The virtual workshop will take place over two days on May 16th – 17th.

Academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology companies play a critical role in rare disease drug development and are often the sponsors for rare disease drug development, but they may lack regulatory experience. This workshop focuses on academic investigators and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of drug development.

This workshop will:

• Provide lessons on the fundamentals of drug development and discuss rare disease case studies shared by academic investigators
• Share experiences and expertise from multi-disciplinary stakeholders
• Discuss regulatory considerations for rare disease clinical trials
• Explore topics such as:
  • Adequate and well-controlled trials
  • Core principles for clinical trials
  • The nuts and bolts of Investigational New Drug (IND) applications and additional considerations
  • Additional pathways to interact with the FDA
  • Case studies

Registration is mandatory to attend the workshop.

More information and registration here: https://events-support.com/events/CDER_NCATS_Regulatory_Rare_Disease_Workshop

The toolbox aims to collect the accumulated knowledge, experience, and resources (collectively termed as ‘tools’) generated by previous projects and/or research infrastructures and other organizations into a practical and guided toolbox to help clinical trialists and R&D managers understand the regulations and requirements for conducting trials, with special focus on investigator-initiated trials for rare diseases and applicable in Europe.

It outlines major considerations for all phases of a clinical trial on medicinal products for human use. It is divided into five sections (research questions, plan, execute, analyse, and end of trial), each of which is further broken down into specific subsections representing the individual steps to follow. The subsections each observe a similar design with a structured explanatory description (What, Why, How, Special Considerations for rare diseases population, Common Pitfalls, and Resources) after which are listed all relevant identified tools.

Check out the Rare Diseases Clinical Trial Toolbox today: https://ecrin.org/tools/rare-diseases-CT-toolbox

CTIS is the backbone of the Clinical Trials Regulation that will harmonise the assessment and supervision of clinical trials in the European Union. The Clinical Trials Regulation and CTIS will strengthen clinical trials in the EU, ensuring better outcomes for patients and supporting the attractiveness of the European Union as a location for clinical research.

Sponsors can visit the public Clinical Trials website at https://euclinicaltrials.eu/home. The “CTIS for sponsors” page provides key information and links for sponsor users.

Patients, healthcare professionals and the general public can also access the public Clinical Trials website. When clinical trial applications have been submitted through CTIS and authorised by Member States,they will be openly accessible via the “Search clinical trials page”. The public searchable database will gradually contain more information as sponsors and Member States use it to initiate and oversee clinical trials.

The webinar will take place on November 29, 2021 from 09.00 – 13.15 CET.

The webinar is being organised as a training event and will present:

• an overview of the Clinical Trials Regulation
• an introduction to the new process for submitting clinical trial information in the European Union/European Economic Area
• functionalities of CTIS, including transparency aspects and safety reporting requirements
• guidance and training material available for sponsors

Attendance at the webinar is by registration only and open to companies that have been assigned SME status by EMA, academia and representatives of stakeholder organisations. There is no registration fee for this event.

More information here: https://www.ema.europa.eu/en/events/webinar-small-medium-sized-enterprises-smes-academia-clinical-trials-regulation-clinical-trials#registration-by-invitation-only-section

The online event will take place over two days: October 5th from 14.00 – 19.00 CET and October 6th from 14.00 – 18.30 CET.

The hands-on workshop format features use of case examples and interactive sessions to facilitate co-creation of solutions and active contribution by participants, and it is targeted towards the following:

• Industry and academic clinical trial researchers (e.g., clinicians, statisticians, and regulatory scientists)
• Representatives from patient advocacy groups
• Members of Ethics Committees and National Competent Authorities
• Representatives from regulatory agencies
• Members of non-profit organisations
• Members of industry/academic associations

More information and registration here: https://www.efpia.eu/news-events/events/efpia-event/accelerating-adoption-of-complex-clinical-trials-in-europe-and-beyond/

The webinar will take place on June 16, 2021 from 09.30 – 13.00 CET.

During the 3.5-hour interactive session focused on the challenges of paediatric multinational clinical trials, several experts from PedCRIN will present the main outcomes of the project and will discuss the supporting tools, methods, identified gaps and future initiatives and collaborations.

More information and registration here: https://ecrin.org/projects/pedcrin/paediatric%20final%20event