c4c – External Training Courses Catalogue

The Translational School contains lectures about the current state of the art of acquired and genetic neuromuscular diseases, the different steps of drug development and the tools needed for this (outcome measures, biomarkers etc). It also stresses how patients should play a role in each of these steps and teaches how to communicate research to patients and the general public during interactive workshop sessions.

A registration fee applies and attendees must pay for and organise their own travel. All meals and accommodation are covered by the organisers.

To educate clinicians and researchers working in the NMD field on aspects relevant for translational therapy development:

• Bench to bedside research
• Regulatory system
• Clinical trials
• Outcome measures
• Patient communication
• Registries and biobanks
• Biomarkers and -omics

Outline and showcase how networks like EURO-NMD and TREATNMD facilitate therapy development:

• Standards of care
• Clinical trial tools
• Outcome measure development
• Interaction with stakeholders

This two-and-a-half day program aims to build research methods knowledge and research capacity by promoting future collaboration and networking between multidisciplinary
healthcare professionals working in paediatric and neonatal acute and critical care. Lectures, workshops, and discussions are led by established professorial faculty with many years of experience in paediatric critical care. In addition to the 2-day educational programme, a half-day Research exchange workshop will be offered to allow clinicians and researchers to share research expertise and discuss the progress of ongoing specific collaborative research projects as well as potential future projects and collaboration in a collegial and multidisciplinary discussion forum. This year nutrition and PICU long-term outcome research will be the focus of this half-day.

The programme provides:

• Opportunities for participants to discuss their projects/ideas in safe and facilitated peer group discussions.
• Keynote plenary lectures, followed by optional choices guided by the participants’ level of existing knowledge and interests. Three tracks are proposed A) Roadmap for conducting clinical research, B) Advanced methods and C) Building Up a research career.
• Various research methodological approaches to answer innovative clinical issues questions.
• Networking opportunities with people who share similar passions and interests
• To build national, European, and international research collaborations in the paediatric & neonatal fields
• To create networks of early career and senior researchers in these fields (PICU and NICU)

This webinar series will cover topics from the basics, providing an introduction to how this new technology can modify diseases, though to a more in-depth look into operational aspects of delivering advanced therapies within the hospital environment. The sessions are delivered as a collaboration between London Advanced Therapies and the ATTC network.

To provide an understanding of advanced therapies and how they are delivered within the hospital environment.

This is a transversal training course for up to 30 participants per year aimed mainly at clinicians, both in training and established, and potentially pharmacists. Patient advocates with a genuine interest and particular aptitude for the regulatory aspects of drug development are also encouraged to attend. The two-year course will include webinars, Q&A online and offline, a case study, as well as CME accreditation.

There are two versions of the training course- an introductory version and a full version. There will be a total of 6 modules spread across the two years:

• The introductory course will include one 1.5hr webinar for each module
• The full course will include four 1.5hr webinars for each module as well as quizzes, Q&A opportunities and Erasmus accreditation

To have an understanding of regulatory science in drug development for childhood cancer.

This webinar will explore why the FAIR data principles are so important for data that is collected during rare and paediatric clinical trials. There will be an introduction to conect4children (c4c). c4c has a number of interesting tasks around the harmonisation of clinical trial data including the development of a paediatric data dictionary, a Paediatric User Guide (developed in collaboration with CDISC) and activities exploring the connectivity between real world data and paediatric clinical trials. At the heart of all of c4c’s data harmonisation efforts are the FAIR data principles, and the challenging task of applying FAIR to data collected at the CRF (or patient) level. This webinar will present the approach c4c is taking to FAIRification, the collaboration with FAIRPlus and how they plan to tackle the harmonisation of disease specific data. The webinar will be interactive with lots of opportunity for questions and discussion.

To provide an understanding of how FAIR data principles can be applied to paediatric clinical trials.

In this training, learners will understand the benefits of adopting CDISC standards. The training will also provide academics with a useful toolkit and helpful information for collecting and organizing research data using CDISC standards. This training also outlines navigating CDISC resources and how to contribute to clinical research standards development.

