Around the Globe
Health Technology Assessment in the EU
Back and Forth in Giant Strides
Thomas Kühler
Sanofi R&D

ore than three years ago, in January 2018, the European Commission published a draft Regulation on Health Technology Assessment for the EU. The intention was to secure timely patient access to novel therapies, to avoid duplicative assessment work at the Member State level, and to provide a predictable and consistent process for the benefit of all stakeholders.

As background, Member States had not adopted an aligned approach but instead applied their own respective procedural measures and scientific standards when conducting Health Technology Assessments (HTAs). This meant duplicative and repetitive work across the EU network and hence higher costs for both companies and society at large. More importantly, it also delayed patient access to novel therapies. It was not a positive benchmark in terms of proper stewardship of available resources.

Accordingly, the European Commission aimed to reinvent EU-wide HTA cooperation, with a particular focus on mandatory joint clinical assessments. The intent was for the network to streamline the process and share resources, and to arrive at one consensus view on assessments, which then would be binding across the EU. While there would be no obligation for a Member State to conduct its own HTA on a product that has undergone a joint clinical assessment, it would now be mandatory to use the joint clinical assessment if the Member State in question would choose to conduct one — thereby effectively preventing Member States from introducing additional national requirements which had long been plaguing the system and resulted in delayed patient access to medicines.

Aside from the joint clinical assessment, the evaluation comprising economic, ethical, and organizational elements of the assessments would remain under the purview of each Member State and would hence not be subject to the subsidiary principle. The subsidiary principle defines the circumstances in which it is preferable for an action to be taken at the EU level rather than at the Member State level. It was an important feature of the Regulation to keep the fiscal sovereignty at the Member State level and not to infringe on their respective mandates to make their own pricing and reimbursement decisions.

It is important to remember that the European Commission is the law-proposing body in the EU (led by appointed Commissioners, one from each Member State) but that any legislative proposal put forward must be ratified first by the European Parliament (made up of representatives elected by the citizens of the Member States) and then by the European Council (with representatives from the governments of the Member States), the latter of which is the ultimate decision-making body.

The draft regulation was quickly adopted by the European Parliament in October 2018 shortly after the EC had published it. While the European Parliament did introduce some additional language to make the proposal even more acceptable to Member States concerned about subsidiarity, they backed the principal proposal of obligatory joint clinical assessments because they thought that a voluntary approach would dilute the value of the regulation, rendering it rather pointless.

But within the European Council the decision process was harder, and it took more than three years to reach its position. This can be attributed to strong opposition primarily by Germany and France, but also Poland and the Czech Republic, against the binding nature of the joint clinical assessment. The requirement for the mandatory acceptance of the joint clinical assessment was therefore softened by the European Council, and they proposed that Member States shall “give due consideration” to the joint clinical assessments. To this end, joint clinical assessments, albeit not mandatory to adopt, will be introduced in a staged approach over several years, starting with oncology treatments, followed by advanced therapeutic medicinal products, and then expanded to the full therapeutic gamut.

A few formal steps remain, including Member States’ validation and adoption at a Council Meeting, but we do not yet know the exact timing of these steps, and it is unlikely they will significantly change the course of action. The European Commission and the European Parliament both had to give up what may very well have been the most important provision in the regulation, namely the introduction and mandatory uptake of the joint clinical assessment. It seems that HTA legislation in the EU is moving back and forth in giant strides.