A study by the European Medicines Agency (EMA) has confirmed that 30 ophthalmic products have been authorised via the Centralised Procedure between 1999 and 2017. The EMA reported that a total of 24 additional approvals for line extensions or additional therapeutic indications were granted – 4 products were approved for orphan indications, including one approval ‘under exceptional circumstances’ and one ‘Conditional Marketing Authorisation’. According to the study, the EMA confirmed that approvals for products were in retina (36%) and glaucoma (28%) indications and that trial end-points predominantly used outcomes for visual acuity and intraocular pressure parameters. Other types of products authorised were in ocular surface disease, inflammation, optic neuropathy and surgical adjuncts.
Under the centralised authorisation procedure in the EU, sponsor companies may submit a single marketing-authorisation application to EMA to make the medicine available to patients and healthcare professionals throughout the EU. The EMA’s Committee for Medicinal products for Human Use (CHMP) or the Committee for Medicinal products for Veterinary Use (CVMP) can carry out a scientific assessment of the application and give a recommendation on whether the medicine should be marketed or not. Once granted by the European Commission, the centralised marketing authorisation is valid in all EU Member States as well as in the European Economic Area (EEA) countries, such as Iceland, Liechtenstein and Norway. Prior to authorisation for marketing approval, clinical trial data is expected to provide convincing evidence of efficacy and an acceptable safety profile.
European regulators often require a quantifiable and objective basis of evidence for products to obtain safety and efficacy and this usually indicates the assessment of visual acuity measurement. However, regulators may accept other research tools such as contrast sensitivity testing, visual fields, anatomical imaging parameters, intraocular pressure (IPO), surrogate end-points and quality-of-life (QoL) parameters for demonstration of clinical benefit. Their paper, published in the European Journal of Ophthalmology (March 2019) stated that, “with 30 ophthalmic medicines and 53 approved product indications through the Centralised Procedure between 1999 and 2017, these products represent a considerable part of the pharmacotherapeutic armoury available to clinicians. Medicines for retina and glaucoma account for the majority of the total indications, demonstrating the high level of interest for clinical development in these therapeutic areas for which there is high and increasing disease burden.”