Morgan-Warren Peter J
Medicines and Healthcare products Regulatory Agency, London, UK.
Eur J Ophthalmol. 2020 Mar;30(2):321-349. doi: 10.1177/1120672119830932. Epub 2019 Mar 4.
Regulatory approval of new medicines requires a thorough assessment of the potential clinical benefits and risks. Study end-points are expected to demonstrate a clinically relevant treatment effect that will translate into direct patient benefits. This study sought to review the ophthalmic medicines with European Union-wide approval granted via the Centralised Procedure and characterise the key efficacy end-points underpinning the demonstration of clinical benefit.
This study was a retrospective review of published data pertaining to the European regulatory authorisation of centrally approved ophthalmic products between 1999 and 2017, inclusive. All sources and data consulted are in the public domain. European Public Assessment Reports published by the European Medicines Agency were consulted for data concerning the pivotal clinical efficacy studies supporting the applications. Data analyses were descriptive.
The European Medicines Agency have authorised 30 products via the Centralised Procedure between 1999 and 2017. For these products, a total of 24 additional approvals for line extensions or additional therapeutic indications were granted. Four products have been approved for orphan indications, including one approval 'under exceptional circumstances' and one 'Conditional Marketing Authorisation'. Approvals for products in retina (36%) and glaucoma (28%) indications together accounted for the majority of authorisations, with trial end-points predominantly based on visual acuity and intraocular pressure parameters, respectively. Products were also approved for indications in ocular surface disease, inflammation, optic neuropathy and surgical adjuncts, with a range of functional and anatomical end-points.
Approvals for ophthalmic medicines have been granted for a range of clinical indications, representing a considerable portion of available major therapeutics for practitioners. Benefit-risk assessments rely on clinical trial data demonstrating a clearly relevant patient benefit.
新药的监管批准需要对潜在的临床益处和风险进行全面评估。研究终点应证明具有临床相关性的治疗效果，这将转化为对患者的直接益处。本研究旨在回顾通过集中程序获得欧盟范围内批准的眼科药物，并确定支持临床益处证明的关键疗效终点。
本研究是对1999年至2017年（含）期间集中批准的眼科产品的欧洲监管授权相关已发表数据的回顾性分析。所有查阅的资料来源和数据均为公开信息。查阅了欧洲药品管理局发布的欧洲公共评估报告，以获取支持申请的关键临床疗效研究的数据。数据分析采用描述性方法。
1999年至2017年期间，欧洲药品管理局通过集中程序批准了30种产品。这些产品共获得了24项关于产品线扩展或额外治疗适应症的批准。有4种产品已获批用于罕见病适应症，其中包括1项“在特殊情况下”的批准和1项“有条件上市授权”。视网膜疾病（36%）和青光眼（28%）适应症产品的批准占授权总数的大部分，试验终点主要分别基于视力和眼压参数。产品还获批用于眼表疾病、炎症、视神经病变和手术辅助等适应症，具有一系列功能和解剖学终点。
眼科药物已获批用于一系列临床适应症，占从业者可用主要治疗药物的相当一部分。获益-风险评估依赖于证明对患者有明确相关益处的临床试验数据。
