Training and capacity buildling of the rare disease research and care community

Provided by European Joint Programme on Rare Diseases (EJP RD)

EJPRD

The EJP RD Training and Empowerment program aims to develop knowledge and build capacity of the rare disease (RD) research and care community through the delivery of training programs on a wide range of relevant topics thereby providing opportunities to a large panel of stakeholders, including:
* DATA MANAGEMENT & QUALITY TRAININGS
The main objectives of the training activities on data management & quality are to decrease rare disease data fragmentation and increase data quality by raising the level of capacities and help data sharing in a FAIR ecosystem. The training activities cover a variety of topics including but not limited to variant interpretation, data management, registries, FAIRfication processes, Orphacodes, biobanking, and undiagnosed cases.
*PATIENTS & RESEARCHERS TRAININGS
The overall objective of this training module is to improve rare disease research and innovation and to enhance the uptake of research results by building the capacity of the patient community and other key stakeholders. Moreover, dedicated educational activities will be aimed to train “expert” paediatric patients on rare diseases, paediatric medicines development and clinical research.

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Pharmacoepidemiology and pharmacovigilance Resource Database

Provided by European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCepp)

ENCePP

The Resource Database is an electronic index of available EU research organisations, networks and data sources, including patient registries, in the fields of pharmacoepidemiology and pharmacovigilance, and is a key component of the ENCePP web portal. All information contained in this database is provided and maintained by the listed organisations, data providers or registry holders.
The Resource Database includes information on expertise and research experience across Europe and serves as a hub for both researchers and study sponsors seeking to identify organisations and data sets for conducting specific pharmacoepidemiology and pharmacovigilance studies in Europe.
The database is fully searchable and allows the identification of research centres, data sources and patient registries by country, type and other relevant criteria.

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Joint Clinical Assessment (JCA) and Scientific Consultations (JSC)

Provided by European Network for Health Technology Assessment (EUNetHTA)

EUNETHTA

EUnetHTA develops several services that help inform the decision-making process around the introduction of pharmaceuticals and other technologies at the national, regional and European levels. These include Joint assessments, early diologues and Post Launch Evidence Generation (PLEG)
* Assessment submission: Health technology companies are encouraged to contact us via the following two channels if interested in a EUnetHTA assessment of their product. These are distinguished by EUnetHTA as Pharmaceuticals and Other Technologies (i.e. non-pharmaceuticals).
*Early dialogue: parallel scientific advice with the European Medicines Agency. This aims to allow medicine developers to obtain feedback from regulators and health technology assessment (HTA) bodies on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.
* Horizon Scanning: topic identificatin, selection and Prioritisation for HTA cooperation

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Guide on patient partnerships in rare diseases research projects

Provided by European Joint Programme on Rare Diseases (EJP RD)

EJPRD

The EJP RD has developed a short guide on patient partnerships in rare diseases research projects. It aims to encourage fruitful, sustainable and enduring partnerships between scientists and patient organisations, co-leading the way for systematic patient-centered research.

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EMA’s Innovation Task Force

Provided by European Medicines Agency

EMA

EMA provides regulatory and scientific support to foster development of new and innovative medicines, from the early phases in the laboratory all the way to the patient.

EMA achieves it through the following resources:

Innovation Task Force (https://www.ema.europa.eu/en/human-regulatory/research-development/innovation-medicines#ema’s-innovation-task-force-(itf)-section)
EU Innovation Network (https://www.ema.europa.eu/en/human-regulatory/research-development/innovation-medicines#eu-innovation-network-section) /
SME Office (https://www.ema.europa.eu/en/human-regulatory/overview/supporting-smes)
PRIME scheme (https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines)
Qualification advice on novel methodologies (https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance/novel-methodologies-biomarkers/opinions-letters-support-qualification-novel-methodologies-medicine-development)
Scientific advice / protocol assistance (https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-advice-protocol-assistance)
Post-authorisation (https://www.ema.europa.eu/en/veterinary-regulatory/post-authorisation)
Pilot for Simultaneous National Scientific Advice (SNSA): : in one single step national scientific and/or regulatory advice can be requested with two NCAs simultaneously. The focus of the SNSA is on innovative developments to identify the needs of the applicants to enhance innovation and avoid gaps in early regulatory support. The pilot aims at exploring the opportunities and interest in providing such coordinated national scientific advice particularly to developers of new medicines and therapies (https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/guidance-applicants-pilot-simultaneous-national-scientific-advice-snsa_en.pdf)

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Innovation offices in national regulatory agencies

Provided by EU Innovation Offices Network

HMA INNOVATION NETWORK

National scientific advice for developers of medicinal products or medical devices and other technologies to optimize prospectively their development programme. • Two in one approach = get two NCA opinions within one application

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Comprehensive Inventory of available regulatory support

Provided by CSA STARS

CSA STARS

a Comprehensive Inventory (CI) has been developed to assist European academic drug developers in finding support on regulatory affairs. The inventory lists various support services provided by national competent authorities, public actors and private entities by:
* Expertise area (ATMP, Biologicals, biomarkers, chemicals, diagnostics, drug repurposing, drug-device combinations, e/m health, ethical aspects, IVD/ Blood products, legal matters, medical devices, paediatric studies, statistics, societal aspects)
* Support activity (assistance in clinical trial application, pre-grant advice, GCP, GLP, GMP, non clinical, pre-clinical, paediatrics, Phase I, PHase II-III, Phase IV, pre-grant advice, quality)

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Strategic Feasibility Advice Service

Provided by conect4children

Conect4Children

4c aims to generate a sustainable infrastructure that optimizes the delivery of clinical trials in children through:
a) a single point of contact for all sponsors, sites and investigators;
b) efficient implementation of trials, adopting consistent approaches, aligned quality standards and coordination of sites at national and international level;
c) collaboration with specialist and national networks;
d) high quality input into study design and preparation, through rigorous strategic and operational feasibility assessment;
e) the promotion of innovative trial design and quantitative scientific methods;
f) an education and training platform to shape the future leaders of paediatric drug development;
g) the development of sustainable support for all these activities.

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Brokerage service Connection application

Provided by SOLVE-RD

Rare Diseases Models & Mechanisms – Europe (RDMM-Europe): This brokerage service will catalyse connections between people discovering new genes in rare disease patients and basic scientists who can study those genes and pathways in model organisms. Solve-RD brokerage service are to:
*provide functional validation of human genetic variants that cause disease;
*supplement clinical disease gene discoveries by generating functional data so as to get higher impact publications;
*develop insights into potential rationale for treatment (e.g. identification of candidate drug targets) via knowledge of a disease gene pathway; and
*establish longer term collaborations between basic scientists and clinicians that will lead to subsequent grant funding in support of outstanding basic and/or applied research

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Clinical Patient Management System for ERNs

Provided by SOLVE-RD

Clinical Patient Management System (CPMS) is a secure IT-Platform used by clinicians across Europe to discuss patient cases without the patients having to travel. The ERN Experts (from one or more ERNs) gather in multidisciplinary panels and use CPMS to carry out e-consultations of rare and complex cases. Patient medical documents can be safely uploaded to the CPMS after a consent form has been signed by the patient.
Integrated tools in CPMS allow peer-to-peer communication, viewing of digital documents and recording expert contributions. This promotes an active collaboration, sharing knowledge and experience within and across ERNs. The health professionals are ultimately aiming an outcome either as a diagnosis, treatment, clinical trial or other.

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