European ‘Call to Action’ for Rare Bone Diseases

Aim

This project aimed to review the literature and engage key stakeholder groups through a series of engagement activities to identify and understand unmet needs and challenges across the R&D landscape, identify potential solutions, and to develop a set of recommendations to overcome these challenges.

Approach

Targeted literature review

First, we conducted a targeted literature review using electronic database and grey literature searches. We then screened the results for relevance and extracted key data to create a summary slide set, where we outlined the methodology and key results.

Gap analysis and stakeholder mapping

Next, we performed a gap analysis by assessing the evidence from the targeted literature review against pre-specified categories. This was supplemented with online searches to identify and map RBC experts across different stakeholder groups and geographical locations.

Multistakeholder focus groups

We arranged and facilitated virtual focus group discussions with multiple stakeholders, including patient representatives, researchers, healthcare professionals, and industry experts to further examine underreported topics of interest identified by the gap analysis.

Online stakeholder survey

We gathered opinions from a broad range of stakeholders through online surveys, which were translated into five different languages and shared via social media to maximise our reach.

White paper report

Evidence and insights gathered across the various project components were presented in a visually-engaging white paper report, presented as a ‘call to action’ to various stakeholders in the RBC space including patient organisations, healthcare professionals, researchers, industry and policymakers.

Impact

The information gathered during this project was used to develop a set of ‘call to action’ recommendations to encourage discussion and help shape future R&D policy in the RBC space, with an aim to address the unmet needs of people living with these complex, debilitating conditions. The wider impact of this work is yet to be determined.