DIA Europe 2023
2023-03-22 - 2023-03-24 | Basel, Switzerland
DIA EUROPE is the largest neutral event in the European life science industry – the knowledge hub between science, healthcare, and regulation. For it’s 35th anniversary, DIA Europe is back in Basel, where all thought leaders meet in person to discuss, inspire, and network.
DIA Europe 2023 is designed to drive insights into action by connecting key policy discussions to real-world knowledge. Gathering innovative thought leaders from the entire life science landscape, DIA will have 4 new content tracks in 2023:

Artificial Intelligence and Data Science
EU Health Policy and Regulatory Strategy
Medical Devices and Combination Products
Professional Development

Learn more about DIA EUROPE 2023 here.

Personalized Health Series - Workshop on Drug Repurposing
2023-05-12 13:30 - 18:15 (Europe/Amsterdam) | Innovation Office Garage, Kreuzstrasse 4, Allschwil
The goal of this workshop series is to foster and strengthen partnerships and collaborations between industry and universities, and to define a few pilot projects that are leading to innovation and societal impact.
This year’s edition will focus on Drug Repurposing. As in previous workshops, the goal is to engage professionals from both academia and the pharma sector, showing their joint initiatives with the aim to inspire new potential partnerships, and to spark interdisciplinary and cross-functional interests.
The presentation of these join initiatives will be followed by the panel discussion “Harnessing the power of collaborations and partnerships”, which will explore how to facilitate and accelerate partnerships and collaborations between academia and industry, as well as how to remove obstacles to pave the way into impactful progess.
AGENDA
13:30 – 13:45 Welcome

Dr. Bram Stieltjes, Head of Department Research & Analysis Services, University Hospital
Basel, Chair of Personalized Health Basel
Dr. Raffaello Ferone, Research Coordinator at Personalized Health Basel
Dr. Alessandro Mazzetti, Team Lead, Innovation Collaborations at Innovation Office

Collaborations between Pharma companies and Academia
13:45 – 14:10 Genome-assisted drug repurposing in psychiatry

Prof. Andreas Papassotiropulos, University of Basel

14:10 – 14:35 Combining Protein & Ligand Centric approaches to predict Drug Repurposing with in-silico
Polypharmacology

Dr. Andrea Beccari, Dompé farmaceutici S.p.A.

14:35 – 15:00 REMEDi4ALL – the European platform for medicines repurposing & REMEDi4ALL Funders Network

Dr. Heleen van der Meer, ZonMw, and Dr. Anton Ussi, EATRIS

15:00 – 15:20 Break
15:20 – 15:50 Pitching session

Small Molecules, Big Opportunities: A Multi-Purpose Platform for Off-Target Analysis
Prof. Martin Smiesko, University of Basel
Using implementation science to power the research pipeline towards faster transla-
tion in real-world settings
Dr. Sabine Valenta, Institute for Nursing Science
Holistic Approach to Personalized Medicine & Host-Response Monitoring: Quantifying Pro-
tein & Nucleic Acid Biomarkers with Diaxxo’s Desktop qPCR Platform
Engr. Joseph Iwasyk, Swiss TPH
Drug repurposing for helminth infections
Prof. Jennifer Keiser, Swiss TPH
Picky Binders: choose the right predictor for your drug or target
Dr. Janani Durairaj, University of Basel

15:50 – 17:10 Panel discussion: Harnessing the power of collaborations and partnerships: What is still needed in Drug Repurposing? From in-silico discoveries to business models – How to
remove obstacles and facilitate and accelerate partnerships and collaborations?
Panelists: Prof. Henriette Meyer zu Schwabedissen (University of Basel), Charlotte Piens
(STADA Arzneimittel AG), Dr. Andrea Beccari, (Dompé farmaceutici S.p.A.), Engr. Silvano
Coletti (CEO Chelonia Applied Science) Moderator: Dr. Benjamin Kasenda (Senior Consul-
tant Medical Oncologist, University Hospital Basel)
17:10 – 18:15 Networking Apéro

Organising Team:

Raffaello Ferone (Personalized Health Basel) – raffaello.ferone@unibas.ch
Alessandro Mazzetti (Innovation Office) – alessandro.mazzetti@unibas.ch

Open Academy Schools 2023
2023-06-19 - 2023-06-23 | Barcelona
The Open Academy School on Medicines Research & Development is a training programme offered by
EURORDIS, designed to help participants become experts in medicines research and development.
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. Following a review of our training offerings, we are excited to announce a new format for our in-person training.
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.
Click here to learn more and to submit your application. The deadline for sending applications is on October 24.

Creating Health Impact from Research: Course of 3 Live Sessions
2023-06-28 10:00 - 2023-07-11 12:00 (Europe/Amsterdam) | Online Event
If you are an Early Career Researcher who aims to enhance your skills to make an impact in healthcare research, this course is for perfect you!
Supported by the Wellcome Trust Institutional Translational Partnership Award (iTPA), we are working with UCL Academic Careers Office to sponsor UCL participants for this online course, with the course fee fully covered. Register now to secure your spot as there is a limited quota available on a first-come-first-served basis.
About this course
This course offers professional commercialization training for Higher Education Institutions across the UK. It consists of a series of live sessions accompanied by learning videos created by Pete Moores.
In this course, you will:

Develop an impact hypothesis for your work
Develop your route to impact skills in three live, online workshops
Get practical support through an online community

About the trainer
Pete is a consultant, mentor, coach and trainer in the translational field with a vast experience in working at the intersection of science and technology, industry application and funding. He’s driven by a passion to create real-world benefits from new science and technology.
He has previously worked as a head coach on international training and mentoring programmes and has mentored, coached, and trained global researchers and entrepreneurs to develop strategic sales and marketing initiatives that unlock their innovation potential.

