EJP RD Advisory Committee for Therapeutics (ACT) workshop (ERN-focused event)

  • 7 juillet 2021 - 14:00
  • Online

Organiser – Newcastle University 
7th-8th July 2021 

The main objective of this workshop is to demonstrate the benefits of adopting the Advisory Committee for Therapeutics (ACT) model in rare disease communities, orientated around the European Reference Network groupings (and subdomains) and to communicate best practices in this respect from the neuromuscular field. Participants will be given dedicated space and time to discuss and explore the feasibility of creating an ACT within their own disease domain and/or between disease domains.   

Context for this workshop 

The neuromuscular trial-readiness network TREAT-NMD established the TREAT-NMD Advisory Committee for Therapeutics (TACT) in 2009. The TACT model centres on a multi-disciplinary panel of experts who provide independent and objective advice on the development pathway of therapeutic programs in neuromuscular diseases. This is a much-needed resource, as many manuscripts on potential therapies for neuromuscular diseases have been published, but very few of these therapies have moved forward into successful clinical trials. TACT provides a unique resource and educational tool to the neuromuscular community that helps to bridge the gap between promising preclinical data and successful clinical trials. 

The TACT model was developed originally for the neuromuscular field and has proven to be a key resource in the quest to optimise the planning and execution of early stage and later phase clinical research in this community. However, the model itself is readily transferable to many other rare disease areas, and this is one of the goals of the EJP RD (namely, taking assets proven to add-value in one field or community and deploy to others). Newcastle University has developed the Advisory Committee for Therapeutics (ACT) toolkit through the EJP RD, in compliance with the EJP RD mission of expanding good practices and innovative assets to other rare disease communities through Pillar 4 activities. The aim of the toolkit is to provide other rare disease networks with procedural advice and template documents to set-up their own ACT. The toolkit will be made available via the Innovation Management Toolbox, a virtual library of self-help resources, which will be made available to all on the EJP RD website. This workshop has been designed to give attendees the opportunity to learn more about the ACT model and the lessons learned from its deployment over the past decade in the neuromuscular field, and will provide the opportunity to explore adopting the ACT model within additional rare disease communities, organised along the lines of the ERNs.  The participants will discuss the added value of such a model in supporting ERNs to achieve their research priorities and will consider opportunities for cross-ERN collaboration.   

The aims of the workshop are: 

  • For participants to gain an in-depth understanding of the ACT model 
  • Provide a forum for participants to engage with experienced TACT reviewers from the neuromuscular fieldto bring the model to life and allow participants to gain an insight into the added value an ACT could bring to their field 
  • Encourage participants to scope-out opportunities and challenges in adopting an ACT within their network’s disease area, and explore the practical steps they would need to take to do so 
  • Encourage participants to consider and scope-out the possibility of collaborating across and between disease domains   

Expected Outputs: 

This workshop aims to provide participants with an in-depth understanding of the ACT model. Participants will be given the opportunity to explore the feasibility of developing an ACT within their ERN domain and/or between ERNs. By the end of this workshop, participants will have scoped-out the following: 

  • Disease areas to prioritise  
  • Potential for cross-collaboration between ACTs, where experts’ skills are transversal   
  • Funding opportunities to pilot an ACT within the EJP RD funding period  
  • Related initiatives ACT could engage with – how does an ACT compliment the wider field of EJP RD mentoring, early dialogue support, etc 

The main participants of this workshop will be a) ERN researchers, based in ERN HCPs; and b) ePAG advocate researchers. A small number of additional invited experts will complete the group. In total, the workshop is expected to unite approximately 30-35 experts.    

For ERN Researchers based in HCPs: Each ERN will be invited to nominate ONE representative to participate in the workshop. This opportunity is being advertised to ERN Coordinators, who should agree who their representative will be (or participate themselves directly). If you are interested in applying, please discuss with your Coordinator in advance.  

NB The participants should ideally be able to contemplate the likelihood of supporting and establishing an ACT in their community; therefore, a reasonable degree of seniority and strategic oversight would be beneficialOnce you have agreement with your ERN Coordinator that you are the nominated representative, please complete the application form below, which will ask you to answer the following two questions 

  • What expertise and insight will you personally bring to this event? (max. 150words)
  • What benefit do you foresee for your ERN from joining the workshop?(max. 150 words) 

For ePAG researchers: A total of 6 places will be available at this workshop for ePAG advocates interested in research. This opportunity is being disseminated by EURORDIS to the ePAG research community. The successful participants should be ready and willing to consider the strengths of the ACT model being presented at this workshop (for instance the extent to which you feel the approach is patient-centred), and share views on how this could add value to the range of initiatives and opportunities which currently exist, in order to streamline and improve rare disease research. Please complete the application form below, which invites you to respond to three questions:   

  • What expertise and insight will you personally bring to this event? (you should aim for, but not exceed, approximately 250 words) 
  • How will the topic of this workshop benefit your ERN? (you should aim for, but not exceed, approximately 250 words.) 
  • What do you feel are the main reasons why clinical trials in rare diseases are unsuccessful, and what actions do you feel are most critical to optimize success? (your answer should be approximately 400 words)  

Due to COVID-19 restrictionsthis workshop will take place online via Zoom or a similar platform. We will utilise break-out rooms in Zoom on day two to allow participants to discuss the feasibility of creating an ACT. We will engage a facilitator to help with the technical aspects of the platform.    

In advance of the meeting, we plan to provide all participants with preparatory materials (including a mock application – of approx. 23 pages- to read), to enable a basic understanding of the aims and objectives of the workshop. This preparatory activity will also include a very short survey (no more than 5 questions) to help us tailor the sessions to the needs of our audience as a whole, plus any specific challenges for individual ERNs. 

Registration are open here until May 10th, 2021. 

The training course is free of charge.  

For ERN researchers: affiliation to an ERN Full HCP or Affiliated Partner institution* 

For ePAG advocate researchers: must be officially an ePAG advocate  

* If you are not the Coordinator of your ERN, you must seek approval from the Coordinator to attend this workshop as the sole representative of your ERN. This information will be verified with the respective Coordinator. 

The organizer together with one member of the WP17 Scientific Evaluation Committee will select the most appropriate workshop participants from amongst the ePAG advocate researcher applicants and the ERN HCP-based researchers (though note that as abovethose applying under the latter category need to secure their Coordinator’s approval in advance, to ensure each ERN is guaranteed a place)All registered participants will be informed if they are selected to attend this training course before Monday 31st May 2021 

At the end of the training workshop a Certificate Of Attendance will be sent to the participants who attended the entire workshop. 
No credits of Continuing Education in Medicine will be issued. 

Workshops will run in English. 


If you have questions, please write to the workshop organizer:
Joanne Lee (joanne.lee@newcastle.ac.uk)
Newcastle University

For general inquiries about the workshops in the ERN RD Support and Training Program please write to:

Download the program HERE.


  • Start Date:7 juillet 2021
  • Start Time:14:00
  • End Date:8 juillet 2021
  • End Time:15:00
  • Location:Online