Do conditional marketing authorisations actually accelerate patient access? Time-to-access of conditional vs. standard marketing authorisations in Italy, Spain, and Germany

有条件上市许可真的能加快患者获得药物的速度吗?意大利、西班牙和德国有条件上市许可与标准上市许可的用药时间对比

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Abstract

BACKGROUND: The European Medicines Agency (EMA) implemented 'fast-track' programmes, like conditional marketing authorisations (CMA), where the benefits of immediate drugs' availability outweigh the risks associated with incomplete evidence. However, payers in the European Union (EU) decide on medicines' coverage based on clinical benefits assessment, cost-effectiveness and/or budget impact. We investigated differences in the time-to-access of drugs approved via CMA vis-à-vis standard marketing authorisation (SMA) in Italy, Germany, and Spain. METHODS: CMA-licenced drugs from 2006 to 2022 were retrieved and matched with comparable SMA drugs. Collected data were as follows: marketing authorisation details, drug characteristics, pivotal trials' characteristics and national reimbursement decision dates. Data sources included European Public Assessment Reports, and country-specific databases (Farmadati®, Lauer-Taxe®, BIFIMED). RESULTS: CMA drugs take longer, in days, to reach reimbursement compared to SMA drugs both in Italy (CMA: median 523, mean 635, standard deviation (SD 364; SMA: median 455, mean 497, SD 242) and Spain (CMA: median 691, mean 779, SD 456; SMA: median 534, mean 568, SD 273). Cox regressions and Kaplan-Meier survival analyses corroborate these findings. CONCLUSIONS: The EMA's intent to accelerate access to promising medicines may be offset by longer timelines to secure national reimbursement in major EU nations.

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