PARADIGM, a collaborative IMI project that aims to make meaningful patient engagement in medicines development a reality. The objective is to develop processes and tools for three key decision-making points: research priority setting, design of clinical trials and early dialogue with regulators and HTA bodies. Share your opinion and experience on patient engagement NOW! Learn more HERE

 

The CORBEL Open Call invites researchers to apply to access technologies and services from more than 20 facilities from 10 different research infrastructures across Europe.
Learn more HERE

 

The International Rare Diseases Research Consortium (IRDiRC) is proud to announce the new vision and goals for 2017-2027. See the publication HERE.

E-Rare Newsletter December 2015

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ERA-Net for Research Programmes on Rare Diseases
E-Rare Newsletter December 2015

The eighth E-Rare joint call for funding multilateral research projects on rare diseases (JTC2016) on ‘Clinical research for new therapeutic uses of already existing molecules (repurposing) in rare diseases’ was launched on the 7th of December, 2015. The following 15 countries participate in this call: Austria, Belgium (Flanders), Canada (including Quebec), France, Germany, Hungary, Israel, Italy, Latvia, Poland, Portugal, Romania, Spain, Switzerland and Turkey, with a total budget of 17M€.

The specific objective of this call is to promote the clinical and pre-clinical proof of concept for the potential application of medicinal products in rare indications either already marketed or having achieved a significant stage in the development process. Two types of projects will be eligible for this call:

  • Type A: Preclinical studies to verify target engagement and to perform additional toxicity testing if necessary (for example in the case of paediatric indications where juvenile animal studies might be warranted) in a disease model for a maximum period of one year followed by the implementation of Phase 1b or Phase 2a clinical trials at the beginning of the second year of the project.
  • Type B: Milestone-driven Phase 2 clinical trials to demonstrate that the Agent modulates the target and has the potential to yield the desired clinical outcome in the proposed disease population for a period up to three years.

Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10.000 persons in the European Union,  Associated States and Canada.

The use of existing European health research infrastructures or initiatives is strongly encouraged when appropriate. The following European Research Infrastructures or Initiatives were identified as potentially useful for this kind of study: ECRIN; EATRIS; BBMRI; ELIXIR; EU-OPENSCREEN; INFRAFRONTIER; RD-Connect and European Medicines Agency.
More information on European Research Infrastructures/Initiatives, their services and costs can be found on www.erare.eu/infrastructures/

EXCLUDED TOPICS ARE: (1) Development of new models of diseases; (2) Discovery and early development of new compounds; (3) Feasibility studies of devices; (4) Preliminary studies of radiation, surgical, behavioural or rehabilitation therapies; (5) Studies on advanced therapies (gene therapy, cell therapy); (6) Clinical studies concerning rare infectious diseases, rare cancers and rare adverse drug events or secondary outcomes in treatments of common diseases; (7) Compassionate use.

The deadline for proposal pre-registration is February 1, 2016.
The deadline for proposal submission is March 3, 2016.

Visit our website www.e-rare.eu

ECRIN, Collaborating with European Countries in E-Rare Clinical Trial Projects
A Partner for Multinational Trial Management
Managing a multinational clinical trial raises multiple challenges for investigators and sponsors. ECRIN, a non-profit research infrastructure, helps organisations to facilitate trials by coordinating various management tasks (regulatory and ethical submissions, adverse event reporting, monitoring, data management, etc). Currently a partner in ongoing trials funded by the 7th Framework Programme for Research and Technological Development (FP7) and Horizon 2020 (H2020), ECRIN is available to support groups in all European countries in clinical trials for treatment repurposing supported by the E-Rare 3 Call for Proposals 2016 (JTC 2016).
For more information on how ECRIN can support E-Rare trial projects, please contact the European Correspondent in your country (see contact list).


ECRIN-On-Board: Supporting E-Rare Funding Applications for ECRIN Member Countries
In addition, ECRIN is offering support for applications to the E-Rare 2016 call to principal investigators from its Member Countries (France, Germany, Hungary, Italy, Portugal, and Spain) through its “ECRIN-On-Board” programme. Support areas include:
1 – Independent methodological recommendations on protocol development
2 – Consulting on trial logistics and management (regulatory and ethical requirements, monitoring strategies, insurance, contracting, costs, investigation sites, etc.)
3 – Advice on E-Rare applications (consortium organisation, work packages, budget, project management)
ECRIN-On-Board support is provided for free on a first-come, first-serve basis. Interested parties should send a summary description of the trial protocol based on pages 6-9 of the Full Proposal Application form (available on the E-Rare website) to the European Correspondent in their respective country as well as to the ECRIN-On-Board focal point (see contacts above) by January 5th, 2016. Applications will be dealt with under the strictest confidentiality. Proposal applications will be reviewed by ECRIN-On-Board and feedback will be provided within three weeks (by end January), giving principal investigators another month to modify their projects as needed before submission to E-Rare (deadline: March 3rd, 2016).

EATRIS Services for E-RARE 3 call
EATRIS ERIC, the European Infrastructure for Translational Medicine, specialises in advancing medicines safely into clinical development. With over 75 leading academic translational research institutions across Europe, EATRIS can find your partner with specialised facilities and long-standing expertise in an array of disciplines, including:

  • In vivo imaging-enabled drug development, supporting pre-clinical and clinical assessment of target expression, modulation and internalisation, drug pharmacokinetics and biodistribution, normal organ uptake, optimisation of drug dose & scheduling, and assessment of inter-patient variation;
  • Drug (re-) formulation expertise, access to an array of drug delivery technologies;
  • Access to a wide array of specialised pre-clinical models, as well as clinical expertise and patient cohorts for many rare diseases.

Please email Florence Bietrix for more information [florencebietrix[at]eatris.eu] or check EATRIS website

Why and How to work with European health research infrastructures or initiatives?
The use of existing European health research infrastructures or initiatives is strongly encouraged when appropriate in the E-Rare-3 JTC 2016. In order to help you to find the right initiatives to work with, the E-Rare website has now a new webpage on describing their services and providing contact points. You can also tell us what you are looking for and we will help you to find the best service to realise your project!
Go now on erare.eu/infrastructures

In December 2014 E-rare launched the seventh E-Rare joint call (JTC 2015) for funding multilateral research projects on rare diseases together with the European Commission (EC) under the ERA-Net E-Rare co-fund mechanism. Seventeen countries joined this call: Austria, Belgium, Canada (including Québec), France, Germany, Greece, Hungary, Israel, Italy, Latvia, Poland, Portugal, Romania, Spain, Switzerland, The Netherlands and Turkey.

The aim of the call was to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach.

The process included a two-step submission and evaluation procedure.

In the first step a total of 231 eligible pre-proposals were submitted. After careful examination by the Scientific Evaluation Committee (SEC), 55 pre-proposals were selected for full submission. Each of the full proposals was then evaluated by at least two additional external experts whose reviews were sent to project coordinators in order to give them the opportunity of studying the assessments and commenting on experts' arguments and evaluations. Both inputs were taken into account in a second SEC meeting.

Following the second SEC evaluation and ranking of the best projects, 19 consortia with a foreseen budget of about 19.35 Mio € were selected for funding.

More information about the selected projects and involved research teams can be found HERE.

www.e-rare.eu contact@erare.eu

E-Rare 2012 - Created by Toussaint Biger