Ensuring value for money in health care: the role of health technology assessment in the European Union

on Health Systems and Policies

European

Corinna Sorenson, Michael Drummond, Panos Kanavos

Observatory Studies Series No 11

ENSURING VALUE FOR MONEY IN HEALTH CARE The role of health technology assessment

in the European Union

Ensuring value for money in health care

The European Observatory on Health Systems and Policies supports and promotes evidence- based health policy-making through comprehensive and rigorous analysis of health systems in Europe. It brings together a wide range of policy-makers, academics and practitioners to analyse trends in health reform, drawing on experience from across Europe to illuminate policy issues.

The European Observatory on Health Systems and Policies is a partnership between the World Health Organization Regional Office for Europe, the Governments of Belgium, Finland, Greece, Norway, Slovenia, Spain and Sweden, the Veneto Region of Italy, the European Investment Bank, the Open Society Institute, the World Bank, the London School of Economics and Political Science and the London School of Hygiene & Tropical Medicine.

Ensuring value for money in health care The role of health technology assessment in the European Union

Corinna Sorenson

Michael Drummond

Panos Kanavos

Keywords:

TECHNOLOGY ASSESSMENT, BIOMEDICAL – organization and administration

OUTCOME AND PROCESS ASSESSMENT (HEALTH CARE)

EVIDENCE-BASED MEDICINE

DECISION MAKING

COST-BENEFIT ANALYSIS

EUROPEAN UNION

© World Health Organization 2008, on behalf of the European Observatory on Health Systems and Policies

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ISBN 978 92 890 7183 3

Printed in the United Kingdom by MPG Books Ltd, Bodmin, Cornwall.

Contents

List of abbreviations vi

List of tables, figures and boxes x

About the authors xi

Acknowledgements xii

Executive summary xiii

1 Project overview, objectives and methods 1

2 Background on innovation and HTA 3

3 HTA and decision-making in Europe 9

4 Conclusions 33

Appendices: Select country case studies

Appendix 1: Sweden 39

Appendix 2: The Netherlands 55

Appendix 3: Finland 72

Appendix 4: France 85

Appendix 5: Germany 100

Appendix 6: United Kingdom 114

References 145

List of abbreviations

ACD Appraisal consultation document.

Afssa Agence Française de Sécurité Sanitaire des Aliments.

Afssaps Agence Française de Sécurité Sanitaire des Produits de Santé.

Afsset Agence Française de Sécurité Sanitaire de l’Environnement et du Travail.

ANAES Agence Nationale d’Accréditation et d’Evaluation en Santé National.

ANDEM Agence Nationale pour le Déement de l’Evaluation Médicale.

ASMR Amélioration du Service Médical Rendu.

ATC Anatomical therapeutic chemical.

BfArM Bundesinstitut für Arzneimittel und Medizinprodukte.

BMG Bundesministerium für Gesundheit.

BZgA Bundeszentrale für gesundheitliche Aufklärung.

CAMTO Centre for Assessment of Medical Technology.

CBA Cost benefit analysis.

CBO Dutch Institute for Healthcare Improvement.

CDC Centers for Disease Control and Prevention.

CE Cost-effectiveness.

CEA Cost-effectiveness analysis.

CEDIT Comite d’Évaluation et de Diffusion des Innovations Technologiques.

CEMTV Center for Evaluering og Medicinsk Teknologivurdering (DACEHTA).

CEPP Commission d’Évaluation des Produits et Prestations.

CEPS Comité Économique des Produits de Santé.

CES College des Économistes de la Santé.

CFH Commissie Farmaceutische Hulp (Pharmaceutical Care Committee).

CMA Cost minimization analysis.

CUA Cost-utility analysis.

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vii

CVZ College voor Zorgverzekeringen (Health Care Insurance Board).

DAHTA Deutsche Agentur für Health Technology Assessment.

DIMDI Deutschen Institut für Medizinische Dokumentation und Information.

ERNIE Evaluation and review of NICE implementation evidence database.

EUnetHTA European network for Health Technology Assessment.

FinOHTA Terveydenhuollon menetelmien arviointiyksikkö (Finnish Office for Health Technology Assessment).

HTA Health technology assessment.

HTAi Health Technology Assessment International.

IHE Institutet för hälso-och sjukvårdsekonomi (Swedish Institute for Health Economics.

IMOR Institute for Medical Outcome Research.

INAHTA International Network of Agencies for Health Technology Assessment.

Inserm Institut National de la Santé et de la Recherche Médicale.

IOM Institute of Medicine.

IQWiG Gemeinsamen Bundesausschuss/Institut für Qualitat und Wirtschaftlichkeit im Gesundheitswesen.

ISPOR International Society for Pharmacoeconomics and Outcomes Research.

ITAS Institut für Technik-folgen-abschätzung und Systemanalyse.

LFN Läkemedelsförmånsnämnden (Pharmaceutical Benefits Board).

MPA Läkemedelsverket (Medical Products Agency).

NAM Lääkelaitos (National Agency for Medicines).

NCC National Collaborating Centre.

NCCHTA National Coordinating Centre for Health Technology Assessment.

NHG Nederlands Huisartsen Genootschap (Dutch College of General Practitioners).

NHI National health insurance.

NHS National Health Service (United Kingdom).

List of abbreviations

Ensuring value for money in health careviii

NICE National Institute for Health and Clinical Excellence.

NoMA Statens legemiddelverk (Norwegian Medicines Agency).

NSF National service framework.

NWO Nederlandse Organisatie voor Wetenschappelijk Onderzoek (Netherlands Organisation for Scientific Research).

OECD Organisation for Economic Co-operation and Development.

PPB Lääkkeiden hintalautakunta (Pharmaceuticals Pricing Board).

PCT Primary care trust.

QALY Quality-adjusted life year.

QIS NHS Quality Improvement Scotland.

QUORUM Improving the quality of reporting of meta-analyses of randomized controlled trials.

R&D Research and development.

RCT Randomized controlled trial.

RFV Riksforsakringsverket (National Social Insurance Board).

ROHTO Lääkehoidon kehittämiskeskus (Centre for Pharmacotherapy Development).

RVZ Raad voor Volksgezondeid & Zorg (Council for Public Health and Health Care).

SBU Statens beredning för medicinsk utvärdering (Swedish Council on Technology Assessment in Health Care).

ScHARR School of Health and Related Research, University of Sheffield.

SHA Strategic health authority.

SHI Statutory health insurance.

SHTAC Southampton Health Technology Assessments Centre.

SMC Scottish Medicines Consortium.

SMR Therapeutic value.

STA Single technology appraisal.

STAKES National Research and Development Centre for Welfare and Health.

TAB Büro für Technikfolgen-Abschätzung beim Deutschen Bundestag.

TAG Technical appraisal guidance.

TAR Technology assessment report.

TNO Kennis voor zaken (Netherlands Organisation for Applied Scientific Research).

ix

UNCAM Union Nationale des Caisses d’Assurance Maladie.

UNOC Union Nationale des Organismes d’Assurance Maladie Complémentaire.

WHO World Health Organization.

WTP Willingness to pay.

ZonMw Nederlandse organisatie voor gezondheidsonderzoek en zorginnovatie (Netherlands Organisation for Health Research and Development).

List of abbreviations

List of tables, figures and boxes

Tables

Table 3.1 Institutions and advisory bodies responsible for HTA activities.

10

Table 3.2 Criteria for assessment. 17

Table A1.1 Overview of HTA governance, processes and role in decision-making in Sweden.

50

Table A2.1 Overview of HTA governance, processes and role in decision-making in the Netherlands.

67

Table A3.1 Overview of HTA governance, processes and role in decision-making in Finland.

81

Table A4.1 Overview of HTA governance, processes and role in decision-making in France.

96

Table A5.1 Overview of HTA governance, processes and role in decision-making in Germany.

110

Table A6.1 Examples of published and planned NICE guidance. 135

Table A6.2 Overview of HTA governance, processes and role in decision-making in the United Kingdom.

139

Figures

Fig. A1.1 Principal reforms in the Swedish pharmaceutical market. 41

Fig. A2.1 Reimbursement decision process in the Netherlands. 57

Boxes

Box 3.1 Methods of disseminating and implementing recommendations.

31

Box A4.1 High Health Authority in France. 86

Box A4.2 Procedures for a standard HTA at ANAES. 91

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About the authors

Corinna Sorenson (MPH, MHSA) is a research officer at LSE Health, London School of Economics and Political Science and a Ph.D. candidate in the Department of Social Policy. Before joining LSE, Ms Sorenson served as a senior policy and planning analyst for the United States’ Food and Drug Administration. Also, she has held various consulting positions in health policy and health economics research.

Michael Drummond (D.Phil.) is a professor of Health Economics at the University of York, Centre for Health Economics. He has undertaken evaluations over a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. In addition, Professor Drummond has authored more than 500 scientific papers and currently serves as President- Elect of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).

Panos Kanavos (Ph.D.) is a senior lecturer in international health policy in the Department of Social Policy and Merck Fellow in Pharmaceutical Economics at LSE Health, London School of Economics and Political Science. He has acted as an adviser to a number of international governmental and non- governmental organizations including the World Bank, WHO, Organisation for Economic Co-operation and Development, American Association of Retired Persons and ministries of health in over 14 transition and developing countries.

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Acknowledgements

This project was part of a year-long study: Financing sustainable health care in Europe. It was endorsed by Luxembourg’s Ministry of Health, Sitra and the Finnish Innovation Fund.

