In the latest installment of our ‘A Life in Science’ series, we’re excited to introduce Crystel Ogier, our Clinical Biomarker Director, who leads the awareness and implementation of companion diagnostics at Ipsen. Crystel’s work is a prime example of how cutting-edge science is making a real impact in shaping the future of drug development and improving patient outcomes.
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Next in our ‘A Life in Science’ series, Dorinne Desposito, Predictive Toxicology Project Manager, shares her insights on driving innovation through creative thinking and the power of shared learning within our dedicated teams. Dorinne’s work exemplifies how cross-functional collaboration at Ipsen is advancing science, making a meaningful impact, and ensuring that science remains both fun and motivating.
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An article by Philippe Ghyssels, Vice President Corporate and Global Public Affairs
For individuals living with a rare disease, every day without treatment is a day too long. At the World Evidence, Pricing and Access (WEPA) congress in Amsterdam, I participated in a panel discussion on long-term access to rare disease innovation. The conversation reminded me that behind every policy, negotiation, and delay are real people whose lives depend on timely access to treatment.
Innovation in rare diseases has come a long way, but as we continue to make progress, we need to ensure that access keeps pace with innovation. A new treatment can only change lives if it consistently reaches the people who need it, when they need it.
And yet, timely access isn’t always straightforward—rare disease treatments don’t fit neatly into the traditional pharmaceutical development and access model. The orphan drug business model is unique—it requires a thoughtful balance between fostering innovation and making therapies available equitably and without delay. While many advances have been made, there are still areas where we can work together to improve:
- More orphan drugs are being approved in the U.S. than in Europe. This highlights an opportunity to strengthen regulatory and market access pathways in the EU, helping to prevent unnecessary delays in treatment availability.
- Even after EMA approval, access timelines vary widely across Europe. Differences in national pricing and reimbursement (P&R) processes mean some people wait significantly longer than others for the same treatment.
- Rare disease access frameworks are evolving, but challenges remain. Many countries are navigating questions around the role of surrogate endpoints, the use of low-cost comparators, and pricing thresholds—which can sometimes slow down access instead of accelerating it.
Rather than seeing these as barriers, we should view them as opportunities to refine and optimize access pathways—so that innovation translates into impact, faster and more consistently.
How can we move forward? Ensuring that rare disease treatments reach people in a sustainable, long-term way requires collaboration and fresh thinking. For me, key areas of focus include:
- Strengthening incentives for innovation. Policies supporting intellectual property protections, tax incentives, and scientific guidance will drive continued investment in rare disease research and development.
- Fostering early, multi-stakeholder collaboration. When people living with rare diseases, payers, regulators, researchers, and industry engage early, we can create a more predictable and effective pathway to access.
- Exploring new funding models. The traditional P&R process doesn’t always account for the full value of rare disease treatments. We need to continue exploring flexible, innovative funding solutions.
- Embracing flexibility in decision-making. Greater acceptance of surrogate endpoints, real-world evidence, and adaptive approvals can help accelerate access while maintaining high safety and efficacy standards.
- Aligning access frameworks with the realities of rare diseases. More inclusive willingness-to-pay (WTP) thresholds—like NICE’s Highly Specialized Technologies (HST) model—recognize the long-term impact of these therapies and should be considered more broadly.
One of my biggest takeaways from the World EPA Congress is that there is a strong, shared commitment to improving rare disease access. Policymakers, healthcare professionals, regulators, patient groups, and industry are all working toward the same goal—making sure that people with rare diseases can benefit from innovation in a sustainable and timely way.
Rather than focusing on what isn’t working, we should channel our efforts into what we can build together. By aligning incentives, fostering collaboration, and keeping people at the center of decision-making, we can shape a future where rare disease access is no longer a challenge but a given.
The solutions are within reach. Now is the time to make them a reality.
