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