EU HTA: Can a one-size-fits all approach really work, and how can manufacturers prepare?

EU HTA marks the beginning of efforts to streamline and harmonize the Health Technology Assessment (HTA) processes and outcomes across European Union (EU) member states.

Despite its imminent rollout (from January 2025*), key uncertainties remain around its implementation and likely success. How are EU member states going to leverage the Joint Clinical Assessment (JCA) as part of their individual market evaluations? Will the initiative achieve key goals of improved efficiency, harmonization, and time to access? What implications will the process have for manufacturers looking to launch in Europe?

EU HTA: Can a one-size-fits all approach really work, and how can manufacturers prepare? Article by Georgina Powell (Senior Consultant) & Daisy Pharoah (Senior Analyst), Windrose Consulting Group

We covered these questions with payers from key European markets (France, Germany, Italy, Spain, Netherlands and Poland), and include insights from these discussions below. We conclude that while EU HTA presents good opportunities in theory, there may be limited change to market-level processes in practice, given that - ultimately - individual evidence requirements are likely to remain the same, at least in the short term. That said, given EU HTA is on the near horizon, manufacturers will need to be well-prepared to avoid any pitfalls, and ensure a smooth JCA evaluation and successful launch in Europe.

What is the JCA Process and Output?

Table 1: PICO requirements from member states, are used to define the assessment scope for the JCA, collected as part of a scoping process: Indicative Pan-EU HTA Timeline [1].

The JCA process is timed in parallel with EMA evaluation. It provides a summary of the available clinical evidence, with the assessment scope defined by the PICO framework (population, intervention, comparator, outcome). In the initial scoping process, all member states outline individual PICO requirements. The PICOs then undergo a consolidation and validation process before the final list is shared with manufacturers and forms the foundation of the JCA dossier. The final output of the JCA is an objective summary of evidence, and member states will still retain responsibility for making their own conclusion of the level of clinical benefit [1].

How Will Markets Use the JCA In Their Assessments, and Why?

Market Assessment

Though designed to ultimately replace individual market evaluations, initially, member states are required to take “due consideration” of the JCA in their own HTAs. However, there is great variation - and indeed uncertainty - around the extent to which market states will leverage the JCA in their assessments, with no markets anticipating immediate shifts in current practices.

Some markets, such as Poland (who have had minimal involvement in the JCA) have not yet started discussions about incorporation of EU-HTA, given competing national priorities. For others, such as the Netherlands, better-established partnerships (e.g., Beneluxa) will likely take precedence over more widespread collaboration. In Italy, the JCA has the potential to impact the level of “innovation status” by informing key parameters of level unmet need and quality of evidence; however, given arguably declining importance of innovation status, this may not make a tangible difference to pricing and reimbursement decisions. In Spain, while the national IPT assessment (if ongoing post-2024 reforms) will consider the JCA evidence across specific populations of interest or comparative benefit, regions are unlikely to use EU-level information given individual formulary considerations.

Even markets that have played a pivotal role in the development of the JCA – France and Germany - are resistant to deviation from their existing practices. The G-BA expect to incorporate the JCA analysis similarly to assessments by IQWiG, but it will not affect their process for determining the level of additional benefit. In France, level or ASMR will remain the key driver of pricing negotiations, with decisions made nationally based on the TC’s national requirements and comparisons to French standard of care.

Key Payer Concerns

Broadly, expectations to continue with market specific HTAs are driven by a commitment to long-established processes and requirements (e.g., around trial length, or acceptability of endpoints). The prevailing belief is that existing HTA procedures effectively serve individual markets, and there is widespread concern regarding the feasibility of standardizing PICO requirements across markets to meet their unique needs. Beyond these obvious concerns, there are more subtle barriers to adoption of the JCA; for example, the nuanced language used in German and French assessments, which payers do not believe will be adequately addressed through a Pan-European approach. There are also uncertainties relating to re-assessments to accommodate evolving evidence, the integration of which in EU HTA is currently unclear.

Overall, payers are more receptive to the idea of establishing the level of unmet need or quality of evidence on a pan-European basis; this could, for example, inform the level of SMR in France or innovation status in Italy. However, by design, the outcome of the PICO process more closely relates to the level of additional clinical value, which will likely remain heavily market-specific (at least in the short term).

What are the Opportunities & Challenges with Meeting the EU HTA Goals?

Efficiency and Avoiding Duplication

A key objective of EU HTA is to improve efficiency by avoiding the duplication of efforts across member states. Crucially, this objective of efficiency hinges on the consistent adoption of the JCA by markets in their HTAs. Given reluctance to adopt a process that is still associated with significant uncertainty, the feasibility of achieving this objective is somewhat doubtful. The PICO process poses another threat to improved efficiency and risks being onerous to both market systems and manufacturers. With almost 90 successful EMA submissions annually2, a European assessment will impose an additional workload on market systems, requiring involvement in the PICO scoping process for each submission. The downstream consequences of such emphasis on local practices may further drive payer expectations for more tailored evidence, ultimately increasing development costs for manufacturers.

