Good Practices for Synthesizing and Using Evidence in Health Care Decision Making?

Transcription

1 Good Practices for Synthesizing and Using Evidence in Health Care Decision Making? An ISPOR Workshop presented by the ISPOR HTA Council Working Group ISPOR 22 nd Annual International Meeting Boston, MA, USA May 22, 2017

2 Good Practices for Synthesizing and Using Evidence in Health Care Decision Making? Finn Børlum Kristensen, MD, PhD, Professor, Faculty of Health Sciences, University of Southern Denmark, Hillerød, Denmark Don Husereau, MSc, BPharm, Senior Associate, Institute of Health Economics, Edmonton, and Adjunct Professor, University of Ottawa, Ottawa, ON, Canada Dan Ollendorf, PhD, Chief Scientific Officer, Institute for Clinical and Economic Review, Boston, MA, USA Marc Berger, MD, Vice President, Real World Data and Analytic, Pfizer, Inc., New York, NY, USA 2

3 Leadership Group Co-Chairs: Finn Børlum Kristensen, MD, PhD, Professor, Faculty of Health Sciences, University of Southern Denmark, Hillerød, Denmark Don Husereau, MSc, BPharm, Senior Associate, Institute of Health Economics, Edmonton, and Adjunct Professor, University of Ottawa, Ottawa, ON, Canada Leadership Group: Federico Augustovski, MD, MS, PhD, Director, Economic Evaluations and HTA Department, Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, Argentina Marc Berger, MD, VP Real World Data and Analytics, Pfizer, Inc., New York, NY, USA Kenneth Bond, MA, BEd, BA, is Director, Patient Engagement, Ethics and International Affairs, Canadian Agency for Drugs and Technologies in Health (CADTH), Ottawa, Canada Andrew Booth, PhD, Reader in Evidence Based Information Practice and Director of Information, ScHARR, The University of Sheffield, Sheffield, England, UK John F. P. Bridges, PhD, Assistant Professor, Department of Health Policy & Management Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA Michael Drummond, DPhil, MCom, BSc, Professor of Health Economics, University of York, York, England, UK Jeremy Grimshaw, MBCHB, PHD, FRCGP, FCAHS, Director, Cochrane Canada and Professor of Medicine, University of Ottawa, Ottawa, Canada 3

4 Leadership Group (Con t) Mirjana Huić, MD, MSc, Assistant Director, Agency for Quality and Accreditation in Health Care and Social Welfare, Zagreb, Croatia Maarten J. IJzerman, PhD, Professor of Clinical Epidemiology & Health Technology Assessment (HTA); Head, Department of Health Technology & Services Research, University of Twente, Enschede, The Netherlands Egon Jonsson, PhD, Executive Director & CEO of the Institute of Health Economics, Edmonton, Canada Daniel Ollendorf, MPH, PhD, Chief Scientific Officer, Institute for Clinical and Economic Review (ICER), Boston, MA, USA Alric Rüther, MD, PhD, Head, International Affairs, Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany Uwe Siebert, MD, MPH, MSc, ScD, Professor of Public Health, Department of Public Health, Medical Decision Making and Health Technology Assessment (HTA), University of Health Sciences, Medical Informatics and Technology (UMIT), Hall in Tirol, Austria Jitendar Sharma, PhD, Director & CEO, AP MedTech Zone & Advisor (Health), Department of Health & Family Welfare, Andhra Pradesh, India Allan Wailoo, PhD, MSc, MA, Professor of Health Economics, ScHARR, University of Sheffield and Director, NICE Decision Support Unit, Sheffield, England, UK 4

5 Rationale Behind the Working Group There is an increasing demand worldwide for capacity building, education, and greater consistency in HTA-based decision making Identifying good practices in using evidence to inform population-based health care decision making (after regulatory approval) is an important step forward 5

6 Objective of the Working Group Provide an up-to-date review of current practices and identify best practices in the use of outcomes research evidence to inform population-based decision making for pharmaceuticals and medical devices, with an emphasis on HTA approaches 6