• Describe the CDISC standards and how they improve the findability, accessibility, interoperability, and reusability of research data in order to recognize their value in academic research.
• Identify individual CDISC standards in order to set the stage for implementation.
• Support the adoption of implementing CDISC standards for data collection and organization in academic research.

This webinar, presented by some of the foremost CDISC experts, will cover the fundamentals to get you up to speed on the what, how, and why of CDISC standards. We will address such topics as:

• The need for global standards in the current industry environment,
• An overview of the CDISC Model
• Regulatory requirements
• Use cases for CDISC Standards.

To provide an introduction to CDISC and the use of data standards.

The training programme consists of several modules, covering the full lifecycle of clinical trial submission, authorisation and supervision.

To prepare and train users in the use of the Clinical Trials Information System (CTIS).

This webinar is designed to introduce the concept of the the FAIR cookbook. The FAIR cookbook is a collection of ‘recipes’ designed to assist both academia and industry to apply FAIR data principles, which could help to build capacity for high quality data management. The FAIR cookbook was developed by FAIR Plus, which is an IMI (Innovative Medicines Initiative) funded project.

1. Learn how to improve FAIRness of data with exemplar datasets
2. Understand the levels and indicators of FAIRness
3. Discover open source technologies, tools and services
4. Find out the required skills
5. Acknowledge the challenges in FAIRification.

This course from the EJP RD offers an exploration of key questions around rare disease diagnostic research and issues today, as well as insights into patient experiences.

You will discover more about the progress made through diagnostic research, as well as the types of genetic tests available for rare diseases, and the impact of having a diagnosis – or lacking a diagnosis – on patients’ lives. You’ll explore key issues relating to rare genetic diseases and undertake your own internet-based investigation into several diagnostic research topics.

On this five-week course from the European Joint Programme on Rare Diseases, students will explore the therapeutic development process from the unique perspective of rare diseases.

Students will follow the journey from discovery to approved treatment, learn how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks.

During this course you will learn:

About each stage of a clinical trial, learning to plan and design a trial and address any regulatory issues.

To examine how to end a clinical trial and how to approach marketing approval and authorisation. Students will also investigate data sharing and reuse post-trial.

By the end of this course, students will understand how, during the process of translational research, you can build evidence that a treatment is safe and effective.

EpiCARE, in collaboration with ILAE Academy, have developed 6 patient-centred interactive e-learning modules, corresponding to diagnosis and management of some rare and complex epilepsies. Some of the modules include paediatric patient cases.

To support the diagnosis and management of some rare and complex epilepsies.

The course aims to provide healthcare professionals and caregivers with the knowledge and skills needed for managing a critically ill child during the first hour of illness, and preventing progression to cardiac arrest. Emphasis is also given to team management and non-technical skills.

• Recognition of a critically ill child
• Knowledge and skills in assessing and treating problems with the airway, breathing and circulation (including bag-mask ventilation)
• Intraosseous infusion Management of shock Scenario-based training in the management of critically ill children
• Basic life support treatment of choking
• Neonatal life support
• Advanced cardiac arrest algorithms Shockable and non-shockable rhythms, defibrillation Scenario-based training in the management of (imminent) cardiac arrest, severe trauma and special circumstances; with emphasis on non-technical skills and team approach Post-resuscitation care and ethics

The 2023 edition of the Open Academy Schools will take place in-person from 19-23 June in Barcelona. Schools are delivered in English in a blended format, comprising e-learning modules, pre-training webinars and face-to-face intensive days.

From 2023, the face-to-face intensive week of the Open Academy School on Medicines Research & Development (formerly EURORDIS Summer School) and the Open Academy School on Scientific Innovation & Translational Research (formerly EURORDIS Winter School) will take place during the same week, incorporating three days of parallel sessions and two days of joint sessions focusing on patient engagement across the whole life-cycle of a medicine. Greater knowledge sharing and networking opportunities will be fostered with both sets of trainees together during the week.

The new format will include off-site visits to research facilities, greater contact time with faculty and EURORDIS staff and the chance to network with fellow patient advocates, supporting each other in learning and practice during the training and beyond.