Live Session Programme

Wednesday 28 June 2023, 10:00-12:00
Building a Bridge to the Market: the foundations of commercialisation
Wednesday 5 July 2023, 10:00-12:00
How to Write a Successful Translational Research Application
Tuesday 11 July 2023, 10:00-12:00
Creating impact from scientific research

Who is this course for?
We encourage Early Career Researchers (e.g. post-docs, fellows and newly established lecturers) with an interest in delivering impact in healthcare research to sign up, but this course is also open to any researcher who has not previously had translational-type project funding independent of their career stage. This course is NOT suitable for PhD or MRes students. If you have not engaged in any previous translational activity, but are keen to do so, this is the course for you!

Course Delivery
This workshop has been arranged by the University of Birmingham Translational Research Team, together with a network of other Universities, each supported by a Wellcome Trust Institutional Translational Partnership Award (iTPA). This event is part of an ongoing effort to support researchers in translating ground-breaking discovery research findings into practical improvements in human health.
UCL participants will join the training sessions alongside researchers from the University of Birmingham, and everyone will be encouraged to ask questions and network with fellow attendees.

Learn more and register here.

Workshop - REMEDi4ALL: what’s in it for funders?
2023-07-05 12:00 - 14:00 (Europe/Amsterdam) | Online Event
Learn and discuss how REMEDi4ALL & the funders network can support your funding activities in drug repurposing
Target audience: Funders who are (willing to be) active in drug repurposing
Within REMEDi4ALL an international funders network for drug repurposing is currently being created by ZonMw. So far, more than 50 different funders from all over the world have expressed their interest to join the network. Many of them also already shared their experiences with and ideas of funding drug repurposing with us via an online survey and in face-to-face interviews. This is the first workshop where funders who are interested in joining the funders network come together. Within an interactive setting we will talk about how REMEDi4ALL and the funders network can support funders in drug repurposing. You will learn about current and upcoming activities in REMEDi4ALL related to funders. Also, we will share and discuss the insights collected from funders on the barriers, solutions and incentives needed for funding drug repurposing.
DOWNLOAD THE EVENT SUMMARY
DOWNLOAD THE FULL PRESENTATION

Your browser does not seem to support iframes. Click here to read this PDF.
Created using FlowPaper Flipbook Maker ↗

Agenda

Time
Topic

12.00-12.05
Welcome
Chair: Heleen van der Meer (ZonMw)

12.05-12.30
Introduction REMEDi4ALL and funders network
Anton Ussi (EATRIS) & Heleen van der Meer (ZonMw)

12.30-12.40
How do you repurpose? REMEDi4ALL’s training programme for the whole repurposing ecosystem
Rick Thomson (BEACON: for rare diseases)
+ input from all participants

12.40-12.50
Introduction to the REMEDi4ALL Funding Decision Tool
Marcell Csanadi (Syreon Research Institute)
+ input from all participants

12.50-13.00
Q&A – break

13.00-13.20
Funding drug repurposing: barriers, solutions & incentives needed
Heleen van der Meer (ZonMw)

13.20-13.45
Paneldiscussion
Moderated by Pan Pantziarka (Anticancer Fund)
Panellists:

Joanna Davidge (LifeArc)
Teun Grooters (Arega Medical)
Sagair Hussain (DEBRA)
Sarah Puddicombe (National Institute for Health and Care Research Coordinating Centre (NIHR CC))

13.45-14.00
Next steps for the funders network & closing
Heleen van der Meer (ZonMw)
+ input from all participants

FIP Brisbane 2023: Pharmacy building a sustainable future for healthcare – Aligning goals to 2030
2023-09-24 - 2023-09-28 | Brisbane, Australia
81ST FIP WORLD CONGRESS OF PHARMACY AND PHARMACEUTICAL SCIENCES
The pharmacist is key in assuring a safe and effective supply of medicines as well as their responsible use. As a direct provider of services, from primary prevention and screening of diseases, immunisations to therapeutic monitoring, the work of this profession ensures that a patient’s medical therapy is appropriate, the most effective, the safest possible and the most convenient.
The International Pharmaceutical Federation (FIP) Development Goals align pharmacists’ education with societal and healthcare needs, taking into consideration the path to universal health coverage and the UN Sustainable Development Goals, and make the case for investing in the pharmacy workforce of tomorrow.
These goals present the profession with the direction for advancement and transformation and establishing its place within the healthcare system. By linking the FIP vision with a needs-based pharmaceutical workforce transformation programme, a sustainable future for healthcare can be built — aligning goals to 2030.
Linked to a healthier world, the “Pharmacy building a sustainable future for healthcare — aligning the goals” theme sets out long-term goals and principles, as well as short-term actions, to transform the role and contribution of pharmacy. That transformation is required to maximise the health gain that healthcare systems derive from their interactions with the pharmacy profession.
Learn more and register for the congress here.

