EMA: Regulatory and procedural guideline: Practical information for sponsors during the early phase of an orphan drug application

This is a short list of practical issues and information for sponsors planning to submit an application for designation of a medicine as an orphan medicinal product. It does not replace the legal requirements as set in the EU Directives, nor the guidelines available on the Agency website. The information is based on issues that frequently arise during the early phases of the application process, and is loosely organised as a simple list of issues.