The European pharmaceutical landscape is preparing for one of the most significant regulatory changes in decades as the EU Health Technology Assessment Regulation (HTAR) approaches implementation. Starting January 2025, all new advanced therapeutic medicinal products and oncology drugs will be subject to mandatory EU-wide joint clinical assessments, fundamentally altering how innovative treatments reach patients across Europe.
The regulation, passed in 2021 under the European Network for Health Technology Assessment (EUnetHTA), aims to harmonize the currently fragmented HTA process where each EU member state conducts separate assessments with varying processes, timelines, and requirements. This nation-by-nation approach has historically resulted in access disparities, with treatments being delayed or blocked in some countries while approved in others.
Regulatory Framework and Timeline
The HTAR introduces a phased implementation schedule that will gradually encompass different product categories. Advanced therapeutic medicinal products and oncology drugs will be the first to undergo mandatory Joint Clinical Assessments (JCAs) in January 2025. Orphan drugs and class IIb or III medical devices will follow by January 2028, with class D in vitro diagnostic devices completing the transition by January 2030.
At the core of the new system are Joint Scientific Consultations (JSCs) that facilitate collaboration between the European Medicines Agency (EMA), EU HTA bodies, and industry stakeholders. These consultations focus on defining the Population, Intervention, Comparator, and Outcomes (PICO) framework that will guide each assessment.
The process begins when stakeholder groups from individual member states submit PICO requests for information to developers. These requests form the foundation for the EU-wide Joint Clinical Assessment, which must be completed within six months of EMA approval. While individual countries retain the option to follow their own HTA pathways, the JCAs will serve as a standardized starting point for national adoption decisions.
Operational Challenges and Timeline Pressures
The compressed timeline presents significant operational challenges for pharmaceutical companies and prospective market authorization holders. With only three months typically required to receive all PICO requests, developers have limited time to prepare comprehensive responses addressing multiple country-specific requirements simultaneously.
The evidence requirements are substantial, as companies may need to provide information supporting different endpoints, subgroup analyses, sensitivity analyses, and indirect treatment comparisons across multiple PICOs. This creates a scenario where organizations must supply large volumes of information within very short timeframes.
For products under accelerated regulatory procedures, the timelines become even more compressed. The process requires a letter of intent at the point of EMA submission, followed by a PICO scoping exercise by the EU consortium, and completion of dossier preparation and submission within 100 days of the finalized scope becoming available.
Strategic Preparation Requirements
Industry experts emphasize that successful adaptation requires early preparation and systematic changes to internal processes. Organizations need to develop new standard operating procedures, reallocate resources, and implement comprehensive team training programs well before the January 2025 implementation date.
Companies are advised to anticipate likely PICO content by leveraging existing data on their products and patient needs. This proactive approach can help accelerate response times when the regulation takes effect. However, the unpredictable nature of some PICO requests means teams must also develop capabilities to quickly analyze data and answer unforeseen questions accurately.
For organizations lacking adequate in-house data science capabilities, partnerships with specialized contract research organizations (CROs) may become essential. These partnerships can provide the methodological expertise needed to rapidly access and analyze relevant insights regardless of request complexity.
Potential Benefits and Market Impact
Despite the operational challenges, the unified HTA framework offers significant potential benefits. Streamlining the process to create a single assessment rather than numerous separate HTAs could create faster routes to market across the EU while reducing administrative burden and costs associated with duplicated efforts.
The PICO framework development by national stakeholder groups, including patients and clinicians, provides valuable insights into the needs and preferences of target countries and patient groups. These insights could inform future product development and "beyond the pill" interventions that better address unmet medical needs.
The regulation also promises improved decision-making through rigorous, scientifically sound evaluations and standardized methodologies. Increased transparency is expected to enhance trust and confidence in assessments among all stakeholders, while greater emphasis on patient and healthcare professional involvement should align assessments more closely with patient needs.
Data Privacy and Quality Considerations
The public nature of JCAs introduces new data privacy considerations that developers must address. Companies need robust systems to ensure data protection while meeting transparency requirements. Additionally, strict quality controls become critical for translated outputs, particularly when operating under tight timelines.
Organizations must ensure their data and analysis programs can adapt to evolving requests and maintain systems that provide immediate understanding of trial design nuances, analyses, endpoints, and data. This flexibility will be crucial for maintaining clinical development pipeline momentum under the new legislation.
The EU-wide HTA regulation represents a fundamental shift toward more harmonized, efficient, and evidence-based health technology assessment. While the transition presents significant operational challenges, successful adaptation could provide pharmaceutical companies with more predictable pathways to European market access and ultimately improve patient access to innovative treatments across the continent.
