SC1-PM-08-2017-New therapies for rare diseases

Objectives of the Call:

• To advance the development of new therapeutic options with concrete benefits for patients living with rare diseases.
• To progress in orphan drug development due to well-prepared clinical trials and a multinational multicentre clinical trial with an appropriate number of patients.
• To develop a preliminary assessment of the potential economic and public health aspects of the new therapeutic option.
• To contribute to growth of SMEs involved in drug development.
• To contribute towards International Rare Diseases Research Consortium (IRDiRC) objectives