Workshop reportFacilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

- Increasing number of drug development programmes present opportunities and challenges.
- Increased focus on rigour and de-risking of both pre-clinical and clinical studies.
- Focus on lessening burden on patients and families, reducing cost of development.
- Increased patient engagement in design, execution, and regulatory processes key.
- TREAT-NMD provides an open discussion for these opportunities and challenges.