REMEDi4ALL MULTI-STAKEHOLDER MEETING: Drug Repurposing, an Attractive Strategy in Pancreatic Cancer Treatment?
2023-11-14 09:00 - 17:30 (Europe/Amsterdam) | Nhow Brussels Bloom, Rue Royale 250, 1210, Brussels, Belgium
REMEDi4ALL is pleased to announce the first Multi-Stakeholder Meeting on drug repurposing in PANCREATIC CANCER.
This one-day in person workshop is aimed to bring together all relevant key stakeholders (patients and patient representatives, academia, industry, regulators, funders, etc.) to discuss the recent state of art on drug repurposing in oncology and specifically in pancreatic cancer. Topics of discussion will include an overview of the disease and the current (preclinical and clinical) drug repurposing programs, biomarkers, the importance of a patient-centred outcome measures and patient engagement activities in the study design and implementation. In addition, current hurdles including financial and regulatory barriers to successfully repurpose non-oncology drugs in pancreatic cancer will also be discussed.
REGISTER HERE
Preliminary Agenda (might be subject to change)

8.30 – 9.00
Welcome and Registration

9.00 – 9.10
Open Ceremony
Claudia Fuchs (REMEDi4ALL – EURORDIS)

SESSION 1: SETTING THE SCENE
Moderator: Claudia Fuchs (REMEDi4ALL – EURORDIS)

9.10 – 9.30
General Introduction on Pancreatic Cancer
Alfredo Carrato (Pancreatic Cancer Europe – PCE)

9.30 – 9.50
Tackling pancreatic cancer: tricks and tips
Ilse Rooman (Anticancer Fund, Vrije Universiteit Brussels)

9.50 – 10.10
Drug repurposing in oncology – Attractive but not that simple
Gauthier Bouche (REMEDi4ALL – Anticancer Fund)

10.10– 10.25
REMEDi4ALL (vision, mission, European Context)
Donald Lo (REMEDi4ALL – EATRIS)

10.25 – 11.10
Patient Perspective on Drug Repurposing in Oncology
Patrycja Rzadkowska (Pancreatic Cancer Europe – PCE and DiCE)
Ali Stunt (Pancreatic Cancer Europe – PCE and Pancreatic Cancer Action UK)

COFFE BREAK 11.10 – 11.40

SESSION 2: TREATMENT OPTIONS AND DRUG REPURPOSING OPPORTUNITIES IN PANCREATIC CANCER
Moderators: Gauthier Bouche/Patricia Vandamme (REMEDi4ALL – Anticancer Fund)

11.40 – 12.00
Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation
Violeta Stoyanova-Beninska (Medicines Evaluation Board, Chair of Committee for Orphan Medicinal Products at EMA)

12.00 – 12.20
Preclinical and Clinical Updates on Drug Repurposing Opportunities in Pancreatic Cancer
Luca Cardone (Istituto Nazionale Tumori Regina Elena and National Research Council of Italy)

12.20– 12.40
Patient Story
Steve Bigelsen (Rutgers New Jersey Medical School, Newark, NJ, USA)

12.40 – 13.00
The VESPA Trial – Repurposing valproic acid combined with simvastatin to potentiate first line gemcitabine/nab-paclitxel-chemotherapy regimen in metastatic pancreatic cancer patients
Alfredo Budillon (REMEDi4ALL – Istituto Nazionale Tumori Fondazione Pascale)

LUNCH BREAK 13.00 – 14.00

SESSION 3: THE PAST, PRESENT AND FUTURE OF PANCREATIC CANCER CLINICAL TRIALS
Moderators: Eve Hewitt/Rick Thompson (REMEDi4ALL- Beacon)

14.00 – 14.40
Financial and Regulatory Barriers and Potential Solutions
– Overview on the current findings within REMEDi4ALL and future perspectives
– Industry perspective
– Funders persepective
Heleen van der Meer (REMEDi4ALL – ZonMW)
Raluca Radu (REMEDi4ALL – Medicines for Europe)
Hans Platteeuw (CEO Galenicap)
Alexandre Bento Costa Alencar (Rising Tide Foundation)

14.40 – 15.00
Biomarkers in Pancreatic Cancer
María Laura Garcia Bermejo (Ramon & Cajal Health Research Institute)

15.00 – 15.20
Patient Centricity in Clinical Trials
Antonella Cardone (Cancer Patients Europe – CPE)
Federica Valsecchi (Fondazione Valsecchi)

COFFE BREAK 15.20 – 15.50

15.50 – 17.20
FINAL ROUNDTABLE AND DISCUSSION
Moderators: Claudia Fuchs (REMEDi4ALL – EURORDIS) – Rick Thompson (REMEDi4ALL – Beacon)

17.20 – 17.30
Closing Remarks
Claudia Fuchs (REMEDi4ALL – EURORDIS)
Rick Thompson (REMEDi4ALL – Beacon)

17.30
End of the Meeting and “GET TOGETHER”

REMEDi4ALL #iDR24 International Drug Repurposing Conference
2024-03-06 08:00 - 2024-03-07 18:00 (Europe/Amsterdam) | Recinte Modernista de Sant Pau, Calle St. Antoni Maria Claret, 167, Barcelona, Spain
Please see this page for more information: https://remedi4all.org/international-drug-repurposing-conference/

REMEDi4ALL Funders Network Meeting
2024-03-07 15:15 - 17:00 (Europe/Amsterdam) | Recinte Modernista de Sant Pau, Calle St. Antoni Maria Claret, 167, Barcelona, Spain
iDR24 Satellite Event – REMEDi4ALL Funders Network Meeting
After the iDR24 the members and observers of the REMEDi4ALL Funders Network will come together to discuss the next steps of the network, including the strategic agenda.
Register here
Location: Recinte Modernista de Sant Pau. Green auditorium.
This will be a hybrid event – possible to join online.
AGENDA

Clinical Pharmacology Guidances Advancing Drug Development and Regulatory Assessment
2024-05-08 09:00 - 14:30 (America/Halifax) | Online & in-person (Maryland, United States), MD, United States
ABOUT THIS EVENT
This public workshop will be hosted by the FDA Office of Clinical Pharmacology (OCP) and the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI). The purpose of this workshop is to serve as a mechanism to engage with the public to share information on existing clinical pharmacology guidances and identify emerging scientific topics that might benefit from the development of scientific research and recommendations.
BACKGROUND
At every step of drug development, clinical pharmacology is applied to generate, evaluate, and use knowledge of drug disposition, pharmacology, and disease biology to progressively reduce regulatory uncertainty and inform public health decision-making. Clinical pharmacology principles form the basis of dosage selection and optimisation and promote therapeutic individualisation by translating the knowledge of patient diversity into clinical recommendations for safe and effective use of medications. Therefore, having clear, pragmatic, and contemporary scientific recommendations to inform drug development and regulatory assessment is critical for the successful and efficient development of therapeutics that protect and promote public health. Developing new or revising existing guidance documents, especially in the broad field of clinical pharmacology, requires a unique and integrated approach centered around multistakeholder partnerships.
WORKSHOP GOALS

Provide an overview of scientific recommendations pertaining to clinical pharmacology applications during drug development and regulatory assessment.

