Key Trends Impacting HTA and Regulatory Interface

Navigating EU HTA Regulation: Opportunities, Challenges, and Optimizing Parallel Joint Clinical Assessments and EMA Reviews
Presenter: Nathalie Largeron, MSc, PharmD – Global Head of HTA Strategy, Sanofi

Description: The implementation of the new EU Health Technology Assessment Regulation in January 2025 marks a significant milestone poised to reshape how stakeholders engage with Health Technology Assessment (HTA) and the evidence requested for access decisions. This shift aims to streamline processes, reduce redundancy, and expedite treatment access for patients. However, this transition will require new approaches from EMA and national HTA agencies, as well as other interested stakeholders, and will necessitate reshaping their interactions to meet the regulation’s requirements.

Crucially, the parallel running of Joint Clinical Assessments and EMA reviews requires a nuanced understanding of their simultaneous execution, mutual impact, and communication pathways. Clear insights into how stakeholders can positively engage with these processes, proactively identifying opportunities and challenges that may surface, and nurturing collaborations, are critical to achieve optimal outcomes for patients without additional delays to access treatment.
This presentation will explore the interaction between these distinct yet related procedures, uniting shared participants and interests and will present opportunities and challenges in the proposed revision and how they will impact the different stakeholders involved.

Disclosure: Nathalie Largeron is a Sanofi employee and may hold stock and/or stock options in the company.

The changing regulatory and HTA landscape of legislation & guidance facing stakeholders when seeking to use heterogenous health data
Presenter: Rebecca Lumsden, PhD – IDERHA task lead & Head of Regulatory Science & Policy EU/AMEE, Sanofi

Description: Over recent years there has been a rapid growth in new data sources that can support both research and healthcare delivery. However, these data can come from diverse sources ranging from new digital technologies e.g. apps & wearables to clinical trials to electronic health records. Efforts to fully leverage the potential of these heterogenous data sources have hit challenges in maximizing their use by third parties. This presentation will explore the changing regulatory landscape of legislation & guidance facing stakeholders when seeking to use heterogenous health data, including key barriers and opportunities.

Case-study: How the IDERHA project is addressing challenges to the use and acceptance of Real-World Data in regulatory and HTA settings
Presenter: Katharine Cresswell, MPH – IDERHA task lead & Scientific Adviser - Science Policy and Research Programme, National Institute for Health and Care Excellence

In 2023, a new Public Private Partnership (PPP), the Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IDERHA) project was launched. The project was one of the first projects funded under the newly launched Innovative Health Initiative’s (IHI) call titled “Access and integration of heterogeneous health data for improved healthcare in disease areas of high unmet public health need”. This presentation will describe the IDERHA project and the steps it is taking to accelerate policy development to support and advance the wider use and acceptance of Real-World Evidence (RWE) to support regulatory and HTA decision making processes. It will also touch on the need for stakeholder consensus and engagement in the policy development and value creation.