Presentation (CTI)

OBJECTIVES: Romania has the slowest access to innovative medicines in Europe: 1,201 days from EU marketing authorization to availability, according to 2025 EFPIA Patients W.A.I.T. Indicator, and one of the continent's lowest availability rates. Funding decisions are made reactively, after authorization, from historical budgets, not anticipating the incoming innovation. We propose a model for national horizon-scanning (HS) system for all innovative medicines that anticipates budget needs and pre-positions access decisions, using national orphan-medicine data to show such planning is both needed and achievable.
METHODS: We designed an HS model from a scoping review of European systems (IHSI, the Dutch Horizonscan, AIFA, the Nordic JNHB, and UK PharmaScan) and mapped it onto Romania's institutions and HTA procedure, without a parallel structure. All orphan dossier-indications under the specific orphan HTA pathway (2014-2026) were analyzed for reimbursement status and time to reimbursement.
RESULTS: Of 199 orphan dossier-indications, 145 were reimbursed, reaching patients a median of 45 months (mean 59) after EU marketing authorization; June 2026: 43 (36 medicines) were awaiting reimbursement for a median of 28 months (mean 39) after authorization, ten for over five years. Targeting this domestic delay, the pilot HS draws on EU-level pipeline data, tracking each molecule from entry into EMA evaluation and mapping every input (with defined data sources, amenable to AI-assisted automation) needed to score unmet need and budget impact. A need-by-impact matrix then assigns a management track, so CHMP opinion and EC authorization become planned triggers, with budget and contracting terms prepared in advance.
CONCLUSIONS: Romania's growing orphan backlog is the cost of reacting rather than anticipating. This first national HS proposal, built on existing institutions and piloted on orphans, scalable to advanced therapies, PRIME-designated products, and all centrally authorized medicines, becomes critical for securing earlier access as budget-driven delays keep growing.