The Home Stretch: Is Europe Any More Ready for the HTAR One Year On?

With less than two months to go until the European Union’s (EU) Regulation on HTA (HTAR; 2021/2282) begins, ISPOR Europe 2024 unsurprisingly continued to have a heavy focus on Joint Clinical Assessments (JCAs). The conference regularly explored how both health technology assessment (HTA) bodies across the Member States and health technology developers (HTDs) are preparing for the starting block.

The uncertainty felt across the HEOR community about how the JCA process will work in practice was palpable at last year’s conference, as highlighted in our post-congress report from ISPOR Europe 2023. There remains a strong sense of trepidation going into 2025 – particularly from HTDs. Despite this, the mood has now shifted; this close to launch, there is a collective acknowledgement that the best way forwards is with a sense of cautious optimism to make the best of the initiative. With the publication of various guidance documents over the past year, is Europe any more ready now for the JCA than this time last year? We explore perspectives from the conference of key stakeholders in the JCA process below.

Are Member States Ready?

During the conference, it was evident that different Member States are at varying levels of preparedness for the implementation of the JCA, reflecting differing national priorities and existing HTA frameworks.

Germany’s Federal Joint Committee (G-BA) and the French Haute Autorité de Santé (HAS) have been leading participants in the development of the JCA process. Both HTA bodies expressed expectations that the introduction of the JCA will cause minimal disruption to their existing processes, with the JCA report complementing the existing Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) process in Germany and the JCA being a seamless transition for HAS considering their existing separation of clinical and economic assessments.

Whilst the HTAR is viewed positively by both G-BA and HAS, there are still aspects of the process that need defining. The AMNOG dossier is submitted, on average, approximately 40 days following the EMA regulatory decision, and there is a desire to ensure that the release of the JCA report does not delay this process in any way. With the JCA being conducted in English, there are also language considerations for HAS, who have not yet determined if the JCA report should be provided as an Appendix in English or if any translations will be required.

Other HTA bodies at ISPOR conveyed their optimism about addressing the access gap through JCA, and the benefits of joint collaboration, in particular HTA bodies based in Eastern Europe. This optimism was balanced with the highlighting of the practical challenges associated with JCA implementation, including the need to build additional capacity, such as at the Spanish Agency of Medicines and Medical Devices (AEMPS) and the Italian Medicines Agency (AIFA), and in some cases the requirement of adaptations to national reimbursement legal frameworks, such as in Poland. Uncertainty about the ability of the JCA to capture the broader perspective of Europe’s diverse demographics was consistently reiterated.

Are HTDs Ready?

From an industry viewpoint, the introduction of the JCA continues to represent both opportunities and significant challenges. There is a consensus that JCA should lead to harmonisation and more predictable outcomes, ultimately leading to expedited market access. The notion of JCA as a tool for improving evidence generation is also embraced.

With the timelines for when an HTD will have products likely to hit the JCA varying – from immediately to a few years down the line – it is unsurprising that the level of preparation for the JCA across HTDs also remains varied. A notable change from this time last year, however, was that many industry representatives were able to talk in more detail, and with tangible examples, regarding preparatory work that they have done. Examples of this included:

• Establishment of cross-functional teams and/or steering committees to lead on JCA and disseminate learnings from Global level to local affiliates and Medical Science Liaisons (MSLs)
• Investment in capacity and capability building across teams, including the up-skilling of teams through internal trainings
• Consideration of the level and timings of local affiliate involvement, particularly for affiliates with very limited resource
• Determination of the required Governance and sign-off processes, and where final responsibilities for submission sign-off lies

Whilst lots of work has been conducted internally to prepare HTDs for next year, the same concerns from industry at last year’s conference recurred again this year. Further guidance from the JCA coordination group across 2024 has provided some practical guidance, but has not yet allayed fears relating to the potential scale of the number of PICOs, the ability to manage the resulting evidence complexity, and the ability to meet the short timelines within the process, to name a few. The unresolved concerns around the limited role of industry within the scoping process echoed around the conference, with some HTDs expressing a frustration at a perceived naivety from the JCA Coordination Group about the challenges of excluding HTDs from that process raises.

Is Early Dialogue the Winning Strategy?

A key recurring theme from both HTA bodies and HTDs was the importance of the role of the Joint Scientific Consultation (JSC) process in allowing HTDs to appropriately prepare for the JCA and understand the evidence requirements they must meet. HTDs who have been through JSCs spoke enthusiastically about the benefits that the process provides, with well-organised input from the Committee. However, it is expected that only up to 10 advice slots will be available in 2025; 5–7 JSCs for pharmaceutical medicines are expected, compared with 25 JCAs.¹ With so few slots available, many products with strategically complex and challenging narratives and evidence bases are likely to be excluded. Research by Costello Medical presented at ISPOR Europe 2024 explored how achievable (or indeed not…) the JCA evidence requirements are for rare and non-rare treatment landscapes. This highlighted, in particular, the additional barriers facing orphan products for the JCA; these products are often developed by smaller HTDs with fewer resources to be able to meet the evidence requirements, however the capacity of the HTD is not considered within the selection criteria for JSC.²

Some HTA bodies, such as HAS, are anticipating further efficiency benefits of JSC, with products that have received JSC advice not subsequently being eligible for HAS Scientific Advice and therefore reducing the resource burden of conducting Scientific Advice at a national level. However, these added benefits at a national level will only be truly achieved in a meaningful way with capacity changes for the JSC.

Whilst the JSC will undoubtedly provide an invaluable source of advice and support for the JCA, the current capacity needs to be substantially increased for it to meet its objectives, and provide a broad, even playing field for all HTDs to benefit from.

It’s Time to Kick-Off

As we head into 2025, HTDs and HTA bodies across the EU are braced for a bumpy ride; no one anticipates the start of the JCA to be smooth, with a period of trial and error expected as the process fully settles in and remaining uncertainties are resolved. Time will tell if the cautious optimism from some quarters at the conference will really translate into reality. The collective willingness of HTDs and HTA bodies to accept the challenges, face them head on, and make the best of the process will help the JCA towards achieving its aims of improved collaboration. On your marks, get set…