The European Parliament (EP) on 13 December 2021 formally adopted, following years of political wrangling, a new Regulation on Health Technology Assessment (HTA), establishing a framework that aims to enable streamlining of HTA processes across European Union (EU) member states. The regulation has been warmly welcomed by stakeholders, but questions nonetheless remain as to whether the new legislation will achieve its stated aims of “allow[ing] vital and innovative health technologies – such as innovative medicines, certain medical devices, medical equipment and prevention and treatment methods – to be more widely available”.

The EP’s decision to adopt the Regulation is certainly significant, coming in the wake of faltering earlier efforts to create a pan-European HTA network on a voluntary or opt-in basis. These have ranged from the establishment of the EU-funded EUnetHTA initiative back in 2009, to ad hoc pricing and reimbursement (P&R) collaborations that have included an HTA component, such as the BeNeLuxA initiative (initially established in 2015 by Belgium and the Netherlands, later expanding to include Luxembourg, Austria and Ireland).

The new Regulation envisages a more formalised system of HTA co-operation, based on proposals put forward by the European Commission (EC) in 2018 and in line with the principles set out in the EC’s November 2020 Pharmaceutical Strategy for Europe. Such co-operation will require healthcare bodies in different EU member states to co-operate in three key areas: joint clinical assessments with common methodologies across the EU, joint scientific consultations, and co-operation in the identification of emerging health technologies. The framework also makes provision for voluntary co-operation in other areas (eg assessment of surgical procedures).

The Regulation thus aims to ultimately supplant existing initiatives, most notably the recently launched EUnetHTA 21 programme, eventually requiring all EU member states to participate in information-sharing and joint working in relation to the assessment of new health technologies. In so doing, the Regulation aims to ultimately create a single point of entry for clinical HTAs in the EU, replacing the patchwork of around 50 national and regional-level HTA processes currently in place.

The legislation is due to take effect from 1 January 2025, though member states will be permitted to delay their participation for a period of up to three years. Compliance will, however, become compulsory after 1 January 2028. Joint assessments under the Regulation are expected initially to focus on products that address unmet clinical needs, most notably cancer and gene therapies.

According to European Commissioner for Health and Food Safety, Stella Kyriakides, the Regulation is key to “building a new way of cooperating on health in the EU,” and major stakeholders have similarly thrown their backing behind the new legislation. Significantly, the European Federation of Pharmaceutical Industries and Associations (EFPIA) has stated that it “strongly supports a system to pool resources and high level expertise so that medicines can be assessed jointly for their relative effectiveness on the basis of clinical evidence and that joint early scientific dialogue can take place with HTA agencies”. The prospect of a less bureaucratic, more centralised European approach to clinical HTAs holds obvious appeal for drug makers who may be frustrated by the variety of national and sub-national approaches currently on offer.

In truth, however, much remains to be decided. In the three years to 1 January 2025, member states will work together to establish governance structures (including a dedicated pan-European HTA secretariat), build stakeholder networks, and draft guidance documents to support the implementation of the new Regulation. The outcome of these deliberations is likely to have a significant impact on the effectiveness of the legislation as a whole.

In addition, the rules give member states significant leeway to conduct their own evaluations of non-clinical factors, such as economic, ethical and social considerations. Indeed, EFPIA cautions that member states must “keep the objective of timely access to innovative treatments in mind and avoid further delaying timelines at national level,” if the Regulation is to fulfil its intended purpose.

Despite these uncertainties, the overall direction of travel is clear. The new Regulation will sit alongside other pan-European initiatives that are changing the overall P&R landscape across the continent. These include the aforementioned BeNeLuxA collaboration – which in late 2021 oversaw the conclusion of the first joint pricing agreement between Belgium, Ireland and the Netherlands, for Zolgensma (onasemnogene abeparvovec) – but also the launch of joint hospital tenders between Norway, Denmark and Iceland, the most recent round of which concluded at the end of January 2022. Significantly, Norway is also involved in the FINOSE initiative, an HTA collaboration with Finland and Sweden.

Against this backdrop, there is therefore much optimism that the passage of the EU Regulation heralds a new era in European HTA, even if there are significant challenges to be overcome before that vision becomes a reality.

IQVIA Pharma Pricing & Reimbursement (PPR) draws on decades of experience in the market access space to bring you the latest global developments in pricing and reimbursement. With access to IQVIA’s network of local offices, and extensive contacts within government and industry, the PPR service offers timely news and articles alongside comprehensive guides to P&R regulations around the world. For more information about IQVIA Pharma Pricing & Reimbursement, contact us here.