Conducting Value for Money Analyses for Non-randomised Interventional Studies Including Service Evaluations: An Educational Review with Recommendations

This article provides an educational review covering the consideration of conducting ‘value for money’ analyses as part of non-randomised study designs including service evaluations. These evaluations represent a vehicle for producing evidence such as value for money of a care intervention or service delivery model. Decision makers including charities and local and national governing bodies often rely on evidence from non-randomised data and service evaluations to inform their resource allocation decision-making. However, as randomised data obtained from randomised controlled trials are considered the ‘gold standard’ for assessing causation, the use of this alternative vehicle for producing an evidence base requires careful consideration. We refer to value for money analyses, but reflect on methods associated with economic evaluations as a form of analysis used to inform resource allocation decision-making alongside a finite budget. Not all forms of value for money analysis are considered a full economic evaluation with implications for the information provided to decision makers. The type of value for money analysis to be conducted requires considerations such as the outcome(s) of interest, study design, statistical methods to control for confounding and bias, and how to quantify and describe uncertainty and opportunity costs to decision makers in any resulting value for money estimates. Service evaluations as vehicles for producing evidence present different challenges to analysts than what is commonly associated with research, randomised controlled trials and health technology appraisals, requiring specific study design and analytic considerations. This educational review describes and discusses these considerations, as overlooking them could affect the information provided to decision makers who may make an ‘ill-informed’ decision based on ‘poor’ or ‘inaccurate’ information with long-term implications. We make direct comparisons between randomised controlled trials relative to non-randomised data as vehicles for assessing causation; given ‘gold standard’ randomised controlled trials have limitations. Although we use UK-based decision makers as examples, we reflect on the needs of decision makers internationally for evidence-based decision-making specific to resource allocation. We make recommendations based on the experiences of the authors in the UK, reflecting on the wide variety of methods available, used as documented in the empirical literature. These methods may not have been fully considered relevant to non-randomised study designs and/or service evaluations, but could improve and aid the analysis conducted to inform the relevant value for money decision problem.

[1]  Karl Claxton,et al.  Methods for assessing the cost-effectiveness of public health interventions: key challenges and recommendations. , 2009, Health policy.

[2]  Tracey A. Young,et al.  Estimating mean total costs in the presence of censoring , 2012, PharmacoEconomics.

[3]  A. Detsky,et al.  Relevance of cost-effectiveness analysis to clinicians and policy makers. , 2007, Journal of the American Medical Association (JAMA).

[4]  D. Altman,et al.  Distinguishing Case Series From Cohort Studies , 2012, Annals of Internal Medicine.

[5]  J. Lellouch,et al.  Explanatory and pragmatic attitudes in therapeutical trials. , 1967, Journal of chronic diseases.

[6]  Yaling Yang,et al.  Review and critical appraisal of studies mapping from quality of life or clinical measures to EQ-5D: an online database and application of the MAPS statement , 2018, Health and Quality of Life Outcomes.

[7]  M. Franklin,et al.  The feasibility of early pulmonary rehabilitation and activity after COPD exacerbations: external pilot randomised controlled trial, qualitative case study and exploratory economic evaluation. , 2018, Health technology assessment.

[8]  Michael Drummond,et al.  Economic evaluation for devices and drugs--same or different? , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[9]  Zoltán Kaló,et al.  Multiple Criteria Decision Analysis for Health Care Decision Making--Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force. , 2016, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[10]  M. Drummond,et al.  Whither trial-based economic evaluation for health care decision making? , 2006, Health economics.

[11]  Maarten J. IJzerman,et al.  Identifying the Need for Good Practices in Health Technology Assessment: Summary of the ISPOR HTA Council Working Group Report on Good Practices in HTA. , 2019, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[12]  B. Prendergast,et al.  The Cost-Effectiveness of Antibiotic Prophylaxis for Patients at Risk of Infective Endocarditis , 2016, Circulation.

[13]  S. Griffin,et al.  Distributional cost-effectiveness analysis: a tutorial , 2016 .

[14]  C. McKevitt,et al.  The potential role of cost-utility analysis in the decision to implement major system change in acute stroke services in metropolitan areas in England , 2018, Health Research Policy and Systems.