Discuss the current scientific challenges and gaps in applying clinical pharmacology principles during drug development.

Identify potential opportunities and priorities for regulatory research and scientific guidance development from a clinical pharmacology perspective.

AGENDA
Click here to see the full agenda.
REGISTRATION
Registration is free. All interested parties are encouraged to register early. Please click here to register.

Click here to learn more.

2nd MULTI-STAKEHOLDER MEETING - Ensuring patient centricity in Osteogenesis Imperfecta research
2024-06-13 08:30 - 18:00 (Europe/Amsterdam) | Savoia Hotel Regency, Via del Pilastro, 2, Bologna, Italy
REMEDi4ALL is hosting our 2nd Multi-stakeholder Meeting (MS) – Ensuring patient centricity in Osteogenesis Imperfecta (OI) research.

Join the MSM
REMEDi4ALL invites all stakeholders in the OI research community to join us for a day of learning, discussion, and inspiration.
We welcome clinicians, academics, patients and patient representatives, pharmaceutical companies, regulators, payers, health economists and all interested in OI and Rare Bone Diseases research. Together, we will discuss and develop patient centric approaches to research and medicines development for OI.
Our MSM will:

Share information and advance learning, in a pre-competitive and cooperative setting
Develop constructive dialogue between all stakeholders
Facilitate collaboration and knowledge exchange
Identify current hurdles and propose solutions to ensure patient centricity in OI research
Publish and disseminate the meeting outcomes to a broader community

The Power of Patients
The patient’s voice is at the heart of this meeting. The OI community and the REMEDi4ALL Patient Advocacy Group have played integral roles in shaping the content and format of this event.
We wanted to ensure that the patients’ perspectives, experiences, and preferences guide our discussions and are at the forefront of OI research and therapeutic development. Over the day, you will hear from numerous patients, patients representative and advocates. They will:

Share their personal journeys of living with OI, providing insights into the day-to-day challenges they face, as well as the triumphs and setbacks they encounter.
Highlight unmet medical needs within the OI community
Discuss patient-relevant research questions and identify research priorities

Patients will have the opportunities to

Learn about the recent advancements in medicines development in OI
Initiate collaborations and partnerships with all stakeholders
Actively participate and engage in scientific discussions about OI

Patient centricity will run as a core-narrative in all our sessions and panel discussions, encouraging each stakeholder to consider the patient perspective in their current and future work.
Agenda (might be subject to change)
WEDNESDAY, 12 JUNE 2024

19.30/20.00
Arrival and Speakers Dinner at the Hotel
BY INVITATION ONLY

THURSDAY, 13 JUNE 2024 (8.30 – 18.00 CEST)

8.00 – 8.30
Registration

8.30 – 8.40
Opening Ceremony and Welcome
Claudia Fuchs (EURORDIS)
Leonardo Panzeri (AS.IT.O.I.)
Donald Lo (EATRIS)

Opening Speech

8.40 – 9.00
What does it mean living with OI?
Gem Turner

SESSION 1: Multi-national strategies for Patient Advocacy in OI
Moderators: Claudia Fuchs

9.00 – 9.10
The European Reference Network on rare bone diseases (ERN-Bond)
Luca Sangiorgi (Istituto Ortopedico Rizzoli)

9.10 – 9.20
The European Registries for Rare Bone and Mineral Conditions (EuRR-Bone)
Claudia Finis (DOIG (German Ostogenesis Imperfecta Organisation) and Berlin Institute of Health at Charité)

9.20 – 9.30
Patient Engagement Initiatives in Rare Diseases: an Italian Perspective
Annalisa Scopinaro (UNIAMO – Federazione Italiana Malattie Rare)

9.30 – 9.45
Q/A session

PANEL DISCUSSION – Ask the Expert: How to become a Patient Advocate in OI?
Moderators: Marina O’Callaghan & Claudia Fuchs

Lightning talks: European and National Patient Engagement Activities in OI

10.00 – 10.05
Stronger Together: OIFE’s Patient Engagement Initiatives
Anna Rossi (OIFE)

10.05 – 10.10
Introduction to Patient Engagement Activities in Italy
Simona Paveri (AS.IT.O.I.)

