Variation in Health Technology Assessment of new medicines: processes and outcomes

Background Health technology assessment (HTA) has become an important policy tool for its ability to inform policy makers regarding the optimal allocation of increasingly limited resources and to ensure evidence-based decision processes. Existing definitions of HTA emphasise its multidisciplinary character, its required robustness as a scientific process and its link with health policy. Objective The objective of this thesis is to investigate the variations in HTA processes and outcomes across jurisdictions, with a focus on oncology versus non-oncology medicines and on Poland, a country with limited resources. Methods We studied the cross-jurisdictional variations in HTA of new medicines and attempted to disentangle these variations by exploring some of their possible determinants. We investigated HTA processes, outcomes and timelines for new drugs across jurisdictions included in our research, as well as differences between HTA for oncology and non-oncology drugs, and the impact of regulatory conditional versus standard pathways on HTA and access, finally focussing on HTA in Poland in the context of a changing HTA environment in a country with limited resources. We also explored the access gap, or the time between regulatory approvals and HTA recommendations for oncology and non-oncology drugs. In our research, HTA processes were mapped and based on in-depth understanding of each jurisdiction’s characteristics and a trichotomous classification of HTA recommendations, positive, positive with restrictions and negative was also developed to enable international comparison of HTA recommendations across jurisdictions. The specific focus of our research was to develop a robust methodology to enable comparative benchmarking across HTA agencies. Results Overall, almost 40% of all HTA recommendations were negative, while over 60% were positive and positive with restrictions across jurisdictions included in our study. However, when this is viewed at a jurisdictional level, about half of HTA recommendations in Scotland, Germany and France were negative. Our study results showed that EU jurisdictions vary substantially in their approach to oncology and non-oncology drugs, with Germany issuing more positive recommendations for oncology drugs and England issuing more positive recommendations for non-oncology drugs. The Netherlands was the only studied jurisdiction with recommendations that were consistent across oncology and non-oncology drugs. This research showed that access gap differs considerably across European jurisdictions, with a median time of less than four months in Germany and over 1.5 years in Poland. Conclusions HTA processes and outcomes vary across jurisdictions, impacting the timely access to new medicines for patients. There are many possible determinants for these variations. Variations in HTA processes and outcomes can be explained by jurisdiction- and agency-specific determinants such as health priorities in a given jurisdiction, a legal framework, the engagement of stakeholders, the use of cost-effectiveness criteria, the assessment of added clinical value, HTA guidelines and evidence required from a pharmaceutical company. Variations can also be explained by drug- and disease-specific determinants such as therapeutic field, available versus required evidence and the magnitude of effect size). We further concluded that both HTA outcomes and timelines can only be interpreted with in-depth understanding of jurisdiction-specific HTA processes

A DECADE OF HEALTH TECHNOLOGY ASSESSMENT IN POLAND

OBJECTIVES: The objective of this study is to illustrate and provide a better understanding of the role of health technology assessment (HTA) processes in decision making for drug reimbursement in Poland and how this approach could be considered by other countries of limited resources. METHODS: We analyzed the evolution of the HTA system and processes in Poland over the past decade and current developments based on publicly available information. RESULTS: The role of HTA in drug-reimbursement process in Poland has increased substantially over the recent decade, starting in 2005 with the formation the Agency for Health Technology Assessment and Tariff System (AOTMiT). The key success factors in this development were effective capacity building based on the use of international expertise, the implementation of transparent criteria into the drug reimbursement processes, and the selective approach to the adoption of innovative medicines based on the cost-effectiveness threshold among other criteria. CONCLUSIONS: While Poland is regarded as a leader in Central and Eastern Europe, there is room for improvement, especially with regard to the quality of HTA processes and the consistency of HTA guidelines with reimbursement law. In the "pragmatic" HTA model use by AOTMiT, the pharmaceutical company is responsible for the preparation of a reimbursement dossier of good quality in line with HTA guidelines while the assessment team in AOTMiT is responsible for critical review of that dossier. Adoption of this model may be considered by other countries with limited resources to balance differing priorities and ensure transparent and objective access to medicines for patients who need them