7 Decision-making steps Health care technology decision problem Analysis Recommendation Decision HTA questions What level of support? What is the problem and what research is needed? How should research be conducted? What does the research say? How should results be put into context? What should the decision be? HTA practices Defining the HTA process - Structure and governance / organizational aspects (e.g., government-/ health insurance based) - Underlying principles (e.g., accountability for reasonableness, formal agreement with decision-maker) - Priority setting process (e.g., application process for new medicines) - Framing and scoping research process - What output from HTA is requested? - Critical Review of Evidence - CEA/CUA Models - Indirect Treatment Comparisons - Budget Impact Models - Summary / Recommendation Interpreting research - Local guidance for conduct of research - Standards / checklists for researchers - Peer review of HTA research - Experts or expert panels Contextualizing evidence - What is the role of HTA vs Decision Marker? - What considerations should be made explicit? - Strength of the evidence base - Affordability - Acceptability - Other - How should these considerations be assessed? - Citizen s councils / surveys; Qualitative research; Using thresholds; Value frameworks; Deliberative processes; Stakeholder engagement; Voting rules / weighted / nominal group techniques Implementing HTA - Communicating the output of HTA (e.g., recommendation) - Defining involvement in HTA process (e.g., arms length) - Transparency

8 Are there best practices in considering equity and use use of CE thresholds? Don Husereau, MSc ISPOR Good Practices for Synthesizing and Using Evidence Workshop May 22 nd, 2017

9 Decision-making steps Health care technology decision problem Analysis Recommendation Decision HTA questions What level of support does the decision-maker need? What is the problem and what research is needed? How should research be conducted? What does the research say? What do we know? What do we infer? What don t we know? How should the results of the research be put into context? What should the decision be? HTA practices Define the HTA process - Structure and governance / organizational aspects (e.g., government-/ health insurance based) - Underlying principles (e.g., accountability for reasonableness, formal agreement with decision-maker) - Priority setting process (e.g., application process for new medicines) - Framing and scoping research process - What output from HTA is requested? - Critical Review of Evidence - CEA/CUA Models - Indirect Treatment Comparisons - Budget Impact Models - Summary / Recommendation Interpreting research - Local guidance for conduct of research - Standards / checklists for researchers - Peer review of HTA research - Experts or expert panels Contextualizing evidence - What is the role of HTA vs Decision Marker? - What considerations should be made explicit? - Strength of the evidence base - Affordability - Acceptability - Other - How should these considerations be assessed? - Citizen s councils / surveys; Qualitative research; Using thresholds; Value frameworks; Deliberative processes; Stakeholder engagement; Voting rules / weighted / nominal group techniques Implementing HTA - Communicating the output of HTA (e.g., recommendation) - Defining involvement in HTA process (e.g., arms length) - Transparency

10 It is the process of evidence assimilation and interpretation, along with the application of contextual judgments of both fact and value, which constitutes the assessment. -A.Culyer [1] [1] Culyer AJ. HTA Algorithm or Process?: Comment on Expanded HTA: Enhancing Fairness and Legitimacy. International Journal of Health Policy and Management Aug;5(8):501.

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12 Consider the Following Intervention 1 Intervention 2 Population coverage Q 0.5Q Cost / person P 2P Total cost PQ=$1.1M PQ=$1.1M QALYs produced ICER $1100/QALY $1000/QALY. Adapted from: Ubel PA, DeKay ML, Baron J, Asch DA. Cost-Effectiveness Analysis in a Setting of Budget Constraints, Is It Equitable? N Engl J Med 1996; 334: International Society for Pharmacoeconomics and Outcomes Research 13

