Cracking the Code: EU Joint Clinical Assessments and the Future of Pharma

The European Union's Health Technology Assessment Regulation (HTAR), effective from January 12, 2025, introduces a transformative framework for evaluating health technologies across member states. Central to this regulation is the implementation of Joint Clinical Assessments (JCAs), aiming to harmonize the evaluation of clinical evidence for new health technologies. This article delves into the nuances of JCAs, their implications for pharmaceutical companies operating within the EU, and recent examples of JCAs in practice.

Understanding Joint Clinical Assessments (JCAs)

Under Regulation (EU HTAR) 201/2282, JCAs are designed to assess four clinical domains centrally, while the remaining (non-clinical) domains will remain part of national assessments. Reimbursement and pricing decisions informed by the JCA and by national HTAs will continue to be made at the national level. The assessments are conducted collaboratively by the Coordination Group on Health Technology Assessment (HTACG), comprising representatives from national HTA bodies.

The rollout of JCAs is phased:

• From January 12, 2025: JCAs are mandatory for new oncology medicines and advanced therapy medicinal products (ATMPs).

• By 2028: Orphan medicinal products will be included.

• By 2030: All other centrally authorized medicinal products will require JCAs.

For medical devices, JCAs will apply to specific high-risk categories starting in 2026.

Recent Examples of JCAs

The first two JCAs under the new regulation commenced in April 2025:

1. Tovorafenib (Ojemda): Developed by Ipsen, this RAF kinase inhibitor is intended for the treatment of pediatric low-grade glioma. The assessment is led by Ireland's National Centre for Pharmacoeconomics (NCPE), with Germany's Institute for Quality and Efficiency in Health Care (IQWiG) as co-assessor.

2. Lifileucel (Amtagvi): An autologous tumor-infiltrating lymphocyte therapy developed by Iovance Biotherapeutics for advanced melanoma. France's Haute Autorité de Santé (HAS) is the assessor, with Poland's Agency for Health Technology Assessment and Tariff System (AOTMiT) as co-assessor.

These assessments are conducted in parallel with the European Medicines Agency's marketing authorization process, aiming to streamline evaluations and facilitate timely patient access to innovative therapies.

Implications for Pharmaceutical Companies

1. Streamlined Access and Reduced Duplication

JCAs aim to minimize redundant evaluations by providing a single clinical assessment accepted across EU member states. This harmonization can expedite market access and reduce the administrative burden associated with multiple national submissions. However, it's important to note that JCAs focus solely on clinical evidence; economic evaluations and reimbursement decisions remain under national jurisdiction.

2. Procedural Shifts JCAs begin with a PICO survey where 27 member states submit their clinical criteria. These diverse requirements are consolidated but come after EMA approval, making it hard to tailor clinical trials to all PICO demands. Early engagement through Joint Scientific Consultations (JSCs) is vital to align development with HTA needs. However, JSC slots are limited, so pharma must also engage local HTA bodies early.

3. Evidence Generation Challenges

With varied PICO inputs and timing mismatches, companies will rely more on indirect treatment comparisons (ITC) and real-world evidence to fill data gaps. Planning the necessary systematic literature reviews to feed into the ITCs will be key to addressing diverse PICO requirements across member states.  

4. Challenges for Small and Mid-Sized Enterprises (SMEs)

The JCA runs alongside EMA submissions, compressing timelines and requiring earlier resource allocation, especially for local market access teams. Smaller companies must build their European infrastructure sooner than before. SMEs may face disproportionate challenges in adapting to the new HTA framework due to resource constraints. The limited number of JSC opportunities and the complexity of aligning clinical development plans with HTA requirements could pose significant hurdles for these companies.

5. Potential for Delayed Access

While JCAs are intended to streamline assessments, the lack of binding authority means that member states can request additional evidence, potentially leading to delays in reimbursement decisions. Moreover, the limited involvement of developers in the JCA process may result in misalignments between submitted data and HTA expectations, further complicating access timelines.

Strategic Considerations for Pharma Companies

To navigate the evolving HTA landscape effectively, pharmaceutical companies should consider the following strategies:

• Proactive Planning: Initiate early engagement with HTA bodies and plan evidence generation strategies that align with both regulatory and HTA requirements.

• Resource Allocation: Invest in building internal capabilities or partnerships to manage the complexities of the JCA process, particularly for SMEs.

• Monitoring and Adaptation: Stay informed about the evolving HTA procedures and be prepared to adapt strategies based on feedback and emerging best practices.

• Cross-Functional Collaboration: Success demands tight coordination between clinical, regulatory, RWE/HEOR, and market access teams. Integrating HTA requirements early in clinical development is crucial to avoid costly misalignments. Collaboration across departments is crucial for early scientific consultations, anticipating PICO requirements, planning evidence generation, and preparing a robust dossier.

  Conclusion

The implementation of JCAs under the EU HTAR marks a significant shift in the evaluation of health technologies, aiming to harmonize assessments and improve patient access to innovative treatments. While the regulation offers opportunities for streamlined processes, pharmaceutical companies must navigate challenges related to limited consultation opportunities, potential delays, and resource constraints. The EU HTA regulation is set to transform market access pathways across Europe. To succeed in this new environment, companies must:

• Engage early: Anticipate PICO demands through JSCs and national consultations.

• Align cross-functionally: Ensure regulatory, RWE/HEOR/Market Access, and clinical teams collaborate on trial and submission strategies.

• Plan for scale: Allocate resources well ahead of submission deadlines and consider bolstering local affiliate support.

• Leverage evidence smartly: Use RWE and ITCs to supplement clinical trial data and meet diverse HTA needs.

Pharma companies are at different stages of readiness, but all must be fully prepared for the EU JCAs. Ongoing uncertainties around procedures and rising resource requirements highlight the need for strategic planning, strong cross-functional collaboration, and proactive engagement with key stakeholders well ahead of time.