PedCRIN developed a series of tools (WP3) and procedures to support the setup and the management of multinational neonatal and paediatric clinical trials in Europe. These tools are developed to train and support researchers and clinicians so they can establish, run, and manage both paediatric and neonatal multinational clinical trials in a more effective and easier way. The removal of barriers to trial management will speed up the evaluation of new and improved therapies.
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Mapi Research Trust’s PROVIDE services are a unique resource for the scientific community. This full-range support is critical for anyone involved in the field of Clinical Outcomes Assessments (COAs).
PROVIDE, which is available on an ad-hoc basis or within the context of annual subscriptions, includes the following in-house easily accessible services (non-exhaustive list):
Copyright for measures (who owns the measure)
Available translated versions & methodology to translate
Translation Certificates
Providing of scoring manuals
Licensing process / permission to use measures
Find measures in a specific therapeutic indication/on-going clinical trials
Original and review copies of thousands of COAs and translations
List of COA concepts
List of recommended COAs from guidelines
PRO Claims: a study of competitor products and COA Labeling claims
Provide reports that come from PROQOLID / PROLabels
Developer contact (for conditions of use, scoring, translation, etc.)
Bibliographic references
See content validity documentation, availability, psychometric properties, etc.
Provided by EURORDIS - EUROPEAN ORGANISATION FOR RARE DISEASES ASSOCIATION
* Mechanism of Coordinated Access to orphan medicinal products (MoCA): is a platform where companies’ issues around reimbursement or financing schemes can be discussed with a variety of jurisdictions and societal perspectives. It provides a mechanism for European countries to collaborate on coordinated access to orphan medicines in a voluntary, dialogue-based approach, intended to create a fluid set of interactions between key stakeholders, across all aspects of a product.
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*The EURORDIS Open Academy brings together all EURORDIS trainings with the goal of building the capacity of rare disease patient advocates.
Through the EURORDIS Open Academy capacity-building programmes, EURORDIS empowers patient advocates to have the confidence and knowledge needed to bring their expertise to discussions on health care, research and medicines development.
EURORDIS promotes the implementation of services adapted to the needs of people living with a rare disease and provides:
* Specialised Social Services are instrumental to the empowerment of people living with rare diseases and essential to the improvement of their well-being and health.
* RareConnect.org, the online network for rare disease communities, brings together patients, families, and groups from around the world who might otherwise be isolated
* Patient Community Advisory Boards (CABs) are groups established by patient advocates to discuss the latest developments, challenges and issues related to medical research in their disease area.
* Rare disease help lines offer social, psychological and information solutions to people living with a rare disease.
Provided by IFB - FRENCH INSTITUTE OF BIOINFORMATICS
IFB provides IFB users (biologists, biomedical and bioinformaticians) with a training offer that takes into account the wide diversity of needs and expectations:
– diversity of computer skills (no command line experience, occasional use, familiar, program a little)
– specific courses on new technologies or on biological applications (e.g. NGS, long reads, metabolomics…)
– generic methodological skills (statistics, good programming practices, etc.)
– advanced use of IT technologies (HPC, virtualisation, cloud computing, interoperability, FAIR practices, integrative bioinformatics…)
– good training practices (pedagogical approaches, reuse of materials, etc.)
The IFB’s services have two main objectives: the deployment of a high-performance bioinformatics service optimized for the needs of the life sciences communities (university laboratories and industries) and the development of innovative bioinformatics resources to address strategic challenges in health, environment and agriculture. As such, IFB provides support, account opening, software and database installation, website and virtual machine hosting, software/database/ information system development and bio-data analysis
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We have developed at I-Stem a fully automated bioproduction platform for research applications (non-GMP) based on the use of CompacT SelecT technology for the amplification and differentiation of hPSCs under feeder-free adherent culture conditions. Process performance and variability are monitored throughout scale out to prevent deviations from specification limits. Cell suspensions are automatically dispensed into cryovials (Fill-it) and frozen using a controlled-rate freezing equipment chamber (Cryomed). Importantly, all cell banks undergo appropriate quality testing according to international guidelines – such as phenotypic characterization, genomic stability and microbiological testing – to ensure they meet release criteria.
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I-Stem has a platform for high-throughput screening of libraries of chemical molecules on embryonic stem cells or iPS with genetic defects.
The laboratory has a high-performance robotic platform with miniaturization and standardization of cellular tests as well as the performance of large-scale screening campaigns (10 000 to 40 000 tests per screening campaign) under sterile and reproducible conditions.
Orphadata provides the scientific community with comprehensive, massive, re-usable and computable quality data sets related to rare diseases from the Orphanet knowledge base, available through 4 distinct services:
* Files: Provided files are a partial extraction of the Orphanet knowledge base.
* APIs
* Customizable services
* Fair Datapoint
