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Attendees of the 2nd Multi-stakeholder meeting on “Ensuring Patient Centricity in Osteogenesis Imperfecta Research.”

Bologna, Italy provided the backdrop for our 2nd Multi-stakeholder Meeting “Ensuring Patient Centricity in Osteogenesis Imperfecta (OI) Research”. This hybrid event brought together over 80 stakeholders, including patients, patient representatives, researchers, clinicians, industry professionals, regulators and consortium partners to discuss and develop strategies to ensure that patient voices remain at the forefront of OI research and therapeutic development. 

The event featured an engaging and fully packed agenda alternating  keynote speeches, presentations, panel discussions, and interactive sessions; all aimed at fostering a collaborative environment where every stakeholder could contribute their unique insights and experiences.

“See the people behind the science”

Patient advocate Gem Turner gives a speech on “what does it mean living with OI?”

Disability rights advocate Gem Turner form the UK kicked off proceedings with an impactful speech on “what does it mean living with OI?”, sharing her personal lived experience. During her talk, she particularly emphasised the importance of acknowledging individuals beyond their disabilities and the importance of appreciating their valuable contributions to society.  “See the people behind the science” was one of the powerful statements by Gem, which not only captured the essence of the event itself, but also called for better multi-stakeholder collaboration to work towards patient-centred treatment options.

Multidisciplinary, multi-stakeholder approaches in developing patient-centred treatment options

The event facilitated inclusive dialogue among all participants, allowing them to share their unique perspectives and experiences. Session 1 focused on multinational strategies for patient advocacy (PA) in OI and included a series of insightful presentations. Representatives of different organisations, including the European Reference Network on Rare Bone Diseases (ERN-BOND), the Italian Federation on Rare Diseases – UNIAMO and the Osteogenesis Imperfecta Federation Europe (OIFE) showcased their strategies to connect and empower OI patients, emphasising the importance of collaboration, education,  support programmes, and efficient information exchange.

Current and emerging treatment options and approaches for OI

The event also included two sessions on impactful initiatives within the OI community and on ongoing clinical trials. Initiatives, like the Impact Survey Initiative, the Adult Health Initiative, the Care4Brittle Bone Key4OI Initiative, were presented and current clinical trials including the MOI-A study and the ORBIT study, were discussed. All speakers underlined the importance of aligning research goals with patient priorities. This entails not only conducting studies that address the most pressing issues faced by individuals with OI but also actively involving patients in research thorugh co-creation processes.

The Multi-stakeholder Meeting also addressed the topic of patient engagement in early Health Technology Assessment (eHTA) and regulatory decision making. Renowned speakers discussed  methodologies, case studies, and best practices for incorporating patient perspectives into this processes.

Importantly, participants also had the chance to discuss their repurposing plans with patient representatives, gaining a greater understanding of patient perspectives and its importance in the development of new treatments for rare and ultra-rare diseases. Insights gained from these discussions led to participants reconsidering potential drugs of interest.

Building a community

The event not only provided the opportunity to catch up on different initiatives and the latest research advancements, but also provided time and space for networking. Attendees enjoyed the opportunity to meet with other stakeholders within the OI research community, sharing ideas and devising new patient-centred solutions within OI research.

 (Left) Time to socialise with old and new connections within the Osteogenesis Imperfecta network. (Right) Patient advocates and presenters Gem Turner and Marie Holm Laursen catching up during the meeting.

Overall, the meeting underscored the importance of collaborative frameworks that bring together different stakeholders in the research and drug developmental process. The Multi-stakeholder Meeting hosted by REMEDi4ALL was a significant step toward in this direction.

The meeting was organised within the  REMEDi4ALL project in collaboration with EURORDIS – Rare Disease EuropeBeacon for Rare DiseasesAssociazione Italiana Osteogenesi Imperfetta – AS.IT.O.I.Osteogenesis Imperfecta Federation Europe – OIFEEuropean Reference Network on rare bone diseases – ERN BOND. To find out more about upcoming Multi-Stakeholder meetings, follow REMEDi4ALL on social media or sign up to our newsletter.