European Medicines Agency (EMA), London, UK.
Spanish Agency for Medicines and Healthcare Products (AEMPS), Madrid, Spain.
Ann Oncol. 2016 Jan;27(1):96-105. doi: 10.1093/annonc/mdv506. Epub 2015 Oct 20.
Patient access to new cancer drugs in the EU involves centralised licensing decisions by regulators as well as reimbursement recommendations in the context of national healthcare systems. Differences in assessment criteria and evidence requirements may result in divergent decisions at central and national levels, ultimately compromising effective access to patients. Early access decisions are particularly challenging due to the limited clinical evidence available to conclude on the benefit-risk and relative (cost-) effectiveness of new high-priced cancer drugs. We describe mechanisms to accelerate approval of promising anticancer drugs that fulfil an unmet medical need, review the experience from the European Medicines Agency, compare timelines and outcomes of reimbursement decisions in major EU markets, and discuss shortcomings of the current system, ongoing initiatives, and future steps to facilitate effective early access.
患者在欧盟获得新癌症药物需要监管机构做出集中许可决定，同时需要在国家医疗体系背景下提出报销建议。评估标准和证据要求的差异可能导致集中和国家层面的决策出现分歧，最终影响患者的有效获得药物的机会。由于新的高价癌症药物在获益风险和相对（成本）有效性方面的临床证据有限，早期获得药物的决策特别具有挑战性。我们描述了加速批准满足未满足医疗需求的有前途的抗癌药物的机制，回顾了欧洲药品管理局的经验，比较了主要欧盟市场报销决策的时间线和结果，并讨论了当前系统的缺点、正在进行的举措以及促进有效早期获得药物的未来步骤。
