For people living with rare diseases, scientific progress matters only when innovation reaches them. Too often, fragmented regulatory, data, and access systems slow that path. Servier Pharmaceuticals recently hosted Pioneering the Path for Rare Diseases: A Transatlantic Policy and Innovation Summit at the Embassy of France in Washington, D.C., to help close those gaps. By convening researchers, regulators, policymakers, patient advocates, and other leaders from across sectors and around the world, Servier created a patient-centered forum to strengthen transatlantic collaboration, elevate patient voices, and identify practical ways to accelerate transformative innovations to the people who need them most.

Watch highlights from Servier’s Pioneering the Path for Rare Diseases: A Transatlantic Policy and Innovation Summit. 

During the summit, stakeholders from across the healthcare ecosystem identified three connected policy priorities for accelerating the path from scientific discovery to patient access: 

• Increase Regulatory Alignment: One central theme emerged – the urgent need for regulatory alignment across Europe and the United States, where appropriate. Earlier dialogue among regulators, investigators, patients, and sponsors combined with greater consistency in data requirements and selection of meaningful endpoints, can support global development programs that satisfy both local and multinational requirements. This can reduce avoidable duplication and ultimately, help accelerate clinical trials.  

• Improve Cross-Border Data Sharing: Participants also called for responsible, privacy-protective approaches to cross-border data sharing. Interoperable natural history databases and the exchange of real-world evidence across Europe and the U.S. could reduce duplication and better inform clinical trials. Fit-for-purpose evidence requirements and trial models designed around the realities of rare disease can preserve scientific rigor while reducing burdens on patients and families. 

• Modernizing Reimbursement to Speed Equitable Access: Advancing access to rare disease innovation requires long-term strategies for patient access and reimbursement. Approval alone does not guarantee impact for patients. Stakeholders discussed the necessity of modernizing reimbursement frameworks to reflect the unique clinical, evidentiary and economic realities of the development of rare disease therapeutics. Incorporating patient-centered outcomes and real-world evidence into value assessments ensures that once a transformative therapy is approved, it can rapidly and equitably reach those in need. 

While each priority addresses a different part of the healthcare ecosystem, they share one measure of success: making the journey to diagnosis, clinical research, approval, and access faster and less burdensome for patients and families. For Servier, the lived experience of the patient is the foundation of all that we do. Our drive to improve how innovation reaches those who need it is fueled by patients’ real-life challenges. For many people living with a rare disease, the diagnostic odyssey can take and average of 5 – 7 years and involve numerous time-consuming and often costly medical encounters.¹ 

At the Summit, patient advocates openly shared their personal journeys, underscoring the impact fragmented healthcare systems have on their lives. Their stories reinforced the central principle of the day’s discussions: that policies must be shaped in partnership with patients, ensuring that their voices lead the conversation in shaping the future of rare disease care.  

As a company with a strong global presence backed by a non-profit foundation, Servier is uniquely positioned to strengthen transatlantic collaboration and help carry this important dialogue forward. Convening stakeholders who share a commitment to patients was an important step; the opportunity now is to turn alignment into sustained action. At the opening of the summit, Servier Pharmaceuticals CEO David K. Lee called on attendees to leave with one new connection and one commitment to act. Whether that action means continuing dialogue across borders and sectors, advancing patient-informed policy solutions, or building new partnerships, each step can help move innovation more efficiently from discovery to access. Scientific breakthroughs alone are not enough. Servier will continue advocating for science-based regulatory alignment and data sharing, modernized value and access models, and robust transatlantic partnerships so that global innovation better serves patients and their families. 

This event came together with the strong partnership of the Embassy of France. Servier thanks all of our speakers, panelists, and attendees for contributing to a productive and impactful summit.