New in our ‘A Life in Science’ series, we hear from Christelle Huguet, Executive Vice President and Head of Research and Development, about what keeps her motivated in the challenging world of pharma. Driven by a deep sense of curiosity and a strong commitment to advancing patient outcomes, her journey is a powerful testament to the incredible impact of a curious mind and science with a purpose.
Every day, millions of people around the world face the immense challenges of living with a rare disease. For these individuals and their families, resilience is essential, and hope becomes a lifeline. Rare Disease Day is a moment to honour their courage, amplify their voices, and reaffirm our shared vision of a future filled with opportunities beyond what anyone affected by a rare disease might have imagined possible.
This year, we embrace the Rare Disease Day theme, “More Than You Can Imagine,” as a call to action for patients, caregivers, advocates, healthcare professionals, policymakers, and industry alike. It serves as a powerful reminder of what is possible when we work together to turn imagination into impact.
Led globally by EURORDIS and supported by countless other organizations, Rare Disease Day serves as a vital platform to spotlight the rare disease community while driving awareness and collective action. Their efforts remind us of the importance of collaboration—not only on this day but throughout the year.
At the heart of this year’s activities is the collective vision of what we can achieve when united by shared purpose. Ipsen employees, inspired by the resilience and determination of those living with rare diseases, came together to share their hopes and dreams for the community. Their perspectives reflect a shared belief that imagining more is not a solitary act—it is a collaboration driven by everyone dedicated to making a difference.
The challenges in rare disease care remain significant. Delayed diagnoses, disparities in access, and barriers to support continue to impact patients around the world. Addressing these issues requires more than innovation; it demands partnerships, advocacy, and a deep understanding of the community’s needs. By working alongside patients, families, and advocates, we strive to ensure their voices shape the future of rare disease care.
Rare Disease Day reminds us of the extraordinary potential we have to make a difference. It is an opportunity to focus not only on what is possible today but on what we can achieve tomorrow. Together, with the rare disease community, we can imagine—and create—a future filled with possibilities beyond anything we can currently imagine.
Because when we imagine more, we can achieve more—not just for the few, but for everyone impacted by rare diseases.
To learn more Rare Disease Day and read stories from the community, visit https://www.rarediseaseday.org/.
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An article by Josep Catlla, Executive Vice President, Chief Corporate Affairs Officer
“Each year, Rare Disease Day is a moment of profound reflection for me. It’s an opportunity to celebrate the resilience of people living with rare diseases, to listen to their stories, and to reaffirm my personal commitment—and Ipsen’s collective commitment—to standing alongside the rare disease community.
This year’s Rare Disease Day Theme ‘More than you Can Imagine’ makes me thing about the complex challenges those living with or caring for a loved one with a rare disease often have to navigate. For many, the journey starts with a delayed diagnosis that can take years to confirm, alongside limited treatment options and disparities in access to care. These obstacles are not confined to one condition or community—they are the lived reality for millions around the world, whether they’re managing a rare liver disease, rare bone condition, or rare cancers.
I recently had the privilege of listening to Francesca, the mother of Eva Luna, a child living with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare cholestatic liver disease. Her strength and perseverance in the face of daily struggles remind me why we do what we do. It’s stories like Francesca’s—and those of Roberta, Stephanie, Gill, Nadine, and so many others that I have heard in recent weeks—that inspire us every day. Their courage to share their experiences and advocate for change motivates us to continue pushing the boundaries of what’s possible.
At Ipsen, we believe in the power of listening. Patients and their families are the true experts in their conditions, and their insights are invaluable. Their lived experiences have shaped our understanding of rare diseases and, in turn, guided our priorities. Through our ongoing collaboration with the Primary Biliary Cholangitis (PBC) community, we co-created patient-reported outcome measures to better capture the impact of symptoms like debilitating fatigue and itching—elements of this rare liver disease that are too often overlooked in traditional clinical assessments. By learning directly from the rare disease community, we are reminded that empathy and collaboration are as vital as innovation in addressing unmet medical needs.
