The Joint Clinical Assessment Dossier: A Fresh Approach or Business as Usual?

The European Union (EU) Joint Clinical Assessment (JCA) is set to launch in January 2025 with the aim of harmonising health technology assessment processes (HTA) across EU Member States. The process intends to expedite access to new health technologies and prevent inefficiencies in the evaluation of clinical evidence across the EU.

The final JCA submission template was published by the European Commission in May 2024 and defines the information that manufacturers are required to present as well as the structure for doing so.

On the one hand, the JCA template does not present any surprises and will be reassuringly familiar to many. On the other hand, the challenges posed by the need to address multiple PICOs, combined with the short period of only three months between PICO finalisation and submission deadline, will require submitting manufacturers to adopt a proactive approach to dossier development and be smart about initiating elements of the write-up ahead of PICO finalisation. Here, we outline this familiarity and the differences observed within the template compared with other HTA bodies, and also consider the potential approaches that manufacturers can take to simplify the dossier development process.

Observations on the Dossier Template: Clinical Scene-Setting

The dossier template will feel familiar to anyone who works with national HTA submissions. Whilst the JCA template closely builds on the previous tried and tested EUnetHTA submission template, the JCA version contains less contextual guidance on exactly what the assessors are looking for manufacturers to present. Referring back to the EUnetHTA template provides useful additional context to ascertain the intentions of the JCA template where it is currently a bit ambiguous. The German Federal Joint Committee (Gemeinsamer Bundesausschuss [G-BA]) template has additionally clearly served as inspiration for both the prior EUnetHTA template and the finalised JCA template – the JCA dossier closely resembles the G-BA template in terms of both structure and language.

One notable difference from the G-BA dossier is that the JCA template includes sections that effectively constitute a ‘Background’ section and are intended to provide a comprehensive overview of the medical condition, its clinical management and estimates of disease burden. This ‘Background’ section echoes the National Institute for Health and Care Excellence’s (NICE) ‘Health condition and position of the technology’ section, with submitting manufacturers being required to detail the pathway of care, the proposed positioning of the technology, and relevant clinical guidelines.

This summary should present an overarching view of the pathway of care across the Member States, and highlight any significant diversions from the standard pathway. Whilst in some therapy areas the clinical pathway is clear and defined, this is not always the case – for example, where clinical practice is highly variable, involves use of off-label comparators and/or comprises nuanced subpopulations within the overall clinical indication. These challenges could be amplified when considering the need to summarise the care pathway across a large number of Member States. Whilst this section of the JCA dossier template initially sounds straightforward, it has the potential to be a difficult section to develop if submitting manufacturers go too far down the rabbit hole with this. As the Population and Comparators will already be defined by the PICOs, this section does not need to establish the rationale for the populations and treatments considered. It is intended to be an overview and therefore a pragmatic approach is likely advisable – leveraging European guidelines where possible to inform the basic standard pathway and only focusing in on major potential discrepancies in certain member states. It will be important though that manufacturers offer an overview of the clinical condition and treatment pathway that is not at odds with the PICOs addressed later in the dossier (for example, summarising the clinical condition through the lens of one disease staging criteria when the PICOs use a different staging criteria to define population subgroups would risk confusion).

Table 1. Comparison of the JCA, G-BA and NICE templates

Table 1: Comparison of the JCA, G-BA and NICE templates

Footnote: A tick has been allocated when the topic is explicitly covered as a defined section in the dossier. Consequently, a cross does not indicate the topic is not mentioned at all in the dossier, but that it is not a defined section of the dossier.

Abbreviations: G-BA: Der Gemeinsame Bundesausschuss; HTA: health technology assessment; JCA: Joint Clinical Assessment; NICE: National Institute for Health and Care Excellence; SLR: systematic literature review.

How Can Submitting Manufacturers Prepare Their Dossiers in a Timely and Efficient Manner?