[15]  William H Crown,et al.  Propensity-Score Matching in Economic Analyses: Comparison with Regression Models, Instrumental Variables, Residual Inclusion, Differences-in-Differences, and Decomposition Methods , 2014, Applied Health Economics and Health Policy.

[16]  A. Schuh,et al.  Issues surrounding the health economic evaluation of genomic technologies. , 2013, Pharmacogenomics.

[17]  R. Edwards,et al.  Public health economics: a systematic review of guidance for the economic evaluation of public health interventions and discussion of key methodological issues , 2013, BMC Public Health.

[18]  S. van den Broucke Implementing health in all policies post Helsinki 2013: why, what, who and how. , 2013, Health promotion international.

[19]  J. Raftery,et al.  NICE’s Cost-Effectiveness Range: Should it be Lowered? , 2014, PharmacoEconomics.

[20]  J. Grimshaw,et al.  Systematic review of economic evaluations and cost analyses of guideline implementation strategies , 2007, The European Journal of Health Economics.

[21]  E. Faerstein,et al.  A DICTIONARY OF EPIDEMIOLOGY , 2016 .

[22]  S. Petrou Rationale and methodology for trial-based economic evaluation , 2012 .

[23]  Andrew R Willan,et al.  Regression methods for cost‐effectiveness analysis with censored data , 2005, Statistics in medicine.

[24]  Karl Claxton,et al.  Methods for the estimation of the National Institute for Health and Care Excellence cost-effectiveness threshold. , 2015, Health technology assessment.

[25]  James Lomas,et al.  Estimating health opportunity costs in low-income and middle-income countries: a novel approach and evidence from cross-country data , 2018, BMJ Global Health.

[26]  P. van Baal,et al.  Future Costs in Cost-Effectiveness Analyses: Past, Present, Future , 2018, PharmacoEconomics.

[27]  Huseyin Naci,et al.  A review of NICE appraisals of pharmaceuticals 2000-2016 found variation in establishing comparative clinical effectiveness. , 2019, Journal of clinical epidemiology.

[28]  Miquel Porta,et al.  A Dictionary of Epidemiology , 2008 .

[29]  Steve Goodacre,et al.  Uncontrolled before-after studies: discouraged by Cochrane and the EMJ , 2015, Emergency Medicine Journal.

[30]  S. Palmer,et al.  After 20 Years of Using Economic Evaluation, Should NICE be Considered a Methods Innovator? , 2020, PharmacoEconomics.

[31]  Philip Jones,et al.  Public Finance and Public Choice (2nd ed) , 1998 .

[32]  K. Marsh,et al.  Assessing the Value of Healthcare Interventions Using Multi-Criteria Decision Analysis: A Review of the Literature , 2014, PharmacoEconomics.

[33]  B. O'brien,et al.  The death of cost-minimization analysis? , 2001, Health economics.

[34]  Jon Sussex,et al.  Critique of CHE Research Paper 81: Methods for the Estimation of the NICE Cost Effectiveness Threshold , 2013 .

[35]  S Bryan,et al.  The use of economic evaluations in NHS decision-making: a review and empirical investigation. , 2008, Health technology assessment.

[36]  A. Fischer,et al.  Additional Elements of Value for Health Technology Assessment Decisions , 2017 .

[37]  J. Brazier,et al.  Improving Cross-Sector Comparisons: Going Beyond the Health-Related QALY , 2015, Applied Health Economics and Health Policy.

[38]  N. Booth On value frameworks and opportunity costs in health technology assessment , 2019, International Journal of Technology Assessment in Health Care.

[39]  A H Briggs,et al.  Handling uncertainty when performing economic evaluation of healthcare interventions. , 1999, Health technology assessment.

[40]  Anthony J Culyer,et al.  Cost-effectiveness thresholds in health care: a bookshelf guide to their meaning and use , 2016, Health Economics, Policy and Law.

[41]  Kelly Hall,et al.  Social Return on Investment (SROI) and Performance Measurement , 2013 .

[42]  M. Cowie National Institute for Health and Care Excellence. , 2015, European heart journal.

[43]  Gianluca Baio,et al.  A Review of Methods for Analysis of the Expected Value of Information , 2015, Medical decision making : an international journal of the Society for Medical Decision Making.

[44]  Mohit Bhandari,et al.  Principles of evidence-based medicine. , 2010, The Orthopedic clinics of North America.

