The EU Joint Clinical Assessment – Ready or Not, Here it Comes…
The Health Technology Assessment (HTA) landscape in Europe is undergoing a significant shift, with the introduction of the Joint Clinical Assessment (JCA) as part of the European Union’s (EU) Regulation on HTA (HTAR; 2021/2282). JCA will be introduced in 2025, initially for oncology products and advanced therapy medicinal products (ATMPs), with expansion to orphan products in 2028 and all new medicines (including, under certain criteria, Class IIb and III medical devices and Class D in vitro diagnostic devices) in 2030.
The Regulation, which introduces a permanent legal framework for joint HTA in the EU, aims to encourage greater cooperation between the 27 EU Member States, culminating in cross-border harmonisation of the clinical assessment that forms part of national HTA processes. The Regulation also states that, through formalising this joint process for clinical assessment, it will:1
- Ensure the efficient use of resources
- Strengthen the quality of HTA across the EU
- Save national HTA bodies and industry from duplicating their efforts
- Reassure business and ensure the long-term sustainability of EU HTA cooperation
As we draw close to the point of JCA commencing “next year”, it is unsurprising that JCA was a dominant presence on the agenda at ISPOR Europe 2023. Most discussions concluded that, although the prospect of joint HTA holds promise, a lot of uncertainty remains around how this will work in practice, contributing to a palpable sense of intrigue (and anxiety?) among the ISPOR audience. Some key topics discussed are highlighted below.
Will the JCA really harmonise decisions between Member States?
It was clear there was some anticipation that the JCA could transform how HTA is conducted on a European level…However, we observed some continuing confusion and misaligned expectations over what the JCA aims to achieve. The expected benefits of the JCA, in terms of reducing duplication of effort for industry and national HTA bodies with regards to preparation and assessment of clinical evidence, was well-communicated and this does – at least in theory – represent a potential benefit of JCA. The extent to which this benefit is realised will depend on the success of the implementation. However, the conference also saw multiple references to “alignment of decisions” and “consistency in access to medicines” across Member States as an expected benefit of JCA. We may see more consistency in reimbursement decisions across Member States, to the extent to which current divergent decisions relate to differences in the clinical evidence assessed and the approaches used to do so. However, the reimbursement decision remains a matter for national consideration and there are a number of reasons these decisions will still differ despite a common JCA:
- Although the JCA will be a single dossier, it will address multiple PICOs (Population, Intervention, Comparator, Outcome). Individual Member States still need to interpret the JCA for their own setting, which means they may focus on different PICOs and indeed may choose to carry out further assessments to determine clinical added value of the product as they see fit. For example, specific country-level considerations regarding product positioning may affect the interpretation of the findings of the JCA
- Many countries will also wish to conduct economic assessments, which can represent significant drivers of decision-making. Economic analyses and price negotiations remain decentralised with the final reimbursement decision made at a national level
- HTA is broader than just clinical and economic components, with other factors such as ethical, legal and social implications also feeding into the decision on adoption and reimbursement (often in a more deliberative manner)
It will be interesting to see if JCA does lead to more consistency in final decisions, but we should avoid assuming this will be the case or putting this forward as an a priori benefit of JCA.
Additionally, given the variety across Member States with regards to factors such as capacity and health system requirements, it remains unclear how the JCA will impact access and uptake on a practical level. There was even discussion that larger Member States could realise the most benefit due to the increased resource they have available to juggle input into JCA alongside national HTA processes, and leave smaller Member States behind; something that would go against the objectives of this Regulation.
Is industry ready?
The feeling in the room during the Q&As was that industry, in general, does not feel it is ready (one comment simply asking directly for a delay summed-up this mood quite succinctly!). Concern was particularly focused on how industry will manage with having to prepare a detailed JCA dossier alongside other market access activities, particularly the European Medicines Agency (EMA) submission (see the Figure below for an overview of how the two processes are likely to interact). The hope for JCA is that there will ultimately be a lower burden on industry and collective European HTA bodies over the course of a product’s HTA lifecycle by reducing the need for multiple different clinical assessments. However, the trade-off is that there is likely to be a greater burden on industry up front, given the timing of the JCA and the potential for the JCA to be significantly more complicated than any national-level clinical submission as a result of potentially needing to address multiple PICOs to meet diverse member state requirements. Indeed, a spotlight session on the first day of ISPOR Europe highlighted a study that simulated consolidated EU PICOs for a hypothetical lung cancer product and found that the JCA would need to address 10 or more PICOs simultaneously. This challenge is compounded by the fact that the final PICOs to be addressed in the dossier (i.e. the scope) will only be confirmed to the manufacturer approximately three months before the JCA dossier submission is due. Inevitably, therefore, manufacturers will have to start work on addressing anticipated JCA PICOs in advance of final scope confirmation, and those at the conference agreed that early engagement with key stakeholders, such as the EMA and Member State representatives, will be critical in guiding these preparatory activities.
Figure 1: Anticipated integration between the JCA and EMA regulatory procedure
How can industry ready themselves?
Although there was a lot of discussion on uncertainties, practical solutions were also covered. Four key themes related to preparation are highlighted below:
- At an organisation level, there will be a need to define a clear process for JCA dossier preparation including outlining what additional resource is required, how different functions and regions should collaborate, timelines for preparation and who is ultimately responsible for the sign-off
- Industry must also focus on their drug pipeline, to ensure that they have resource, established responsibilities and timelines in place in time to support the HTA of assets at both an EU and national level
- With regards to the overall process, industry were encouraged to have a plan in place for anticipating Member States PICOs, during which early engagement with Member States will be key. Additionally, a need for industry to consider how best to integrate and link the JCA process with other activities, such as the clinical development program, was highlighted
- Industry are encouraged to monitor the JCA landscape, maintaining an awareness of the guidelines and implementation acts that are soon to be published and may clarify some of the existing uncertainties
And what about the JCA Coordination Group?
Industry will need to embrace preparation for the JCA process (it is happening!). However, there are some clear areas in which the approach to the JCA from the JCA Coordination Group, responsible for the scoping phase of a JCA, will have an important bearing on the success of the process:
- Changes in scope from PICO drafting/scoping workshop to issuing of finalised scope will need to be limited, to allow industry to prepare evidence with confidence in a timely manner
- In cases with potential for a large number of PICOs, there will need to be a level of pragmatism applied by the JCA Coordination Group, in order to keep PICOs to a reasonable level. This will require effective and strong project management, with clear demarcation of what is “important” versus what is “nice-to-have” when digesting Member State PICO survey responses. Hopefully, EUnetHTA and BeNeLuxA(Ir) initiatives have provided some experience to build upon in this regard. It was noted at the conference that the specifics of the approach to project management on the JCA side are still being developed. Whilst there is still plenty of time before January 2025, it feels important to the success of the JCA that preparatory activities between now and then imbue the JCA Coordination Group with sufficient confidence, capacity and derived experience from other similar initiatives that they are able to effectively translate potentially highly divergent Member State PICOs into something that works for a joint process