10.10 – 10.15
Introduction to Patient Engagement Activities in Norway
Inger-Margrethe Stavdal Paulsen (Norsk Forening for Osteogenesis Imperfecta)

10.15 – 10.20
Introduction to Patient Engagement Activities in Spain
Miguel Rodríguez Molina (Asociación Nacional Huesos de Cristal OI España – AHUCE)

10.20 – 10.25
Brittle Bone Society – Support that matters most
Patricia Osborne (Brittle Bone Society – BBS)

10.25 – 11.00
Panel Discussion – Ask the Expert: How to become a Patient Advocate in OI?Panelists: Gem Turner, Annalisa Scopinaro, Anna Rossi, Miguel Rodríguez Molina, Patricia Osborne, Emilija and Dace Liepina, Marie Holm Laursen

11.00 – 11.30 Coffee Break
SESSION 2: Initiatives in the OI community
Moderators: Anna Rossi & Luca Sangiorgi

11.30 – 11.40
The Impact Survey Initiative
Oliver Semler (University of Cologne)

11.40 – 11.50
Adult Health Initiative
Stuart Ralson (University of Edinburgh)

11.50 – 12.00
Care4BrittleBones Initiative Key4OI
Lida Zhytnik (Amsterdam UMC)

12.00 – 12.15
Q/A session

SESSION 3: Current Treatment Options in OI – a multistakeholder perspective
Moderators: Anna Rossi & Luca Sangiorgi

12.20 – 12.40
Introduction to different treatment options and approaches for OI
Nick Bishop (The University of Sheffield)

12.40 – 14.00 Lunch Break
CONTINUATION SESSION 3

Lightning talks: Current ongoing clinical trials with specific focus on challenges and opportunities of Patient Engagement

14.00 – 14.10
REMEDi4ALL MOI-A Study
Judith Cohen (University of Hull)

14.10 – 14.20
TOPaZ trial
Stuart Ralson (University of Edinburgh)

14.20 – 14.30
The ORBIT Phase 2/3 study: a collaborative design to study outcomes in OI
Luigi Picaro (Mereo BioPharma)

14.30 – 14.40
BOOSTB4 Trial: Boost Brittle Bones Before Birth
Cecilia Götherström (Karolinska Institute)

14.40 – 14.55
Q/A session

SESSION 4:”Patient-centered clinical trial design”
Moderators: Nick Bishop & Claudia Fuchs

Lightining talks: Patient perception and experience in clinical trials

15.00 – 15.10
Patient Perspective as a Teenager
Emilija and Dace Liepina

15.10 – 15.20
Patient Experience on the ASTEROID Trial
Marie Holm Laursen

15.20 – 15.30
Patient Engagement activites within the R4ALL demonstrator (MOI-A Study)
Marina O’Callaghan

15.30 – 15.45
Q/A session

15.45– 16.15 Coffee Break
PANEL DISCUSSION – Innovative outcome measures in clinical trials
Moderators: Inger-Margrethe Stavdal Paulsennger & Claudia Fuchs

Lightning talks: Quality of Life assessment (QoL) and patient related innovative outcome measures in clinical trials in OI

16.15 – 16.25
QoL assessment in OI
Nick Bishop (The University of Sheffield)

16.25 – 16.35
Developing patient related outcome measures on mobility in rare bone disorders
Luca Sangiorgi (Istituto Ortopedico Rizzoli)

16.35 – 16.45
Regulatory perspective on innovative outcome measures – how can patients contribute?
Violeta Stoyanova-Beninska (Committee for Orphan Medicinal Products)

16.45 – 16.55
Patient perspective on innovative outcome measure – how to advocate?
Inger-Margrethe Stavdal Paulsen (Norsk Forening for Osteogenesis Imperfecta)

17.00 – 17.40
Panel Discussion – innovative outcome measures in clinical trials
Panelists: Nick Bishop, Stuart Ralson, Luigi Picaro, Cecilia Götherström, Oliver Semmler, Lida Zhytnik, Violeta Stoyanova-Beninska

17.40 – 17.50
Patient engagement in early Health Technology Assessment (eHTA)
Zsuzsanna Petyko (Syreon Research Institute)

17.50 – 18.00
Wrap Up and Closing Remarks
Claudia Fuchs (EURORDIS)

18.00 Get Together
FRIDAY, 14 JUNE 2024
9.00 – 11.00 CEST

9.00 – 11.00
Workshop on early Health Technology Assessment (eHTA)
BY INVITATION ONLY
Dalma Hosszu (Syreon Research Institute)

*Please note that only meals (lunch, coffee breaks and get-together) are covered by the event organisers. Participants need to arrange their own travel and accomodation.
Download the programme here.
Download the event flier here.
Speakers
Click here to see the complete list of speakers and their bios.
Confirmed speakers:

Organisers
Organised within the REMEDi4ALL project in collaboration with EURORDIS – Rare Disease Europe, Beacon for Rare Diseases, Associazione Italiana Osteogenesi Imperfetta – AS.IT.O.I., Osteogenesis Imperfecta Federation Europe – OIFE, European Reference Network on rare bone diseases – ERN BOND.

Registration
This event will be a hybrid event, hosted both online and in-person (Bologna, Italy). Registration closes on 31 May, 2024.
Register here

ICCBH 2024 - 11th International Conference on Children’s Bone Health
2024-06-22 - 2024-06-25 | Salzburg Congress, Auerspergstraße 6, 5020 Salzburg, Austria, Salzburg, Austria
This conference will bring together scientists, clinicians, trainees, allied health professionals and others from a wide range of disciplines to gain a better understanding of the growing skeleton in relation to childhood health and disease and to discuss the most recent developments in clinical outcomes, novel therapies, and genetic discoveries. The conference scope ranges from basic molecular mechanisms to clinical aspects, from bone pathophysiology to prevention and medical and surgical treatment.
This conference is for anyone with an interest in bone metabolism and bone mass in children, adolescents and young adults to attend.
The ICCBH conference takes place every two years. The 2022 meeting in Dublin was attended by over 400 participants from across the globe, making it truly multinational and multidisciplinary.
The scientific programme is designed to present researchers, physicians, and allied health professionals with the newest research, with sessions including plenary lectures by world-leading experts, symposia, workshops, oral abstract communications, poster sessions and meet the expert sessions. The conference will offer investigators from around the world an opportunity to meet with each other and with industry representatives.
ICCBH 2024 is your opportunity to hear about and discuss the newest developments in our understanding of paediatric bone health.
Learn more and register here.

