Over two years have passed since the EU Health Technology Assessment – EU HTA regulation was announced. The first draft implementation act was published in March 2024 for public consultation, with a go-live date set for January 12, 2025. This timeline leaves manufacturers of oncology and advanced therapy medicinal products (ATMPs) with less than 10 months to prepare for the new regulation. While we await further clarity, there are several proactive steps manufacturers can take now to optimise their opportunities under this regulation.
This article is based on the content presented in our two webinars:
Organisational Preparation
Effective organisational preparation is crucial for success due to the tight Joint Clinical Assessment (JCA) timelines aligned with European Medicines Agency (EMA) submissions. Currently, the regulatory and HTA processes across the EU are linear and sequential, with global teams handing over responsibility to regulatory teams and then to affiliates. Consequently, market access strategies are developed similarly. However, the EU HTA will change this, as EMA and JCA submissions will occur simultaneously. The JCA will assess clinical effectiveness for all EU member states concurrently, necessitating effective collaboration among global, regulatory, and affiliate teams to achieve market access.
Given the tight timelines and the range of internal stakeholders involved, a robust EU HTA process is critical. Developing this process requires clear communication of roles and responsibilities to facilitate collaboration and avoid duplication of work. New roles and responsibilities, along with new hierarchical management structures, may be necessary to ensure the process is efficient and effective. Resource requirements are likely to increase, as the JCA process will run in parallel with EMA evaluation. It is also essential to reevaluate the development of value materials to ensure they are fit for purpose and ready on time.
Although the optimal structure for aligning EMA labels and JCA evidence needs is still unknown, manufacturers must start early to establish the right organizational structure for JCA dossier development. This may involve learning from affiliates’ HTA structures and processes. Additionally, internal education is essential to ensure awareness of EU HTA across all functions and to emphasise the collaborative nature of market access activities to secure patient access.
Joint Scientific Consultation (JSC) Advice
To meet JCA requirements, seeking JSC advice before the start of pivotal trials can improve the quality and relevance of data and clarify the focus on relevant PICOs (Population, Intervention, Comparator, Outcome). Not all products will be selected for JSC advice, as selection criteria include unmet medical needs, major EU-wide added value, and Union research priorities.
Currently, very few JCA slots are available, and it is uncertain when more will open. Manufacturers should assess how their products could benefit from JSC advice and prepare a strong case for inclusion in the program. If JSC advice is not utilised for JCA submission, manufacturers must justify this decision, though the impact of not following the advice remains unclear. Furthermore, PICOs may change between JSC and JCA submission, raising questions about the relevance of the initial advice.
To optimise pivotal trial design for efficient EU patient access, manufacturers should aim to obtain JSC and EMA/HTA advice simultaneously. The existing joint parallel advice process is well-regarded and allows for alignment of EU regulatory and access requirements, rationalising internal resources and decision-making.
PICO Identification
PICO is a crucial component of the new regulation, though it is unclear if all markets will contribute to PICOs given their resourcing capacity and how these will be consolidated. Considering the tight submission timelines, potential PICOs need to be identified early to ensure relevant data analysis is conducted. Early scoping activities can help manufacturers gain clarity on clinical trial design feasibility and indirect treatment comparisons, optimising patient access in the EU. This scoping will provide insights into which markets represent which PICO and how this affects local access considerations, reducing duplication of information and workload.
Engaging with EU HTA during the scoping process allows manufacturers to obtain early input from all European markets, contributing to cohesive JCA and local HTA dossier development.
JCA Dossier Development
The JCA dossier will follow a structure similar to the previous EUnetHTA relative efficacy assessment and G-BA dossiers. However, the consequences of not submitting a dossier and the accepted methodologies are not yet known. Developing the dossier will require significant resources from manufacturers, who must carefully consider the inclusion of data due to its impact on local submissions. Fortunately, a second submission option will be available if the first is insufficient or data quality is inadequate.
Given the extensive data required for the dossier, concerns arise about the resource burden, especially for small and mid-sized pharma companies managing dossier development alongside the parallel EMA process. Additionally, evidence to support the product’s true clinical value may not be available at the time of JCA dossier submission, complicating the process.
Local HTA Submissions
Local HTA dossiers remain essential, as the EU HTA assessment will not cover value judgments or rank health outcomes. Countries are likely to weigh different domains in value judgments differently. Furthermore, chosen PICOs may not capture the needs of all countries, necessitating additional country-level evidence to facilitate access to all EU markets, especially those not contributing to the PICO scoping process due to limited resources.
Manufacturers must bridge the gap between accepted methodologies in member states and those in the EU HTA, developing mitigation strategies to reduce duplication of work and resource burden. Anticipating additional evidence requested by each country will facilitate smooth access to all EU markets.
Conclusion and Impact
In summary, manufacturers must act now to meet the opportunities and challenges of the JCA. Here are five key points to keep in mind:
- Be prepared: Ensure the budget and resources required for the EU HTA are in place.
- Be organised: Clearly define roles and responsibilities and communicate them across functions.
- Be adaptable: Anticipate changes to the process and consider differences between EU and local HTA submissions.
- Be early: Start market access preparations for EU HTA around one year before EMA submissions.
- Be willing to learn: Learn from others, both internally and externally.
Still have questions? Reach out to see how Remap Consulting can help you achieve your best EU HTA outcomes.
Sources:
- Health Technology Assessment – Joint Clinical Assessments of Medicinal Products – European Commission Link (Accessed March 20, 2024)
- Craddy P, Foxon G. Webinar: Implications of the EU HTA process for manufacturers. Remap Consulting. Published April 27, 2023. Accessed March 18, 2024. https://remapconsulting.com/hta/implications-of-the-eu-hta-process-for-manufacturers/
- Boland L. EU HTA and what it means for manufacturers. Remap Consulting. Published April 20, 2023. Accessed March 15, 2024. https://remapconsulting.com/hta/eu-hta-and-what-it-means-for-manufacturers/