority setting for health technology assessments: a systematic review of current practical approaches.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/17579932
 Title: NICE Technology appraisal topic selection
Source:  http://www.nice.org.uk/proxy/?
sourceUrl=http%3a%2f%2fwww.nice.org.uk
%2fgetinvolved%2ftopicselection
%2fTechnologyAppraisalTopicSelection.jsp
  
 Title: Washinton State Health Care Authority, Health Technology Assesment - HTA, Prioritization Criteria
Source:  http://www.hca.wa.gov/hta/
documents/prioritization_criteria.pdf

  
E. Determining evidence requirements / framing questionsTitle: SUPPORT Tools for evidence-informed health Policymaking (STP) 4: Using research evidence to clarify a problem
Source:  Health Research Policy and Systems 2009, 7(Suppl 1):S4 doi:10.1186/1478-4505-7-S1-S4
Title: A model for evidence-informed decision making in public health.
Source:  http://www.nccmt.ca/pubs/
FactSheet_EIDM_EN_WEB.pdf
Title: Best practice in undertaking and reporting health technology assessments. Working group 4 report.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/12053427
 Title: NICE, Guide to the methods of technology appraisal 2013; Evidence
Source:  http://www.nice.org.uk/article/
PMG9/chapter/3-Evidence
 Title: Observational studies in systemic reviews of comparative effectiveness: AHRQ and the Effective Health Care Program 
Source:  http://www.sciencedirect.com/
science/ article/pii/S0895435611000618
 Title: Danish Centre for Health Technology Assessment, Health technology assessment handbook 
Source:  http://www.dacehta.dk
  
1. Scope of HTA - determining feasibility, availability of evidence or potential duplicationTitle: The HTA Core Model 2.0 – www.corehta.info [Internet]. EUnetHTA Work Package 8; 2013. 
Source:  http://www.corehta.info/model/
HTACoreModel2.0.pdf
  
 Title: Danish Centre for Health Technology Assessment, Health technology assessment handbook 
Source:  http://www.dacehta.dk
  
F. Evidence appraisalTitle: NICE, Guide to the methods of technology appraisal 2013
Source:  http://www.nice.org.uk/article/
pmg9/chapter/1-introduction
Title: A Framework for Science Advice on Health: Principles and Guidelines.
Source:  http://www.eusanh.eu/
wp/wp-content/uploads/2011/
12/EuSANH_principlesandguidelines.pdf
 
 Title: NICE, Guide to the methods of technology appraisal 2013; The appraisal of the evidence and structured decision-making
Source:  http://www.nice.org.uk/article/PMG9/
chapter/6-The-appraisal-of-the-evidence-and-structured-decision-making
  
 Title: The HTA Core Model 2.0 – www.corehta.info [Internet]. EUnetHTA Work Package 8; 2013.
Source:  http://www.corehta.info/model/
HTACoreModel2.0.pdf
  
1. Outcomes research studies for decision-making   
i. Observational studies - prospective and retrospectiveTitle: Assessing Retrospective Observational Studies for Health Care Decisions Task Force
Source:  Assessing Retrospective Observational Studies for Health Care Decisions Task Force
Title: Users' Guides to the Medical Literature: a Manual for Evidence-based Clinical Practice by Guyatt, G., Rennie, D.
Source: Chicago, IL: American Medical Association, eds. 2002
 
 Title: Assessing Prospective Observational Studies for Health Care Decisions Task Force
Source:  Assessing Prospective Observational Studies for Health Care Decisions Task Force
Title: Interpreting the Medical Literature by Gehlbach, S.H. 
Source: 5th ed. New York, NY: McGraw-Hill, Professions Division, 2006 
Comments:
 
ii. Modeling studiesTitle: Assessing Modeling Studies for Health Care Decisions Task Force
Source:  Assessing Modeling Studies for Health Care Decisions Task Force
 Title: Reporting guidelines for modelling studies                       
Source:  http://www.biomedcentral.com/
1471-2288/12/168
iii. Feasibility / health services delivery / studies  Title: How do new technologies impact on workforce organisation? A rapid review of international evidence 2011.  
Source:  http://www.skillsforhealth.org.uk/
component/docman/doc_view/
1834-how-do-new-technologies-impact-on-workforce-organiastion-082011.html
iv. Systematic reviews, meta-analyses, decision analysis, and qualitative synthesis studiesTitle: Guide to the Methods of Technology Appraisal (reference N0515)
Source:  Guide to the Methods of Technology Appraisal (reference N0515)
Title: Finding what works in health care: standards for systematic reviews. Eden J, editor. Washington, D.C: National Academies Press; 2011. 317 p.
Source: http://www.iom.edu/Reports/2011/Finding-What-Works-in-Health-Care-Standards-for-Systematic-Reviews.aspx
Title: Deciding how much confidence to place in a systematic review. 
Source:  http://www.health-policy-systems.com/content/pdf/
1478-4505-7-S1-s8.pdf
   Title: Towards systematic reviews that inform health care management and policy-making
Source:  http://www.ncbi.nlm.nih.gov/pubmed/
16053582?dopt=Abstract

v. Indirect treatment comparison studiesTitle: Interpreting Indirect Treatment Comparisons and Network Meta-Analysis for Health-Care Decision Making: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: Part 1
Source:  Interpreting Indirect Treatment Comparisons and Network Meta-Analysis for Health-Care Decision Making: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: Part 1
  
