Welcome to the Rare Bone Disease Summit

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Rare Bone Disease Summit Logo

About RBD Summit

The RBD (Rare Bone Disease) Summit is a global multi-stakeholder group meeting that aims to provide a platform to enable contributors working in the rare bone disease field (patient organizations, experts, pharmaceutical industry, and scientific and medical societies) to collaborate towards improving the lives of people living with these conditions. This is planned to be an annual event. The format and organisation of the summit will evolve reflecting on objectives and scope each year based on ongoing discussions and challenges of RBD with a shared goal towards improving the lives of patients living with RBD.

The RBD Summit 2021

The RBD Summit 2021 was the first edition of an initiative led and funded by Ipsen Pharma, with Kyowa Kirin, Alexion and Ultragenyx as the supporting strategic partners, in close collaboration with patients organisations, experts, and medical and scientific societies. The RBD Summit 2021 was a treatment and disease agnostic event which will cover multiple areas of RBDs.

Meeting Objectives

  • Provide a platform to build a robust and valuable annual program to enable stakeholders working in the area of rare bone disease to collaborate towards improving the lives of patients living with RBDs
  • Discuss the patient experience and the impact of RBDs on patients and caregivers
  • Highlight the bottlenecks in the patient journey (diagnosis, care pathways) and advance knowledge, awareness and communication about RBDs
  • Facilitate dialogue between stakeholders to exchange ideas that drive tangible outputs and provide a synergistic action plan for 2022 and beyond
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RBD Summit Highlights Video


RDB Summit Newsletter



Participation for Contribution at the Summit is by Invite Only.

2021 Agenda Summary

Day 1

Welcome and introductions

Presentation by Chairpersons

Presentation - Outlining the key unmet needs for patients living with Rare Bone Diseases

Discussion with all facilitated by Chairpersons

Working session 1 – Contributors split into 3 workshops

Workshop topics:

Workshop A: Overcoming obstacles to diagnosis: Improving awareness of rare bone diseases

Workshop B: Optimizing pathways to care by Implementing a person-centered care pathway (PCCP)

Workshop C - Understanding the patient experience: How can we narrow the gap between the patient experience and how it’s perceived by HCPs

Summary and close

Presentation by Chairpersons

Day 2

Welcome and introductions

Presentation by Chairpersons

Working session 2 – Workshop feedback altogether

Feedback from breakout session to wider group

Actions for 2022 and beyond

Group discussion led by Chairpersons

Summary and closing remarks

Presentation by Chairpersons

RBD Summit 2021 Steering Committee

Committee member Inês Alves European Rare Bone Forum (ERBF)
Committee member Charlene Waldman Rare Bone Disease Consultant
Committee member Manju Chandran International Osteoporosis
Foundation/osteogenesis imperfecta
(OI) and X-linked hypophosphataemia Expert
Committee member Oliver Semler The Osteogenesis Imperfecta
Federation Europe
Committee member Michelle Davis International Fibrodysplasia
Ossificans Progressiva
Association (IFOPA)
Committee member Federico Moscogiuri International Federation of
Musculoskeletal Research Societies
Committee member Ed Hsiao The International Clinical Council (ICC)
on Fibrodysplasia Ossificans
Progressiva (FOP)
Committee member Eric Rush Hypophosphatasia (HPP)
Committee member Tom Carpenter X-linked hypophosphatemia (XLH)
Committee member Sue Wood Global Medical Affairs,
Kyowa Kirin
Committee member Anna Petryk Global Medical Affairs,
Committee member Jenny McCue Global Patient Affairs,
Committee member Marwan Sleiman Global Medical Affairs,
Ipsen Pharma

RBD Summit 2021 Partners