Directory of professionals and expert centers

Provided by ORPHANET

ORPHANET

Orphanet provides information on professionals whose activity(ies) in the field of rare diseases is (are) registered in Orphanet. This includes, for example, consultant and physicians in charge of an expert centre, biologists in a laboratory, researchers, representatives of patient organisations, coordinators of networks, principal investigators of clinical trials, managers or contact person of registries and biobanks, and experts writing or peer-reviewing data for Orphanet.

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Drug development accelerator for rare diseases

Provided by ORPHANDEV

ORPHANDEV

ORPHANDEV provides its scientific and regulatory expertise in Orphan Designation and Protocol Assistance Applications; its logistical and methodological support for clinical trials in rare diseases with a specificity in the patient’s recruitment strategy; and its experience in national and European calls for projects. This set of services and tools allows the acceleration of the development of therapies for rare diseases.

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Mouse models

Provided by PHENOMIN

Phenomin provides access to more than 50 mouse rare disease models. These models are useful for decrypting the mechanisms behind of the specific disease and form a platform for testing therapies

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High Throughput Screening, chemical and target libraries

Provided by PCBIS - PLATFORM OF INTEGRATIVE CHEMICAL BIOLOGY OF STRASBOURG

The Platform of Integrative Chemical Biology of Strasbourg (PCBIS) gives access to High Throughput Screening (HTS), chemical libraries and target libraries to academic and private laboratories. The platform evaluates the ADME properties of active molecules: Ciblothèque

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RaDiCo supports the implementation of national programs in any country that would need it, by sharing its expertise in database management and implementation of national cohorts on rare diseases. Beyond its own projects, RaDiCo promotes the commitment of French actors to European and international projects needing the implementation of borderless cohorts. We advise and support you for the setting up of propositions and for the management of your programs in this field..).

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Paediatric clinical trial management

Provided by PedCRIN

PEDCRIN

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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Clinical Outcome Assessments support services

Provided by Mapi Trust

MAPI trust

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.

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Mechanism of Coordinated Access to orphan medicinal products (MoCA)

Provided by EURORDIS - EUROPEAN ORGANISATION FOR RARE DISEASES ASSOCIATION

Eurordis

* 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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Training on translational medicine for patient advocates

Provided by EURORDIS - EUROPEAN ORGANISATION FOR RARE DISEASES ASSOCIATION

Eurordis

*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.

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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.

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