White Paper
Collaboratively address existing gap, driving meaningful progress, presenting challenges and future steps.
This white paper is presented as a call to action to all rare bone condition (RBC) stakeholders, including patient organisations, healthcare professionals (HCPs), researchers, industry and policymakers. The call to action aims to accelerate processes in the research and develop (R&D) pathway to provoke meaningful changes and address unmet needs within the RBC community.
Reducing the time associated with R&D is expected to improve access to effective treatments and clinical care for people with an RBC (pwRBC). This document was informed by evidence gathered between January 2022 and February 2023, via a review of recent literature, a series of multistakeholder focus groups, and an online stakeholder survey available in five languages.
Each project component was designed to gather evidence and insights into unmet needs for pwRBCs and to identify opportunities to improve the current R&D landscape for RBCs.
This important work, developed in collaboration with Costello Medical, provides a comprehensive overview of the current challenges, gaps, and opportunities in advancing therapies for RBCs across Europe.
Moreover, offers an in-depth analysis of the current landscape of research and innovation in this field, identifying key barriers to the development of effective therapies, highlighting disparities in access to care across Europe, and outlining actionable recommendations to accelerate progress through policy, funding, and multi-stakeholder collaboration.
Costello Medical provides scientific support in the analysis, interpretation and communication of clinical and health data. With experience with a variety of leading pharmaceutical and device companies across an extensive range of therapy areas and geographies.