65 days to go until January 12, 2025
As we reflect on Tuesday’s seventh and final regional meeting organised by the European Commission (EC), From Theory to Practice: implementing the EU Health Technology Assessment Regulation (HTAR) in Paris, we recall that participants were looking for clarity and (practical) information. With representatives from the HAS, G-BA and IQWIG as well as the HTA coordination group (HTACG) and its subgroups, expectations for answers were high. In addition, the panel sessions included a range of stakeholders including clinical experts, patient advocacy groups (PAGs) as well as the industry organisations EFPIA and MedTech Europe were all represented. While representatives of national HTA bodies, feel ready for the implementation on 12 January 2025, health technology developers (HTDs), patient organisations and clinical expert organisations acknowledge that there are still some remaining steps to be taken (e.g. finalisation of implementing acts and guidance documents).
All attending stakeholders agreed that this will be a challenging process with tight timelines. However, Roisin Adams (Chair HTACG) emphasised that we are on a journey with patients as our North Star. These tight timelines are necessary to achieve the goal of accelerating access for patients. Representatives of HTA bodies highlighted the need for mutual trust and the importance of avoiding duplication. The importance of having high quality dossiers was also stressed. When HTDs asked how this could be achieved within 100 days, the answer was that HTDs should stick to their current timetable of starting to prepare the national HTA dossiers well in advance of submission. Furthermore, the requested PICO (population, intervention, comparator and outcome) should not come as a surprise, according to the representatives of the subgroups. With regard to this PICO, the chair of the subgroup for JCAs emphasised that an assessment can only be carried out if all four aspects of a PICO are present. He referred to situations where there is no comparator (e.g. in single-arm trials). It is of paramount importance that there is a comparator to perform the JCA. If you are wondering how to do this, they referred to the methodological and practical guidance that has been available since the 8 March 2024.
With regard to these guidance documents, we noted that several additional guidance documents and templates will be approved at the next HTACG meeting on 28 November. As only 5/19 guidance documents have been adopted so far, this will be key for HTDs to fully prepare for January, as it will include templates for the dossiers and procedural steps and timeframes for both procedures. Other practical takeaways relate to the IT platform, which is operational and already being used by the subgroups for information exchange. A completeness checklist for the dossier will also be provided, but is not yet available.
Sustainability. This is a key objective and it was reiterated several times that it is very important to ensure that this new process works in the long term. Therefore, Natalie Moll from EFPIA stressed the importance of keeping the conversation open (e.g. at the EMA).
Moving on to another important aspect, patient involvement, we found that this is an area where many uncertainties remain. Firstly, it is not clear how it will work in practice, as the involvement of patient organisations in PICO scoping seems to be national. However, many Member States do not have a process in place for this to happen, so a European approach may be better to ensure involvement in this matter, given the short timeframe for PICO scoping. Secondly, there is uncertainty about how patient organisations should prepare and adapt their way of working and data collection. Some examples that have come up are the types of data required (e.g. questionnaires, interviews, PROs, …) and formal guidance on what is expected from the PAG. These organisations really want to be involved and to develop their ways of working so that they can be an active partner in the processes, but therefore additional guidance on robust evidence generation for PAG is needed. Some of them are already much more advanced and can provide more data than just anecdotes from patients, but some additional coaching/guidance on evidence gathering could easily improve the involvement. In conclusion, patient involvement in the PICO scoping process remains ‘woolly’, but Roisin Adams acknowledged that this needs further consideration.
Looking at Medical Devices (MD), we note that in 2025 2-3 slots are foreseen for a JSC and only in 2026 will the first MD undergo a JCA. Which ones will be selected after 12 January 2025 by the EC after consultation of the HTACG and the subgroup as foreseen in the Regulation. The last implementing act on JCAs for MDs is still in the drafting phase, but is well developed and almost ready to be circulated. The draft implementing act on JCAs for MDs is currently under public consultation. It has been clearly stated that the methodological guidance will be the same for MD and pharmaceuticals as it is based on the principles of evidence-based medicine.
To elaborate on the additional figures provided during the meeting, with regard to medicinal products, the number of JCAs cannot be predicted and will depend on the number of medicines subject to a JCA under the Regulation, so there will not be a limiting cap. For this reason, the number of JSCs will be limited to 7-10 slots in 2025, but will increase in the following years (as more resources and assessors become available). Exact numbers will be made available in the annual work programme to be published shortly.
In a final survey of participants, the session was seen as providing some answers and a good update. However, there is still a lot of “we will see” and many unanswered questions. Roisin Adams summed up the session: “Feel the fear and deal with it”.
If you would like to learn more about the different perspectives of the stakeholders and representatives who participated, their definition of success and the gaps they identified, please contact the MAPxAxtalis team at info@axtalis.com and we will provide you with additional insights. Additionally, on Thursday, November 14, Claire and Sven will host the Access Forum, a pre-conference workshop titled EU HTA Countdown to Implementation – Opportunities and Challenges. This interactive session will provide the latest updates on the HTAR implementation, covering legislation, timelines, and challenges, especially for ATMPs and anti-cancer medicinal products.
A segment will focus on HTDs preparation for upcoming changes, offering practical advice on overcoming hurdles related to PICOs, timelines, and engagement. The workshop concludes with an international panel of industry and assessor experts, discussing the integration of EU HTAR into national HTA, balancing EU and local perspectives.
