Newsletter - May 2018
 
 
Translational Summer School Special 2018!!
 
SSLogoPost
 

Our translational summer school is fast approaching and we have a few places still available for those who are interested in attending. This month's newsletter features a comprehensive overview of the programme, highlighting the key stakeholders attendees as well as the objectives of each session.

The course, which is run in association with TREAT-NMD, costs €500 will be held 2-6 July in Newcastle-upon-Tyne, UK.

Attendees will benefit from a comprehensive grounding in the translational research pathway from bench to bedside. The final programme is highlighted below in this special newsletter.

Although this course focuses on translational research in the context of neuromuscular diseases, the processes, principles and concepts that underpin this summer school are applicable to many different disease areas. We would warmly welcome attendees from these different disease areas as their experiences, understanding and input would only go further to enhance the learning environment for all concerned and we would encourage them to apply.

 
 
 
 
 
 
 
 
 
 

"Our comprehensive course will be especially beneficial to those people working in clinical trials."

 
 
 
 
pathway
 

Session 1 - The Translational Lifecycle

 
 
 
 
 

Overview of bench to
bedside

Annemieke Aartsma-Rus
Leiden University Medical Center
Netherlands

Objective: to outline the different steps of therapy development from idea, to proof of concept studies in model systems, to preclinical optimization studies, clinical trials, drug approval and post marketing surveillance studies

Tools of the trade for preclinical Research

Volker Straub &
Annemieke Aartsma-Rus
Institute of Genetic Medicine
Newcastle University, UK & Leiden University Medical Center, Netherlands

Objective: outline different models in preclinical research, their opportunities and limitations, and the need for standardized tests and the TREAT-NMD advisory committee for therapeutics (TACT)

 
 

How the regulatory systems works

Violeta Stoyanova-Beninska
Medicines Evaluation Board
Netherlands

Objective: explain the centralized system, how it is organized and how regulators decide whether drugs are eligible for marketing authorization, outline patient involvement in committees – focus on rare diseases






Mock TACT review
session

Annemieke Aartsma-Rus, Volker Straub, & Cathy Turner
Leiden University Medical Center, Netherlands & Institute of Genetic Medicine, Newcastle University, UK

Objective:
learning to have a critical look at preclinical research. In this mock session participants will have been provided with a fictitious TACT application from a company planning a clinical trial in the NMD space. Participants will be split into groups of 8-10 participants to discuss the strengths and limitations and outstanding questions of the application.

 
 
 
 
om20018
 

Session 2 - Clinical Trials

 
 
 
 
 

Introduction to clinical
trials

Michela Guglieri
Institute of Genetic Medicine
Newcastle University, UK


Objective: introduce how and why clinical trials are conducted, the objective of different trial phases, trial design, primary endpoints, secondary endpoints, clinical significance, ethical concerns and informed consent

Ethical discussion &
role play

Silvere van der Maarel, Becca Leary
& Teresinha Evangelista
Leiden University Medical Center, Netherlands & Institute of Genetic Medicine, Newcastle University, UK

Participants will be provided with a scenario for a clinical trial plan for a drug to be tested in children. Different roles will be given to different participants.
Objective: gain insight in ethical discussions related to clinical trials and the perspectives of different stakeholders

 
 

Outcome
measures

Anna Mayhew
Institute of Genetic Medicine
Newcastle University, UK

Objective: overview of the different types of functional outcome measures used in clinical trials for neuromuscular, their opportunities and limitations, need for standardization and training, TREAT-NMD efforts in their area, natural history studies and their usefulness in trial design

Showcase on outcome measure development & labelling & marketing

Annemieke Aartsma-Rus
Leiden University Medical Center, Netherlands

Objective: to illustrate the consequences of using outcome measures limited to certain disease stages with regards to extrapolation and limited indications

 
 

Showcase on outcome
measure development

Anna Mayhew
Institute of Genetic Medicine
Newcastle University, UK

Objective: to outline the steps and stakeholders involved in developing and validating functional outcome measures that assess what patient find important using upper limb function outcome measure development for DMD and SMA as showcases

TREAT-NMD tools to facilitate clinical trials

Michela Guglieri & Becca Leary
Institute of Genetic Medicine
Newcastle University, UK


Objective:
to gain insight in available tools and services for planning and conducting clinical trials

 
 

Biomarkers

Pietro Spitali & Andreas Roos
Leden University Medical Center, Netherlands & Institute of Genetic Medicine, Newcastle University, UK

Objective: to explain why types of biomarkers exist and how they can be used in trial planning and as outcome measures, the regulatory perspective on biomarkers, highlighting ongoing networking efforts (ENMC workshops)

Showcase: validation of MRI as a biomarker in clinical trials

Volker Straub
Institute of Genetic Medicine,
Newcastle University, UK


Objective:
introduce MRI as an outcome measure in trials, ongoing efforts to validate this biomarker for assessment of muscle quality in neuromuscular diseases

 
 

Showcase:
PROM development

Nathalie Goemans
University Hospitals Leuven, Belgium
DVC St Jozef Antwerp, Belgium


Objective: explain what happens after a drug is approved, marketing, postmarketing studies, reimbursement and challenges around drug access

After EMA approval: lessons learned, challenges

Ria Broekgaarden
VSN; Dutch Patient Society of Neuromuscular Diseases

Interactive workshop with Ria Broekgaarden (VSN Netherlands, PAB member EURO-NMD) to discuss challenges around drug access, start stop criteria and drug migration

 
arrow
 

Role of patient organisations in research: examples, achievements from start up to registries

Ria Broekgaarden
VSN; Dutch Patient Society of Neuromuscular Diseases

Objective: to learn how patients are equal partners in drug development and can be instrumental in the processes involved in drug development

Clinical trial practicality forum

Teresinha Evangelista
Institute of Genetic Medicine,
Newcastle University, UK

Participants: trial nurse, clinician involved in clinical trials, patients involved in clinical trial, patient representatives

In this panel session participants will briefly introduce themselves and their personal experience with being involved in a clinical trial as a health care professional or a patient.

What is it like? What is challenging? What is burdensome? Where things going as expected? This is followed by an interactive panel discussion where participants can ask questions and contribute to the discussion

 
 
 
 
debate
 

Patient communication workshop:
Importance of patient communication

 
 
 
 
 

Communication workshop by behavioural scientists

Objective: During this workshop participants will learn about unconscious processes, association and framing, which influence how what is communicated is received by the recipient.
 
 

Participant presentations

 
 

Communication workshop by behavioural scientists

We ask each group of 3-4 participants to prepare a 10-15 minute talk - Who they are and what they expected from the summer school

 - The things they learnt
 - How this will influence their daily work?
 - What we should keep in future summer schools?
 - What we should drop/improve?
What they were missing?
 
 
 
 
classroom-1910014_1280
 
 
 
 
So, are you interested?

If you are interested in attending, we recommend you fill out our application form to ensure you don't miss out on this opportunity to provide yourself with an excellent grounding in translational research.

Apply here to attend the
2018 Translational Summer School