A new approach from 2025 to health technology assessment in Europe

The EU Regulation on Health Technology Assessment (HTA) will enter into force in Europe on 12 January 2025, marking a watershed in the way health technologies and medicines are currently assessed in Europe. This new regulatory framework is designed to revolutionise the assessment of new medicines and health technologies, improving collaboration, efficiency and inclusiveness, with promising implications for orphan communities. By streamlining processes, the Regulation could help speed up access to essential treatments in the EU Member States.

What changes are expected? First, let’s define what a Health Technology Assessment (HTA) is – a multidisciplinary approach that compares a new technology with an existing one to assess whether it is more, equally or less effective. HTA helps EU governments decide which healthcare methods and technologies to prioritise, taking into account medical, social and economic factors. For patients with rare diseases, HTA is crucial as currently 94% of patients with rare diseases do not have access to specific treatments and most have not received them because they are not available.

The new EU Regulation on Health Technology Assessment (HTA) provides a historic opportunity to start harmonising processes across EU countries for funding innovative treatments and the cost of medicines for patients with rare diseases. Currently, each country carries out its own funding assessments, leading to inconsistent results and delays in access to treatment and the availability of essential medicines.

The introduction of a joint clinical assessment (JCA) under the new Regulation (EU) will change this dynamic by streamlining the NTA process across the European Union, although some powers (such as deciding whether to reimburse a new technology) will remain the prerogative of each country. From January 2025, JCA will be mandatory for new oncology medicines and all advanced therapy medicines, including innovative therapies for cystic fibrosis.