Webinar: Welcome to the REMEDi4ALL Digital Academy
2025-01-29 12:30 - 13:30 (Europe/Amsterdam) | Online Event
Are you ready to embark on an exciting digital learning journey with us? Join us on Wed Jan 29 2025 at 12:30 PM CET for our online event where we will introduce you to our innovative academy.
During this event, you will have the opportunity to learn about our cutting-edge digital courses, interact with our experienced instructors, and discover how REMEDi4ALL can help you enhance your skills and knowledge in drug repurposing.
Don’t miss out on this chance to learn all about our repurposing curriculum and our upcoming events! Register now and get ready to take your drug repurposing knowledge to the next level with REMEDi4ALL Digital Academy.
Learn more about the launch of the Digital Academy here.

REMEDi4ALL Clinical Trials Webinar Series: Introduction to Clinical Trials
2025-02-21 11:00 - 12:30 (Europe/Amsterdam) | Online Event
Register for Webinar 1 here!

Mark your calendars for 11.00 – 12.30 CET on 21 February, 28 February and 7 March as we take an early lunch to discuss clinical trials with REMEDi4ALL partners Beacon for Rare Diseases, EURORDIS and ECRIN!
Each session will build on the knowledge gained in the previous session – with session 1 acting as a beginners guide and session 3 involving a more in-depth look at who and what makes a clinical trial run successfully.
Webinar 1: Introduction to Clinical Trials – 21 Feb (11:00-12:30 CET)
Why we do clinical research and what the process looks like for participants.
This webinar will explain why we do clinical research and what the process looks like for participants and answer some of the most common questions raised by trial participants and patients interested in clinical research. Find out more here.
See our other event listings to register for webinars 2 (rare diseases) and 3 (support and management of clinical trials).

REMEDi4ALL Clinical Trials Webinar Series: Clinical Trials for Rare Diseases
2025-02-28 11:00 - 12:30 (Europe/Amsterdam) | Online Event
Register for Webinar 2 here!

Webinar 2: Clinical Trials for Rare Diseases – 28 Feb (11:00-12:30 CET)
Exploring different ways of conducting clinical trials, particularly for rare disease.
Our Rare Disease Day special! This webinar will explore different ways of conducting clinical trials in smaller populations where ‘traditional’ methods might not be as appropriate. We will describe what endpoints are and how these can guide trial design in a patient-centric way. We will explore how the patient voice can help inform trial design and outcomes. Find out more here!

DREAMS Project Webinar - Clinical Trials in Focus: Innovations and Patient Perspectives in Rare Diseases
2025-02-28 12:00 - 13:30 (Europe/Amsterdam) | Online Event
Join leading experts as they explore advancements in clinical trial methodologies, outcome measures, and lessons learned from research in muscular disorders.
During this session, experts in the field will share insights on:
• The need for innovation in clinical trial methodologies for muscular disorders.
• An overview of outcome measures in clinical trials for neuromuscular disorders.
• Lessons learned from a decade of clinical trials on Duchenne muscular dystrophy (DMD).
As part of the event, a patient will share their personal journey and insights on participating in clinical trials. Following their testimony, there will be an interactive Q&A session where you can engage directly and ask questions.

REMEDi4ALL Clinical Trials Webinar Series: Support and Management of Clinical Trials
2025-03-07 11:00 - 12:30 (Europe/Amsterdam) | Online Event
Register for Webinar 3 here!

Webinar 3: Support and Management of Clinical Trials – 7 March (11:00-12:30 CET)
Exploring different organisations, processes and pathways for expert support when planning, setting up and running a clinical trial.
In this third and final webinar in the series we will highlight different collaborations that are required in running a clinical trial efficiently. Find out more here!

REMEDi4ALL Drug Repurposing Bootcamp for Academics
2025-03-17 - 2025-03-18 | Hinxton Hall, Wellcome Genome Campus, Hinxton, Cambridge, CB10 1RQ, United Kingdom
Drug repurposing offers a faster, more cost-effective way to develop treatments, addressing urgent patient needs and unlocking opportunities for academic innovation. However, the journey from a repurposing concept to a market-ready product is complex, presenting significant challenges for researchers. The REMEDi4ALL Drug Repurposing Bootcamp for Academics is designed to support senior post-doctoral researchers and early-career principal investigators leading promising projects by equipping them with the skills and knowledge to navigate this pathway successfully.
This immersive, day-and-a-half training programme combines expert lectures, interactive discussions, and working groups to cover key topics such as regulatory engagement, collaboration building, funding applications, and science communication. By participating, attendees will not only develop a clearer understanding of how to deliver real patient impact but also join the growing REMEDi4ALL community.
Twenty fully funded places are available, covering travel, accommodation, and access to this free training. Applications are open until February, with reviews conducted on a rolling basis.
Find out more information here: REMEDi4ALL Drug Repurposing Bootcamp for Academics
Register here!

Unlocking research opportunities: Partnering with academics
2025-04-01 12:00 - 13:30 (Europe/Amsterdam) | Online Event
Are you looking to engage in research but don’t know where to start?
Research is a key priority for many patient groups but connecting with the right academic partners is not often straightforward. With limited awareness of rare conditions in academia, knowing where to start, initiating conversations, and building lasting relationships with the right research teams can be challenging.
What will you learn in this webinar?

How academic research works and the structures that shape it
Where to find researchers working on your condition and how to connect with them
Practical ways to boost the appeal of your patient group to researchers
A real-world example of a patient group successfully partnering with academics and getting involved in research

For more information visit the event page here.

