The results in this study point to a number of key features of HTA processes and their relationship to coverage decisions. First, HTA is not a single mechanism but consists of several salient features that can differ substantially across countries and further determine the way it is implemented to inform coverage decisions. HTA operates mainly at national level except in countries with decentralised systems (e.g. Italy, Spain, UK, Sweden) or autonomous regions/provinces (e.g. Canada). As such, HTA infrastructure and activities reflect the structure of the healthcare systems in which they operate. The scope of HTA systems can directly mirror the administrative division of a country (highly regional systems vs centralised ones). In countries where HTA activities are performed both at national and regional level, assessments of clinical benefit for the same technology could amount to duplication of effort. Similarly, when considering comparable HTA structures across Europe, assessment of clinical evidence performed by HTA agencies duplicates effort as manufacturers tend to submit very similar, if not the same, evidence. Therefore, EU HTA cooperation may be able to streamline HTA activities and homogenise methodologies and procedures in assessing health technologies within the European Union .
Second, institutions performing HTA at national level are mostly independent from the competent authorities they serve (e.g. Ministries of Health, health insurance organisations, pricing committees), even though their activities may be sometimes supervised by these authorities. Considering that HTA activities can, in general, be grouped into (a) assessment, (b) appraisal, (c) coverage recommendations, and (d) funding negotiations, the remit of independent bodies covers (a), (b) and (c), while integrated bodies cover (a), (b) and (d). Given that HTA recommendations by integrated bodies can result in negotiations, they can play a key role in funding decisions. However, this depends on the role of the HTA and whether recommendations are binding or not. Overall, HTA bodies operating at arm’s length are present in more developed HTA systems and tend to be transparent and independent, avoid conflicts of interest and offer dispassionate advice on the costs and/or effects of assessed technologies adapted for contextual considerations. By contrast, newly founded and less well-developed HTA systems such as those found in Greece, Cyprus and Malta tend to be integrated within existing competent authorities. They may lack transparency as assessments are internalised, and recommendations are not reported in publicly available documents, rendering decision-making and negotiation processes unclear and non-transparent. Nevertheless, it could be argued that integrated HTA functions can be very useful as a starting point in the implementation of HTA activities, particularly in circumstances where there is a lack of capacity for the development of an independent HTA body. Unsurprisingly, most independent bodies make their HTA reports and outcomes publicly available (see Appendix in the ESM), whereas integrated bodies tend to keep their reports confidential. Overall, more transparency improves the extent to which a decision can be controlled by the organisation that has commissioned it in the first place .
Third, irrespective of operating at arm’s length or being integrated with competent authorities, the majority of HTA bodies have an advisory role where HTA outcomes act only as recommendations and can be used as supplementary tools or additional criteria during negotiations. HTA bodies with a coordination role may operate in an advisory capacity to competent authorities when asked to assess technologies, however, the extent of their contribution to the final coverage decision is unclear and the consideration of their recommendation is mostly made in a non-systematic manner. Coordination bodies are few and, in our opinion, entities with this role are urgently needed to assist with the coordination and interoperability of HTA activities as well as to generate evidence on how new technologies impact society, including cost–benefit analyses. Coordination bodies can also assist in the transformation of HTA recommendations into clinical guidelines contributing to optimal resource allocation by positioning new technologies along treatment pathways, monitoring their use and assessing the impact they have.
Fourth, the type of HTA evaluation plays an essential role in the way HTA outcomes are translated into funding decisions. During the assessment phase, comparative clinical and/or economic evidence is reviewed, whereas during the appraisal phase, the evidence is assessed and interpreted based on its scientific rigour, the achievement of the endpoints of interest, the design of the included studies, the economic effectiveness, the budget and/or economic model submitted and a other contextual considerations that may be relevant to the setting in question. Value dimensions are examined to investigate the extent to which a new technology is relevant for the healthcare system of interest . Under appraisal these dimensions are always taken into consideration regardless of their nature and the disease context. Therefore, when appraisals are performed, recommendations are context-specific as they take into consideration how a technology can be adopted at national or regional level and what budget impact it will have. Appraisals can inform purchaser–manufacturer negotiations by providing steer, among others, on whether any risk mitigation strategies should be implemented.
Fifth, the vast majority of HTA bodies or HTA-performing institutions have some form of stakeholder involvement or engagement. Whether stakeholders were involved in the HTA process itself or whether they were part of the HTA committee varied, with more developed and well established systems giving patients, carers, citizens and health experts the possibility to act as external stakeholders through public calls. However, engagement of external stakeholders does not always serve the same purpose and ranges across systems from opinions and insights on topic selection and scoping to consultations during HTA assessment or appeals on the final recommendations. In many systems where patient representatives or patient organisations are not involved in HTA, members who are ethicists are bringing the societal perspective into decision-making. However, even though, in theory, stakeholder participation or engagement can result in better uptake of HTA recommendations, there is no evidence establishing a direct link. Undoubtedly, participation of healthcare experts and professionals, experts on ethics, patients, their families and carers in either the HTA process itself or decision-making can ensure transparency, inclusiveness and the reflection of different perspectives in the final recommendations.
