The EU Health Technology Assessment Regulation (HTAR), approved in 2021, will become effective as of 12 January 2025. Its main objectives are to improve and speed up access for European patients to innovative health technologies —including both new medicines and medical devices— and to increase transparency and efficiency of the assessment processes. By establishing a centralised and harmonised system, the new HTAR aims to make more efficient use of public resources and achieve faster market access for health technologies across the EU.
The HTAR is one of the important pieces of new pharmaceutical legislation, the revision of which was identified as one of the key actions of the Europe Health Union, an ambitious joint strategy established in the aftermath of the coronavirus pandemic.
The EU is committed to better protect the health of its citizens, to be equipped to better prevent and address future pandemics, and to improve the resilience of European health systems. To achieve this, the EU wants the pharmaceutical legislative framework to favour innovation and transparency, to reduce bureaucratic burdens, to ensure patient access to medicines, to establish a Single Market for medicines, and to make the production of medicines environmentally friendly, among other objectives.
The new HTAR is aligned with the purpose of ensuring faster authorisation of innovative medicines by implementing simpler rules and procedures and through more patient involvement in the medicines assessment processes without compromising safety. Application of the EU HTAR will start with oncology and advanced therapy medical products (ATMPs) in January 2025, orphan drugs will follow in 2028, and all other drugs in 2030.
From PICO to JCA
The new HTA procedure is launched once the dossier for market authorisation has been submitted to the EMA. The EU HTA Coordination Group —made up by representatives of HTA national and regional authorities— gathers requests from member states about the so-called PICO questions (Patient/population, Intervention, Comparison and Outcomes). Once consolidated, this information is communicated to the Health Technology Developer (HTD), which has a maximum of 100 days to prepare the dossier to be submitted to the Joint Clinical Assessment (JCA). The JCA outcomes are released after the European Commission (EC) decision on market authorisation, and member countries can then adopt the JCA report or ask for further evidence.
HiTT Foundation is member of the HTA Stakeholder Network that supports the implementation of the HTA Regulation. The network provides advice and expertise as required on issues of general relevance for the joint work and gives feed-back as appropriate on the outputs of the EU HTA Coordination Group.
While recognising the positive development that a centralised assessment system can bring, the implementation of the EU HTAR raises doubts among HTDs. There is, for example, uncertainty around evidence requirements for JCA. PICO requests from member countries could be very heterogeneous, requiring a lot of very diverse information to be included in the dossier for JCA. This adds to the perception that the time for the preparation of the dossier (100 days) is very tight. Another issue that raises questions is how the new regulation will affect local pricing and reimbursement processes.
EU HTAR is seen as an exercise in cooperation aimed at reducing fragmentation and duplication in the assessment of medicines by the EMA and national bodies, but it is true its implementation will challenge the resources needed from HTD and that there are still open questions about Joint Clinical Assessments and its impact in the health technology market at national scale.
HTA in Spain
Some EU member countries have been reviewing their legislative rules on HTA to adapt them to the new framework established by the EU HTAR. The Spanish government is in the process of drafting a Royal Decree on Health Technology Assessment, which closed its public consultation phase on 20 September 2024. HiTT Foundation contributed to this phase with some proposals aimed at improving the new Spanish regulation in different areas.
It is worth to underline that the RD on HTA draft is an important step forward, bringing Spanish legislation in this area into line with that of neighbouring countries. However, we believe that there is room for improvement in aspects such as the promotion of innovation, for which specific incentives should be established. In this regard, it would be very interesting to create mechanisms that would allow positive discrimination of the most innovative therapeutic alternatives in the assessment processes.
It would also be important for the new Spanish HTA regulation to include mechanisms that give it flexibility and guarantee its ability to adapt to the evolution of an extremely dynamic environment.
We also believe that it would be interesting to consider the possibility of including measures such as those in force in Germany, where HTDs systematically turn to health authorities to consult on new developments due to the binding nature of their design recommendations.
Furthermore, the RD on HTA should include more specifically the importance of considering the life cycle and environmental impact of the health technology being evaluated. It should also provide greater patient participation in the evaluation processes and greater involvement in decision-making.