EVENT RECAP – Life Science Lectures: Episode Three “‘’EU HTA: How will the revised Pharma Package impact the value assessment of therapies?’’

On 7 September, EUCOPE – the European Confederation of Pharmaceutical Entrepreneurs, hosted the third episode of its #LifeScienceLectures webinar series.  The focus of the webinar, titled ‘’EU HTA: How will the revised Pharma Package impact the value assessment of therapies?’’ was to shed light on the interactions between the Regulation (EU) 2021/2282 on Health Technology Assessment (HTA) and the revision of the pharmaceutical legislation. As both legislative pieces will play a key role in supporting better health outcomes, the webinar explored how these two crucial legal reforms could enhance patient access to innovative therapies across the European Union.  

The Commission’s Perspective

The discussion was kicked off by Dirk van den Steen, Deputy Head of Unit ‘State of Health, European Semester, Health Technology Assessment’, DG SANTE, who elaborated on the opportunities and implications of the EU HTA Regulation and potential synergies with the EU Pharmaceutical Package. Dirk began by underscoring the shared objectives of both legislations: supporting expeditious access to more cost-effective medicines and a streamlined assessment process for medicinal products. He emphasised that “While both regulations work toward a common goal, they achieve this in different ways.” Dirk highlighted the vital interconnection between the EU Pharmaceutical Package and the EU HTA Regulation, stressing the need for consistency between upstream and downstream decisions, notably as regards the possible uptake of concepts under the pharma proposal such as possible guidance on (High) Unmet Needs and how this relates to EU-level and national level HTA work. According to van den Steen, “A well-coordinated relationship between the Pharmaceutical Package and HTA is essential for success”. 

According to van den Steen, the EU HTA procedure aims to create transparency and inclusivity by establishing the Coordination Group on HTA, consisting of national or regional authorities, the Stakeholder Network, and by defining rules for Joint Clinical Assessments (JCAs) and joint scientific consultations (JSCs) involving patients, clinical experts, and other relevant experts. The procedure intends to eliminate redundancy for national HTA authorities and the industry, ensuring predictability and the long-term sustainability of EU HTA cooperation.  

The Industry’s Perspective (Small & Mid-sized)

Ana Palma, Senior Director Global Head of Patient Access & Access Policy, Sobi, praised the ambitious goals of the EU HTA procedure to improve access and reduce burdens for both HTA bodies and the pharmaceutical industry. However, she cautioned that, “To succeed, we need sufficient harmonisation of methodologies to avoid additional requests for data and scientific evidence at the national level. In order to further improve the system, JSCs should be offered to all relevant stakeholders. Moreover, the methodologies should be flexible to reflect the specificities of particular therapies, such as Advanced Therapy Medicinal Products (ATMPs) and Orphan Medicinal Products (OMPs), while the Stakeholder Network should be a co-creating partner in drafting such procedures and its implementation. Finally, timelines have to be practical. I see great opportunities for adapted clinical trials, Real World Evidence (RWE) and regulatory sandboxes”. She highlighted the challenges of evidence generation for certain therapies, especially in rare diseases, and stressed the need for the EU HTA to endorse suitable approaches like single-arm trials and use of RWE to avoid further delays in access to medicines. 

Along these lines, Alexander Natz, Secretary General, EUCOPE, emphasised the need for predictability. He also drew attention to the importance of implementing existing legislation, such as Directive 89/105 EEC, the so-called Transparency Directive, to ensure timely access to medicines for patients. Alexander noted that “Timelines at the Member States’ level are not always met. We should look into the Transparency Directive because timely access to medicine is key. The JCA report cannot provide value for national decision-making when the timelines for reimbursement decisions at the national level are not followed. The discussion around its application should not be forgotten”. 

The Patients’ Perspective

Ultimately, the patients are the primary beneficiaries of these legislations.  Antonella Cardone, Chief Executive Officer, Cancer Patients Europe (CPE), speaking on behalf of patients, stated “We are watching the actual implementation of the JCA process, and we believe that relevant governance should be set up in a way that is as inclusive as possible, and all relevant stakeholders should be part of it”. Cardone added that “The unmet clinical needs should be fostered throughout the process. We believe that the definition of unmet need should be broader and go beyond clinical and medical aspects and consider societal aspects and quality of life for patients”.  

In conclusion, the webinar explored the complex interplay between the new EU HTA procedure and the Pharmaceutical Package, emphasizing the need to ensure timely access to innovative therapies and better healthcare outcomes for patients across the EU. The ongoing work on implementing acts and the careful consideration of specific therapies and evidence-generation approaches are critical in achieving these objectives. As underlined by Palma and Cardone, fast and systematic adoption of JCAs by Member States is key to preventing duplication of work and accelerating access to medicines.  

EUCOPE will continue working to ensure the success of the EU HTA Regulation and its implementation, as a member of the HTA Stakeholder Network.