REMEDi4ALL Funders Network Event 2025
2025-05-06 | Royal Netherlands Academy of Arts and Sciences (KNAW), Het Trippenhuis, Kloveniersburgwal 29, 1011 JV, Amsterdam, 1011 JV, Netherlands
Pre-iDR25 REMEDi4ALL Funders Network Event
In an exclusive pre-conference meeting, members and observers of the REMEDi4ALL Funders Network will come together to share best practices and discuss important topics. This meeting will be followed by an evening boat trip on the Amsterdam canals and a Funders Network dinner at IJver restaurant.
Register here!

Meeting location: Royal Netherlands Academy of Arts and Sciences (KNAW), Het Trippenhuis, Kloveniersburgwal 29, Amsterdam
Dinner location: IJver Restaurant, Scheepsbouwkade 72, Amsterdam

#iDR25 Pre-Conference Workshops
2025-05-06 12:00 - 18:30 (Europe/Amsterdam) | Royal Netherlands Academy of Arts and Sciences (KNAW), Het Trippenhuis, Kloveniersburgwal 29, 1011 JV, Amsterdam, 1011 JV, Netherlands
To kick off the iDR25 conference series, two exclusive pre-conference workshops will be held on 6th May 2025 at the Royal Netherlands Academy of Arts and Sciences (KNAW). The workshops are primarily aimed at academics, early career researchers and patient representatives looking to increase the understanding of key topics related to medicines repurposing and gain practical skills to apply to current and future drug repurposing projects.
Register today for the pre-iDR25 workshop series!

Workshop 1: Health Technology Assessment: a key tool to patient access
Tuesday 6th May 2025
12:30-14:30

After regulatory approval by the EMA, Health Technology Assessment (HTA) becomes a pivotal process in determining whether new treatments will be accessible to patients, influencing their affordability, availability, and overall impact on healthcare systems. The HTA dossier encapsulates critical information, including the medical, economic, social, and ethical considerations of using a health technology—such as a repurposed medicine. Prepared by the product developer or sponsor, these dossiers undergo evaluation by HTA agencies, like EUnetHTA 21 in the EU or NICE in the UK, to assess their value and feasibility.
This workshop will equip researchers, patient representatives and product developers with the knowledge and tools to design their repurposing projects to meet HTA standards from the onset. With a focus on early alignment with HTA requirements, evidence planning, and incorporating patient engagement into the HTA process from the beginning, attendees will gain actionable insights to streamline their projects through HTA evaluations, enhancing the likelihood of timely patient access.
This workshop offers a unique opportunity to learn proactive strategies to maximise the success of drug repurposing projects in achieving their ultimate goal—reaching and benefiting patients.

Workshop 2: Navigating the regulatory path for medicinal product repurposing
Tuesday 6th May 2025
15:00-18:30

In this workshop, we will discuss the regulatory path and key milestones for advancing a repurposed/repositioned medicinal product from preclinical development to market authorisation in Europe, tailored for academic researchers and early-career developers navigating such processes for the first time. Whether you are preparing for your first clinical trial application or strategising for market authorisation, this session will provide crucial tools and insights to help build your regulatory strategy including:

Core elements of a Clinical Trial Application (CTA)
Obtaining Orphan Drug Designations for rare diseases
Effective engagement with regulatory authorities (e.g. scientific advice)
Overview of possible registration pathways to bring promising therapies to patients faster
Building the Common Technical Document (CTD) for a European Marketing Authorisation Application (MAA)

Confirmed speakers

Register today for the pre-iDR25 workshop series!

Note: this event is separate to the main conference. Find out more about iDR25: Medicines Reimagined here.
Questions? Contact conference@remedi4all.org.

International Drug Repurposing Conference 2025 - #iDR25
2025-05-07 - 2025-05-08 | Amsterdam, the Netherlands
Exciting news for anyone passionate about healthcare innovation—iDR25, the International Drug Repurposing Conference, is coming up on 7-8th May 2025 in the beautiful city of Amsterdam in The Netherlands!

Recurrent Respiratory Papillomatosis (RRP) Awareness Day Webcast
2025-06-11 19:00 - 20:30 (Europe/Amsterdam) | Online Event
RRP Awareness Day is an international event to raise awareness about recurrent respiratory papillomatosis (RRP), a debilitating disease driven by chronic HPV 6/11 viral infection.
Join the RRP Awareness Day global webcast on June 11th to hear from RRP patients, their caregivers, and the healthcare community supporting them about:

Patient and caregiver experience with RRP
Significant burden of living with RRP
Challenges of repeated surgeries and risk of irreversible injury
Need for new innovations

CO-HOSTS

RRP Foundation
Kim McClellan, President

PRECIGEN
Helen Sabzevari, PhD, President and CEO

Who should attend?

RRP patients
Caregivers and family members
Healthcare providers
Community supporting RRP patients

Webinar: Cures Within Reach new funding call: Clinical trials to validate AI-driven drug repurposing
2025-07-31 16:30 - 17:30 (Europe/Amsterdam) | Online Event
Artificial intelligence (AI) is increasingly seen as a powerful tool in the search for new treatments – especially through drug repurposing. By analysing large sets of data, AI can help identify existing medicines that might work for different conditions – something particularly valuable in rare diseases.
However, do AI-generated predictions actually work when tested in real-life clinical trials?
Cures Within Reach is helping to explore this question through a new funding call. They’re inviting letters of intent for Phase I/IIA clinical trials that will test and validate AI-based repurposing ideas, with a preference for rare disease.
Learn more about the call here.