 Title: Assessing Indirect Treatment Comparison Studies
Source:  Assessing Indirect Treatment Comparison Studies
  
 Title: A GRADE Working Group approach for rating the quality of treatment effect estimates from network meta-analysis 
Source:  http://www.bmj.com/content/349/bmj.g5630
  
vi. Cost-effectivenessTitle: IQWIG General Methods
Source:  General Methods
Title: Methods for the economic evaluation of health care programmes by Drummond MF, Sculpher MJ, Torrance GW, O’Brien BJ, Stoddart GL 
Source: Oxford University Press, USA; 3rd edition, 2005.
Title: Health Economic Assessment: Cost-Effectiveness Thresholds and Other Decision Criteria
Source: Int J Environ Res Public Health
http://www.ncbi.nlm.nih.gov/pmc/
articles/PMC2872336/
vii. Budget Impact Analysis   
viii. Equity issues  Title: Taking equity into consideration when assessing the findings of a systematic review.
Source:  http://www.health-policy-systems.com/content/pdf/1478-4505-7-S1-s10.pdf
2. Methods for integrating stakeholders (e.g., patients, clinicians) values / perspectives Title: Health Technology Assessment Handbook
Source:  Chapter 7 in Health Technology Assessment Handbook
Title: Public engagement in health technology assessment and coverage decisions: a study of experiences in France, Germany, and the United Kingdom.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/23052686
i. Deliberative processesTitle: Evaluating Deliberative Processes
Source:  http://www.ncchpp.ca/docs/
DeliberativeFS3_evaluation_EN.pdf
 Title: Deliberative processes in decisions about health care technologies: combining different types of evidence, values, algorithms and people.
Source:  http://www.ohe.org/publications/
article/deliberative-processes-in-decisions
-about-health-care-technologies-30.cfm
 Title: What is a deliberative process?
Source:  http://www.ncchpp.ca/docs/
DeliberativeDoc1_EN_pdf.pdf
 

Title: Deliberations about deliberative methods: issues in the design and evaluation of public participation processes
Source:  http://www.cprn.org/download.cfm
?doc=353&file=20819_en.pdf&
format=pdf&l=en

 Title: Deliberative Processes - Inventory of Resources
Source:  http://www.ncchpp.ca/docs/
DeliberativeInventory_EN_pdf.pdf
  
ii. Multi-criteria decision analysisTitle: Multiple criteria decision analysis for HTA
Source:  http://www.nicedsu.org.uk/
MCDA%20for%20HTA%20DSU.pdf
 Title: Multi-criteria Analysis Manual for 
Making Government Policy
Source:  https://www.gov.uk/government/
publications/ multi-criteria-analysis-
manual-for-making-government-policy
   Title: Comparative Weight Elicitation Methods
Source:  https://www.evidem.org/docs/2012/
Weight-Elicitation-Techniques.pdf
   Title: International Study on Decision Criteria
Source:  https://www.evidem.org/
international-study.php
iii. Patient engagement - patient advisory groups Title: Understanding Health Technology
Assessment (HTA) 
Source:  http://img.eurordis.org/newsletter
/pdf/nov-2010/58-1%20HEE%20Guide%20
To%20HTA %20for%20Patients%20English.pdf
Title: New Era Of Patient Engagement 
Source: Special Issues of  Health Affairs,February 2013; Volume 32, Issue 2
G. Implementation of HTA   
1. Managed entry agreements: risk-sharing/ performance-based, coverage with evidence development agreementsTitle: Performance-Based Risk-Sharing Arrangements - Good Practices for Design, Implementation and Evaluation: A Report of the ISPOR Good Practices for Performance-Based Risk-Sharing Arrangements Task Force 
Source:  https://www.ispor.org/docs/default-source/resources/outcomes-research-guidelines-index/performance-based-risk-sharing-guidelines.pdf?sfvrsn=dba8bdcf_2
 Title: Managed entry agreements for pharmaceuticals:
The European experience 
Source:  http://ec.europa.eu/enterprise/
sectors/healthcare/files/docs/
mea_report_en.pdf
   Title: What principles should govern the use of managed entry agreements?>
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/?term=21262072
   Title: Principles of design of access with evidence development approaches: a consensus statement from the Banff Summit.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/20085388
   Title: Current status and trends in performance-based risk-sharing arrangements between healthcare payers and medical product manufacturers.
Source:  http://www.ncbi.nlm.nih.gov/
pubmed/24664994
2. Supporting disinvestment decisionsTitle: Identification, prioritization and assessment of obsolete health technologies. A methodological guideline.
Source:  Identification, prioritization and assessment of obsolete health technologies. A methodological guideline.
  