The authors greatly appreciate the helpful comments and suggestions and time taken by the final reviewers – Dr David Taylor and Dr Frans Rutten. We are also grateful to Dr Elias Mossialos for his insightful guidance and contributions to the project.

This study was made possible with the financial support of Pfizer Inc.

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This report addresses the concepts and controversy surrounding health technology assessment (HTA) in Europe, with a particular focus on selected Member States including Sweden, the Netherlands, Finland, France, Germany and the United Kingdom.

Extensive review of these HTA systems produced a number of key findings relevant to a wide range of stakeholders including policy-makers, HTA bodies, manufacturers, health-care professionals and patient organizations.

1. HTA plays a major part in evidence-based decision-making. Without good evidence, the uptake and diffusion of health technologies is likely to be influenced by a range of social, financial and institutional factors. This may result in suboptimal health outcomes and inefficient use of resources.

2. Innovation and the actual needs of the health-care system should be linked more closely. Products that provide the most value for investment must be identified and supported and their manufacturers rewarded with appropriate reimbursement and pricing schemes.

3. Many countries have dedicated HTA bodies, but with somewhat unclear and disparate roles and responsibilities. Groups involved in reimbursement and pricing decisions often differ from those affiliated with independent HTA assessment and clinical guidance development. Divergent processes and roles may hinder the effectiveness and efficacy of the decision-making process and lead to unnecessary duplication of efforts and resource use.

4. Most review bodies involve a range of stakeholders including physicians, health economists, pharmacists and patient group representatives. Most

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Executive summary

Ensuring value for money in health carexiv

agencies support some level of involvement from patients and consumers, and a greater role for industry representatives has been proposed. More stakeholder involvement is needed to improve the HTA processes and the implementation of decisions and related policy. This is true of broad HTA networks and partnerships (such as EUnetHTA) that can enhance collaboration between agencies and facilitate innovation in HTA processes and methods.

5. Some countries consider the evidence and resources required to conduct an assessment, as well as its relevance to the primary clinical and/or policy question. An assessment will not be helpful if there are insufficient data, and may delay access to new treatments. Clinical and policy relevance is especially important as HTAs are useful only if they are expected to contribute to the decision-making process.

6. Generally, HTA processes lack transparency – from prioritizing decision criteria to stakeholder involvement. More transparency is necessary to ensure an open, systematic and unbiased decision-making process.

7. There is limited information on HTA’s use in identifying areas of disinvestment. More attention should be paid to identifying topics for potential disinvestment so that ineffective and obsolete products and practices do not remain in the health-care system.

8. Assessments should adopt a broader definition of value and product benefit by considering patient preferences, quality, equity, efficiency and product acceptability among a wide range of stakeholders. Further exploration to reveal how non-quantifiable factors (e.g. preferences, equity concerns) are accounted for in assessments and decision-making will enable the social implications and constraints of efficient and equitable health to be addressed effectively. In pursuit of this aim, the opinions and experiences of health professionals and individual patients are needed to understand the real-world application and use of a product.

9. Different countries have diverse technical requirements so it is important that the choice of parameters and methods is substantiated and well- documented. The model and resulting analysis should be as transparent as possible, and shared with all parties involved in its development.

10. Several technical and methodological hurdles remain. These require further investigation and include the summary measures’ ability to capture other benefits important to patients and the public; generalizability of studies beyond the particular setting or country; inability to account for the opportunity costs of new and expensive technologies; and comparability of measures to ascertain patient preferences. Moreover, as the modelling of cost-effectiveness (CE) becomes more complex, more resources

xv

should be devoted to assessing new methods and the resulting impact on uncertainty in decision-making.

11. Assessments should take account of indirect benefits and costs. It would be helpful if review bodies could agree on the inclusion of additional years of life provided by new treatments, as well as opportunity costs related to leisure activities. A better understanding of threshold values, other decision criteria and their application to the overall decision process is needed.

12. The timing of assessments can affect significantly the decision-making process and patient access. Programmes have been introduced to provide more timely information on important products. These programmes should be monitored and evaluated for effectiveness and resulting impact on access to new technologies.

13. In order to maintain the accuracy of assessments and ensure that the most valuable products are on the market, re-evaluation is key to the HTA process. Often the data needed to confirm the cost- and clinical- effectiveness of a technology can be found only after practical application in the market. This is particularly true for novel products and technologies undergoing fast-track assessment. Systems should be created to allow new clinical and health economic information to be introduced during the assessment process and following market entry. However, there must be safeguards to prevent any re-evaluation leading to inefficiency, resource burden and delayed access to treatments.

14. There is a lack of understanding and evidence on the practical application of HTA from both a process (decision-making) and an outcome (health outcomes, care delivery, costs, research innovation) perspective. More focused research is needed in these areas.

15. The scope of HTA has focused predominantly on pharmaceuticals and, less frequently, on other medical technologies such as devices. There should be further exploration of applying the principles and methods of economic evaluation to preventive measures. Additional research should establish whether (and in what circumstances) such assessments have been conducted, and identify the opportunities and challenges.

Overall, HTA can play a valuable role in health care decision-making, but the process must be transparent, timely, relevant, in-depth and usable. Assessments need to use robust methods and be supplemented by other important criteria. Maximization of HTA will enhance potential decision-makers’ ability to implement decisions that capture the benefits of new technologies, overcome uncertainties and recognize the value of innovation, all within the constraints of overall health system resources.

Executive summary

Almost all Member States have experienced exponential growth in the introduction and uptake of health technologies in recent years – new medicines and diagnostic tools, telemedicine and surgical equipment. Such innovation provides enormous opportunity for governments, providers and patients to realize improved health-care services and outcomes.

The rapid diffusion of health technologies has presented governments with unprecedented challenges to provide high quality and innovative care to meet population health needs most effectively while managing health-care budgets and safeguarding the basic principles of equity, access and choice. Consequently, governments increasingly are required to manage scarce resources strategically by investing in those services that deliver the best health outcomes. This equates to care that is affordable, effective, safe and patient- centred. Moreover, innovation is supported adequately, with sufficient market access to new treatments.

In recent years, various Member States have developed systems to identify the innovations that provide the best value. The National Institute for Health and Clinical Excellence (NICE) in the United Kingdom was the first national attempt to provide faster access to cost-effective treatments through an evidence-based review process. Review bodies, such as NICE, employ health technology assessment (HTA) to ascertain the relative costs and benefits of health technologies. The resulting evidence is used to support various forms of decision-making, such as reimbursement and pricing. In other words, this information can aid priority-setting for access to limited health-care resources. Beyond ascertaining value, increased use of HTA in this setting signals a desire for a more systematic and transparent process to allocate health-care resources.

Chapter 1

Project overview, objectives and methods

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Ensuring value for money in health care2

The operations of NICE and its international counterparts have generated controversy. There are concerns regarding the methods employed during the assessment process; HTA’s role and utility in decision-making and priority- setting; and the resulting impacts on health care. How are assessments prioritized and who decides? What do authorities mean by evidence? How do HTA methods differ across agencies? What, if any, impact do they have on the assessment results? Is HTA actually employed in a way that aids decision-making? What is HTA’s effect on health care in terms of patient care, innovation and costs?

Spearheaded by the London School of Economics, this report aims to address the concepts and controversy surrounding HTA in Europe. The report reviews HTA organizations and processes throughout the European Union (EU) and within selected Member States including extensive case studies on Sweden, the Netherlands, Finland, France, Germany and the United Kingdom, the latter focusing on England and Wales (see Appendices).

Broadly, the report is intended to identify and address current considerations regarding HTA methodological and process issues related to the prioritization and financing of modern health care. In particular, it describes the processes and challenges for identifying and prioritizing assessments; assesses and compares current assessment methods and procedures; and highlights the barriers to effective implementation. The report also ascertains the roles and terms of engagement of key stakeholders, and captures the opportunities and challenges for the use of HTA guidance in general priority-setting, decision- making and health-care provision.

Current literature related to HTA was reviewed systematically, including peer- reviewed journals and grey literature sources. Where necessary, reports and other information sources were translated into English. Experts in HTA were consulted in order to supplement the secondary data collection and address any gaps in the evidence available.

In conclusion, the review of HTA in Europe and the overarching themes identified in the report should assist in improving the HTA process in Europe and its role in supporting value in health care.

Overview of innovation in health care

Health technology is an indispensable part of any nation’s health-care system. During the past half-century, all Member States and several other countries have increased their technological base for health care – in knowledge and through investments in equipment, devices and pharmaceuticals. As a result, national health-care systems have become increasingly advanced as health-care delivery has introduced a range of technological innovations, such as new medicines and diagnostic tools, telemedicine and surgical equipment.

The introduction of new technologies has brought remarkable improvements. Many innovations result in applicable new therapies with significant benefits for patients including improved health, enhanced quality of life and reduced adverse or side effects. Moreover, innovations in clinical practice provide enormous opportunity for physicians and other health-care providers to improve the effectiveness, safety and quality of treatment. On a broader level, technological innovation provides governments with mechanisms to improve the quality and outcomes of national health-care objectives.

