For decades, pricing and reimbursement activities have been handled separately from the regulatory process and typically only began once regulatory approval was secured. This separation has largely been driving the heterogeneity of pricing and reimbursement decisions and policies across nations in Europe, where decisions are shaped by national policies and country-specific procedures rather than a centralized European framework.
The EU HTA Regulation (HTAR), implemented last year and now in its first year of operation, marks a significant shift toward earlier and more coordinated clinical assessments in Health Technology Assessments (HTAs) across EU member states. By harmonizing the clinical evaluation of HTA at the EU level, the regulation increases the importance of initiating pricing and reimbursement planning earlier and in parallel with regulatory activities.
This blog outlines the key pricing and reimbursement steps that run alongside and after EU market authorization, helping companies achieve timely, efficient patient access and sustained market uptake.
The EU HTAR in a Nutshell
The EU Regulation 2021/2282 on Health Technology Assessment (HTAR) establishes a joint clinical assessment framework for medicines and high-risk medical devices across the European Economic Area (EEA). Its aim is to harmonize HTA processes, reduce duplication across Member States, accelerate patient access to innovation, and support evidence-based pricing and reimbursement decisions.
A central feature of the HTAR is the Joint Clinical Assessment (JCA), a coordinated EU-level evaluation of the clinical effectiveness and safety of new technologies. As of January 2025, JCAs are mandatory for new cancer treatments and advanced therapy medicinal products (ATMPs) and will gradually extend to all new medicines undergoing EMA marketing authorization. The JCA begins once EMA validates the marketing authorization application and focuses on comparative clinical effectiveness and safety, and the outcomes are expected to be highly influential for national reimbursement decisions.
While JCAs assess only comparative clinical evidence, economic evaluations, pricing, and reimbursement decisions remain the responsibility of national authorities. As all Health Technology Developers (HTDs) will eventually need to use the JCA framework when introducing new technologies in Europe, its implementation marks a significant shift in EU HTA processes and in the responsibilities of companies pursuing a Marketing Authorization (MA).
Points to Consider When Pursuing Marketing Authorization and Market Access
The Marketing Authorization Holder (MAH) plays a central role in securing and sustaining market access for a medicinal product. After obtaining regulatory approval, the MAH is responsible for coordinating the activities that enable the product to reach patients in a timely and cost-efficient way. With the JCA process now running in parallel with the EMA approval process, ensuring that market access activities are fully aligned with regulatory milestones is more important than ever.
Aligning Regulatory and Market Access Strategies
During the pre-market approval phase, it is essential to ensure that the clinical evidence generated for the EMA regulatory assessment is designed to fully support and align with the relative efficacy and safety evaluation assessed by the European HTA Coordination Group (CG) during the JCA.
This same evidence base must also be suitable for meeting subsequent national pricing and reimbursement requirements. This means that clinical development, regulatory strategy, and market access planning need to be closely coordinated from the outset to avoid evidence gaps that could delay or limit patient access.
One topic for alignment is that the pivotal phase III trial uses the standard of care recommended in EU clinical guidelines as the comparator arm, which EMA considers appropriate for assessing benefit–risk. At the same time, the phase III comparator is preferable also the one used in most EU Member States' HTA assessments, ensuring relevance for relative efficacy comparisons across countries. Moreover, the pivotal trial endpoints should include both primary endpoints that are validated and acceptable for EMA's regulatory decision-making (e.g., overall survival) and key secondary endpoints essential for HTA bodies when evaluating added therapeutic value and patient-centered benefit (e.g., patient-reported outcomes and health-related quality-of-life measures).
Preparing and Submitting HTA, Pricing, and Reimbursement Dossiers
In many European countries, the preparation of reimbursement dossiers can, and ideally should, begin well before market approval. Early planning allows companies to anticipate evidence needs, align internal functions, and avoid delays at launch. However, depending on the planned launch sequence and national requirements, some activities may also be prepared post-approval. When preparing reimbursement dossiers in general, it is essential that the relative clinical assessment from the JCA is consistent with the evidence used in national health economic evaluations and reimbursement assessments, where applicable.
For instance, the JCA can conclude that the new oncology product provides improved progression free survival (PFS) and similar overall survival (OS) compared with the comparator. The same hazard ratios and subgroup results used in the JCA are then preferably incorporated into national economic models to avoid discrepancies that could undermine credibility. Immaturity of data can be expected to be highlighted in both the regulatory as well as the HTA assessment. National dossiers will typically address this by including scenario analyses (e.g., alternative survival extrapolations), demonstrating that the company has considered the same uncertainties identified at the EU level.
