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2nd MULTI-STAKEHOLDER MEETING – Ensuring patient centricity in Osteogenesis Imperfecta research

13 June 2024 @ 8:30 am - 6:00 pm

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)


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

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.


Click here to see the complete list of speakers and their bios.

Confirmed speakers:



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.



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

Register here