Crommelin Daan Ja, Broich Karl, Holloway Chris, Meesen Bianca, Lizrova Preiningerova Jana, Prugnaud Jean-Louis, Silva-Lima Beatriz
Department of Pharmaceutics, Pharmaceutical Sciences, Utrecht Institute for Pharmaceutical Sciences (UIPS), Faculty of Sciences, Utrecht University, Utrecht, The Netherlands
President and Head of the Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM), Bonn, Germany.
Mult Scler. 2016 Aug;22(2 Suppl):47-59. doi: 10.1177/1352458516650744.
Although there is still no cure for multiple sclerosis (MS), the introduction of several innovative drugs with modes of action different from that of the existing drug arsenal and the progress in monitoring disease progression by imaging and using biomarkers are currently causing a knowledge surge. This provides opportunities for improving patient disease management. New therapies are also under development and pose challenges to the regulatory bodies regarding the optimal design of clinical trials with more patient-focused clinical endpoints. Moreover, with the upcoming patent expiry of some of the key first-line MS treatments in Europe, regulatory bodies will also face the challenge of recommending marketing authorisation for generic and abridged versions based on appropriate requirements for demonstrating equality/similarity to the innovator's product.
The goal of this article is to improve the understanding of the relevant guidance documents of the European Medicines Agency (EMA) on clinical investigation of medicinal products and to highlight the issues that the agency will need to clarify regarding follow-on products of first-line MS treatments.
Today, it is clear that close collaboration between patients, healthcare professionals, regulatory bodies and industry is crucial for developing new safe and effective drugs, which satisfy the needs of MS patients.
尽管多发性硬化症（MS）仍无法治愈，但几种作用方式不同于现有药物库的创新药物的推出，以及通过成像和使用生物标志物监测疾病进展方面的进展，目前正在引发知识的激增。这为改善患者疾病管理提供了机会。新疗法也在研发中，在以患者为中心的临床终点进行临床试验的最佳设计方面给监管机构带来了挑战。此外，随着欧洲一些关键一线MS治疗药物即将到期，监管机构还将面临根据证明与创新产品等效/相似的适当要求，为仿制药和简化版产品推荐上市许可的挑战。
本文的目的是增进对欧洲药品管理局（EMA）关于药品临床研究的相关指导文件的理解，并突出该机构在一线MS治疗后续产品方面需要澄清的问题。
如今，很明显患者、医疗保健专业人员、监管机构和行业之间的密切合作对于开发满足MS患者需求的新的安全有效药物至关重要。