The JCA process allows for only a three month period between the manufacturer receiving confirmation of the PICOs they need to address and the submission deadline. Three months does not provide much time to produce a high quality, clear and compelling dossier from scratch, and the task of achieving clarity in the presentation of evidence addressing multiple PICOs should not be underestimated. We suggest that manufacturers take a highly proactive approach to identifying which sections of the submission template can be drafted in advance of the PICOs being finalised (in a similar manner to how work on a NICE submission needs to start before the final scope is issued). Fortunately, there is ample scope for this, for example:

  • Information about the medicinal product under assessment and the HTD, and previous assessment under the HTAR (Sections 1.1–2)
  • Description of the medical condition and characterisation of the target patient population (Sections 2.1.1–1.2) –whilst prepared in advance, this section should be revisited after PICO finalisation to ensure that the description of the medical condition is coherent with any patient population definitions provide by the PICOs (e.g. with regards to reference to staging criteria, mutation biomarkers or lines of therapy)
  • Overview of the medical product and characteristics of the medical product (Section 2.2)
  • Overview of the clinical management of the condition (Section 2.1.3) –this section should be revisited after PICO finalisation to ensure that broadly the description of the clinical management of the condition is not at odds with the populations and comparators defined by the PICOs
  • Description of methods used in study selection and information retrieval (Sections 4.1–2.2)
  • Results of the literature review, if an initial literature review has already been performed prior to PICO finalisation as per the suggestion from our colleagues in this post (Section 5.1) – this will then need revisiting in line with the completion of the literature review update
  • Description of the design, methodology and results of the included original clinical studies (Sections 4.3.1–3.2)

As noted above, once the final PICOs are established these sections may need adjustments to account for nuances in PICO definitions or to include new, relevant elements that were not initially anticipated, but some important groundwork can be laid before PICO finalisation, allowing the final 100 days of submission development to be focussed on addressing the PICOs in the clearest and most compelling manner and ensuring all relevant internal stakeholders (of which there could be many!) are happy with the final submission.

In addition to proactively drafting some sections of the dossier, submitting manufacturers can also help get ahead by creating a skeleton outline of the dossier based on predicted final PICOs from the assessors. It is easy to think that Section 5 of the dossier, where clinical study results by PICO will be presented, requires no advance preparation in terms of writing the dossier because it will be a case of simply plugging in clinical results. However, this underestimates the realities of completing this section. For example, there will be key questions regarding how to order the PICOs, particularly to balance the different views of stakeholders within submitting manufacturer companies, and precisely which evidence pieces will be brought to bear in support of each PICO and how these will be presented in a way that avoids too much repetition and hence risk of confusion. We expect that most manufacturers will run their own PICO scoping studies to try to predict the final PICO list with some level of certainty. With these probable PICOs in mind, manufacturers can use an outline to get ahead on starting to think about these questions and obtaining internal alignment on these key decisions.

The modular structure of the JCA template, with the clear sectioning by different PICOs, additionally allows for different PICOs to be easily and efficiently removed from or added to the dossier in the final 100 days of submission development. This would allow for more certain PICOs from the manufacturer’s scoping to be prepared ahead of receipt of the JCA’s final PICO list, to aid in minimising the workload during the end stages of the dossier development. This modular structure can also bring challenges of potential repetition in the dossier, which will require careful crafting to ensure the submission remains concise, and with a clear narrative.

The need to invest time in preliminary dossier write-up activities should be assessed on a case-by-case basis as the risk-benefit ratio is likely to differ between submissions, depending on the complexity of the disease pathway, treatment landscape and clinical evidence available. Submitting manufacturers should carefully weigh the risks and benefits of each activity, and decide which ones should be undertaken before the finalisation of the PICOs.

Key Takeaways

The JCA dossier template represents an evolution in clinical evidence requirements for HTA, rather than a complete reinvention of existing approaches. The requirement for submitting manufacturers to present clinical evidence by PICO will require careful planning and collaborative approaches to evidence generation and dossier development. To meet these demands, it is imperative for manufacturers to adopt a proactive approach, initiating parts of the dossier development early on, and planning in advance their strategy for reporting their clinical evidence within the PICO-orientated results section of the dossier.

If you would like to discuss the above approaches in more detail, or how Costello Medical can support you with the upcoming JCA process, please do not hesitate to contact Matt Griffiths, Global Head of HTA (LinkedIn), Abigail Lampkin, Senior Analyst (LinkedIn) or Fern Woodhouse, Consultant (LinkedIn). The views/opinions expressed here are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners.

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