Since last year, a European Union (EU)-wide health technology assessment (HTA) regulation has been passed. Could you explain the fundamentals of this regulation and how it differs from previous approaches?

The EU now has a legislative framework for cooperation on HTA. This framework has been voluntary and in development since 2006, taking various forms such as joint actions under the EUnetHTA initiative. The recent establishment of a service contract, won by a group of partners from the previous EUnetHTA Joint Action Three, marks a significant milestone. While the full regulation will be implemented in January 2025, the period leading up to it will be busy, particularly for the newly formed Coordination Group. This group, composed of representatives from each EU member state, will be the main decision-making body under the new regulation.

What are the main challenges that industry sponsors will face in adapting to the new legislation by 2025?

We are conducting formal consultations for each deliverable to ensure that industry stakeholders are not taken by surprise. The pharma industry has actively advocated for a more harmonized European HTA system, and while there will be changes and uncertainties during the transition, the establishment of an EU-wide system should provide more certainty in terms of HTA assessments of effectiveness, safety, and timelines. Health economics and pricing negotiations will remain within national and regional systems.

The pharma industry has criticized EUnetHTA 21 for allegedly sidelining their concerns and comments. How do you respond to this criticism and how do you see this dynamic evolving?

We have strived to include comments and make consultations while adhering to the principles of the European legislative system. Every single comment has been read and considered, although it can be challenging to respond individually when faced with hundreds of pages of comments. While some disappointment has been expressed about the magnitude of changes, especially in early deliverables, this criticism has been less pronounced with the publication of more recent ones.

In our previous conversation, you mentioned the goal of creating a more predictable and flexible HTA system. Do you believe that the system being put in place represents significant progress toward these goals?

Yes, that is our intention. The transition from voluntary to mandatory joint HTAs will naturally bring changes, and the decisions will ultimately rest with the Coordination Group. As EUnetHTA 21, we aim to provide clear, transparent, and predictable products to the European Commission (EC) and are curious to see how they will be utilized by the Coordination Group.

Real-world evidence (RWE) usage in HTA assessments reached a record high in 2021. How well do you feel RWE is being utilized today, and how effective is it for assessing costly new therapies?

The joint EU regulation allows for the inclusion of post-licensing evidence generation and RWD, but there is no strict legal framework for it. While discussions about RWE take place, its quality and applicability must be closely scrutinized. The use of RWE varies across the EU, but there is an increasing realization that it is unavoidable in many contexts. Progress has been made in establishing standard operating procedures and reporting, similar to good clinical practice in randomized clinical trials. However, there is still work to be done, particularly in determining the strength of the data generated and reaching consensus on its use in decision-making.

What are the main roadblocks ahead until 2025, and what excites you the most about the future of HTA?

The implementation of the EU HTA legislation is an exciting development after almost two decades of work. It will enable joint work on scientific consultations, clinical assessments, horizon scanning, and other tasks. This shift is necessary to handle the influx of new products, which would be challenging for individual member states. However, the transformational phase will require considerable effort, with national agencies and companies facing resource constraints. The next two years will be a significant challenge for all stakeholders.

Do you have a final message for our international, industry-focused audience?

We have long desired a more collaborative European approach to HTA, recognizing its potential benefits for efficiency and equity across the EU. Let us not lose sight of this vision amidst minor details. Collaboration and harmonization remain key goals to strive for.