EU HTA regulation
The recent European Union’s Regulation (EU) 2021/2282 on Health Technology Assessment (EU HTA) aims to simplify and harmonise patient access to new medicines and medical devices. Previously, after market authorization by the European Medicines Agency (EMA), pharmaceutical companies had to submit Health Technology Assessment applications in each Member State (MS) in which they wished to commercialise their product. This led to delays and inequalities in access between countries as each MS had very different ways to analyse and process these applications.
The EU HTA regulation was adopted by the European Parliament and the Council of the European Union in December 2021. Following the regulation coming into force, a 3-year transition period started in January 2022 and included activities preparing the ground for the successful implementation of the regulation:
- Drafting implementation and guidance documents
- The formation of the HTA Stakeholder Network
- Setting up the Coordination Group which includes representatives from EU MSs (divided into subgroups on Joint Clinical Assessments, Joint Scientific Consultations and Identification of Emerging Health Technologies and Methodology)
1. What is the EU HTA Regulation?
The new EU HTA regulation aims to address previous delays and inequalities in access by mainly:
- establishing a support framework and procedures for cooperation at Union level through the Joint Scientific Consultations (JSCs)
- making only ONE evidence submission for clinical assessments at Union level through the Joint Clinical Assessments (JCAs)
- setting up common rules and methodologies for JCAs
The regulation still leaves non-clinical domains (please see figure below) and drawing conclusions on the overall clinical value of the assessed HTA the sole responsibility of MSs.
The regulation will start its application on 12 January 2025 by focusing on cancer medicines and advanced therapy medicinal products for the first 3 years, followed by a further inclusion of orphan medicinal products from 2028 and including all medicines from 2030.
To support the implementation of this regulation, the European Commission is preparing six implementing acts released throughout 2024.
2. Why is this important for patients/patient organisations?
Not only is the new regulation aiming to simplify and speed approval processes to increase access, it is also giving high importance to patient input. The new regulation details the inclusion of patient experts and patient organisation representatives in decision-making processes. In addition, the HTA implementation governance structure gives an important role to the HTA Stakeholder Network, to which MPE is a member (please see figure below).
However, it is important to ensure that the patient voice remains truly represented and taken into account in decision-making, with the process not being relegated to a tick-boxing exercise. In addition, all stakeholders involved, including patients, European and national regulators, industry and healthcare providers, would need to work together to safeguard achieving the main aim of this regulation: increasing access to medicines.
The governance structure is set up as depicted in the figure below:
Source: European Commission 2023 Factsheet – Implementing the EU Health Technology Assessment Regulation, https://health.ec.europa.eu/publications/factsheet-implementing-eu-health-technology-assessment-regulation_en.
3. What is MPE doing?
While the regulation aims to improve patient access to medicines and the implementation phase aims to set the stage for the full functioning of the regulation after January 2025, it is important that patient advocates are part of the process to ensure that patients’ opinions, concerns and expertise are an integral part of the regulation’s implementation and functioning. MPE already provided comments to open consultations for implementing acts focused on Joint Clinical Assessments and conflicts of interest.
- On the first implementing act supporting the EU health technology assessment (HTA) regulation focusing on the process for conducting joint clinical assessments (JCA) of new medicines and how patient experts will be involved, MPE called for:
- Stronger wording and clearer timelines on patient involvement
- Clarity on the involvement of patient organisations
- Better alignment between patient involvement in the European Medicines Agency (EMA – the European medicines regulator) and JCA processes
- Provision of lay summaries to patient experts to ensure they have the information needed to comment effectively
- Ensuring the conflict-of-interest policy allows for the appropriate declaration of any conflict, whilst also ensuring the contribution of the best and most appropriate patient and clinical experts
See MPE’s full response here.
- On the second implementing act, focused on procedural rules for assessing and managing conflicts of interest (CoI) for representatives and individual experts (including patient experts), MPE commented that:
- In rare and ultra-rare diseases, there are often a limited number of patients with clinical and regulatory expertise in their disease area and are thus involved in a wide range of activities, many made possible with financial contributions from industry
- Requirements for conflicts of interest would thus disqualify the vast majority of patient experts in the area of rare and ultra-rare diseases from being involved in HTA assessments
- Patients with the best available expertise could be left out of HTA assessments under these rules
- A flexible and pragmatic approach towards the CoI, one that balances the need to ensure transparency, impartiality and independence with the realities of patient expertise should be pursued
See MPE’s full response here.
Please contact us at access@mpeurope.org if you have further questions about this regulation and MPE’s efforts to engage with it.