A DECADE OF HEALTH TECHNOLOGY ASSESSMENT IN POLAND

OBJECTIVES: The objective of this study is to illustrate and provide a better understanding of the role of health technology assessment (HTA) processes in decision making for drug reimbursement in Poland and how this approach could be considered by other countries of limited resources. METHODS: We analyzed the evolution of the HTA system and processes in Poland over the past decade and current developments based on publicly available information. RESULTS: The role of HTA in drug-reimbursement process in Poland has increased substantially over the recent decade, starting in 2005 with the formation the Agency for Health Technology Assessment and Tariff System (AOTMiT). The key success factors in this development were effective capacity building based on the use of international expertise, the implementation of transparent criteria into the drug reimbursement processes, and the selective approach to the adoption of innovative medicines based on the cost-effectiveness threshold among other criteria. CONCLUSIONS: While Poland is regarded as a leader in Central and Eastern Europe, there is room for improvement, especially with regard to the quality of HTA processes and the consistency of HTA guidelines with reimbursement law. In the "pragmatic" HTA model use by AOTMiT, the pharmaceutical company is responsible for the preparation of a reimbursement dossier of good quality in line with HTA guidelines while the assessment team in AOTMiT is responsible for critical review of that dossier. Adoption of this model may be considered by other countries with limited resources to balance differing priorities and ensure transparent and objective access to medicines for patients who need them

The impact of quality-of-life data in relative effectiveness assessments of new anti-cancer drugs in European countries

PURPOSE: The aim of this study is to investigate the role of health-related quality-of-life (QoL) data in relative effectiveness assessments (REAs) of new anti-cancer drugs across European jurisdictions, during health technology assessment procedures. METHODS: Comparative analysis of guidelines and publicly available REAs in six European jurisdictions of anti-cancer drugs approved by EMA between 2011 and 2013. RESULTS: Fourteen anti-cancer drugs were included, adding up to 79 REAs. Whilst all guidelines state that QoL is a relevant endpoint to determine the relative effectiveness of new cancer drugs, QoL data were included in only 54% of the 79 reports and their impact on the recommendations was limited. CONCLUSIONS: Whilst national guidelines recognize the relevance of QoL to determine the relative effectiveness of new anti-cancer drugs, this is not well-reflected in current assessments. Developing and implementing into REAs specific evidence requirements for QoL data would improve the use of this patient-centred outcome in future reimbursement and pricing decisions

The transferability of health technology assessment: the European perspective with focus on central and Eastern European countries

INTRODUCTION: Lower-income European countries have a worse health status and less funds for health care compared to Western Europe. Despite their limited human and financial capacities for conducting Health Technology Assessment (HTA), the need for evidence-based decision-making is growing. Two main approaches emerged as potential solutions: joint clinical assessments on the European level, and simplified procedures relying on the judgments of well-established HTA agencies of Western countries. AREAS COVERED: Based on considerations of transferability, the European Network for Health Technology Assessment (EUnetHTA) was built up to harmonize HTA methodologies across the European Union, and to develop an HTA Core Model by focusing on joint production of relative effectiveness assessment, which can be used as a basis for national value assessments. The second approach has been suggested in various forms without considering transferability issues. EXPERT OPINION: Joint clinical assessments reduce duplication of efforts based on appropriate scientific rationale. On the other hand, recent examples show that relying on judgments of HTA agencies from wealthier countries with potentially different health-care priorities can lead to suboptimal allocation decisions. In the short term, some stakeholders may benefit from ignoring transferability, but it will ultimately lead to limited access in other disease areas

The transferability of health technology assessment: the European perspective with focus on central and Eastern European countries

INTRODUCTION: Lower-income European countries have a worse health status and less funds for health care compared to Western Europe. Despite their limited human and financial capacities for conducting Health Technology Assessment (HTA), the need for evidence-based decision-making is growing. Two main approaches emerged as potential solutions: joint clinical assessments on the European level, and simplified procedures relying on the judgments of well-established HTA agencies of Western countries. AREAS COVERED: Based on considerations of transferability, the European Network for Health Technology Assessment (EUnetHTA) was built up to harmonize HTA methodologies across the European Union, and to develop an HTA Core Model by focusing on joint production of relative effectiveness assessment, which can be used as a basis for national value assessments. The second approach has been suggested in various forms without considering transferability issues. EXPERT OPINION: Joint clinical assessments reduce duplication of efforts based on appropriate scientific rationale. On the other hand, recent examples show that relying on judgments of HTA agencies from wealthier countries with potentially different health-care priorities can lead to suboptimal allocation decisions. In the short term, some stakeholders may benefit from ignoring transferability, but it will ultimately lead to limited access in other disease areas