[45]  T C Chalmers,et al.  Randomized versus historical controls for clinical trials. , 1982, The American journal of medicine.

[46]  J. Greene,et al.  Assessing the Gold Standard--Lessons from the History of RCTs. , 2016, The New England journal of medicine.

[47]  H. C. Mann,et al.  The Impact Test , 1938 .

[48]  S. Dopson,et al.  The limitations of QALY: a literature review , 2016 .

[49]  A. Akobeng Understanding randomised controlled trials , 2005, Archives of Disease in Childhood.

[50]  A. Street,et al.  Economic analysis of service and delivery interventions in health care , 2018 .

[51]  J. Oakley,et al.  Estimating multi-parameter partial Expected Value of Perfect Information from a probabilistic sensitivity analysis sample : a non-parametric regression approach , 2013 .

[52]  T. Frieden Evidence for Health Decision Making — Beyond Randomized, Controlled Trials: The Changing Face of Clinical Trials , 2017, The New England journal of medicine.

[53]  John Brazier,et al.  Development of the Scharr HUD (Health Utilities Database) , 2013 .

[54]  E. Wilson A Practical Guide to Value of Information Analysis , 2015, PharmacoEconomics.

[55]  S. Giordano,et al.  Randomized Clinical Trials and Observational Studies: Is There a Battle? , 2016, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[56]  J. Coast,et al.  Use of economic evaluation in local health care decision-making in England: a qualitative investigation. , 2009, Health policy.

[57]  Jeremy E. Oakley,et al.  Estimating the Expected Value of Sample Information Using the Probabilistic Sensitivity Analysis Sample , 2015, Medical decision making : an international journal of the Society for Medical Decision Making.

[58]  G. Tomlinson,et al.  Techniques for estimating health care costs with censored data: an overview for the health services researcher , 2012, ClinicoEconomics and outcomes research : CEOR.

[59]  The NIHR at 10: transforming clinical research . , 2016, Clinical medicine.

[60]  S. Capewell,et al.  Return on investment of public health interventions: a systematic review , 2017, Journal of Epidemiology & Community Health.

[61]  M. Kelly,et al.  Designing and Undertaking a Health Economics Study of Digital Health Interventions. , 2016, American journal of preventive medicine.

[62]  Maarten J. IJzerman,et al.  Multiple Criteria Decision Analysis for Health Care Decision Making--An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force. , 2016, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[63]  M. Soares,et al.  Resolving the "Cost-Effective but Unaffordable" Paradox: Estimating the Health Opportunity Costs of Nonmarginal Budget Impacts. , 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[64]  E. McIntosh,et al.  Applied Health Economics for Public Health Practice and Research , 2019 .

[65]  A. Gasparrini,et al.  Interrupted time series regression for the evaluation of public health interventions: a tutorial , 2016, International journal of epidemiology.

[66]  M. Franklin,et al.  A modelling-based economic evaluation of primary-care-based fall-risk screening followed by fall-prevention intervention: a cohort-based Markov model stratified by older age groups , 2019, Age and ageing.

[67]  D. Rowen,et al.  International Regulations and Recommendations for Utility Data for Health Technology Assessment , 2017, PharmacoEconomics.

[68]  Christiane,et al.  World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects. , 2004, Journal international de bioethique = International journal of bioethics.

[69]  D. Spiegelhalter,et al.  Cancer Drugs Fund requires further reform , 2016, British Medical Journal.

[70]  C. Carroll,et al.  Evidence Review Group approaches to the critical appraisal of manufacturer submissions for the NICE STA process: a mapping study and thematic analysis. , 2011, Health technology assessment.

[71]  G. Richardson,et al.  Economic Evaluation of Environmental Interventions: Reflections on Methodological Challenges and Developments , 2018, International journal of environmental research and public health.

[72]  Joshua T. Cohen,et al.  When cost-effective interventions are unaffordable: Integrating cost-effectiveness and budget impact in priority setting for global health programs , 2017, PLoS medicine.

[73]  Rob Anderson,et al.  Realism and resources: Towards more explanatory economic evaluation , 2016, Evaluation.

[74]  D. Rovithis Do health economic evaluations using observational data provide reliable assessment of treatment effects? , 2013, Health Economics Review.