Supporting the rare disease community requires more than goodwill—it calls for meaningful and systemic improvements. Challenges such as policy gaps, delays in accessing therapies, and disparities in treatment options remain areas where progress is needed. In recent times, we are unfortunately seeing some difficult-to-understand delays and barriers in bringing those new medicines to patients in some countries around the world. When the most respected international regulators (FDA and EMA) have validated the safety and efficacy of a new innovative medicine, it is unfortunate that some find additional arguments to further delay patients accessing to new medicines. Unfortunately a rare disease is a rare space for new innovation and it takes a lot of commitment, resources and determination for researchers like those at Ipsen who are fully committed to go the extra mile to bring new therapies in areas of such unmet need.
Ipsen’s commitment to innovation in rare diseases extends beyond developing treatments; it’s about creating a more supportive environment for rare disease innovation and care. Together, as patients, advocates, policymakers, and industry leaders, we must work collectively to ensure that the needs of the rare disease community are prioritized. This isn’t just a challenge—it’s an opportunity to build a future that meets and exceeds their expectations.
On the eve of this year’s Rare Disease Day, my wish for the rare disease community is one of hope, progress, and solidarity. A future where individuals like Luna, Roberta, Stephanie, Gill, and Nadine, and their families receive the care, understanding, and dignity they deserve. A future where barriers are broken down, and the potential for life-changing innovation is fully realised.
“More Than You Can Imagine,” captures the essence of what we’re striving for.
To all those living with a rare disease and their loved ones: thank you for inspiring us to push boundaries every day. And to my colleagues, peers, and partners, let’s come together to reimagine what’s possible—together. Our commitment to rare disease is… Rare.”
#RareDiseaseDay2025 #MoreThanYouCanImagine #Ipsen
Next on our ‘A Life in Science’ series, meet Sandra Silvestri, our Executive Vice President and Chief Medical Officer, as she shares her journey in science—starting with uncertainty but shaped by a deep commitment to bridging research and patient care. Her path highlights the power of following your passions to create meaningful change in healthcare.
How did you find your path in science, and how can we better support women and girls as they navigate their own STEM journeys? How can we contribute to make Ipsen an even more inclusive workplace where we can all be our true selves, embrace “the real us” and thrive together? Share your insights below!
Ipsen is proud to launch our ‘A Life in Science’ campaign, spotlighting the talented and passionate individuals who are making their mark and driving innovation.
Join Anu Connor, our Vice President and Head of Oncology External Innovation, as she kicks off the series by reflecting on her career in science—a journey driven by passion, curiosity, and a dedication to creating solutions that make a lasting impact on patients’ lives.
Our performance in 2024 confirmed that our strategic initiatives are paying off, with significant advancements and robust financial growth across all three therapeutic areas: Oncology, Rare Disease and Neuroscience. We reported strong financial growth, with total sales growth of 9.9% at constant exchange rate, across all three therapy areas. With four regulatory approvals in 2024 in Oncology and Rare Disease, our portfolio continued to expand. To fuel our pipeline, we closed six new external innovation deals.
We made notable advances in our sustainability commitments, reducing our carbon footprint by 45% in Scope 1 and 2 emissions, and we continued to cultivate a global and inclusive culture.
See what CEO David Loew has to say about our 2024 performance.
The accomplishments of 2024 provide a solid foundation for future innovations and advancements. With much more to come in 2025, we are ready to build on this year’s success as we Focus. Together. For patients and society.
Despite progress in gender equality, women remain underrepresented in STEM, making up just 1/3 of the workforce compared to 1/2 in non-STEM fields.1
At Ipsen, we celebrate the legacy of women in science and recognize our role in amplifying the contributions of women for even greater impact.
Watch our video below to learn more about our commitment and join us in encouraging more women and girls to pursue their passions in STEM—pioneering today to lead tomorrow.
Stay tuned for the launch of our ‘A Life in Science’ campaign next week, where we’ll share personal stories from our employees!