Harmonization of HTA Outcomes Across the EU

In theory, EU HTA provides an opportunity for standardization of the relevance and interpretation of clinical outcomes between European markets, and relative weight placed on different factors in the decision-making process. Some outcomes may increase in importance universally; for example, patient-reported outcomes, given the influence of Germany and France. However, in practice, the JCA is unlikely to lead to materially different state-level outputs. Firstly, the JCA lacks a conclusion on relative therapeutic benefit, leaving markets to make their own decisions. Secondly, achieving alignment necessitates widespread adoption, with markets leveraging the JCA in their own evaluations (ultimately streamlining evidence requests). Without prominent markets like France and Germany taking the lead, it is doubtful that smaller markets will follow suit, hindering the potential for the initiative to gather enough momentum to make substantial change.

Implementation and Time to Access

The JCA (if timed in parallel with the EMA) has potential to shorten time-to-access across markets. However, if multiple PICO requirements are received, the consolidation process could cause significant market access delays. Another layer of complexity could be added to this if, for example, the EMA requested a label change once the JCA was already underway. The JCA also provides opportunity to facilitate unified timelines for reimbursement across Europe; however, some markets benefit from a later submission. An example of this is Poland, who often leverage real-world evidence, and/or HTA and pricing decisions in other markets to inform their own evaluations.

Critically for manufacturers, the JCA dossier will likely necessitate increased EU-level resources to facilitate submission of both EMA and JCA dossiers in parallel during a traditionally busy period. While there will be time during dossier review for both commercial and access teams to work on the access strategy, early alignment on positioning may be required to support the JCA submission.

Recommendations for Manufacturers

In summary, the imminent EU HTA process is intended to reduce the duplication of submissions, streamline workflows, and improve time to access. However, mounting demand for more complex evidence, likely need for increased resource capacity, and risk of delayed access all present significant hurdles to manufacturers. Nevertheless, EU-HTA is on the near horizon, so manufacturers should proactively consider the following steps to ensure continued success in the EU at launch:

Define potential PICO requirements early: This will enable manufacturers to optimally shape clinical trial design. To do so, monitor the first products to go through the JCA to understand evolving clinical-trial criteria. Additionally, given that KOLs and patient groups are set to play a role in the PICO scoping process, engagement with these stakeholders may be helpful to identify requirements ahead of time, in case evidence generation is needed prior to launch.
Optimize the use of the JCA in market-specific dossiers: As payers remain uncertain on how to use the JCA, there is potential for manufacturers to be involved in shaping how markets leverage it in their dossiers. For example, by targeting optimal positioning in JCA submissions, and leveraging any positive outputs in their individual dossiers (keeping in mind that it is not yet clear the level of conclusion that the JCA will draw).
Ensure sufficient EU resources: Forward thinking and the need to plan collaborations and investments is highly important in this evolving environment. Smaller manufacturers may need to look to collaboration if additional resources (at EU-level) are not feasible; similarly, multi-national players can seek opportunities to grow their capabilities through partnership with a diverse range of smaller developers.
Work with JCA committee early to shape requirements: Payers recommend manufacturers should work with the JCA committee to help shape the process of consolidation of PICOs and outputs. However, it currently remains unclear how feasible this is, or at what point the JCA will request manufacturer engagement. It is likely that learnings can be taken from smaller-scale yet similar initiatives, such as Beneluxa and Finose HTA.

How Can Windrose Help?

Windrose is helping clients to navigate the upcoming EU HTA and complex uncertainty that it brings. Through our ongoing work in market access, extensive experience working across the European markets, and commitment to staying atop of developments in this space, we endeavour to ensure Global Market Access teams are equipped with strategies that align with the criteria for success. For more information, please get in touch via email at info@windrosecg.com.

Credits

Many thanks to the contributions of Bernard Avouac, Jean-Francois Bergmann, Stefano Capri, Miguel Angel Calleja Hernandez, Mathias Flume, Frank-Ulrich Fricke, Fabrizio Gianfrate, Dominik Golicki, Peter Kolominsky-Rabas, Maarten Postma, and Joan Antoni Valles Callol.

Article written by Georgina Powell (Left) and Daisy Pharoah (Right), Windrose Consulting Group

Footnotes

*ATMPS and oncology drugs in 2025, orphan drugs by 2028, and all EMA-registered drugs by 2030

References

1. EUnetHTA: Practical Guidelines (D4.2 Scoping Process); available here: eunethta.eu

2. European Medicines Agency: Human Medicines Highlights 2022; available here: ema.europa.eu