Insights from Pharma 2025: Navigating the evolution of EU Health Technology Assessment (HTA) Regulation

The EU HTA regulation, which came into effect in January 2025, marks a significant step toward harmonizing HTAs across Europe. Initially focusing on oncology therapies and advanced therapy medicinal products (ATMPs), the regulation aims to streamline the clinical assessment process through the joint clinical assessment (JCA) and to offer companies to engage in an early development phase through the joint scientific consultation (JSC). By reducing duplication of effort among member states and facilitating early dialogue between stakeholders, the regulation ultimately seeks to accelerate patient access to innovative treatments. By fostering greater collaboration and consistency in HTA evaluations, the new framework is expected to reduce the administrative burden for pharmaceutical companies while supporting more equitable access to medicines across the EU. 

Key challenges and opportunities

1.  Right to be heard: A primary concern highlighted by both experts is ensuring that smaller companies can access JSCs. As noted by Natz, “It’s crucial that smaller companies have opportunities for joint scientific consultations to align clinical trial designs with HTA expectations.” He emphasized, “The right to be heard is the key issue in any HTA procedure.” However, the current limitations on the number of available JSC slots may hinder this process. 
2. Resource limitations: Both the industry and HTA bodies face resource constraints. There is a pressing need for increased slots for JSCs and improved coordination among national HTA institutions to leverage collective expertise. Natz pointed out, “It’s about the lack of resources… the HTA bodies and the coordination group seem not to have the resources to actually bring people into not just doing the evaluations in the JCA but also doing the JSCs.”
3. Learning system: The EU HTA is envisioned as a learning system involving manufacturers, patient organizations, and physician groups. Continuous dialogue is vital for improving the regulation's implementation. As Altmann stated, “It’s important to go into this dialogue so that every participant can learn and improve the system.” 

The phased rollout of the EU HTA, starting with complex ATMPs and oncology products, is designed to allow the system to mature before expanding to all products by 2030. The regulation is interconnected with other frameworks, such as the Critical Medicines Act and General Pharmaceutical Legislation (GPL), emphasizing joint procurement and price negotiations.

Ensuring that patient and industry voices are integral to the decision-making process is crucial. Natz stated, “It’s very important that industry gets a voice in this interaction because, based on my experience with many manufacturers, they don't really feel heard in this process.” This involvement will help tailor the regulation to meet the diverse needs of stakeholders across Europe.

As the EU HTA regulation takes shape, the next five years will be pivotal in determining its success. By addressing challenges, leveraging opportunities, and fostering collaboration among stakeholders, the regulation has the potential to significantly enhance patient access to innovative medicines across Europe. 

The dialogue from Pharma 2025 underscores the importance of these efforts and the collective commitment to improving healthcare outcomes. Altmann encapsulated this vision by stating, "If we get the EU HTA right, we can create a competitive advantage for Europe in the global market for innovative medicines."