Many innovations offer significant potential benefits to patients and the health- care system, but their diffusion can prove problematic in resource-constrained health-care environments. Some innovations produce similar or improved effectiveness and quality of care at significantly lower costs; others increase overall health expenditure (Cutler & McClellan, 2001; Newhouse, 1992). Indeed, the nature and strength of the relationship between health technology and costs are complex and evolving. Moreover, demographic transition (the ageing population) and better-educated health consumers have resulted in

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Chapter 2

Background on innovation and HTA

Ensuring value for money in health care4

increased demand for new medical products and services (Deyo, 2002). This technological imperative frequently is accompanied by expectations of public financing and access that will continue to exert pressure on public budgets in the context of lower economic growth. Governments must strive to attain a balance between innovation, medical progress and productivity gains through more efficient management of health-care systems.

New innovations can significantly improve clinical practice, but the rapid growth of medical technology, and the increasing volume of new knowledge from basic and applied clinical research, have made it virtually impossible for care providers to keep pace with treatment advancements. Inappropriate practices and variations in the use of new and existing technologies have encroached into health-care provision across Europe so that the most effective and efficient technologies may not always be employed. Often, inertia and reluctance to change long-standing practices and outdated education restrict the uptake of new, cost-effective interventions.

Accordingly, many EU countries face the significant policy challenge of harnessing the benefits of technology and innovation while managing health- care budgets and meeting public demand and expectations. Countries employ a wide array of approaches to control the costs of health technology and support the optimal use of such products and HTA has assumed an increasing role in national priority-setting and health-policy processes. In recent years, various Member States have developed systems to evaluate innovations – determining their relative value for investment and mechanisms for equitable and accessible treatment provision. In the United Kingdom, NICE was one of the first review bodies established to provide faster access to modern treatments through a systematic review process and to promulgate evidence-based decision-making.

HTA: overview and key objectives

HTA originated from growing concern about the expansive diffusion of costly medical equipment in the 1970s and taxpayers and health insurers’ ability and willingness to fund their use (Jonsson & Banta, 1999). Moreover, greater public awareness of health-care rationing decisions and a growing consumerist position on health-care policy required more accountability, transparency and legitimacy in decision-making processes. Decision-makers needed a more comprehensive approach to set priorities and obtain maximum benefit from limited resources, without compromising the ethical and social values underpinning health systems (Hutton et al., 2006). The growth and development of HTA reflected this demand for well-founded information to support decisions on the development, uptake and diffusion of health technologies.

5

Since the 1970s, HTA has broadened to encompass a wide range of health technologies including drugs; medical devices; medical and surgical procedures; and the organizational and support systems for care provision (Jonsson, 2002). However, the majority of HTAs have been conducted on pharmaceuticals rather than other technologies such as medical devices and surgical procedures (Hutton et al., 2006).

On a broad level, HTA can be defined as: The systematic evaluation of the properties, effects, and/or other impacts of health care technology (International Society of Technology Assessment in Health Care, 2002).

More specifically, HTA involves the evaluation of an intervention through the production, synthesis and/or systematic review of a range of scientific and non-scientific evidence.1 The type of evidence considered typically includes the safety, efficacy, cost and cost-effectiveness (CE) of a product. However, HTA is also concerned with the societal, organizational, legal and ethical implications of implementing health technologies or interventions within the health system (Velasco-Garrido & Busse, 2005; INAHTA, 2002). For example, HTA often considers health technologies’ broader macroeconomic impacts on national health-care budgets; resource allocation among different health programmes; regulation; and other policy changes for technological innovation, investment, technology transfer and employment (Goodman, 1998).

In addition to ascertaining technologies of value, an effective HTA can reduce or eliminate the use of interventions that are not safe and/or effective, or have insufficient cost-benefits. HTA can also be used to identify existing technologies that may be harmful or ineffective. Less commonly, HTA can also identify underused technologies (e.g. preventive screening, smoking-cessation interventions) and the reasons for this (Asch et al., 2000; McNeil, 2001).

For a systematic review of the available evidence on a health technology(s), HTA employs a multidisciplinary framework to address four principal questions (UK National Health Service R&D Health Technology Assessment Programme, 2003).

• Is the technology effective?

• For whom does the technology work?

• What costs are entailed in its use?

• How does the technology compare with available treatment alternatives?

Background on innovation and HTA

1. HTA typically entails 1) identifying the policy question, 2) systematic retrieval of scientific and non-scientific evidence, and analysis, and 3) appraisal of evidence, including judgments regarding the meaning of the evidence. The evidence and its applications then inform the decision-making process.

Ensuring value for money in health care6

An HTA’s principal aim is to provide a range of stakeholders (typically those involved in the funding, planning, purchasing and investment of health care) with accessible, usable and evidence-based information to guide decisions about the use and diffusion of technology and the efficient allocation of resources. In light of these objectives, HTA has been called “the bridge between evidence and policy-making”, as it provides information for health-care decision-makers at macro-, meso- and micro-levels (Battista & Hodge, 1999). Decision-makers have increasingly relied on the use of HTA to support reimbursement and pricing decisions regarding existing and new pharmaceuticals. HTA also contributes greatly to the knowledge base for improving quality of care, especially by supporting the development (or updating) of clinical practice guidelines and standards for health-service provision (Zentner et al., 2005).

Without sufficient, high-quality evidence the uptake and diffusion of technologies are more likely to be influenced by a range of social, financial, professional and institutional factors. This may not produce optimum levels of health outcomes or efficient use of scarce resources.

Interface between HTA and innovation

The variety and emerging complexity of health technologies has combined with limited national budgets to produce tensions between delivering cost- effective health care and improving or sustaining a country’s manufacturing and research base. The importance of achieving a balance between affordable health care and the use of innovative health technologies has increased. This requires not only consideration of the medical and economic value of a product, but also who benefits from innovations, optimal usage2 and appropriate placement in the spectrum of care (Drummond, 2003).

HTA provides important benefits by empowering governments to make value- driven decisions, supporting innovation and providing patients and physicians with the information for making the best treatment choices.

However, HTA’s effectiveness (particularly in encouraging innovation) rests on accurate assessments and the appropriate implementation and use of subsequent recommendations. HTA can encourage innovation if assessments of new technology are performed properly and consider a wide range of

2. Variation in uptake and diffusion can signify the sub-optimal use of technology. Excess use is signified when the costs outweigh the benefits for any additional level of technology diffusion or use. Under-use can occur when the foregone benefits outweigh the costs of additional diffusion or use. Both scenarios are sub-optimal, potentially resulting in economic costs and/or reduced health outcomes.

7

associated costs and benefits rather than focusing solely on acquisition costs. In particular, adoption costs need to be measured against the potential broader benefits of integrating the new technology into the health system; budget-driven constraints do not necessarily result in the selection of the most effective or cost-effective products. This may require governments to allow additional funding and flexibility between budgets so that expenditure levels are driven by value rather than arbitrary spending caps (Drummond, 2003).

As mentioned, HTA’s value in encouraging innovation and value-added health care also depends on the assessment process – including when and how the review was performed and the resulting decision-making procedures. In particular, the following issues can impact on HTA’s effective use for meeting key objectives (Drummond, 2006; Zentner et al., 2005; Anell, 2004; Busse et al., 2002).

• Delays in the HTA process can result in deferred reimbursement decisions, restricting patient access to treatments needed.

• Evidence requirements can be a significant hurdle for manufacturers, particularly small, innovative companies. These may discourage pursuit of breakthrough technologies.

• As HTA becomes increasingly widespread, assessments are made earlier in the technology diffusion process. This may introduce greater uncertainty into the process and the potential for innovations to appear more, or less, beneficial.

• Current assessment methodologies may limit comparability and transferability across countries and studies.

• Lack of transparency, accountability and stakeholder involvement in the HTA process can decrease the acceptance and implementation of assessment results.

• Low numbers of skilled HTA personnel and limited international collaboration between review agencies can reduce the efficiency and effectiveness of assessments.

• Separate processes and organizations for economic assessments, reimbursement/pricing decisions and practice-guideline development may hinder the effectiveness and efficacy of the overall decision-making process through unnecessary use of resources and duplication of efforts.

Decision-makers are more likely to utilize HTAs if there are established policy instruments (e.g. reports, practice guidelines) and commitments to use them effectively. Moreover, patient demand and the CE of a technology can change so it is important to review HTA recommendations on a

Background on innovation and HTA

Ensuring value for money in health care8

consistent basis. This requires greater participation and collaboration among stakeholders particularly HTA personnel, government officials, the industry, health providers and patients. Without adequate input and understanding of the HTA process, stakeholders may mistrust the evidence and subsequent recommendations.

For HTA to be of optimal benefit, the assessment process needs to be linked with innovation and other aspects of policy-making – recognizing the complexities of decision-making that require consideration of subjective and normative concerns. Without these links, HTA may have limited power to inform the policy process and facilitate access to new and effective products. HTA’s role in encouraging innovation and value in health care could be improved by greater understanding of the challenges inherent in the HTA process, as outlined below.

HTA dates from the late 1970s when the expansion of technology and health- care costs began to capture the attention of decision-makers (Jonsson, 2002). The introduction and subsequent growth within Europe runs alongside health policies that place greater emphasis on measurement, accountability, value for money and evidence-based policies and practices. Moreover, the advent of randomized control trials (RCTs) and subsequent availability of data; growth in medical research and information technology; and increased decentralization of health system decision-making, all contributed to an increased need for HTA activities (OECD, 2005).

