Research & Innovation - Participant Portal


TOPIC : Pilot programme on a Clinical Compound Bank for Repurposing: Rare/orphan diseases

Topic identifier: IMI2-2017-13-15
Publication date: 30 November 2017

Types of action: IMI2-RIA Research and Innovation action
Opening date:
30 November 2017
2nd stage Deadline:
28 February 2018 17:00:00
06 September 2018 17:00:00

Time Zone : (Brussels time)
  Horizon 2020
Topic Description
Specific Challenge:

On average it takes about 14 years for a new drug to travel from the research lab to market approval at an average cost of ≥€2 billion. Only 10% of compounds that enter preclinical testing ever make it into clinical trials, with only 20% of these achieving marketing approval. It is therefore very important to advance approaches to decrease the time, reduce costs and improve success rates during the development of drugs.

One approach is the repurposing of high-quality pharmaceutical industry compounds that have stalled at some stage during research or development. Many of these compounds have already undergone preliminary testing in humans, but have not been progressed further because they were not found to be sufficiently effective in the indication for which they were originally developed.

These compounds represent valuable tools that researchers can use to test their novel hypotheses for alternative therapeutic indications, with the ultimate aim of identifying alternative uses for these compounds in other indications. Since partial preclinical and clinical documentation packages have been developed for these assets, any positive findings hold the opportunity to progress towards the market more quickly and cost-effectively, with the ultimate goal of benefiting patients in diseases of high unmet need.

This asset-sharing repurposing programme through IMI2 JU aims to provide researchers across the EU to form hypothesis, to engage in collaborative research with industry and to access discontinued compounds that have already passed through several stages of the drug development process.

In this call, compounds, together with key information on their mechanism of action, pharmacology, safety, tolerability and exclusions, are made available for exploration in the area of Rare/orphan diseases (please find more details in the topic text). More precisely, applicants are invited to submit proposals to utilise these assets to test their hypotheses, to generate clinical data and, if needed, prerequisite preclinical data, with the ultimate aim of taking these assets to the market in alternative indications to those that they were originally developed for.


The overall objective of this pilot programme is to take one of the previously deprioritised clinical compounds and investigate their therapeutic potential in new clinical indications in the area of Rare/orphan diseases.

These compounds are listed in the topic text together with key information including mechanism of action, original indication, route of administration pharmacology, safety, tolerability and links to previous clinical studies and publications, to facilitate idea generation by investigators with hypotheses for novel uses. These compounds have all been through clinical phase 1 studies.

  • Proposals should cover clinical Phase 2A proof of concept studies, though larger Phase 2 studies are also in scope if these are within the budget. Clinical submissions should aim at moving towards the next stage of development and positive data should be a starting point for further investment into developing a drug towards clinic and regulatory approval;
  • If preclinical work is deemed necessary to provide additional support and confidence before moving into a clinical study in an alternative indication, proof-of-concept/feasibility preclinical studies of up to a year in duration can be included in the proposal. These studies should have clear go/no-go criteria for progressing in to the clinical phase of the project.

This programme intends to support only innovative clinical development for the compounds. This means that proposals for clinical development should not be considered in an indication which has been already tested (i.e. original primary indication or additional studies) or if there are already ongoing or planned clinical studies on identical or related disease indications with the compound or with a compound with overlapping mechanism of action that impacts the novelty of a given proposal.

Information on original primary indications, already tested indications, ongoing and/or planned clinical studies for each of these ten compounds can be found in the topic text.

Expected Impact:
  • Achieving early proof-of-concept for new mechanisms with the potential to rapidly bring novel drugs to patients in areas of high unmet need and/or those with greatest disease burden;
  • Generation of ideas and/or data licensed from the research organisation, leading to further development of the compound in the new indication;
  • Added value by repurposing pharmaceutical assets which have already passed through several stages of the R&D process. This can offer significant time, cost and risk savings over embarking on discovery programmes with novel targets;
  • Supporting EU academic institutions to conduct well-designed and high standard translational and drug development research with quality compounds under GCP conditions, resulting in high impact publications and patents when possible;
  • Pooling of resources and greater collaboration between the public and private sectors, with the potential for pharmaceutical involvement or establishment of SMEs following in/out-licencing;
  • Boosting the discovery and development of therapeutics in the area of Rare/orphan diseases using a more cost-effective approach to drug development;
  • Advancing science and knowledge of disease (patho)physiology through testing of new hypothesis;
  • Boosting European competiveness by contributing to the establishment of closer links between industry and academia across the EU, and ensuring Europe is competitive in line with initiatives already in place in other leading scientific regions around the world.
Topic conditions and documents

Please read carefully all provisions below before the preparation of your application.

The IMI2 13th Call for proposals topic text as well as the Call Conditions are available here.

 The budget breakdown for this Call is given at the end of the Call topics text, in the Call Condtions section, as well as the following information:

1.   Eligible countries: described in article 10(2) of the Rules for participation in Horizon 2020 and in article 1 of the Commission Delegated Regulation related to IMI JU.

2.   Eligibility and admissibility conditions: described in the IMI2 Manual for evaluation, submission and grant award. See also the Commission Delegated Regulation related to IMI JU.

Proposal page limits and layout: Please refer to Part B of the proposal template in the submission tool below.

3.   Evaluation:
Submission and evaluation process, including evaluation criteria and procedure, scoring and threshold are described in the IMI2 Manual for submission, evaluation and grant award. See also the proposal templates for your specific action in section 5, below.

4.   Indicative time for evaluation and grant agreement:
Notification of outcomes of stage 1 evaluations: maximum 5 months from deadline for submitting proposals.
Notification of outcomes of stage 2 evaluations: maximum 5 months from deadline for submitting full proposals.

Signature of grant agreements: maximum 3 months from the date of informing successful applicants.

5.   Proposal templates, evaluation forms and model grant agreements (MGA), clinical trials template:

IMI2 Research and Innovation Action (IMI2-RIA) and Innovation Action (IMI2-IA):

Proposal templates are available after entering the submission tool.

Standard evaluation form RIA 

IMI2 Model Grant Agreement
Members of consortium are required to conclude a consortium agreement prior to the signature of the grant agreement.

Clinical trial template:
The Clinical Trial template is compulsory at stage 2 only !

IMI2 Coordination and Support Action (IMI2-CSA):

Proposal templates are available after entering the submission tool.

Standard evaluation form CSA

IMI2 Model Grant Agreement
Members of consortium are required to conclude a consortium agreement prior to the signature of the grant agreement.

Clinical trial template:
The Clinical Trial template is compulsory at stage 2 only !

6.   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 H2020 main 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. See the Online Manual.

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.

7. Additional documents:

Summary of the most relevant provisions for participating in IMI2 actions

IMI2 - 2nd Amendment to the Annual Work Plan 2017

IMI2 Regulators Guidance tool for researchers

IMI JU derogation to H2020 Rules for Participation  

Horizon 2020 Rules for Participation 

Horizon 2020 Regulation of Establishment

Horizon 2020 Specific Programme


Members of consortium are required to conclude a consortium agreement prior to the signature of the grant agreement.

Additional documents

  • IMI2 Call 13 stage 1 - Flash Call Info Report en
  • IMI2 Call 13 stage 2 - Flash Call Info Report en

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