When Project Mercury launched in 2023, the challenge was clear: promising treatments in the pipeline would mean little if the systems needed to deliver them — registries, clinical trial sites, healthcare pathways, and access frameworks — were not ready. No single organization could solve this alone. What was needed was global, structured collaboration.
Two years on, the results speak for themselves.
Shifting the access paradigm
Project Mercury has also succeeded in shifting the patient-access paradigm — from sponsor-led, product-level evidence to advocacy-led, disease-level readiness. Rather than replacing manufacturer-led HTA submissions, this approach is designed to complement them, enabling patient advocacy organizations, clinicians, and manufacturers to engage earlier and more consistently with HTA bodies, payers, and healthcare systems. Work focused on building a shared evidence infrastructure across stakeholders: beginning development of an HTA disease progression model, agreeing on a burden-of-illness evidence roadmap, and assessing health-related quality-of-life measures used in FSHD.
A model built to last
Perhaps as importantly, Project Mercury has demonstrated that global rare disease collaboration can be done efficiently and sustainably. Operating through locally adapted models rather than a single prescriptive approach, the initiative has minimized duplication, reduced start-up costs for participating countries, and maximized the impact of every dollar invested. It has also secured biopharma partnerships and a significant public-private grant through the European Union Innovative Health Initiative.
What comes next
With the first generation of FSHD therapies now entering Phase 3 clinical trials, the focus is firmly on delivery. The work ahead includes completing the FSHD registry dataset update, building a global education resource hub for patients and healthcare providers, finalizing the HTA disease progression model, and helping healthcare systems across participating countries prepare for treatment arrival.
The interim report is available to download [here]. The journey continues — stronger than ever.