In Europe, the first institutions or organizational bodies dedicated to the evaluation of health-care technologies were established in France and Spain in the early 1980s and in Sweden in 1987 (Velasco-Garrido & Busse, 2005; Garcia-Altes et al., 2004). Over the following decade HTA programmes were established in almost all European countries, either in new agencies or institutes or in established academic governmental and non-governmental units (Table 3.1). Broadly speaking, such bodies fall into two categories: (1) independent (arms-length) review bodies that produce and disseminate assessment reports on a breadth of topics, including health technologies and interventions; and (2) entities under government mandates (e.g. from health ministries) with responsibilities for decision-making and priority-setting, typically pertaining to the reimbursement and pricing of heath technologies. The latter serve an advisory or a regulatory function.

Many EU countries are supporting these efforts by investing resources in HTA and associated evaluation activities. For example, in 2007 Sweden spent around

Chapter 3

HTA and decision- making in Europe

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Ensuring value for money in health care10

€5.7 million on its national agency, the Swedish Council on Technology Assessment in Health Care (SBU); and the United Kingdom Department of Health allocated £35 million to NICE (SBU, 2007; United Kingdom House of Commons, 2007).

Table 3.1. Institutions and advisory bodies responsible for HTA activities

Country

Austria Federation of Austrian Social Insurance Institutions/Drug Evaluation Committee [Hauptverband der österreichischen Sozialversicherungs träger/Heilmittel-Evaluierungs-Kommission]

Belgium National Institute for Sickness and Invalidity Insurance (INAMI)/ Commission for Reimbursement of Medicines [Institut National d’Assurance Maladie-Invalidité/Commission de Remboursement des Médicaments]

Denmark Reimbursement Committee/Danish Centre for Evaluation and Health Technology Assessment (CEMTV)

Finland Pharmaceuticals Pricing Board (PPB)/Finnish Office for Health Technology Assessment (FinOHTA)

France Economic Committee on Health Products (CEPS)/Transparency Commission [Commission de la Transparence]

Germany Federal Joint Committee/Institute for Quality and Efficiency in Health Care (IQWiG)/German Agency for Health Technology Assessment (DAHTA).

Italy Committee on Pharmaceuticals/Italian Medicines Agency (AIFA) [CIP Farmaci/Agenzia Italiana del Farmaco]

The Netherlands

Pharmaceutical Care Committee (CFH) /Health Care Insurance Board (CVZ)

Norway Pharmaceuticals Pricing Board (PPB)/Norwegian Medicines Agency (NoMA)

Spain Spanish Agency for Health Technology Assessment (AETS)/Catalan Agency for Health Technology Assessment and Research (CAHTA) [Agència de Evaluación de Tecnologías Sanitarias/ Agència d’Avaluació de Tecnología Mèdica i Reçerca]

Sweden Pharmaceutical Benefits Board (LFN)/Swedish Council on Technology Assessment in Health Care (SBU)

Switzerland Federal Office of Public Health (BAG)/Confederal Drug Commission [Bundesamt für Gesundheit /Eidgenössische Arzneimittelkommission]

United Kingdom

NICE/National Coordinating Centre for Health Technology Assessment (NCCHTA) /Scottish Medicines Consortium (SMC)

Source: Velasco-Garrido & Busse, 2005; Zentner et al., 2005.

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Additional investment brings growing recognition that the HTA process must be scientifically sound, consistent across applications, transparent and of practical use in both policy-making and health-care practice (Zentner et al., 2005; Jonnson, 2002). Further, more countries are placing greater emphasis on ensuring that the results of HTA are considered in key decision- making processes.

While European HTA agencies share many of the same basic objectives, their structures have developed separately and currently operate differently across countries. In particular, there are variations in:

• responsibility and membership of HTA bodies (governance, decision- making, priority-setting);

• assessment procedures and methods;

• application of HTA evidence to decision-making (criteria and timing of assessments);

• HTA dissemination and implementation.

Moreover, transparency and accountability are encapsulated in each of these elements of the HTA process.

The heterogeneity of HTA activities in EU countries reflects their individual health-care and political environments with differing mandates, funding mechanisms and policy-formulation roles (Velasco-Garrido & Busse, 2005; Banta, 2003). Further, HTA’s use in decisions that influence the diffusion and uptake of technologies can be influenced by a myriad of factors such as income levels, reimbursement mechanisms, regulatory environments and behavioural determinants (e.g. cultural imperative for new technology). As HTA strives to connect policy and evidence, it also reflects the specific needs of decision- makers within a certain system.

Responsibility and membership of HTA entities

Most national HTA bodies can be categorized as serving either an advisory or a regulatory role in the decision-making process, depending on the intent and type of assessment required (Zentner et al., 2005). Advisory bodies, such as those in the Netherlands and Denmark, make reimbursement or pricing recommendations to a national or regional government, ministerial department or self-governing body (Zentner et al., 2005). Regulatory bodies are accountable to health ministries and are responsible for listing and pricing drugs, medical devices and other related services (Zentner et al., 2005). This is the role of HTA agencies in Finland, France, Sweden

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Ensuring value for money in health care12

(LFN) and the United Kingdom. Other groups mainly coordinate HTA assessments and produce and disseminate reports (e.g. Health Council of the Netherlands, Sweden (SBU)).

The mandates or responsibilities of the assessment bodies vary according to their general mission and overall policy objectives (Anell, 2004). As one component in the broader health-care decision-making process, HTA programmes typically reflect the current national policy landscape such as the need to contain costs or improve access to a given intervention or service. Economic evaluations often coincide with policies on the reimbursement, pricing and utilization of health technologies (Hutton et al., 2006). Assessments frequently assume a role in providing information to providers through practice guidelines and in supporting decisions on the investment and acquisition of health technology (OECD, 2005).

In many countries the health ministry oversees the appraisal process, although independent institutions (e.g. NICE) often are involved in managing various aspects of the assessment (Hutton et al., 2006). In many social insurance- funded health systems, the process is driven predominantly by insurance organizations (Hutton et al., 2006). However, the health ministry provides some degree of oversight even in these countries and, often, the social affairs or security ministry is involved.

Evaluation practices also differ. In general, the nature of an assessment determines which organization will conduct the evaluation. Some HTA bodies conduct assessments via in-house committees; others coordinate independent reviews by external bodies such as university research institutions or expert groups (Anell, 2004). Independent reviews present benefits and drawbacks to the assessment process. They may provide greater transparency and help to prevent or resolve disputes (Drummond, 2006; Goodman, 1998). Moreover, decentralization can widen the expertise available and bring a broader range of perspectives to the process. However, independent reviews may introduce certain methodological challenges, such as use of particular study designs (e.g. RCTs) and potential disconnections between the economic model and systematic review. A decentralization of responsibilities may also result in coordination inefficiencies; divergent agendas and methodologies; and opportunities for miscommunication between those conducting the assessment and the ultimate decision-maker(s).

HTA entities also have differing roles in the decision-making process when assessments are complete. In some countries (e.g. United Kingdom) the HTA body develops guidance and/or recommends reimbursement status; in other systems this is determined primarily by national authorities, insurance representatives or independent self-governing bodies. Moreover, some HTA

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committees (e.g. in Finland and France) are also involved in negotiating product prices and reimbursement with manufacturers.

All HTAs have multiple technology-related policy-making needs and perspectives across diverse stakeholder groups. Thus, HTA involves a variety of stakeholders including physicians, pharmacists, health economists, insurance and industry representatives and patients. Anell (2004) found that most reimbursement status recommendations are determined firstly by scientific members (e.g. physicians, epidemiologists) with expertise in evaluation of medicines. These decisions are corroborated by academic entities, representatives from patient organizations, health economists and (in the case of NICE) NHS managers (Anell, 2004). Participation differs across HTA bodies, although all these agencies have some level of stakeholder involvement. A recent OECD study (2005) reported that patients and consumer groups were least involved in the assessment and decision-making process. While patient perspectives were taken into account indirectly through the inclusion of safety, effectiveness and quality-of-life measures, such indicators may not adequately reflect important broader patient values (e.g. preference for one treatment, acceptability of various side effects). Measures of such preferences can play a substantial role in the assessment of new technologies and may provide useful insights into the real-world value of technologies. Greater participation of patients and consumers has been advocated in light of these potential benefits (Coulter, 2004) and some HTA systems support an increased role for patients in assessments and decision-making. NICE has established a Citizens’ Council to gather public perspectives on key issues that inform the development of guidance documents. This assists the development of the social value judgments that should underpin NHS guidance.

A greater role for industry representatives has also been suggested. This is controversial as there is concern that greater collaboration between HTA entities and industry might influence the objectivity and transparency of the assessment process, particularly in the use of commercial in-confidence data. As a result, the implementation of recommendations could be hindered by appeals and general disagreement from various stakeholders.

Stakeholder involvement is generally resource-intensive, but it can improve relevancy and produce greater trust in the assessment. Accordingly, increased engagement may facilitate better overall assessments, reduce the number of appeals and improve implementation of HTA recommendations and guidance (Drummond, 2006). In particular, by playing a more integral role in the prioritization and assessment of health technologies, patients and their organizations can drive a more consensus-based policy process, especially at the macro-level of the health-care system.

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Ensuring value for money in health care14

Assessment procedures and methods

Assessment processes within the EU differ on a variety of issues such as topic selection, evidence/data requirements, analytical design and the methodological approach(es) employed.