[75]  S. Griffin,et al.  Estimating Social Variation in the Health Effects of Changes in Health Care Expenditure , 2020, Medical decision making : an international journal of the Society for Medical Decision Making.

[76]  Jessica M Franklin,et al.  Alternative approaches for confounding adjustment in observational studies using weighting based on the propensity score: a primer for practitioners , 2019, BMJ.

[77]  Bengt Jönsson,et al.  Ten arguments for a societal perspective in the economic evaluation of medical innovations , 2009, The European Journal of Health Economics.

[78]  Elisabeth Fenwick,et al.  Value of Information Analytical Methods: Report 2 of the ISPOR Value of Information Analysis Emerging Good Practices Task Force. , 2020, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[79]  R. Meacock Methods for the economic evaluation of changes to the organisation and delivery of health services: principal challenges and recommendations , 2018, Health Economics, Policy and Law.

[80]  G. Rinkel,et al.  Decision analysis to complete diagnostic research by closing the gap between test characteristics and cost-effectiveness. , 2009, Journal of clinical epidemiology.

[81]  Wolzt,et al.  World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects. , 2003, The Journal of the American College of Dentists.

[82]  Matthew Franklin,et al.  Self-reported and routinely collected electronic healthcare resource-use data for trial-based economic evaluations: the current state of play in England and considerations for the future , 2019, BMC Medical Research Methodology.

[83]  J. Poterba PUBLIC FINANCE AND PUBLIC CHOICE , 1998, National Tax Journal.

[84]  Nicholas R Latimer,et al.  Survival Analysis for Economic Evaluations Alongside Clinical Trials—Extrapolation with Patient-Level Data , 2013, Medical decision making : an international journal of the Society for Medical Decision Making.

[85]  Anthony J. Culyer,et al.  The NICE Cost-Effectiveness Threshold , 2012, PharmacoEconomics.

[86]  Jeremy Cherfas,et al.  Parachute use to prevent death and major trauma when jumping from aircraft: randomized controlled trial , 2018, British Medical Journal.

[87]  David Moher,et al.  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. , 2013, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[88]  S. Whyte,et al.  Distributional cost-effectiveness analysis of health care programmes--a methodological case study of the UK Bowel Cancer Screening Programme. , 2013, Health economics.

[89]  S. Bryan,et al.  Cyclooxygenase-2 selective non-steroidal anti-inflammatory drugs (etodolac, meloxicam, celecoxib, rofecoxib, etoricoxib, valdecoxib and lumiracoxib) for osteoarthritis and rheumatoid arthritis: a systematic review and economic evaluation. , 2008, Health technology assessment.

[90]  Joanna Coast,et al.  A systematic review of the use of economic evaluation in local decision-making. , 2008, Health policy.

[91]  P. McFarlane,et al.  Cost-Effectiveness Analysis of Therapies for Chronic Kidney Disease Patients on Dialysis , 2012, PharmacoEconomics.

[92]  J. Brazier,et al.  An Updated Systematic Review of Studies Mapping (or Cross-Walking) Measures of Health-Related Quality of Life to Generic Preference-Based Measures to Generate Utility Values , 2019, Applied Health Economics and Health Policy.

[93]  P. Austin,et al.  Using propensity score methods to analyse individual patient-level cost-effectiveness data from observational studies , 2008 .

[94]  D. H. Lees,et al.  Epidemiology for the Uninitiated , 1980 .

[95]  Christopher Jackson,et al.  Value of Information: Sensitivity Analysis and Research Design in Bayesian Evidence Synthesis , 2017, Journal of the American Statistical Association.

[96]  Identifying patient-level health and social care costs for older adults discharged from acute medical units in England. , 2014, Age and ageing.

[97]  K. Schulz,et al.  Descriptive studies: what they can and cannot do , 2002, The Lancet.

[98]  D. Meltzer,et al.  Pharmacoeconomic Guidelines Should Prescribe Inclusion of Indirect Medical Costs! A Response to Grima et al. , 2013, PharmacoEconomics.

[99]  R. Cookson,et al.  Distributional cost-effectiveness analysis in low- and middle-income countries: illustrative example of rotavirus vaccination in Ethiopia , 2018, Health policy and planning.

