Companies developing drugs in rare disease face well-known challenges in clinical trial readiness and global access strategies. These challenges can include identifying and optimizing clinical trial sites, defining outcome measures, engaging the patient community, and educating HTA bodies and payors on burden of disease and unmet medical need. In response to these challenges, the FSHD Society recently launched Project Mercury – a multi-stakeholder initiative to solve challenges in FSHD clinical trial readiness and therapeutic access through global collaboration. Project Mercury is governed by patient advocacy organizations representing 10 countries, includes representation across FSHD key-opinion leaders, subject-matter experts, and pharmaceutical companies, and is supported by program management from the FSHD Society.
Project Mercury pursues three foundational workstreams:
1. Clinical trial readiness: ensuring effective tools and infrastructure in the clinical community and education and engagement in the patient community.
2. Therapeutic access: producing an effective evidence base for HTA and payor decision-making.
3. Sustainability: equipping the patient community with education and tools to participate in, advocate for, and sustain workstreams #1 and #2.
Since its launch in 2023, Project Mercury has succeeded in creating alignment and even excitement among numerous global stakeholders; launched key collaborative projects, including an update of the FSHD registry dataset, the creation of an FSHD disease model for HTA decision-making, and the creation of training toolkits for patient advocacy organizations; and helped obtain funding for these projects via a collaborative European Union Horizon grant. Altogether, Project Mercury aims to accelerate and de-risk the delivery of therapeutics for FSHD globally. Project Mercury is also a framework that other rare disease organizations can employ to advance similar goals in their disease areas.