Participants to the Open Academy School on Medicines research and development for patient advocates and researchers will learn about clinical trials methodology, clinical research, ethics in medicines development, regulatory affairs, health technology assessment and marketing authorisation.

Participants to the Open Academy Scientific Innovation and Translational research for patient advocates will gain an understanding of how pre-clinical research translates into real benefits for rare disease patients.

Take this course to learn more about the three main EU institutions: the European Commission, the European Parliament and the Council of the EU. This course will guide you through this unique system, from its creation to the role of each institution.

To gain a better understanding of the main EU institutions.

The EU CTR (European Clinical Trial Regulation) comes into effect on January 31 2022 and affects everyone involved in clinical drug research in Europe, including paediatric drug trials. The EU CTR Expert course for clinical drug trials in Europe helps you understand all the vital aspects of the EU CTR regulation. You’ll learn all strategic elements, from study design, requirements to the paediatric investigation plan (PIP) to submission, to clinical conduct in our course, making you the EU CTR expert. Includes 4 interactive modules,1 test and takes 7.5 hours to complete.

• Principles of EU CTR (EU Clinical Trial Regulation)Transition period and expected implementation EU CTR
• Contents of submission dossier Part I and II, incl paediatric investigation plan (PIP)
• Preparing and submitting of the research dossier in the CTIS portal
• Role of the submitter, the reviewer,and the investigator
• Critical timelines for submission and review of a dossier
• How to answer questions from the reviewing ethics committee
• Requirements for the conduct of clinical drug research
• Safety reporting requirements
• Submitting substantial amendments via the CTIS
• End of trial reporting, summary of Clinical Trial Results for Laypersons
• Publication of trial data: requirements and transparency

This conference series focuses on global regulatory cooperation with an eye to building on existing models to strengthen the global regulatory ecosystem for paediatric drug approval.

This two-part webinar will take place on 29 November 2022 at 9-11:30am ET and 30 November 2022 at 9-11am ET. You are encouraged to attend both parts of this virtual meeting as part two builds on part one.

To understand how the global regulatory ecosystem can be strengthened for paediatric drug approval.

This online course provides recommendations on the competencies (understood as knowledge, skills and behaviours) and resources that each stakeholder organisation should aspire to have in place in order to plan, implement and evaluate meaningful and sustainable patient engagement activities across the medicines lifecycle.

This course is based on one of the main outcomes of the IMI funded multi-stakeholder consortium PARADIGM (Patients active in research and dialogues for an improved generation of medicines).

The objective of this online course is to increase the preparedness of stakeholder organisations by identifying the capabilities required by those individuals involved in implementing patient engagement activities and the resources (processes, tools and systems, organisational structure) needed within the organisation.

The TMex Winter School consists of introductory e learning (~12 hours student investment time) followed by a 5-day face-to-face workshop. The course is intended for PhD/MD students in the second half of their PhD and postdocs who are involved in biomedical research.

For students to learn more about translational medicine, as well as about job profiles of scientists in industry and other sectors relating to translational medicine.

This webinar explores why FAIR data is valuable by giving presentations from the perspective of different stakeholders including academia, industry, regulators and funders. This webinar was hosted by FAIR Plus, which is an IMI (Innovative Medicines Initiative) funded project.

To be able to understand the value of making data FAIR, from the perspective of different stakeholders.

Fondazione Gianni Benzi is jointly organising with Università degli Studi di Bari Aldo Moro, the XV Foresight Training Course that will run as a hybrid meeting on 15-16 December 2022 in Bari (Italy) and online.
The course aims to highlight the efforts made in Europe to foster research and innovation in a changing scientific, social and economic setting with a focus on: clinical research; innovative research methodologies, including digital and in-silico approaches; the use of Real-World Data. More information on the course on the website here.

The learning objectives are:
1) to investigate the clinical research scenario, as expected from the new Clinical Trials Regulation implementation and the Accelerating Clinical Trials in the EU initiative
2) to learn how innovative methodologies are implemented into all the phases of drugs development, with an insight on the paediatric and rare diseases research
3) to learn the challenges and opportunities of the use of Real-World Data and of a patient-centric model to boost research and innovation