[100]  J. Grimshaw,et al.  The methodological quality of economic evaluations of guideline implementation into clinical practice: a systematic review of empiric studies. , 2007, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[101]  M. Deidda,et al.  A framework for conducting economic evaluations alongside natural experiments , 2019, Social science & medicine.

[102]  Grazyna Adamiak,et al.  Methods for the economic evaluation of health care programmes, 3rd ed , 2006 .

[103]  Ties Hoomans,et al.  Economic evaluation of implementation strategies in health care , 2014, Implementation Science.

[104]  J. Concato,et al.  Randomized, controlled trials, observational studies, and the hierarchy of research designs. , 2000, The New England journal of medicine.

[105]  Peter Littlejohns,et al.  Cost effective but unaffordable: an emerging challenge for health systems , 2017, British Medical Journal.

[106]  J. Nelson,et al.  Evidence of bias in estimates of influenza vaccine effectiveness in seniors. , 2006, International journal of epidemiology.

[107]  M. Franklin,et al.  Economic Evaluations Alongside Efficient Study Designs Using Large Observational Datasets: the PLEASANT Trial Case Study , 2017, PharmacoEconomics.

[108]  S. Wilson Methods for the economic evaluation of health care programmes , 1987 .

[109]  Andy Stergachis,et al.  The Role of Cost—Consequence Analysis in Healthcare Decision—Making , 1998, PharmacoEconomics.

[110]  J. Lewis,et al.  Statistical principles for clinical trials (ICH E9): an introductory note on an international guideline. , 1999, Statistics in medicine.

[111]  T J Ulahannan,et al.  Decision Making in Health and Medicine: Integrating Evidence and Values , 2002 .

[112]  P. Kind,et al.  The use of QALYs in clinical and patient decision-making: issues and prospects. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[113]  S. Sullivan,et al.  Budget impact analysis-principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force. , 2014, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[114]  J. Sekhon,et al.  Regression-adjusted matching and double-robust methods for estimating average treatment effects in health economic evaluation , 2013, Health Services and Outcomes Research Methodology.

[115]  S. Hinde,et al.  Understanding and addressing the challenges of conducting quantitative evaluation at a local level: a worked example of the available approaches , 2019, BMJ Open.

[116]  G. Loomes,et al.  The use of QALYs in health care decision making. , 1989, Social science & medicine.

[117]  Matthew Franklin,et al.  An Educational Review About Using Cost Data for the Purpose of Cost-Effectiveness Analysis , 2019, PharmacoEconomics.

[118]  James Lomas,et al.  Incorporating Affordability Concerns Within Cost-Effectiveness Analysis for Health Technology Assessment. , 2019, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[119]  S. Dixon,et al.  Estimating the Cost-Effectiveness of Implementation: Is Sufficient Evidence Available? , 2016, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[120]  S. Walker,et al.  Striving for a Societal Perspective: A Framework for Economic Evaluations When Costs and Effects Fall on Multiple Sectors and Decision Makers , 2019, Applied Health Economics and Health Policy.

[121]  Jeremy E. Oakley,et al.  Estimating Multiparameter Partial Expected Value of Perfect Information from a Probabilistic Sensitivity Analysis Sample , 2013, Medical decision making : an international journal of the Society for Medical Decision Making.

[122]  N. Freemantle,et al.  An Educational Review of the Statistical Issues in Analysing Utility Data for Cost-Utility Analysis , 2015, PharmacoEconomics.

[123]  Timo Siepmann,et al.  Randomized controlled trials – a matter of design , 2016, Neuropsychiatric disease and treatment.

[124]  R. Faria,et al.  Daclatasvir for the Treatment of Chronic Hepatitis C: A Critique of the Clinical and Economic Evidence , 2016, PharmacoEconomics.

[125]  A. Culyer COST, CONTEXT, AND DECISIONS IN HEALTH ECONOMICS AND HEALTH TECHNOLOGY ASSESSMENT , 2018, International Journal of Technology Assessment in Health Care.

[126]  D. Hunter Efficiency and the proposed reforms to the NHS research ethics system , 2007, Journal of Medical Ethics.

[127]  S. Pearson The ICER Value Framework: Integrating Cost Effectiveness and Affordability in the Assessment of Health Care Value. , 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.