A new position paper from Vaccines Europe calls for an EU-wide committee and effort to implement clinical health technology assessment (HTA) methodologies that are specific to vaccines.
The vaccine-specific initiative would be part of a broader effort by the European Commission (EC) to institute common HTA methodologies and tools throughout the EU. Vaccines Europe, together with the European Federation of Pharmaceutical Industries and Associations’ (EFPIA), is seeking to ensure that “the unique specificities of vaccines” are taken into account when any proposed regulation is implemented; the groups also seek to avoid duplication of effort and “unnecessary administrative and regulatory barriers,” according to the position paper.
Vaccines Europe is calling for a European vaccine clinical HTA committee to be established; the committee “should reflect vaccine specific expertise and capacities and include all relevant stakeholders,” with vaccine and public health experts and members of relevant immunization advisory groups and the European Centre for Disease Prevention and Control all represented.
Clinical HTA assessment of vaccines should engage vaccine and public health experts at all stages “to ensure that the direct and indirect impact of vaccines on individuals, society and public health will be properly assessed,” according to the position paper. As Vaccines Europe envisions the process, initial steps will be to flesh out processes and develop guidelines for vaccine HTA, so the actual join clinical assessment process will come at a later stage.
During the implementation of the proposed regulation, the vaccines industry should be engaged and “closely involved” in the development of clinical assessment methodologies that are vaccine-specific.
The position paper lays out some of the many attributes of vaccines that contribute to their broad societal and health value, including the reduction of morbidity and mortality, reducing cross-border threats, the promotion of herd immunity to protect the vulnerable, reducing the risk of antimicrobial resistance, and broader societal benefits such as “societal peace of mind” and reducing the risk of disruption from unpredictable outbreaks.
Among the core vaccine features to be considered in a vaccine-specific clinical HTA framework are those that address a health problem or unmet need. These include the short- and long-term consequences of disease for individuals and those caring for them, as well as the disease burden at the population level. The clinical HTA methodology must identify which individuals should be targeted for routine vaccination “based on risk factors and transmission patterns.
In terms of technical characteristics, the HTA framework should take into account how complex the research and development process is for a particular vaccine, as well as what its storage requirements will be. Additionally, the “goal, scope and intensity” of the immunization strategy for the vaccine should be factored in, as should the optimal dose and dosing interval “to secure high uptake.”
When assessing clinical effectiveness of a vaccine, composite endpoints should be considered, since they enable “robust estimation of the vaccine impact on health-related quality of life.” The position paper gives the example of burden of illness as one such composite endpoint.
Surrogate endpoints may also be needed if long-term benefits, such as reduction in cancers seen only years after vaccination, are not detectable within the time frame of a clinical trial, or when efficacy data for a new vaccine is not yet available.
Modern and evolving epidemiologic techniques such as population-based modeling and the use of real-world data should also be considered. However, Vaccines Europe cautions that in all cases, “quality of the evidence must be assessed and robust analyses using rigorous standards must be considered.”
Safety, the last of the four “Relative Effectiveness Assessment” domains, should be assessed in light of existing vaccine-specific regulatory requirements, and assessors should also take into account how the public views the safety of a particular vaccination.
Finally, wrote Vaccines Europe, “Considering especially the insights from COVID-19 pandemic and vaccination against SARS-CoV-2, additional vaccine features beyond the four domains such as the prevention of inequities should be thoroughly assessed for their potential inclusion in clinical HTA.”
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