Topic selection

Most HTA agencies struggle to keep pace with new technology therefore priority-setting has become an important aspect of the process to determine which products are assessed. Countries set priorities using a number of different mechanisms and criteria, through the emphasis given to different approaches (i.e. proactive, reactive, mixed) and in the process of needs identification. The topic agendas of some review bodies are set by national authorities – typically, the health minister or department of health. However, Germany and the United Kingdom have established processes for suggestions to be submitted by a wide range of stakeholders. In Germany, a board of trustees comprised of public administrators, patients and industry representatives determines HTA topics using a Delphi process (OECD, 2005). Within the United Kingdom’s NHIR Health Technology Assessment Programme, advisory panels recommend priorities to the Director of Research and Development. The Scottish Medicines Consortium (SMC) aims to evaluate every new drug, formulation and indication within 12 weeks of market launch.

For review bodies responsible for reimbursement decisions, topics of assessment are based upon manufacturer submissions (a dossier of clinical and health economic evidence to support reimbursement determinations). The breadth of assessment topics varies too – some HTA agencies focus on health technologies (specifically drugs and/or devices); others attend to particular disease areas or health conditions. Several organizations conduct assessments on both products and broader health issues (e.g. SBU in Sweden). The criteria used to select topics vary across agencies, but generally include health benefit(s); impact on other health-related government policies (e.g. reduction in health inequality, improving access); uncertainty about effectiveness/CE; disease burden; potential benefits and impact of the assessment; and innovation capacity (Garcia-Altes et al., 2004; Taylor, 2001; Oortwijn et al., 1999).

Generally it is considered not cost effective to evaluate all existing technologies and interventions. Review bodies incorporate various approaches to ensure the efficiency of the assessment process in order to provide important and timely information for decision-makers. In the United Kingdom, NICE allows groups of similar technologies to be compared; the Netherlands requires certain procedures to guide proper use of drugs that are not appraised. If a

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drug provides several approved indications then review bodies in Sweden, the Netherlands, Norway and the United Kingdom commonly evaluate the therapy for all conditions.

Although HTA agencies cover a quite broad range of topics, some areas are studied less (e.g. low-technology and preventive interventions). This is also true for research on ineffective and obsolete technologies and interventions. Moreover, HTA bodies rarely undertake assessments to keep abreast of new areas of research and development (R&D), presumably because of limited resources (OECD, 2005). Assessments conducted earlier in a product’s life-cycle have had some impact by identifying areas of uncertainty and highlighting areas for further research (OECD, 2005). Similarly, some HTA agencies have developed early-warning and horizon-scanning systems to identify new and emerging technologies that might require urgent evaluation, consideration of clinical and cost impacts, or modification of clinical guidance activities (Douw & Vonderling, 2006; Douw et al., 2003; Carlsson et al., 1998). Criteria used most often to identify candidates for early warning assessments are listed below.

• Requires attention of (or action by) politicians, hospitals and health-care administrators within certain time limits.

• Deemed cost-demanding or controversial.

• Expected to spread more rapidly than desired, according to current scientific knowledge base.

• Expected to spread more slowly than desired given technology’s potential benefit.

This type of programme has been established nationally in the Netherlands, Sweden, Finland and the United Kingdom; and internationally through the European Information Network on New and Changing Health Technologies (EuroScan).3 There is limited evidence of impacts on decision-making, but there is some concern that early assessment may be biased against new technologies, especially those of higher cost (AdvaMed, 2000).

Several studies have highlighted a lack of transparency in the topic selection process (Garcia-Altes et al., 2004; Hagenfeldt et al., 2002; Henshall et al., 1997). Many HTA organizations lack explicit processes for prioritization, including selection methods and stakeholder involvement (Garcia-Altes et al., 2004). It is important to identify the factors involved in the priority-setting process

3. EuroScan has 12 members (predominantly HTA agencies) in 10 countries – 2 outside Europe (Canada and Israel).

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Ensuring value for money in health care16

and the specific objectives as these affect the criteria for selecting assessments. Typically, there is limited mention of any political deliberation (or other normative considerations) that drives the assessment of certain technologies. Given limited resources and greater accountability, it is increasingly important to state how assessment topics are selected. A certain level of transparency is needed for an open, systematic and unbiased assessment prioritization process (Hagenfeldt et al., 2002). Perceived lack of transparency may exacerbate existing tensions about balancing access to technologies, product innovation and health expenditures between manufacturers, patients and the stewards of health-care budgets.

Evidence/data requirements

HTA systems require various types and qualities of evidence for economic evaluations (Hutton et al., 2006). Typically, manufacturers are required to submit a comprehensive summary of a product’s effectiveness and CE but these data play different roles in the assessment process. In Austria, Norway and the Netherlands, HTA bodies review and validate industry data, which must be based on systematic review of available clinical and economic evidence (Zentner et al., 2005). Other organizations (e.g. NICE, SBU) perform systematic reviews in-house or through an independent evaluation group. Evidence used in these assessments may or may not include manufacturer data and generally involves broader review of various information sources. Some countries (e.g. France, Switzerland, Finland) prefer, but do not require, systematic reviews. Their assessments are based primarily on a definite number of studies (e.g. pivotal clinical trials) provided by industry (Zentner et al., 2005). Assessment of unpublished evidence (e.g. commercial in-confidence data) is considered explicitly in Austria, the Netherlands, Sweden and the United Kingdom.

Manufacturers generally submit evidence comprised of systematic literature searches and analyses of clinical and economic studies, which may or may not include modelling. The majority of HTA institutions have published guidelines to outline the methodological requirements for manufacturers and reviewers. However, such documented procedures require varying levels of detail and transparency (Zentner et al., 2005). It is typical for the pharmacoeconomic methodologies used in assessments to be described in more detail than clinical- review procedures or the evaluation of other product characteristics (Zentner et al., 2005). Most guidelines cover preferred clinical and economic evidence, comparators, specification of the outcome variable(s), sub-group analyses, costs to be included, time horizon, discounting and use of sensitivity analyses and modelling.

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Differences in timing for evidence requirements have developed recently, i.e. the point at which manufacturers submit CE data . The Swedish LFN requires manufacturers to submit evidence on cost-efficacy. If this is acceptable, the product under review is allowed provisional reimbursement while CE data is collected and submitted.

Analytical design

Countries employ different analytical frameworks to guide their assessments (Hutton et al., 2006). Most evaluations assess a variety of criteria including safety and clinical effectiveness; patient need and benefit; and CE and cost of therapy (typically in relation to benefit) (Zentner, 2005; OECD, 2005; Anell, 2004). Some HTA bodies also frame the evaluation around other factors, listed and compared in Table 3.2:

Table 3.2. Criteria for assessment

Criteria AT 4 BE CH DE FI FR NL NO SE UK

Therapeutic benefit X X X X X X X X X X

Patient benefit X X X X X X X X X X

CE X X X X X X X

Budget impact X X X X X X

Pharmaceutical/innovative characteristics

X X X X X

Availability of therapeutic alternatives

X X X X

Equity considerations X X X

Public health impact X

R&D X

Source: Adapted from Zentner et al. (2005) and case studies.

4. AT=Austria, BE=Belgium, CH=Switzerland, DE=Denmark, FI=Finland, FR=France, NL=Netherlands, NO=Norway, SE=Sweden, UK=United Kingdom.

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Ensuring value for money in health care18

While the particular analytical framework may depend on the specific policy question, almost all assessments consider therapeutic and patient benefit. There is also agreement that economic evaluations should be conducted from a societal perspective, taking account of costs and benefits outside the health sector rather than a narrow budget perspective on resource use (Zentner et al., 2005; Anell & Svarvar, 2000).

Assessment methods5

HTA uses diverse methods, but most programmes employ an integrative approach. The majority of agencies share similar methodologies and emphasize the most rigorous types of studies (e.g. use of RCTs and cost-utility analyses), but there is no standard approach for conducting assessments. Given their varying orientations, resource constraints and other factors, assessment programmes tend to rely on different combinations of methods. In particular, assessments often differ according to the (Zentner et al., 2005):6

• type of economic assessment required

• classification of product benefit (benefit vs. harm) – hierarchy of evidence

• choice of comparator

• specification of the outcome variable

• costs included in the analysis

• discounting

• use of CE threshold

• allowance for uncertainty

• missing and incomplete data

Type of economic assessment

In general, different countries have similar requirements for economic assessments (Zentner et al., 2005). Typically, these are guidelines that product sponsors must follow to select the type of economic assessment used in submissions. However, some countries (e.g. Switzerland) do not require

5. This section refers primarily to decision-making bodies that review clinical and economic evidence for product reimbursement and pricing.

6. Methodologies can also differ on sub-group analyses; time horizons; instruments to measure quality of life; and methods for calculating costs.

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assessments and so no guidelines are applied.7 Among existing guidance, CE or cost-utility analyses are most often considered appropriate analytical designs, particularly when the proposed product has significant clinical advantages over the comparator and relative benefits need to be considered against costs. Cost-utility analysis measures health outcomes in terms of quality-adjusted life years (QALYs).8 Increasingly this has become the preferred indicator of effectiveness as it can be applied in comparisons of different therapies and, consequently, employed for priority-setting (Zentner et al., 2005). Moreover, cost-utility analysis is deemed to be associated with fewer issues than other methodological approaches, such as cost-benefit analysis. Although many assessment bodies (such as NICE) have deemed QALYs the principal measure of health outcome, still only a limited number of studies report QALYs based on the actual measurement of patients’ health-related quality of life (HRQoL)9

(Rasanen et al., 2006; Rawlins & Culyer, 2004).