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14 Equity issues in HTA? Review of 98 reports from 19 agencies revealed that equity was not a standard consideration in HTA report production. [2] Typivally ignored in economic evaluation [3] [4] although when when performed focus on: (i) review of background information on equity, (ii) health inequality impact assessment, (iii) analysis of the opportunity cost of equity, and (iv) equity weighting of health outcomes. Equity can also be embedded into scoping, ethical analyses, and methods for contextualizing and evaluating evidence.[1] [2] Panteli D, Kreis J, Busse R. CONSIDERING EQUITY IN HEALTH TECHNOLOGY ASSESSMENT: AN EXPLORATORY ANALYSIS OF AGENCY PRACTICES. Int J Technol Assess Health Care Jan;31(5): [3] Johri M, Norheim OF. Can cost-effectiveness analysis integrate concerns for equity? Systematic review. Int J Technol Assess Health Care Apr;28(2): [4] Cookson R, Drummond M, Weatherly H. Explicit incorporation of equity considerations into economic evaluation of public health interventions. Health Econ Policy Law Apr;4(Pt 2): [1] Culyer AJ. HTA Algorithm or Process?: Comment on Expanded HTA: Enhancing Fairness and Legitimacy. International Journal of Health Policy and Management Aug;5(8):

15 Some tools, no consensus A WHO tool highlighted the need to consider equity throughout the process[5] Culyer and Bombard developed a 13-question checklist intended to be used as part of the HTA process which can be used for [6]: scoping of the agenda prior to the selection of candidate interventions and their comparators for HTA; the accompanying background briefing for decision makers, including systematic and other reviews; a tool to help to structure the discussion and composition of professional and lay advisory groups during the assessment process. [5] Equity-Oriented Toolkit [Internet]. [cited 2017 May 17]. Available from: [6] Culyer AJ, Bombard Y. An equity framework for health technology assessments. Med Decis Making Jun;32(3):

16 Are there best practices for incorporating societal values? Don Husereau, MSc ISPOR Good Practices for Synthesizing and Using Evidence Workshop May 22 nd, 2017

17 Incorporating Social Values/Moral Principles When conducting an appraisal of a technology, where societal values and moral principles are considered in a recommendation, we should aspire to use an approach that reaches these ideals to the greatest extent possible (time and resource limited). Recall Drummond HTA Principles Those conducting HTAs should actively engage all stakeholder groups (e.g., professional bodies, patient organizations, manufacturers) The link between HTA findings and decision-making processes needs to be transparent and clearly defined 2016 International Society for Pharmacoeconomics and Outcomes Research 17

18 Some approaches, no HTA standards Stakeholder engagement increasingly used A growing number of HTA agencies are now incorporating the perspective of patients and citizens through patient and stakeholder engagement, qualitative research, or quantitative research on patient preference Principles of patient engagement developed [7] Deliberative processes have been adopted as a mechanism to bring appropriate actors together when there are important societal decisions that involve evidence and value. Weighted preferences and MCDA techniques GRADE and ISPOR guidance [7] [18] [8] Culyer AJ. Deliberative Processes in Decisions About Health Care Technologies: Combining Different Types of Evidence, Values, Algorithms and People

19 Stakeholder Engagement: What do Best Practices Look Like? Dan Ollendorf, PhD ISPOR Good Practices for Synthesizing and Using Evidence Workshop May 22 nd, 2017

20 Stakeholder Engagement Who? Most formalized processes involve all topic-relevant stakeholders Patients, patient advocacy groups, manufacturers, payers, health systems, clinical experts and clinical societies Often missing: consumers (i.e., those without the disease who might be affected by priority-setting) When? Ideally, all the time (suggest topics, consult on process improvements) For each topic, some involvement from beginning to end HTA should not expect stakeholders to just show up 20

21 Stakeholder Engagement: Patients Informal/Qualitative: Written or spoken testimonials Consult/comment on processes, documents Survey their own communities Quantitative: Preference elicitation, conjoint analysis, discrete choice experiments MCDA or other preference-weighting methods Deliberative: Weigh evidence as a lay committee member Join implementation workgroup 21

22 Stakeholder Engagement: Industry Data: Supply data to support evidence synthesis / economic evaluation Complete manufacturer submission Review and comment on HTA organization analysis Informal/Qualitative: Suggest clinical experts for consultation Consult/comment on processes, documents Attend public deliberation, comment as necessary Join HTA advisory council Deliberative: Weigh evidence as an ex-officio committee member Join implementation workgroup 22

