Enhancing Development Strategies Through Early Scientific Advice from HTA Agencies-Experiences, Expectations and Best Practices from Health Technology Developers.

Introduction: Early health technology assessment (HTA) advice provides value to pharmaceutical companies during drug development by identifying potential data gaps, refining study designs, and improving understanding of HTA agencies' evidentiary requirements. This study evaluated the landscape, benefits, and challenges of early HTA advice, with a focus on European agencies and global practices amid the transition to the EU HTA Regulation.

Methods: A perception survey involving 12 pharmaceutical companies explored engagement with HTA agencies, challenges, and strategic priorities. Additionally, a forum was conducted with 22 company representatives, discussing internal and external best practices. Key topics included the evolving role of Joint Scientific Consultations (JSCs), integration of real-world evidence (RWE), and incorporation of patient-reported outcomes (PROs) into advice processes.

Results: The findings highlighted active engagement with national agencies such as NICE and G-BA and European initiatives like EUnetHTA Joint Actions. NICE advice was valued for cost-effectiveness insights, but its post-Brexit absence reduced collaboration at the European level. JSCs under the EU HTA Regulation were seen as critical, but resource constraints and limited availability posed challenges. Companies prioritized topics such as comparator choices, outcome measures, and RWE but faced internal barriers like resource allocation and unclear decision criteria. Outside Europe, uptake of advice services, such as those from CDA-AMC, remained limited but underscored the potential of lifecycle approaches for iterative learning.

Conclusions: Early HTA advice is essential in pharmaceutical development. Enhancing JSC capacity, stakeholder engagement, and feedback mechanisms will strengthen alignment between companies and HTA agencies, fostering evidence-based decision-making and improved health outcomes.