Evidence to classify product benefit

Zentner et al. (2005) found that all countries consider head-to-head RCTs with a high degree of internal and external validity to be the most reliable and objective evidence of a product’s relative therapeutic benefit. This also applies to systematic reviews and meta-analyses of RCTs. Moreover, the majority of review bodies favour RCTs in naturalistic settings as they reflect daily routines and country-specific care delivery. Where definitive primary studies exist, limitations must be considered. For example, elderly people and patients with co-morbidities often are excluded from clinical trials even though they are major consumers of medical products. Additionally, trials do not always collect a full range of economic data (e.g. indirect costs, health-utility measures) and the study time horizon is often too short to detect longer- term outcomes. Findings from different types of studies should be combined or synthesized to supplement available clinical data in order to formulate

7. In the case of Switzerland, the assessment body applies a cost-analysis approach, whereby a new product is compared with the same therapeutic category and the price is compared with those in several other EU countries (Denmark, Germany, Holland, United Kingdom, France, Austria, and Italy).

8. QALYs provide a common unit of evaluation across multiple domains, including estimating the overall burden of disease; comparing the relative impact of specific diseases, conditions, and health care interventions; and, making economic comparisons, such as the CE of different health care interventions.

9. HRQoL measures are used increasingly alongside more traditional outcome measures to assess health technologies. These capture dimensions such as: physical, social and cognitive function; anxiety/distress; pain; sleep/rest; energy/fatigue; and perception of general health. HRQoL measures may be disease-specific or general.

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Ensuring value for money in health care20

effective and comprehensive policies. To that end, other types of studies (e.g. case series, registries) may be preferred to RCTs for different policy questions. For instance, modelling is useful when making decisions under uncertainty.

When conducting literature searches, selecting studies and assessing the internal and external validity of clinical trials and systematic reviews/meta-analyses, all review bodies apply internationally established standards. These may be guidelines from the Cochrane Collaboration, CONSORT (consolidated standards of reporting trials), QUORUM (improving the quality of reports of meta-analyses of randomized controlled trials) or their own comparable standards (Zentner et al., 2005).

Choice of comparator

Assessments are almost always comparative – the product under review is evaluated against some specified standard of performance or other products and treatments. The choice of comparator is significant in determining the outcomes of clinical and pharmacoeconomic analyses so it is crucial to select an appropriate comparative treatment. Moreover, all relevant options must be included adequately to inform decision-making. Zentner et al. (2005) found that HTA bodies typically use two different selection procedures. Some institutions (Finland, Sweden – new pharmaceuticals only) require a product to be compared with up to three well-defined comparators; others (e.g. United Kingdom) require all relevant comparators. The most cost- effective existing therapy usually is deemed the most appropriate comparator. However, for practical considerations, HTA bodies often accept an evaluation against routine treatment or the least expensive therapy. Routine treatment for pharmaceuticals is identified by prescription or sales volume; the dosage and delivery of medication must be therapeutically equivalent. Few agencies provide information on how usual practice is determined for non- pharmaceutical treatments. Other review bodies (Switzerland, Sweden) require products to be compared to all therapies of the same therapeutic group, based on the WHO ATC (anatomical therapeutic chemical) classification system.10 This means that only products that are reimbursed or marketed currently can be employed as comparators. An equivalent dosage form should be defined for comparator medications. France combines both approaches by considering drugs in the same therapeutic group; the most frequently prescribed, the least expensive; and the most recently listed (positive list for reimbursement).

10. The ATC classification system is used for the classification of drugs. It is controlled by the WHO and was first published in 1976. Drugs are divided into different groups according to the organ or system upon which they act and/or their therapeutic and chemical characteristics.

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The selectors of the assessment comparator vary between countries (OECD, 2003): specified by either the assessment body (e.g. in United Kingdom) or the product sponsor (e.g. in France and Sweden). In the latter case, HTA bodies often require manufacturers and other relevant reviewers to follow specified guidelines as closely as possible when selecting a comparative treatment. Therefore, it is important that manufacturers communicate with review bodies early in the process (i.e. initial phases of study design) (Zentner et al., 2005). In the Netherlands, product sponsors often discuss the comparator a priori with the assessment body, especially in cases considering a more narrow indication than the product’s label for reimbursement approval. In the United Kingdom, Portugal and Switzerland the comparator selection varies between the product sponsor and assessment entity. In most countries the government plays some role in determining the comparator selected. In the United Kingdom, the comparator increasingly is specified by the Department of Health, particularly when it recommends topics to NICE (OECD, 2003). Other review bodies, particularly NICE in the United Kingdom, consider input from stakeholders when scoping study design and comparator selection.

Selection of the outcome variable

Assessments tend to use a variety of health and economic outcome measures. As with comparator selection, the outcome measure(s) specification can influence the conclusions of the assessment. Generally, final outcome parameters that reflect long-term treatment objectives (e.g. changes in mortality, morbidity, quality of life) are preferred, but countries have differing selection procedures and specification processes. In the Netherlands, the outcome variable is outlined by the assessment body; in most countries the product sponsors are the key decision-makers for specifying this.11 The choice of outcome variable may depend on the type of analysis to be conducted and intermediate measures are generally accepted if there is a lack of outcome data available (OECD, 2003). However, this type of study requires a strong and scientifically-based association between intermediate effect and final outcome (Zenter et al., 2005).

Costs included in the analysis

HTA bodies and governments differ on the type of costs allowed in assessments. The specification of costs typically is related to the purpose of the analysis and

HTA and decision-making in Europe

11. Portugal and Switzerland do not directly specify an outcome variable.

Ensuring value for money in health care22

the overall objectives of the assessment (OECD, 2003). The differences lie in the inclusion of direct and indirect costs. Some countries (e.g. Sweden) allow all costs to be included in assessments; others (e.g. the Netherlands, United Kingdom) use only direct costs.12 There is a lack of agreement on how to account for productivity loss for indirect costs – whether a human capital or friction cost approach13 (Zentner et al., 2005). Moreover, only some (e.g. SBU) include the costs of additional years of life due to a longer lifespan (as a result of treatment). There is also inconsistency on the inclusion of opportunity losses in changes to the quality of life related to leisure activities and time spent on household duties. Some systems (e.g. in the Netherlands) assume a societal-cost perspective despite such costs extending beyond budget constraints. However, wider costs typically are presented separately from system-related costs, therefore non-system costs may have limited direct impact on decisions.

Some countries issue guidelines. Zentner et al. (2005) found that HTA entities or governments provide guidelines on the inclusion of costs associated with other diseases resulting from prolonged life and placing utility measures against the preferences of a country’s population. As data are not always transferable across countries, HTA bodies often request resource consumption and related costs based on national data. Moreover, most guidelines require a high degree of transparency in cost calculations. This entails identifying costs accurately, separating the quantity of resources consumed and the respective cost, and detailing adequately any data sources (Zentner et al., 2005; OECD, 2003).

Discounting

The use and effects of many products extend for years, especially those for chronic conditions. Where a product impacts health and treatment utilization for over one year it is considered good practice to employ discounting to assess appropriately the changes in costs and benefits over time (OECD, 2003).

Almost all HTA bodies employ discounting in assessments, typically applying an annual rate between 2.5% and 10% to both costs and benefits (Zentner et

12. United Kingdom includes only direct costs to the NHS and personal social services.

13. Human capital approach employs the principal that a profit-maximizing firm will employ labour up to a point when the value of the marginal product of labour is equal to the gross wage. Thus, it treats the value of each unit of time lost to ill health to be equal to the gross wage and any additional employment costs. The friction cost approach considers that workers who withdraw from work due to ill health or death will be replaced, after some period of adaptation. Even with very short-term absence, a firm can use existing capacity in its labour pool to compensate for lost work due to ill health.

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al., 2005; OECD, 2003).14 However, a discount rate of 5% is recommended to reflect a societal perspective. Either the payer or the product sponsor determines the discounting rate, according to the country. As a general rule, institutions require the discount rate to be included in the sensitivity analysis in order to determine the effects on outcomes.

Use of CE threshold

In economic evaluation, the results of a CE analysis are summarized by the CE ratio.15 This compares the incremental cost of an intervention with the corresponding incremental health improvement. The health improvements typically are measured in QALYs gained, so the CE ratio usually is expressed as a cost per QALY gained. Treatments with a relatively lower CE ratio are considered most cost-effective. Essentially, CE ratios indicate which health technologies will provide health improvements most efficiently (Garber, 2000).

It can be problematic to interpret the results of CE analyses and, therefore, difficult to decide whether to adopt a particular treatment. As a result, a CE or willingness-to-pay threshold often serves as a general decision rule for ascertaining value for money. An intervention’s CE ratio often is compared to the threshold in order to recommend inclusion or exclusion in the benefits package. Interventions with unfavourable CE ratios may be adopted if other factors (e.g. disease burden, health equity) are a consideration.

Few countries employ a formal or fixed threshold, or at least do not make this explicit. For example, NICE maintains that there is no formal threshold, but recent comments by officials and in particular guidances (e.g. on orlistat) indicate a threshold of £20 000 to £30 000/QALY (Devlin & Parkin, 2004). Rawlins and Culyer (2004) suggest that NICE bases decisions primarily on CE ratios below £20 000/QALY. However, as the CE ratio increases there is increased likelihood of rejection on the grounds of CE. Beyond NICE, the available evidence suggests that the threshold for adoption is between US$ 20 000/QALY and US$ 100 000/QALY, with thresholds of US$ 50 000 – US$ 60 000/QALY frequently proposed (Bell et al., 2006).