Engaging with National Authorities and Payers
As a part of the reimbursement decision-making process, national reimbursement recommendations for high-cost, centrally approved medicines typically involve price negotiations and confidential agreements. To support these discussions effectively, the value of the medicine must be clearly demonstrated through robust clinical and economic evidence. Any uncertainties or gaps in the evidence base need to be identified early and addressed proactively, as they can significantly influence the outcome of negotiations.
For illustration, a company may present pivotal oncology trial results showing a statistically significant improvement in PFS compared with the standard of care, along with clinically meaningful improvements in patient reported outcomes, such as improved quality of life. This could then be complemented by a cost-effectiveness model demonstrating that the therapy delivers additional clinical benefits at a cost within the country's acceptable range, and a budget impact analysis showing that, despite a higher unit price, the therapy may reduce downstream costs (e.g., fewer hospitalizations and reduced need for supportive care).
Ensuring ongoing Compliance with Post-Launch Pricing and Reimbursement Obligations
Once reimbursement is secured and the medicine is launched, a continuous cycle of market maintenance activities begins. These activities are essential for sustaining access, meeting national obligations, and ensuring long-term commercial success. They typically include price registration and updates, monitoring and participation in tendering processes, and managing reimbursement renewals or re-assessments as required by national authorities.
In many cases, national pricing and reimbursement authorities request updated data to confirm the medicine's ongoing clinical value. To prepare for this, an oncology company may, for example, update OS and PFS data from long-term follow-up of the pivotal trial and provide new real-world evidence showing treatment patterns, adherence, and outcomes in clinical practice.
Together, these activities ensure that the medicine remains compliant, competitively positioned, and aligned with evolving payer expectations and clinical practice.
Summarising How to Manage HTAR Requirements
As the JCA now runs in parallel to the EMA approval process, it is important that a company pursuing a market authorization also fully understands and effectively manages the HTAR requirements established by the European Commission.
A successful approach begins with early alignment of evidence strategies. Clinical development plans should be designed to meet the expectations of both the EMA and the HTA CG, ensuring that endpoints, comparators, and study designs are suitable for regulatory approval as well as for demonstrating relative efficacy and safety in the JCA. This requires anticipating methodological requirements, understanding evolving HTA guidance, and identifying potential evidence gaps long before submission.
Another important approach is cross-functional coordination. HTAR compliance cannot sit solely within regulatory or market access teams; it requires close collaboration across clinical, regulatory, market access, health economic outcome research (HEOR), medical affairs, and commercial functions. Establishing internal governance structures, clear roles and responsibilities, and shared timelines will ensure that data packages are consistent across EMA and HTA CG.
Market authorization holders must also prepare for the downstream impact of the JCA. While the JCA focuses on clinical evidence, its conclusions will heavily influence national HTA bodies and payer negotiations. Understanding how different countries interpret JCA outcomes, and how they integrate them into their own assessments, is essential for shaping pricing and reimbursement strategies. This includes scenario planning, early payer engagement where possible, and developing value communication materials that address both European level findings and local requirements.
By embedding HTAR planning into the development and launch pathway early, MAHs can reduce delays, strengthen value demonstration, and support faster, more consistent patient access across Europe.
ProPharma's Own Experience
With over 20 years of experience in commercializing medicinal products, ProPharma has successfully supported pharmaceutical companies worldwide in achieving marketing authorization, achieving reimbursement, and maintaining compliance. ProPharma is a trusted partner for all MAH responsibilities, including acting as your MAH. Read more about our MAH activities in our blog Reclaiming the European Market: How to Secure EU/UK MAH Status Without the Local Infrastructure. Read more about how to navigate HTAR and the procedures and timelines for the JCA process in our blog Navigating the New EU Health Technology Assessment Regulation (HTAR): Are You Ready?
ProPharma can provide expert guidance tailored to your specific regulatory and market access needs. Contact one of ProPharma's market access experts today to explore how we can guide you through the European market access landscape as an MAH, especially in light of the European HTAR.
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TAGS: Marketing Authorization Holder (MAH) Health Technology Assessment (HTA) Regulatory Sciences Market Access
