The European Union (EU) adopted the Regulation on health technology assessment (HTA), Regulation (EU) 2021/2282, in December 2021. The Regulation aims to establish joint clinical assessments (JCA) of health technologies in the EU. Two weeks ago saw the first in-person meeting at the European Commission since the Regulation was adopted, and this meeting provided insights into what key national HTA bodies are thinking about how the Regulation should be adopted. Two weeks ago, the newly appointed Coordination Group – a group consisting of representatives from Member States who will be responsible for overseeing the joint work – also met for the first time. It was the first concrete step towards implementing the Regulation, a step that marked the start of discussions on how to bridge differences in assessment perspectives and methodology, alongside a call for investment both at the European and the Member State level.
Lumanity’s Suzette Matthijsse (Director and EU Head of Health Economics Analysis) and Linda Murphy (Senior Director, Insight) attended the meeting in person and remotely, respectively. Key learnings are summarized below.
A ‘top priority’, but we are a long way from clarity
During the meeting, the European Commission stressed that one of its top priorities was implementing the new Regulation. The Commission urged Member States and stakeholders to implement the Regulation effectively by raising awareness, adapting processes, and thinking ‘joint work’. All participants agreed that inclusiveness of adoption is critical with Western countries bearing a responsibility to ensure the deliverables meet the needs for neighbours with fewer resources.
The EU HTA Regulation is unique in that 27 countries will unite and accept shared responsibility for HTA. Alongside aiming to reduce duplication of work and speed up patient access, participants aim to produce guidelines that will set criteria and a minimum standard for assessment of the evidence base included in manufacturer submissions. This process will still allow Member States to determine what they will and will not accept.
Many meeting participants noted that successful implementation of the Regulation will be based on confidence and trust. The European Federation of Pharmaceutical Industries and Associations (EFPIA) emphasized the need for clarity and consideration of big industry changes. These changes are all moving in parallel, including changes to the European health data space, clinical development processes and European Medicines Agency (EMA) requirements. We support this viewpoint at Lumanity, and we recognize that it is still unclear how the Regulation will be implemented. Without some clarity on this point, the risk remains that the Regulation will result in the currently fragmented decision-making process being exported to the EU level, rather than delivering a meaningful EU joint assessment. HTA will then become a further hurdle, with evidence generation and HTA bodies’ requirements continuing to suffer a mismatch.
10 key takeaways from the meeting
All applications for oncology and advanced therapy medicinal products (ATMP) from January 2025 will fall within the scope of the Regulation. It will take some time before the results from the first applications will be seen, possibly towards the end of the year.
While we are already seeing the first sets of methodological guidelines being released for comment, the intention is that the Regulation and the methods involved will be reactive, both in terms of adopting new methods and reassessment with updated data. Extensive comments have been received for the deliverables so far, including 196 pages of comments for a single deliverable.
The French health regulator, the Haute Autorité de Santé (HAS), has stated that JCA will contain all it needs for clinical assessment. However, additional analysis requests beyond JCA are likely in other situations. Expected examples include:
a. Additional subgroups for Germany
b. Early access programme requests before a JCA
c. Health economic assessment
The joint information technology platform (using SharePoint) will be launched either this autumn or by Spring 2023, which reflects the timeframe for the Stakeholder Network being set up. This is vital for communication between all stakeholders.
Considerable uncertainty remains, as no straightforward answer was provided to the legal requirement to give JCA ‘due consideration’ at the national level. Instead, the focus is on Member States updating the European Commission on how they considered the outputs. While the European Commission agreed on the importance of translated materials, it did not confirm whether it would support the translation process – this is key to uptake in some countries
There are two planned calls for joint scientific consultation (JSC) this year. These calls will be the last opportunity for manufacturers to shape the future process:
a. The current open call ends on 31 August 2022
b. A second call will open in November 2022
c. Manufacturers may have additional flexibility to get involved outside of these times if they can present a strong enough case
d. It is still unclear whether any funding for JSC will be available between 2023 and the Regulation’s launch in 2025
The next bilateral meeting between the EMA and the European Network for Health Technology Assessment (EUnetHTA) 21 will focus on patient-reported outcomes
The meeting confirmed that vaccines and vaccine-specific guidelines will be included in JCA
To respond to lengthier patient access processes, an EU Accelerated Access Pathway was discussed for high-risk medical devices. This would be expected to follow a similar model to the Breakthrough Device Program in the US
There is a preference for separate searches per Population, Intervention, Comparison, Outcomes and Study (PICOS) where there are multiple populations/decision problems
The meeting ended with a call for involvement in the ongoing public consultations, as well as for involvement in the JCA and JSC pilots. The public consultation for key deliverables opened on Monday 4 July 2022: these deliverables are the JCA/clinical assessments (CA) Submission Dossier Template and methodological guidance on the applicability of evidence and validity of clinical studies. To find out more and take part in the consultations, visit: https://www.eunethta.eu/jointhtawork/.
Lumanity and independent HTA expert Neil Grubert have joined forces to deliver a cross-functional training programme that raises awareness of the new Regulation’s impact. We are also providing support in responding to the consultation, in addition to planning for how the new Regulation will impact manufacturers’ portfolios.
If you would like to understand more about the impact of the Regulation on your work, or if you would like to register your interest in our training programme, please contact us.