TOPIC : New therapies for rare diseases
|Publication date:||14 October 2015|
|Types of action:||RIA Research and Innovation action|
|DeadlineModel: Opening date:||two-stage 28 July 2016||Deadline: 2nd stage Deadline:||
04 October 2016 17:00:00
11 April 2017 17:00:00
|Time Zone : (Brussels time)|
02 February 2017 13:26
Generalised feedback for successful applicants after stage 1, with information and tips for preparing the full proposal, is now available under the "Topic conditions and documents" section.
25 January 2017 15:16
An overview of the evaluation results of the first stage (flash call info) that closed on 04 October 2016 is now available under the "Topic conditions and documents" section.
13 January 2017 09:35
Please note that Switzerland is now officially associated to the Horizon SC1 as well. Swiss participants are therefore eligible to receive Horizon 2020 SC1 support. Please see details in the note here.
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.Cross-cutting Priorities:
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 (to check funding conditions, 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 the second stage of a two-stage call 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 two-stage evaluation:
For stage 1: maximum 3 months from the deadline for submission.
For stage 2: 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
Essential information for clinical studies
- Additional provisions:
Horizon 2020 budget flexibility
- Open access must be granted to all scientific publications resulting from Horizon 2020 actions.
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.
Open access to research data
The Open Research Data Pilot has been extended to cover all Horizon 2020 topics for which the submission is opened on 26 July 2016 or later. Projects funded under this topic will therefore by default provide open access to the research data they generate, except if they decide to opt-out under the conditions described in annex L of the Work Programme. Projects can opt-out at any stage, that is both before and after the grant signature.
Note that the evaluation phase proposals will not be evaluated more favourably because they plan to open or share their data, and will not be penalised for opting out.
Open research data sharing applies to the data needed to validate the results presented in scientific publications. Additionally, projects can choose to make other data available open access and need to describe their approach in a Data Management Plan.
- Projects need to create a Data Management Plan (DMP), except if they opt-out of making their research data open access. A first version of the DMP must be provided as an early deliverable within six months of the project and should be updated during the project as appropriate. The Commission already provides guidance documents, including a template for DMPs.
- Eligibility of costs: costs related to data management and data sharing are eligible for reimbursement during the project duration.
The legal requirements for projects participating in this pilot are in the article 29.3 of the Model Grant Agreement.
- 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
No submission system is open for this topic.
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.
Contact the EIT for further assistance related to the call, topics and the content of proposals via the Contact Page on the EIT website.
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.
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The European Charter for Researchers and the Code of Conduct for their recruitment
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