Our recent webinar explored the complexities of Health Technology Assessments (HTA), their current role in evaluating healthcare technologies, and the upcoming changes to HTA regulations in Europe.
The upcoming regulatory shift will require developers to generate a substantial body of evidence within much shorter timelines than current guidelines require. To help you prepare for this transition, the webinar explored key aspects of EU HTA and its potential implications for developers preparing for HTA submissions.
Overview of HTA and EMA
Stephen Corson, PhD, Associate Director, Statistics & Technical Solutions at Phastar, began by introducing the European Medicines Agency (EMA), which assesses the efficacy and safety of medicinal products across the EU under a single licensing system. The EMA’s main focus is to ask whether a product works and is safe.
On the other hand, HTAs focus on assessing the added value of health technologies by summarizing medical, patient, social, economic, and ethical aspects of its use. They help determine whether a new medicine or device is more effective than the current standard of care and whether it offers value for money when compared to other interventions. HTAs influence decisions around pricing, reimbursement, and allocation of healthcare budgets which can help create and maintain sustainable healthcare systems which deliver better outcomes for patients.
Current Challenges in HTA
Currently, HTAs are conducted at the national level in EU member states. Each state has its own HTA body, which has country-specific submission requirements. As a result, developers often face multiple and divergent data requests, sometimes with duplications and variation in outcomes. This creates a significant burden for developers, particularly for smaller companies lacking the resources of larger pharmaceutical firms. The process can result in unpredictable business timelines, increased costs, and potential barriers to innovation.
New EU HTA Regulations Coming in 2025
In 2025, the EU will begin to introduce mandatory joint clinical assessments (JCAs) for all new therapeutic medicines and oncology drugs, streamlining the current nation-by-nation approach. Instead of submitting separate dossiers to each country, developers will submit a single dossier, which includes a project plan and the extra analyses that have been requested for review. From 2028, orphan drugs and class IIb or III medical devices will be included in the mandatory JCAs process, with class D in-vitro devices following by 2030.
This new regulation aims to simplify the process, reduce the burden of multiple submissions, and accelerate access to the market across EU member states. However, individual member states may still request additional analyses over those in the JCA to meet specific national requirements.
Key Timelines and Considerations
The new EU HTA process will require developers to start planning much earlier in the drug development cycle. A letter of intent is submitted at the time of EMA submission for regulatory approval. This is followed by a PICO (Population, Intervention, Comparators, Outcomes) scoping exercise by the EU consortium to finalize the scope of the JCA. Once the scope is known, there is the dossier preparation and submission phase with submission expected within 100 days of the scope being available. For drugs under the accelerated regulatory procedure, the timeline is even shorter, requiring faster dossier preparation.
Technical and Operational Considerations
Lauren Rojas, Principal Statistician at Phastar, highlighted the technical aspects of the JCA process. The PICO framework will be central to defining research questions across member states, with each country submitting its specific requests. While the joint assessment will simplify processes, some states may still request additional country-specific analysis, particularly for unique patient subgroups.
Lauren also discussed opportunities for faster market access through the consolidated EU-wide process and the need to plan for extensive data collection and analysis in a compressed timeframe. The sheer volume of evidence required for multiple PICOs may introduce challenges, particularly in ensuring data privacy, given that the JCAs will be public.
Practical Lessons for Implementation
Melanie McGuigan, FSP Manager/Programming Manager at Phastar drew on Phastar’s considerable experience with the German HTA body (G-BA), and emphasized the importance of early preparation, clear documentation, and flexible programming to accommodate the new requirements. As the submission packages for JCAs will be large and complex, developers will need to manage timelines carefully and ensure that data and analysis programs can adapt to evolving requests.
One major challenge is language-specific requirements, where translations of tables, figures, and outputs may be necessary depending on the state. The importance of efficient programming and quality control for translated outputs was stressed, especially under tight timelines.
Conclusion
The new EU-wide HTA regulations will streamline the clinical assessment process, reduce duplication of efforts, and potentially shorten the time to market for healthcare innovations. However, developers will face new challenges, including earlier submission timelines, extensive evidence requirements, and maintaining flexibility in their data analysis processes. By proactively planning and ensuring clear communication between stakeholders, companies can successfully navigate this regulatory shift and seize opportunities in the evolving HTA landscape.
At Phastar, we are able to leverage our extensive experience in preparing submissions with the GBA, the highest decision-making body in the German healthcare system. Our insights into the complexities of these submissions and the large analysis packages involved can provide valuable lessons for navigating HTA submissions under the new regulations.
