The Critical Role of the Global Value Dossier in EU Joint Clinical Assessment

The introduction of the European Union (EU) Joint Clinical Assessment (JCA) seeks to create efficient and unified cross-border clinical assessments that inform national health technology assessment (HTA) processes, as summarised in our previously published report on EU JCA discussions at ISPOR Europe 2023. For manufacturers, the EU JCA process will demand increased collaboration and the integration of diverse workstreams across multiple cross-functional teams to deliver a compelling and evidence-based JCA dossier which addresses all relevant PICOs (Population, Intervention, Comparator, Outcomes) identified by the JCA assessor and co-assessor, in line with the submission dossier template and in adherence to HTA coordination group methodological and reporting guidance. Importantly, this dossier will need to be finalised under considerable time pressure, as there are only ~three months between the confirmation of the PICOs to be addressed in the JCA dossier and the submission deadline.

In the context of these tight timelines and the acute workload implications for manufacturers, the importance of developing a robust Global Value Dossier (GVD) as early as possible is greater than ever. A timely and well-written GVD will ensure that the relevant strategic considerations and value narrative have been explored in advance of JCA dossier initiation whilst also saving manufacturers time and resources during the JCA dossier writing process.

The role of the GVD in JCA dossier development

A typical GVD structure covers all clinical domains required for the JCA dossier, as illustrated in Figure 1. A high-quality GVD designed with JCA requirements in mind could therefore significantly ease the JCA dossier development process by providing readily adaptable content for the JCA dossier (as outlined below). We also anticipate that the GVD will take on a significant role in the EU JCA process prior to JCA dossier development; early development of a high-quality GVD provides an opportunity for key functions, including value and access, commercial, medical and regulatory to align on a clear value narrative for a new technology, key areas of differentiation from competitor treatments and any outstanding evidence gaps well in advance of the JCA dossier writing process. The agreed narrative can then be leveraged consistently within the JCA dossier and other HTA submissions, and evidence can be generated to address any outstanding gaps.

Figure 1. EU JCA dossier and GVD overlap

Abbreviations: EU: European Union; GVD: Global Value Dossier; HTAR; Health Technology Assessment Regulation; HTD: Health Technology Developer; JCA: Joint Clinical Assessment; JSC: Joint Scientific Consolation.

Creating a “JCA-ready” GVD

Whilst a typical GVD structure is fairly well-suited to providing easily adaptable content for JCA dossier development, there are several key areas where consideration of JCA requirements can optimise the usefulness of the GVD to the JCA dossier writing team (as outlined below). However, it is important to balance JCA requirements with the needs of the diverse range of markets outside of Europe; it is impractical for a GVD to comply with HTA dossier requirements for every country and region globally, but thoughtful planning can ensure that GVDs are as useful as possible for all potential stakeholders. In our view, careful consideration of the following points can ensure a GVD is as ‘JCA-ready’ as possible while still remaining useful for other markets:

• Addressing multiple PICOs – JCA dossiers will likely need to address multiple PICOs (as determined during the scoping process). Although the final PICOs will not be confirmed at the time of GVD development, manufacturers should develop a list of potentially relevant PICOs in advance of GVD development through PICO scoping exercises.
  • The best approach to addressing these multiple PICOs in a GVD will depend on the extent and nature of the anticipated PICOs, but it is clear that including relevant supporting data for every potential PICO could result in a lengthy and potentially hard to navigate document, and risks diluting or confusing the core value story. We suggest that ‘JCA-ready’ GVDs should only cover the ‘priority’ PICOs identified by the manufacturer as relevant to multiple countries/regions in the main body, and should provide a summary of other potentially relevant PICOs in a tabular format in an Appendix and/or specific sub-section, including a clear indication of any available clinical and economic data which can be used to support each PICO.
• Epidemiology Data – the JCA dossier must present epidemiology estimates for all countries within the JCA scope. However, we suggest that a ‘JCA-ready’ GVD should include country-specific epidemiology data in a tabular format in an Appendix and remain focused on European (and global) epidemiology data in the main body content, to strike the right balance between detail and readability.
• Treatment Pathways – the JCA dossier will need to describe any major differences in clinical pathways for the target condition between European countries. GVDs typically focus on major regional clinical society guidelines and provide a list of other relevant guidelines in an Appendix,  as providing a summary of all country-specific treatment pathways for a specific condition within a single document is impractical. We suggest that a ‘JCA-ready’ GVD will retain this approach, but with a greater emphasis on ensuring that summaries of country-specific guidelines and treatment pathways provided in the GVD Appendix are complete and any major differences between countries are highlighted.
• Product Characteristics – the JCA dossier requires the presentation of specific information about the health technology (e.g. mode of administration, therapeutic class) in a tabular format. This information is also likely to be useful to other markets, and a ‘JCA-ready’ GVD can therefore incorporate these tables into the GVD structure.
• Clinical and Humanistic Value Data – as highlighted above, JCA dossiers will need to address multiple PICOs and comply with HTA coordination group methodological and reporting guidance (e.g. on subgroup analysis reporting). A ‘JCA-ready’ GVD should therefore appropriately report data on relative efficacy and safety in line with these requirements, and provide a clear indication of which clinical data (e.g. subgroup analyses, indirect treatment comparisons [ITCs]) are available to address the anticipated PICOs in a tabular format in an Appendix and/or specific sub-section.

JCA requirements and HTA coordination group methodological and reporting guidance are also likely to have a significant impact on the evidence generation activities which feed into the GVD, including systematic literature reviews and ITCs. Our thoughts on how JCA may affect systematic literature reviews are summarised here, and we will also be publishing our thoughts on how JCA may affect ITCs on our website in the future.

JCA dossier and GVD development timelines

The finalisation of global economic models is often a key roadblock to GVD finalisation. As a result, we often suggest that GVDs are developed in two phases, with a Phase I GVD developed first to provide all relevant clinical data (Disease Background and Unmet Need, Product Characteristics, Clinical and Humanistic Value) and a Phase II GVD developed later to add non-clinical domains (Economic and Societal Value). Early delivery of the clinical sections of a GVD is helpful for all markets to help inform discussions around clinical messaging and HTA submission development, but this approach is likely to become even more relevant in the era of JCA given that the JCA dossier focuses purely on clinical domains.

For more information about best practices in GVD development, look out for our upcoming piece on GVD development considerations outside of EU JCA requirements.