   Title: Reducing the use of ineffective health care interventions
Source:  Reducing the use of ineffective health care interventions
3. Pricing and value-based pricing Title: Pricing. Prices of reimbursed drugs, negotiations, and risk sharing
PDF file - 3.5 MB 
Scientific editing: Krzysztof Łanda 
Source:  http://www.ceestahc.org/en/
activity,publishing.html?i7
Title: Reimbursement of pharmaceuticals: reference pricing versus health technology assessment.
Source:  http://www.ncbi.nlm.
nih.gov/pmc/articles/
PMC3078322/
4. Evidence-based / strategic procurement  Title: Concept of evidence-based public procurement 
Source:  http://www.unpcdc.org/media/4476/
global%20evidence%20based%20pp.pdf
H. Impact of HTA   
1. Measuring impactTitle: Planning monitoring and evaluation of policies.
Source:  http://www.health-policy-systems.com/content/pdf/1478-4505-7-S1-s18.pdf
 Title: Bridging the gap: The role of monitoring and evaluation in evidence-based policy making. UNICEF, the World Bank and the International Develop- ment Evaluation Association.
Source:  http://www.unicef.org/ ceecis/evidence_based_policy_making.pdf
 Title: HTA agencies and. decision makers. an INAHTA guidance document.
Source:    http://www.inahta.org/wp-content/themes/inahta/img/
HTA%20%20Decision%20Makers.pdf
 Title: How can the impact of HTA be enhanced?
Source:  http://www.euro.who.int/__data/
assets/pdf_file/0019/73225/E93420.pdf

II. Individual Health Care Decisions by Clinicians & Patients

MethodsGUIDELINESTEXTBOOKSREVIEWS/KEYPAPERS
A. Risk-benefit – efficacy vs. safety  Title: A framework for organizing and selecting quantitative approaches for benefit-harm assessment by Puhan MA, Singh S, Weiss CO, Varadhan R, Boyd CM. 
Source:  BMC Medical Research Methodology 2012; 12:173  http://www.biomedcentral.com/1471-2288/12/173
   Title: Benefit-risk evaluation: A tiered approach to an enhanced scope for safety, efficacy, and quality evidence2007.
Source: Health Canada.    http://www.hc-sc.gc.ca/dhp-mps/homologation-licensing/model/evaluation-eng.php. Accessed February 24, 2011.
   Title: Benefit-Risk methodology project - Description of the current practice of benefit-risk assessment for centralised procedure products in the EU regulatory network.
Source: European Medicines Agency. London UK: 2010.
   Title: A United States Regulator's Perspective on Risk-Benefit Considerations by Jenking J.
Source: Center for Drug Evaluation and ResearchFood and Drug Administration; 2010.
   Title: Draft Guidance for Industry and Food and Drug Administration Staff. Factors to Consider when Making Benefit-Risk Determinations in Medical Device Premarket Review
Source: Department of Health and Human Services Food and Drug Administration, Center for Devices and Radiological Health, Center for Biologics Evaluation and Research; August 15, 2011.
   Title:  Benefit-Risk methodology project work package 2 report: Applicability of current tools and processes for regulatory benefit-risk assessment.
Source: European Medicines Agency, London UK:2010
B. Patient engagement in treatment decisions   
i. Shared decision making instruments   
C. Using behavioral methods to help clinicians and patients fulfill decisions made Title: Decision making in health and medicine: Integrating evidence and values by Myriam Hunink MG, Glasziou PP, Siegel JE, Weeks JC, Pliskin JS, Elstein AS, Weinstein MC
Source: Cambridge University Press, 2001
 
D. Evidence-based decision making for clinicians Title: Evidence-based Medicine: How to Practice and Teach EBM by Sackett, D.L., Strauss, S.E., Richardson, W.S., Rosenberg, W., Haynes, R.B. 
Source: Scotland: Elsevier, 2005
 
  Title: Decision Making in Health and Medicine: Integrating Evidence and Values 
Source: Cambridge University Press, 2001
 
  Title: Medical Tests-White Paper Series
Source: Agency for Healthcare Research and Quality (US); Rockville (MD): 2009 -
 
  Title: Users' Guide to the Medical Literature.  A manual for Evidence -Based Clinical Practice.  2nd edition 2008 by Gordon Guyatt, MD, Drummond Rennie, MD, Maureen O Meade, MD and Deborah J Cook, MD.