HTA and National Immunisation Program: an overview of competent bodies and of the impact of HTA on the decision-making process

Abstract Issue/problem The WHO has recommended for many years to adopt transparent and standardized methods to decide upon the introduction of vaccines at national level. For this purpose, the Global Vaccine Action Plan has envisaged the establishment of National Immunization Technical Advisory Groups (NITAGs) to issue independent advice relied on the assessment of vaccines efficacy and safety and health technology assessment (HTA). Description of the problem So far, 172 countries worldwide have a NITAG. Despite the growth in the number of NITAGs worldwide and their strengthening following process indicators identified by the WHO, the way how NITAGs issue recommendations and how they are considered in the development of vaccine policy still represent a matter to the attention of the scientific and policy community. Results The evidence shows that assessment frameworks vary across countries. Furthermore, the legal basis and the authority, namely binding or advisory, recognised to them could influence the way how NITAGs work and impact on vaccine policy. Eventually, the final decision is also led by other aspects, including financial and contextual factors that are less standardizable but should be always taken into consideration in health policy. In fact, the final decision on the inclusion of a vaccination into the national immunization program is made by the Ministry of Health also based on the evaluation of HTA bodies working in the country. Lessons Considering the central role of NITAGs in vaccine policy, some efforts should be done in order to make NITAGs more authoritative and collaborative. This could help, from one side, to make the relationship between final decision and NITAGs recommendations clearer and, on other one, to make it possible to have shared and timely evaluations. In this respect, Joint Clinical Assessment envisaged by the new EU regulation on HTA could prepare the way for a more transparent and timely vaccine policy at European level.

In January 2022, the EU Regulation on Health Technology Assessment (HTAR) entered into force. The HTAR provides for the centralized conduct of EU-wide joint clinical assessment (JCA) for oncology treatments (2025 onwards), and for other medicinal products incl. vaccines (2030 onwards). HTA is a multidisciplinary process aiming to disentangle the value of a health technology in comparison to other. It has been used, to different extent across EU countries, to inform vaccine price and reimbursement decisions. Nevertheless, existing assessment frameworks utilized by HTA bodies, incl. National Immunization Technical Advisory Groups (NITAGs), as well as the relative effectiveness assessment (REA) framework developed by the European Network for Health Technology Assessment (EUnetHTA), do not fully account for the specificities of vaccines. In fact, to account for the broad value of vaccines several aspects ought to be considered, including societal benefits. Attention has been paid to these topics for years by the public health community, and in particular EUPHA-HTA, and also Vaccines Europe, a specialized vaccines group within the European Federation of Pharmaceutical Industries and Associations (EFPIA), has recently released a position statement on the need to tackle vaccine specificities in future JCA. Furthermore, as the COVID-19 pandemic has highlighted, such aspects are critical for joint procurement efforts to combat the current and future pandemics. The objective of this workshop is to address the current and future role of HTA in vaccine regulation across EU countries, inform on key specificities of vaccines in respect to JCA and HTA as a whole and discuss opportunities to develop vaccine-specific methodological guidelines within the HTAR. Following an introduction, the following topics will be addressed: Competent bodies and HTA impact on vaccine decisionmaking process, incl. a discussion of implementation challenges and potential impacts of the HTAR. JCA specificities in respect to vaccines and the importance of addressing the whole value of vaccines and vaccinations.
The proposal of Vaccines Europe for guiding principles to account for vaccines specificities in JCA built on the outcomes of an 'across-the-industry' project incl. also nonindustry experts. The presentations will be followed by a panel discussion to further explore the themes, consider next steps and address questions from the audience.

Key messages:
The new EU HTAR is paving the way for JCAs of several medicinal products, incl. vaccines necessitating expert guidance in terms of determining their whole value. Vaccines specificities should be duly considered in JCAs to inform national and EU-wide decision-making for vaccination programmes, reimbursement, and pricing.
Abstract citation ID: ckac129.327 HTA and National Immunisation Program: an overview of competent bodies and of the impact of HTA on the decision-making process Results: The evidence shows that assessment frameworks vary across countries. Furthermore, the legal basis and the authority, namely binding or advisory, recognised to them could influence the way how NITAGs work and impact on vaccine policy. Eventually, the final decision is also led by other aspects, including financial and contextual factors that are less standardizable but should be always taken into consideration in health policy. In fact, the final decision on the inclusion of a vaccination into the national immunization program is made by the Ministry of Health also based on the evaluation of HTA bodies working in the country.

Lessons:
Considering the central role of NITAGs in vaccine policy, some efforts should be done in order to make NITAGs more authoritative and collaborative. This could help, from one side, to make the relationship between final decision and NITAGs recommendations clearer and, on other one, to make it possible to have shared and timely evaluations. In this respect, Joint Clinical Assessment envisaged by the new EU regulation on HTA could prepare the way for a more transparent and timely vaccine policy at European level.

Issue/problem:
Vaccines are an important public health intervention protecting the population against infectious diseases. The value of vaccines is broad ranging from individual to societal as achieving community immunity protects the unvaccinated, minimizes the risk of outbreaks, reduces the emergence of antimicrobial resistance, and leads to broader societal benefits. Description of the problem: Vaccines' market access processes are characterized by the development of recommendations by NITAGs followed by the assessment of health technology assessment (HTA) bodies in less than half of 27 EU member states. Despite that HTA for therapeutic drugs is well established, there is very limited experience in applying HTA methodologies to vaccines, especially for clinical assessments, as HTA methods and frameworks are traditionally geared toward therapeutics. However, following the adoption of the EU regulation on HTA, Joint Clinical Assessments (JCAs) of vaccines are expected.

Results:
To support a discussion on how to account for vaccine specificities in the JCA, Vaccines Europe has performed a project which aimed at developing a proposal for high-level guiding principles on processes and methodologies for clinical HTA for vaccines. The proposal is informed by findings of literature reviews on currently applied processes, methods, and clinical assessment frameworks of vaccines as well as the outcomes of an advisory board with scientific experts. Lessons: A proposal for high-level guiding principles for clinical HTA for vaccines is being developed based on both evidence and the advice from scientific experts which focuses on processes (e.g., horizon scanning, early advice, consideration of vaccinespecific expertise) and methods (e.g., unmet need, safety, efficacy/effectiveness, real-world evidence and technical characteristics of the technology). Lastly, the implementation of vaccines specificities in JCA represents a call for action. Description of the problem: The evaluation of vaccines has become more and more complex. This is not only because the number of vaccines on the market and the diseases they target has increased, but also because a much wider range of population subgroups can be targeted, and for some diseases several vaccines are available and need to be compared. In addition, vaccines not only have an impact on individual well-being, but can also induce population-level effects and broader socioeconomic value.

Results:
NITAGs consider to a large extent similar key factors in their evaluation processes. These can be divided into context-free and context-specific aspects. Context-free aspects include effectiveness and safety. Local disease epidemiology, potential vaccine impact at population-level, cost-effectiveness and societal or cultural values and preferences are context-specific aspects. Collaboration is possible when jointly assessing the evidence related to context-free aspects, since this is usually easily transferrable across countries. In 2019, a network of NITAGs in the European Union and Economic Area countries was established under the coordination of the European Centre for Disease Prevention and Control. Within this network, working groups have been established and either performed themselves or supported external contractors in conducting systematic reviews on vaccines effectiveness and safety.

Lessons:
The increasing workload of NITAGs and the prospect of joint clinical assessments draw the attention to a number of practical issues to be solved, e.g., consensus on methodological guidelines for systematic reviews, processes to ensure that the products meet NITAGs expectations (content, quality, timing), and approaches how to consider unpublished data.