HTA and decision-making in Europe

14. It is recommended that the same rate be applied to both costs and benefits but some countries use different rates (e.g. United Kingdom, Sweden).

15. The intervention under study and its alternative are denoted as 1 and 0 respectively. If C1 and C0 are the net present values of costs that result when the intervention and alternative are used, and E1 and E0 their respective health outcomes, the incremental CE ratio is simply: CE ratio=(C1-C0)/(E1-E0). This ratio, a cost per unit of incremental health effects, is used often as a measure of value.

Ensuring value for money in health care24

Allowing for uncertainty

Most review bodies either conduct or require sensitivity analyses on all variables that could potentially influence the overall results, or on a subset of inputs (e.g. imprecise estimates only). This is based on the need to test or verify the robustness of assessment findings. Countries have different requirements for sensitivity analyses (e.g. application of uni- or multi-variate methods), so it is important that the choice of parameters and methods employed are substantiated and well-documented. This is especially important when assessing new technologies for which necessary data are seldom evident.

Most countries also require some form of modelling to allow for uncertainty in the variables and estimates used in assessments. Typically, models are generated by either manufacturers or the review body, sometimes both. Many review bodies develop models to substantiate the estimates provided in manufacturers’ submissions and to compensate for missing or incomplete data. Increasingly, complex decision analysis models are used to ascertain CE, although these vary in quality. Many lack adequate transparency, thus making it essential to continue independent assessment of models used in economic evaluations.

Sensitivity analyses and modelling (as well as sub-group analyses) also may be used to predict the effect of certain patient characteristics (e.g. age, sex, ethnicity) on CE and equity (Michaels, 2006). Guidance from some review bodies (e.g. NICE) suggests that it might be appropriate to provide modelling of the clinical- and cost-effectiveness of treatments for subgroups of patients, but make no explicit recommendations on which variables would be considered ethical. Clear criteria for subgroup analyses, based on specific variables, could provide a framework for incorporating social values into decision-making in an explicit, transparent and consistent way.

Missing and incomplete data

Many HTA agencies face analytical challenges when dealing with data from manufacturers or sponsors. Failure to follow specific assessment guidelines may produce data that are incomplete, poorly presented or lack transparency (OECD, 2005). Moreover, sponsors may be asked to produce the same information in various formats for different countries, presumably increasing the costs of compliance and reducing efficiency.

The choice of methods can influence significantly the result of an assessment and its comparability across studies and countries. Ultimately they may impact on HTA’s utility in the decision-making process (Boulenger et al., 2005). Unfortunately, there is minimal information on how agencies handle these data issues.

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Application of HTA evidence to decision-making: criteria and timing of assessments

Countries employ a variety of HTA evidence to support priority-setting and other modes of decision-making (see HTA dissemination and implementation). In a comparative study by Zentner et al. (2005), all countries16 compared a drug’s therapeutic benefit17 with available treatment alternatives. In fact, this tended to be the leading criterion for assessing a product’s added value in the majority of evaluations. Health-related quality of life is deemed the most appropriate criterion for a technology’s added value from the patient perspective. NICE is one of the few review bodies that has made explicit commitments to include this measure in its assessments and recommendations.

As discussed, many decision-makers do not consider CE against a fixed threshold as an absolute decision rule. Other factors are often considered beside efficacy and CE evidence:

• necessity (e.g. disease burden and severity)

• public health impact

• availability of alternative treatments

• equity

• financial/budget impact

• projected product utilization

• innovation of product (e.g. pharmacological characteristics, ease of use)

• affordability.

Rawlins and Culyer (2004) report that NICE usually requires additional justification for CE ratios over £25 000/QALY such as the degree of uncertainty; wider societal costs and benefits; and the particular features of the condition and population using the technology. The Netherlands has an ongoing discussion about adopting a decision framework based on both efficiency and equity criteria. Different thresholds would apply according to disease burden (essentially necessity) with higher CE ratios allowed for the most severe diseases.

16. Austria, Australia, Canada, Switzerland, France, Netherlands, Norway, New Zealand, Sweden and the United Kingdom.

17. A product is considered as having a therapeutic benefit if it demonstrates an improved benefit/ risk profile compared to existing treatment alternatives. In the case of therapeutic equivalence, a drug is typically not accepted for public reimbursement or is subject to a reference pricing system. A therapy with an inferior benefit/risk profile than other viable therapies are not typically reimbursed, even in the case of lower costs.

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Ensuring value for money in health care26

Many countries lack transparency in their decision-making criteria. An analysis by Anell (2004) found that some review bodies rarely, if ever, outline explicitly the relative weight and importance of the criteria used for recommendations. This is especially true of societal-related and non-quantifiable considerations, such as equity and patient’s quality of life. These tend to follow efficacy and CE in their importance in the overall decision process (Zentner et al., 2005). More explicit understanding of both the threshold value and the accompanying criteria and decision rules is important for a transparent and coherent decision- making process. It has also been suggested that the CE threshold should be consistent with overall budget constraints and consider equity and fairness as well as efficiency (Rawlins & Culyer, 2004; Towse, 2003).

The time required to complete an HTA can affect the application of its evidence. Specifically, there may be a conflict between ensuring comprehensive evaluations and providing timely information to decision-makers. As different countries have divergent approaches to HTA, so the time allocated and required to complete an assessment varies. More specifically, assessments range from two weeks to a few years; although the average is between three months and one year (OECD, 2005). French assessments tend to take less time (e.g. two weeks) than those in the United Kingdom (NICE) and Sweden where one-year assessments are typical (Zentner et al., 2005). The European Commission Transparency Directive (89/105/ EC) requires Member States to make decisions on reimbursement and pricing for new pharmaceuticals to be made within 180 days of marketing authorization (Zentner et al., 2005).

Some agencies have addressed the length of time required to complete assessments. Both the SBU and FinOHTA have introduced rapid reviews to facilitate the assessment process and report on emerging technologies. NICE recently instigated single technology appraisals (STAs), a new fast-track procedure to address time concerns regarding standard assessments. STAs place more emphasis on evidence submitted by manufacturers and less on extensive external review. The SMC typically applies an STA approach to its assessments, providing the NHS in Scotland with guidance based on rapid early assessment of the evidence.

Variations in the duration of assessments can be attributed to a number of factors. Depending on the overall mission, mandate and policy objectives, some agencies conduct more of an overview, where results can be delivered rapidly. In addition, some countries may have the resources to conduct primary research in situations lacking key data, but this can prolong the assessment (OECD, 2005). The rapid pace of technological development can extend or delay timelines as HTA results become obsolete or require the development of new evaluation methodologies to reflect advances. Skilled

27

HTA personnel may be unavailable due to resource constraints or the pace of technologies under review. The United Kingdom has striven to address this by offering training fellowships and providing a steady stream of funding for NICE appraisals. This has enabled academic units to build a critical mass of skilled personnel (Drummond, 2006).

Early appraisals can have a number of consequences. Generally, less information is available early in a product’s life-cycle and these assessments rely more on manufacturers’ submissions. Early review may also be less able to consider sub-groups and other restrictions, unless they are highlighted in the company submissions.

HTA dissemination and implementation

As mentioned, the results or evidence associated with HTA are used on a wide range of decisions to:

• plan resource capacities

• shape the benefit catalogue

• guide treatment provision

• inform corporate investment decisions

• identify R&D priorities and spending levels

• change regulatory and payment policy

• acquire or adopt new technology(ies).

Almost all countries require assessments to ascertain reimbursement status, although they place differing importance on the economic evidence (Anell, 2004) – France rarely considers such information when determining reimbursement status. Alternatively, some reimbursement committees may require assessments only for patented drugs and new indications, or varying requirements for different types of products such as generic drugs (Anell, 2004). Overall, health economic evidence appears to have the most impact for decisions on drugs with broad use (thus, significant potential budget impact) and when CE varies by indication or patient sub-population.

Economic evidence is also used to restrict the use of products, especially innovative and expensive technologies that may not meet firm decision parameters. Reimbursement of these can be confined to certain indications, patient populations, treatment settings and therapeutic positioning (i.e. first- or second-line therapy) (Zentner et al., 2005). In the Netherlands, expensive inpatient drugs that meet certain criteria after an initial assessment (e.g. projected sales higher than 0.5% of total drug sales in the hospital) are granted

HTA and decision-making in Europe

Ensuring value for money in health care28

conditional reimbursement. Additional information on the drug’s real-world CE is gathered during this three-year period. Reimbursement is withdrawn if further evidence does not demonstrate value for money. Conditional approvals can play an important role in minimizing uncertainty by allowing the use of technology under limited conditions. However, their utility is contingent upon gathering additional data and subsequent re-evaluation of the product (OECD, 2005). In general, technologies are reimbursed without conditions when CE and marginal therapeutic and patient benefits against competing alternatives have been established (Anell & Persson, 2005). However, some drugs for severe disease (with a small patient population) or conditions lacking treatment alternatives (e.g. orphan drugs) are covered even if they have poor CE.

