Accounting for vaccines specificities in the Joint Clinical Assessment (JCA): a proposal for guiding principles

Abstract 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 vaccine-specific 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.


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. Issue/problem: NITAGs are independent expert advisory committees that provide evidence-based recommendations to the Ministry of Health to guide the introduction of new vaccines and formulate national immunization plans.

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.
iii134 European Journal of Public Health, Volume 32 Supplement 3, 2022