23 Role of Affordability and Use of HTA to Inform Pricing Marc L Berger, MD ISPOR Good Practices for Synthesizing and Using Evidence Workshop May 22 nd, 2017

24 Affordability, Price, and Value Affordable: that which can be afforded; believed to be within one s financial means Value: relative worth, merit, or importance; monetary or material worth, as in commerce or trade; the worth of something in terms of the amount of other things which it can be exchanged or in terms of some medium of exchange Price: the sum or amount of money or its equivalent for which anything is bought, sold, or offered for sale. Underlying Assumption of Market based Assessment of Value including Availability of Credit Budget Impact Depends on Size of Budget & Financing Options 24

25 Affordability, Price, and Value Affordable: that which can be afforded; believed to be within one s financial means Value: relative worth, merit, or importance; monetary or material worth, as in commerce or trade; the worth of something in terms of the amount of other things which it can be exchanged or in terms of some medium of exchange Price: the sum or amount of money or its equivalent for which anything is bought, sold, or offered for sale. Underlying Assumption of Market based Assessment of Value including Availability of Credit Budget Impact Depends on Size of Budget & Financing Options 25

26 Implementing HTA Professor Finn Børlum Kristensen, MD, PhD ISPOR Good Practices for Synthesizing and Using Evidence Workshop May 22 nd, 2017

27 Decision-making steps Health care technology decision problem Analysis Recommendation Decision HTA questions What level of support does the decision-maker need? What is the problem and what research is needed? How should research be conducted? What does the research say? What do we know? What do we infer? What don t we know? How should the results of the research be put into context? What should the decision be? HTA practices Define the HTA process - Structure and governance / organizational aspects (e.g., government-/ health insurance based) - Underlying principles (e.g., accountability for reasonableness, formal agreement with decision-maker) - Priority setting process (e.g., application process for new medicines) - Framing and scoping research process - What output from HTA is requested? - Critical Review of Evidence - CEA/CUA Models - Indirect Treatment Comparisons - Budget Impact Models - Summary / Recommendation Interpreting research - Local guidance for conduct of research - Standards / checklists for researchers - Peer review of HTA research - Experts or expert panels Contextualizing evidence - What is the role of HTA vs Decision Marker? - What considerations should be made explicit? - Strength of the evidence base - Affordability - Acceptability - Other - How should these considerations be assessed? - Citizen s councils / surveys; Qualitative research; Using thresholds; Value frameworks; Deliberative processes; Stakeholder engagement; Voting rules / weighted / nominal group techniques Implementing HTA - Communicating the output of HTA (e.g., recommendation) - Defining involvement in HTA process (e.g., arms length) - Transparency

28 Decision-making steps Health care technology decision problem Analysis Recommendation Decision HTA questions What level of support does the decision-maker need? What is the problem and what research is needed? How should research be conducted? What does the research say? What do we know? What do we infer? What don t we know? How should the results of the research be put into context? What should the decision be? HTA practices Define the HTA process - Structure and governance / organizational aspects (e.g., government-/ health insurance based) - Underlying principles (e.g., accountability for reasonableness, formal agreement with decision-maker) - Priority setting process (e.g., application process for new medicines) - Framing and scoping research process - What output from HTA is requested? - Critical Review of Evidence - CEA/CUA Models - Indirect Treatment Comparisons - Budget Impact Models - Summary / Recommendation Interpreting research - Local guidance for conduct of research - Standards / checklists for researchers - Peer review of HTA research - Experts or expert panels Contextualizing evidence - What is the role of HTA vs Decision Marker? - What considerations should be made explicit? - Strength of the evidence base - Affordability - Acceptability - Other - How should these considerations be assessed? - Citizen s councils / surveys; Qualitative research; Using thresholds; Value frameworks; Deliberative processes; Stakeholder engagement; Voting rules / weighted / nominal group techniques Implementing HTA - Communicating the output of HTA (e.g., recommendation) - Defining involvement in HTA process (e.g., arms length) - Transparency