TOPIC : New therapies for rare diseases
|Publication date:||14 October 2015|
|Types of action:||RIA Research and Innovation action|
|DeadlineModel: Planned opening date:||two-stage 29 July 2016||Deadline: 2nd stage Deadline:||
04 October 2016 17:00:00
11 April 2017 17:00:00
|Time Zone : (Brussels time)|
Topic DescriptionSpecific Challenge:
Rare diseases are diseases which affect not more than 5 per 10 000 persons in the European Union, as defined in the context of the EU legislation. A considerable amount of knowledge has been generated by biomedical research in recent years, yet most of the 6 000 to 8 000 rare diseases are lacking therapies despite many of these diseases being life-threatening or chronically debilitating.
Specific problems posed in therapy development for rare diseases include the small and dispersed patient populations and the nature of the therapies proposed, which are often highly specialised and novel. Amongst other challenges, this leads to the requirement for seeking early advice of regulatory authorities during development. In addition, despite the special incentives for the development of orphan medicinal products, and the often high prices of some of the developed therapies, the limited market for such therapies lead to a low commercial return, and/or limited access.Scope:
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.
Selected proposals shall contribute to the objectives of, and follow the guidelines and policies of the International Rare Diseases Research Consortium, IRDiRC (www.irdirc.org).
The Commission considers that proposals requesting a contribution from the EU of between EUR 4 and 6 million would allow this specific challenge to be addressed appropriately. Nonetheless, this does not preclude submission and selection of proposals requesting other amounts.Expected Impact:
• In line with the objectives of the Union pharmaceutical legislation on orphan medicinal products, proposals shall contribute to advance the development of new therapeutic options with concrete benefits for patients living with rare diseases.
• Rapid progress in orphan drug development due to well-prepared clinical trials and a multinational multicentre clinical trial with an appropriate number of patients.
• Develop a preliminary assessment of the potential economic and public health aspects of the new therapeutic option.
• Contribute to growth of SMEs involved in drug development.
• In line with the Union’s strategy for international cooperation in research and innovation, proposals shall contribute towards IRDiRC objectives.
Topic conditions and documents
Please read carefully all provisions below before the preparation of your application.
- List of countries and applicable rules for funding: described in part A of the General Annexes of the General Work Programme.
Note also that a number of non-EU/non-Associated Countries that are not automatically eligible for funding have made specific provisions for making funding available for their participants in some Horizon 2020 topics (follow the links to Australia, Brazil, Canada, China, Hong Kong & Macau, India, Japan, Republic of Korea, Mexico, Russia, Taiwan, USA*).
*Note that US participants in projects under the Horizon 2020 "Health, Demographic Change and Wellbeing" Societal Challenge are automatically eligible for EU-funding according to the provisions in the relevant work programme
- Eligibility and admissibility conditions: described in part B and C of the General Annexes of the General Work Programme.
Proposal page limits and layout: Please refer to Part B of the standard proposal template.
3.1 Evaluation criteria and procedure, scoring and threshold: described in part H of the General Annexes of the General Work Programme, with the following exceptions: The thresholds for each criterion in a single stage process will be 4, 4 and 3. The cumulative threshold will be 12.
3.2 Submission and evaluation process: Guide to the submission and evaluation process
- Indicative timetable for evaluation and grant agreement:
Information on the outcome of single-stage evaluation: maximum 5 months from the deadline for submission.
Signature of grant agreements: maximum 8 months from the deadline for submission.
- Provisions, proposal templates and evaluation forms for the type(s) of action(s) under this topic:
Research and Innovation Action:
Specific provisions and funding rates
Standard proposal template
Specific evaluation form
H2020 General MGA -Multi-Beneficiary
Annotated Grant Agreement
- Additional provisions:
Horizon 2020 budget flexibility
- Open access must be granted to all scientific publications resulting from Horizon 2020 actions, and proposals must refer to measures envisaged. Where relevant, proposals should also provide information on how the participants will manage the research data generated and/or collected during the project, such as details on what types of data the project will generate, whether and how this data will be exploited or made accessible for verification and re-use, and how it will be curated and preserved.
- Additional documents:
H2020 Work Programme 2016-17: Health, demographic change and wellbeing
H2020 Work Programme 2016-17: Dissemination, Exploitation and Evaluation
H2020 Work Programme 2016-17: General Annexes
Legal basis: Horizon 2020 - Regulation of Establishment
Legal basis: Horizon 2020 Rules for Participation
Legal basis: Horizon 2020 Specific Programme
The submission system is planned to be opened on the date stated on the topic header.
H2020 Online Manual your online guide on the procedures from proposal submission to managing your grant.
Participant Portal FAQ – Submission of proposals.
National Contact Points (NCP) - contact your NCP for further assistance.
Research Enquiry Service – ask questions about any aspect of European research in general and the EU Research Framework Programmes in particular.
Enterprise Europe Network – contact your EEN national contact for advice to businesses with special focus on SMEs. The support includes guidance on the EU research funding.
IT Helpdesk- contact the Participant Portal IT helpdesk for questions such as forgotten passwords, access rights and roles, technical aspects of submission of proposals, etc.
Clean Sky 2 National Contact Points - Clean Sky 2 JU States Representative Group
Clean Sky 2 Helpdesk: Info-Call-CPW-2014-01@Cleansky.EU
ECSEL National Contact Points – consult Annex G of the ECSEL Work Plan
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IMI States Representative Group (SRG) – contact you SRG member for assistance.
IMI JU IT Helpdesk – for IMI2 topics contact the SOFIA IT helpdesk for questions such as forgotten passwords, access rights and roles, technical aspects of submission of proposals, etc.
Ethics – for compliance with ethical issues, see the Participant Portal and Science and Society Portal
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CEN and CENELEC, the European Standards Organisations, advise you how to tackle standardisation in your project proposal. Contact CEN-CENELEC Research Helpdesk at firstname.lastname@example.org.
The European Charter for Researchers and the Code of Conduct for their recruitment
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