Sixth, to assess whether HTA recommendations are translated into funding decisions, we looked at whether HTA outcomes across our sample countries are legally binding. This means that decision-makers are legally bound to respect and follow the final HTA recommendations when making coverage decisions. Based on our findings, the majority of HTA systems issue non-binding recommendations; however, the importance and the weight these recommendations might have on the decision-making process vary across countries. In less-developed HTA systems, such as that of Greece, the role and the impact of HTA recommendations, which are non-binding, is still unknown due to lack of transparency in the decision-making process. Nevertheless, from our sample we identified countries such as Poland, France, England, Scotland, Australia and Romania where recommendations are non-binding but their role during pricing and reimbursement processes at national level is considered crucial.
In order to be able to capture all types of recommendations, we created three alternative scenarios on how HTA outcomes feed into final funding decisions and what their contribution to the final coverage decision of technologies is. Fig. 3 shows three categories of recommendations: (i) binding; (ii) non-binding but as impactful as binding and (iii) non-binding. We created the second category to be able to include the HTA systems of England, Scotland, Australia, France, Poland and Romania, which have an advisory role according to legal statutes, however (positive) recommendations are considered binding for coverage purposes. For instance, the NHS in England is legally obliged to fund technologies recommended by NICE and ensure they are available within 3 months from the date of the NICE recommendation being published .
HTA recommendations that do not fall into the first two categories might jeopardise transparency by creating uncertainty on how evidence-based information is used meaningfully during negotiations or is translated into either price discounts or any other type of Managed Entry Agreements. Importantly, however, non-binding HTAs might inform decision processes further upstream. For instance, in the Greek context, the HTA committee’s recommendations inform the negotiation committee, which has decision-making power over what is reimbursed. In Poland, HTA recommendations play an important role and are extensively used by the MoH, which decides on reimbursement based on negotiations between the Economic Commission of the MoH and the manufacturer .
Seventh, while HTA systems across the study countries seem to have well developed processes for the assessment of pharmaceuticals, these do not appear to be in place for medical devices and other technologies, including public health interventions; there is significant need for refinement in the assessment of both medical devices and other technologies . Among other reasons, this is due to the highly fragmented market structure of medical devices, the lack of clear guidance on evidence requirements and the inconsistency in the methods employed in their assessment . Overall, the range of relevant technologies undergoing HTA is determined by budget holders wishing to optimise the available resources. The identification of more than one HTA agency at national level often coincided with the identified HTA bodies having different remits and assessing different health technologies. For instance, in Wales the All Wales Medicines Strategy Group (AWMSG) assesses pharmaceuticals only, while Health Technology Wales (HTW) assesses medical devices and other technologies. This was further validated by experts who participated in the consultation round: according to primary evidence, the majority of countries reporting more than one body/institution performing HTA at national level, except Belgium and Estonia, have different national bodies for the assessment of different technologies.
Finally, there are different avenues for how HTA recommendations can be used depending on the way HTA systems operate, their role, and the technologies undergoing assessment, such as (i) reimbursement and coverage, (ii) price setting, (iii) strategic purchasing and procurement, especially for medical devices, and (iv) to inform clinical guidance. For instance, HTA bodies with a coordination role can rarely impact coverage decisions but their recommendations can be used for the update of clinical guidelines. HTA systems across our sample could be further divided into several categories in terms of the way HTA recommendations are implemented in decision-making. We observed systems where HTA outputs provide a fundamental basis for pricing and reimbursement; for example, in France, HTA recommendations are used both for reimbursement decisions by the national insurance fund and for pricing decisions by the TC. Other systems, such as that of England, operate in a manner whereby the HTA body makes a recommendation that eventually might trigger negotiations if the cost-effectiveness threshold is higher than the acceptable range or when there is considerable clinical uncertainty around the technology under evaluation. In this case, negotiations take place outside the remit of the HTA body between the purchaser/commissioner of care and the manufacturer. Lastly, there are HTA systems such as that of Australia or HTA by health insurance funds that internalise the decision-making process. Under these systems, negotiations, risk-sharing agreements or strategic purchasing and procuring take place within the HTA body based on the HTA outcome of the body itself. Despite how HTA recommendations are implemented, it is important to highlight that the ultimate ‘client’ of the HTA bodies is the healthcare system they operate in.
Our study is not without limitations. (a) Due to unavailability of data and limited access, we were not able to identify all regional HTA bodies across study countries. (b) Reliance on secondary sources has meant that it may not have been possible to capture HTA processes and implementation in detail. (c) Some HTA bodies may consider additional dimensions of value beyond clinical benefit and cost-effectiveness. Nevertheless, it has not always been possible to determine whether these features have an explicit impact on HTA recommendations through literature or expert opinion. (d) Even though we tracked the HTA systems across countries, the actual implementation and uptake of HTA activities during funding decisions were not fully captured. In order to address the latter two limitations, we performed the round of expert consultation to improve our understanding of the role and extent of HTA uptake at the national/regional level.