HTAs also play a role in product pricing and in negotiating special agreements with manufacturers (e.g. price-volume, cash rebates) (Anell, 2004). However, the closeness of these links differs from country to country. Some countries make reimbursement decisions prior to pricing, others (e.g. Sweden, the Netherlands, Finland) consider the reimbursement and price of a product simultaneously before making a final decision. However, the different HTA schemes and cost-containment strategies adopted by countries may not have significant impacts on individual drug prices. Rather, their effect on drug costs may be more indirect, through better definition of the appropriate clinical indications for the use of treatments (Taylor et al., 2004).

Assessment results are also used to develop clinical or practice guidelines. Typically, these include recommendations on priority-setting and provide national support to assist decision-makers (e.g. policy-makers, providers) to determine effective models of treatment delivery. However, health economic evidence is currently not used optimally in developing guidelines; a minority of overall recommendations employ guidelines grounded in HTA. Berg et al. (2004) suggest that the lack of integration between HTA evidence and guideline development may be attributable to a number of factors including a disconnect between the requirements of clinical practice and data generated by HTA; the medical profession’s aversion to combining economics and health; and guideline development’s reliance on efficacy and effectiveness data, rather than CE. Consequently, the authors suggest that guidelines are a limited mechanism for influencing the use or uptake of new health technology (Berg et al., 2004). This is likely exacerbated by minimal coordination between guideline-producing bodies and those that set priorities and fund HTA studies, and by limited resources for their implementation. However, guideline development and HTA are beginning to converge in many countries.

Despite increasing support for the use of HTA in national priority-setting and health-care policy-making, there remains a paucity of evidence on the

29HTA and decision-making in Europe

real-world effectiveness of economic evaluation in improving health-care planning, clinical practice, diffusion of technologies or overall health costs. The use of HTA has produced advances in technical and methodological issues, but decision-makers continue to diverge frequently from the principles of economic evaluation (Goddard et al., 2006). In addition, there is relatively weak evidence on the impact of HTA and research development, an explicit link is found in only two countries – the Netherlands and the United Kingdom (Henshall et al., 2002).

A wide range of factors may prevent decision-makers from using strict CE criteria to set priorities, or other stakeholders from using HTA products (e.g. reports, practice guidelines) in decisions on health-care provision and innovation. Goddard et al. (2006) argue that methodological shortcomings are not necessarily the main reason for lack of impact. Rather, the wider context of public-sector decision-making places political, institutional and environmental constraints on decisions. While decision-makers may value health economic information (even requesting or requiring its inclusion in the overall evidence base), other aspects of the public policy process result in sporadic and unsystematic application of HTA.

On a macro-level, HTA’s orientation in the decision-making process can affect the extent to which evidence is used to inform policy and related priority- setting. In particular, countries often hold differing views on the use of HTA recommendations (Draborg & Andersen, 2006; Garcia-Altes et al., 2004). Some countries support recommendations on the grounds that experts are best suited to inform decision-making; others prefer decision-makers to be responsible for interpreting evidence and formulating conclusions to reflect political contexts, country-specific or regional conditions, or other normative circumstances (Draborg & Andersen, 2006). However, decision-makers may lack the technical expertise necessary to understand adequately the methodological strengths and weaknesses of a given assessment. Assessment bodies have done much to enhance HTA’s accessibility and usability among different audiences (e.g. policy-makers, health professionals, general public), but improvements are still needed.

Although different decision structures provide policy-makers with a wide range of discretion, failure to use available HTA evidence may produce policies that lead to inefficient, ineffective and inequitable health care. Jacob and McGregor (1997) note, “however excellent an HTA may be, if it fails to be used to influence the working of the health-care system, it is without impact and must be considered without value”.

The influence of HTA depends on several other considerations, including the information needs of decision-makers; transparency of the economic evaluation

Ensuring value for money in health care30

and subsequent decision-making; mechanisms for disseminating decisions; and processes for monitoring and reappraising evidence (Hutton et al., 2006; Zentner et al., 2005). The usefulness of recommendations can be limited by incongruities between the societal and long-term perspective of assessments and the short-term horizon of policy-makers (the moving-target problem)18 (Neumann, 2004). The uncertainty inherent in HTA may also hinder its use in decision-making – effective assessments identify areas of under- and over- use, and can have ambiguous effects on price determination (Crookson & Maynard, 2000). Moreover, best use of economic evaluation may be prevented by broader health-system characteristics such as decentralized management; inadequate public resources or “silo” budgeting; and existing incentives for manufacturers and academics to deliver research that is interesting rather than practical and focused (Rutten et al., 2005; OECD, 2003; Cookson & Maynard, 2000).

It has been suggested that interest groups exert significant influence on the process. Decision-making may benefit some groups at the expense of others, and particular groups may have sufficient power to affect government choices (Goddard et al., 2006). The Dutch Council for Public Health and Health Care (RVZ) (2006) noted that, thus far:

…decisions regarding payment or non-payment for medical treatment are only based on a limited degree on ‘hard’ factors, such as cost- effectiveness, and much more on less transparent considerations, as a result of pressure by lobby groups, such as consumer organizations, the media, and so on. This means that limits are indeed being set at present, but on an ad hoc and somewhat random basis. The result is that the available resources are not being deployed as efficiently as possible.

However, effective implementation of HTA requires the involvement of key stakeholders such as providers and patients. A recent OECD study found stakeholder acceptance to be one of the key determinants of whether decisions are actually put into practice within health systems (OECD, 2005).

It is difficult to assess in practice HTA's ability to maximize health for a given budget. In fact, few countries have formal processes to measure the impact of HTA. The long-term nature of some effects of HTA (e.g. changes in expectations and behaviour patterns of users) and the fact that economic evaluation is just one of many factors influencing policy and practice decisions are obstacles to measuring the impact of assessments (Hailey et al., 1990).

18. Often there is the possibility that by the time an HTA has been conducted, reviewed and disseminated, its findings may be outdated by changes in a technology, how it is employed or its technological alternative for a given problem.

31HTA and decision-making in Europe

A clear and well-communicated decision-making process must be in place before recommendations can be implemented. Lack of a defined process can create doubts about the legitimacy of decisions and make them less likely to be supported by stakeholders. This may increase the risk of appeal procedures (Drummond, 2006; Neumann, 2004). Furthermore, ill-defined decision- making processes may prevent the producers of evidence delivering timely and relevant advice. A clear decision-making process requires identification of an assessment framework that aligns incentives with evidence and health system objectives.

Improved transparency and effective dissemination of recommendations also depends on the methods used for implementing decisions (Box 3.1).

Box 3.1. Methods for disseminating and implementing

recommendations

• Coverage/reimbursement policy

• Formulary restrictions

• Medical audit/peer review

• Clinical guidance

• Accreditation

• Standards

• Media campaigns

• Conferences/workshops

• Professional education

• Web sites and newsletters

HTAs with well-chosen and appropriate policy instruments; a prior commitment to use assessment findings; stakeholder involvement; and real- world applicability (e.g. not too narrowly focused) of the resulting decisions are more likely to influence practice and, ultimately, health outcomes (OECD, 2005; Henshall, 2002). Clinical guidance documents on the use of health technologies are more likely to be adopted when there is strong professional and financial support, in organizations that have established systems for tracking implementation, and when they reflect the appropriate clinical context (Sheldon et al., 2004).

Recommendations by HTA agencies and any resulting decisions must be reviewed and re-evaluated regularly to avoid the moving-target problem. This applies to new technologies and to those already on the market. Some countries

Ensuring value for money in health care32

(e.g. Finland, France, United Kingdom) have a more structured process for reappraisals and conduct re-evaluations at fixed or variable intervals. Others (e.g. Austria, Switzerland) initiate reviews if new characteristics of products emerge (e.g. new or broader indication) or new or better clinical and/or economic evidence becomes available (Zentner et al., 2005).

National and international collaboration is one final area that may improve HTA’s impact on decision-making. Improved cooperation between assessment groups can facilitate the development of methodologies; enhance the transferability and transparency of HTA results and recommendations; and potentially improve the efficiency and accountability of the process itself. The variety of HTA activities and multiplicity of customers also necessitate strong collaboration within and between agencies and different entities dealing with HTA. Several countries have increased collaboration by creating standardized assessment guidelines or standards; convening periodic meetings to discuss assessment issues; devising new channels to encourage communication among national HTA groups; and strengthening the role of international assessment organizations (e.g. Health Technology Assessment International – HTAi) operating at the global level (OECD, 2003). Most countries engaged in HTA activities are involved in one or more international organizations. The European Network for HTA (EUnetHTA) was formed recently to connect public national/regional HTA agencies, research institutions and health ministries, in order to enable effective exchange of information and support Member States’ policy decisions. EUnetHTA represents 59 partner organizations, including FinOHTA, IQWiG, DAHTA, the National Coordinating Centre for Health Technology Assessment (NCCHTA) and the SBU.

Overall implementation of HTA could be enhanced by ensuring that it adapts to the policy question and needs of decision-makers. Timely, methodologically sound evidence should be available in line with decision priorities, recognizing the various dynamics of health technology markets and the public policy process. Indeed, responsibility for achieving better alignment between assessments and stakeholder needs requires collaboration and effort from users and producers. Increasing engagement of key constituencies (e.g. patients, providers, industry) will make decision-making processes more acceptable, relevant and transparent.

Without high-quality evidence, the uptake and diffusion of technologies is likely to be influenced by a range of social, financial and institutional factors. This may not produce optimum health outcomes or efficient use of limited resources. HTA is a significant aid to evidence-based decision-making, but it must address the challenges of delivering timely and relevant information that reflects adequately the dynamics of technology and the health-care system i