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H2020 Call – New therapies for rare diseases


Topic identifier: SC1-PM-08-2017
Types of action: RIA Research and Innovation action

Deadline: April 11th 2017

Support will be provided to clinical trials on substances where orphan designation has been given by the European Commission, where the proposed clinical trial design takes into account recommendations from protocol assistance given by the European Medicines Agency, and where a clear patient recruitment strategy is presented. Clinical trials may focus on a range of interventions with an orphan designation, from small molecule to gene or cell therapy, may include novel interventions and/or repurposing of existing and known interventions. The intervention must have been granted the EU orphan designation at the latest on the date of the full proposal call closure. A concise feasibility assessment justified by available published and preliminary preclinical or clinical results and supporting data shall also be provided. Appropriate plans to engage with patient organisations, Member States health authorities and considerations of efficacy/potential clinical benefit as well as early indication on health economics should be integrated in the application. In addition to the clinical trial, proposals may also include limited elements of late stage preclinical research and/or experimental evaluation of potential risks which must be complementary/contribute to the clinical trial(s) carried out within the proposal. The centre of gravity must clearly be the clinical trial(s). The participation of SMEs is encouraged.

More information and call documents here

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