This webinar hosted by Beacon for Rare Diseases in collaboration with REMEDi4ALL and Cures within Reach will cover:

A breakdown of what this funding call is about
Advice from Cures Within Reach on what makes a strong application
How patient groups can get involved in applications
A practical example of a patient group contributing to an AI-led initiative
The opportunity to ask any questions you might have to the Cures Within Reach team

Register for the webinar here!

2025 REMEDi4ALL Hackathon
2025-10-06 14:00 - 2025-10-10 17:00 (Europe/Amsterdam) | Hotel Checkin Montserrat, Carrer Querol, 08293 Collbató, Barcelona, Spain
Are you an early career researcher (ECR) who wants to delve into drug repurposing?

Join us for the second REMEDi4ALL Hackathon, 6-10 October 2025, held at Hotel Checkin Montserrat, near Barcelona, Spain.
The REMEDi4ALL Repurposing Hackathon is designed to help early career researchers learn by doing. Join a small team of international trainees keen to learn about the repurposing pathway on a week-long residential course where you aim to plan the development of a repurposing candidate for a specific patient community.
Who is the Hackathon for?

Students and researchers in the early stages of their career up to 2 years post-doc
Individuals with an interest in drug repurposing
Individuals with an excellent scientific track record
Previous experience in the drug repurposing field will be considered a plus

What will you learn?
The REMEDi4ALL Hackathon aims to teach participants how to:

Use computational tools to generate repurposing candidates suitable for a defined indication
Narrow down potential repurposed drug options tailored to a particular indication
Identify needs and knowledge gaps in a repurposing project, helping to define the path for a drug candidate
Work together with other ECRs to complete a repurposing challenge

Training format

In-person, residential training for 15 participants
Advance registration required
Small teams of early career researchers work on a project throughout the week
Support through lectures and guidance of REMEDi4ALL experts, and external advisers, including patient experts

Interested in learning more? Check out our highlights video from 2024 or explore the Hackathon website within the REMEDi4ALL Repurposing Academy.

Webinar: IP Strategies for Repurposing
2025-11-21 14:00 - 15:30 (Europe/Amsterdam) | Online Event
Discover the value of having an intellectual property (IP) strategy in your repurposing project and how to use this to drive success!
Not sure where to start with intellectual property (IP) in your drug repurposing project? REMEDi4ALL’s Repurposing Academy is here to guide you!
Join us for our upcoming webinar where we will explore the importance of considering IP as part of your broader drug repurposing strategy.
We’ll discuss common challenges you may encounter and key considerations to keep in mind throughout your repurposing project. Additionally, we’ll outline practical steps for securing IP protection for repurposed drugs and show how a strong IP strategy can help drive your project forward.

ProvenConnect REMEDi4ALL Webinar
2025-11-27 13:00 - 14:00 (Europe/Amsterdam) | Online Event
* This is an online event that will take place 1200–1300 UK time (1300-1400 CET) on Zoom. *
REMEDi4ALL is presenting to the TAR Network in a Proven Connect webinar. Speakers Alicia Soler (Lead Scientific Operations at REMEDi4ALL /EATRIS), Heleen van der Meer (Senior programme manager international drug repurposing at ZonMw) and Pan Pantziarka (Director of Drug Repurposing at Anticancer Fund) will present the REMEDi4ALL project and Funder’s Network.

REMEDi4ALL Drug Repurposing Bootcamp for Academics 2026
2026-03-11 08:00 - 2026-03-12 17:00 (Europe/London) | Hinxton Hall, Wellcome Genome Campus, Hinxton, Cambridge, CB10 1RQ, United Kingdom
Join us for the second REMEDi4ALL Drug Repurposing Bootcamp for Academics!
From Hypothesis to Impact: Navigating the Repurposing Pathway
Drug repurposing represents a vital pathway for treatment development, both for patient communities and academic innovators. Finding new uses for existing drugs can provide a more rapid pathway to patient impact, both by reducing the number of steps to a clinical trial and reducing the financial barrier to research.
While the potential of repurposing is huge, the pathway to deliver a repurposed medicine from concept to market remains a hugely challenging one. This is particularly hard for academic researchers to navigate – employing their scientific skill set to the complex and highly regulated world of drug development unsupported can lead to projects quickly becoming mired in challenges or uncertainty.
The REMEDi4ALL Drug Repurposing Bootcamp for Academics, held at the Wellcome Genome Campus in Cambridge, aims to support researchers embarking on this journey. This pilot training course will provide face to face training to explain the full drug repurposing pathway, helping researchers understand the steps involved in turning a repurposing hypothesis into a product capable of delivering real impact to patients.
Registrations open now!

REMEDi4ALL Funders Network Event 2026
2026-05-11 | Maison de la Poste, Brussels, Belgium, Rue Picard 7, Brussels, Belgium
Pre-iDR26 REMEDi4ALL Funders Network Event
As in previous editions, #iDR26 will feature a pre-conference meeting dedicated to the Funders Network on Monday 11 May.
During this meeting, funders will:

discuss important topics, including innovative co-funding models,
share best funding practices,
enjoy a networking lunch, and
conclude the day with drinks & bites.

The meeting is open to all members of the Funders Network. Register here for the meeting (limited places available)!
Are you a funder who is not yet a member but interested in joining? Contact us at remedi4all@zonmw.nl.

International Drug Repurposing Conference 2026 - #iDR26
2026-05-12 - 2026-05-13 | Maison de la Poste, Brussels, Belgium, Rue Picard 7, Brussels, Belgium
Join iDR26, the International Drug Repurposing Conference, on 12-13 May 2026 in Brussels, Belgium to explore the future of patient-centric medicines repurposing.
From rare diseases and AI-driven drug discovery, to policy, funding and community-led success stories, iDR26 brings together the global drug repurposing community to navigate the next chapter in maximising patient benefit.
Explore the full conference here!
