Presentation (CTI)

OBJECTIVES: Following implementation of the EU Health Technology Assessment Regulation (HTAR) in January 2025, the industry’s focus now shifts to its upcoming expansion into orphan diseases in 2028. Given the investment into preparations for oncology, insights will be invaluable to manufacturers in the orphan space. Our research examines the scope of manufacturer’s preparatory activities head of the 2025 implementation, providing recommendations on when and how orphan medicine manufacturers should act to ensure their readiness.
METHODS: Forty-four projects relating to governance, training and strategic decision-making, at manufacturer and asset level, were analysed to identify the types, timings and duration of work conducted to prepare for joint clinical assessments (JCA) and joint scientific consultations (JSC). Findings were synthesized into a timeline for preparedness.
RESULTS: Preparations encompassed organizational readiness (e.g., benchmarking, impact assessment & ways of working), JSC and JCA process preparations (e.g., PICO simulations, briefing book, dossier) and training, thought leadership and PMO support. Whilst early PICO simulations and JSC/scientific advice are the earliest activities, prior to registrational trial protocol lock, most activities occur 12 - 24 months before submission. Assuming the earliest submission date in January 2028, activities could begin as early as July 2025 with significant investment required from early 2026. Notably, the peak in activities aligns with typical timelines required for an external comparator study (24-30 months), which are often required to inform comparative effectiveness for orphan products with a single arm trial design.
CONCLUSIONS: With such a broad range of activities to be executed to prepare for launch, orphan drug manufacturers should start preparations this year to sufficiently prepare a robust evidence package for 2028 JCA submissions. Considering the complex and resource-intensive processes of evidence planning and generation for orphan medicines, it is essential to strategically align efforts to optimize resource allocation and manage timelines effectively.