A review of the current collaboration that the European Medicines Agency (EMA) has with other regulators to designate orphan drugs is described in Nature Reviews: Drug Discovery. Since the inception of the possibility of joint applications with the EMA and the United States in 2007, the two agencies have worked together to understand areas of similarity at the time of submission for an orphan drug designation. Even though there are some differences in the way both parties make interpretations, there is a constant effort “to setting the boundaries of a rare disease or condition at the time of designation.” According to the authors, currently ~50% of submissions to the EMA are done in parallel with the FDA, with ~30–40% of applicants using the joint FDA–EMA application form.
The EMA also collaborates with the Ministry of Health, Labor and Welfare/Pharmaceuticals and Medical Devices Agency (MHLW/PMDA) in Japan which began in late 2010, even though the collaboration is limited in comparison to FDA. This is due to several administrative differences, however many strides are being made to make this process feasible which includes an English website for the MHLW and some links to it on the EMA website. According to the authors, there are some similarities with the scientific submission, some evidence of medical plausibility can be interchangeably used even though the prevalence threshold in Japan is different to the one required in Europe.
The authors hope for an increase in the global approach towards orphan medicine as it would mean greater benefits for rare disease patients.
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