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

The call is currently open but will close on July 15th at 17:00 CET.

Applicants who are selected can avail the following benefits:

• Facilitation of communication with patients, their carers and their families
• Support for patients and carers throughout the research process
• Data storage with secure environment and GDPR-compliant protocols and procedures
• Support for ethical submission and approval
• Ensuring that validated users will be able to complete questionnaires
• Data analysis not only for your study, but also for the users who contribute with their data, so they can visualise where they stand compared to their peers in an anonymous and aggregated way
• Provision of a data science report at the end of research study with all the results

More information and application process here: https://www.share4rare.org/news/share4rare-launches-1st-open-call-patient-driven-research-projects

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 in-person event will take place over two days on 30th September and 1st October 2021 at the Hotel Golden Tulip Leiden Centre, Schipholweg 3, 2316 XB Leiden, the Netherlands.

The workshop is open by prior registration and selection to medical specialists affiliated to an ERN Full Member or Affiliated Partner institution and who have a special interest in clinical research with databases. Registration closes on 18th June 2021, and those selected to participate from among the applicants will be informed by 9th July 2021 of their selection.

More information and registration here: https://www.ejprarediseases.org/event/clinical-research-with-databases-the-basics-beyond-workshop-in-clinical-epidemiological-research-for-erns/