The European Medicines Agency is a decentralized agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union. It began operating in 1995.
Which services are provided for rare disease researchers? - Orphan designation Since the pharmaceutical industry has little interest, under normal market conditions, in developing and marketing medicines intended for small numbers of patients, the EU offers a range of incentives to encourage the development of orphan medicines. EMA is responsible for reviewing applications from sponsors for the designation of medicines for rare diseases. Sponsors of designated orphan medicines are eligible to benefit from the incentives offered, including assistance with development of the medicine, reduced fees for marketing-authorization applications, protection from market competition once the medicine is authorized. Click here to apply for an orphan designation
- Scientific advice and protocol assistance Scientific advice is when the Agency gives advice to a company on the appropriate tests and studies in the development of a medicine. This is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines, for the benefit of patients. Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases. In addition to scientific advice, companies developing an orphan medicinal product can receive answers to questions relating to the criteria for authorization of an orphan medicine. These include: the demonstration of significant benefit within the scope of the designated orphan indication; similarity or clinical superiority over other medicines. This is relevant if other orphan medicinal products exist that might be similar to the product concerned and which have market exclusivity in the same indication. Click here for details on how to apply
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