Jennifer Schranz, SVP, Global Head of Rare Diseases
Small populations, high stakes
For years, rare disease research has been shaped by the belief that small patient populations inevitably produce limited evidence, forcing scientific rigor to yield to the constraints of scarcity. This narrative is now being actively challenged. The FDA’s 2025 rare disease guidance framework signaled a clear shift, formally recognizing that single pivotal trials and innovative study designs can meet approval standards even when populations are small. In many ways, constraint has catalyzed a more precise and intentional form of science. This has driven the field toward tighter, more meaningful endpoints, adaptive methodologies and outcome measures engineered to detect real clinical signals from very small numbers, proving that rarity does not have to mean compromise.
When numbers are limited, trial design does what volume cannot
When patient numbers are limited, thoughtful clinical trial design becomes essential. For Ipsen, this starts with designing for the disease itself, with a focus on how it affects patients’ lives, endpoints shaped around that reality and regulatory strategy led by the science. In conditions like primary biliary cholangitis, progressive familial intrahepatic cholestasis and Alagille syndrome, where patients often wait years for an accurate diagnosis, those who engage in research do so as active partners with us, shaping what we measure and how we measure it. In cholestatic liver diseases, that means capturing not only biomarkers of disease activity but the symptoms that define daily life – most notably the crushing fatigue and relentless itch – through validated tools held to the same rigor as any clinical measure. We have seen this approach work; randomized controlled trials across rare cholestatic conditions in both children and adults have already achieved regulatory approvals in the EU, US and beyond.
A new standard, not a lower one
We are witnessing the emergence of a new standard in rare liver disease research – one that Ipsen has helped to shape through more innovative, adaptable scientific approaches. This progress is not defined by incremental change, but by a willingness to challenge established boundaries and rethink how evidence is generated in small populations. The legacy of this work will extend far beyond the therapies available today. It will be seen in the scientific and statistical limits the field was required to push – redefining what rigorous, meaningful evidence can look like in rare disease research. Through this approach, Ipsen is helping to reshape expectations across the rare disease landscape, demonstrating how much more can be achieved when science is designed around both biological complexity and patient reality.
Every person’s experience of living with neuroendocrine tumors (NETs) is different.
Nadia, a 57-year-old mother and chef from Italy, was diagnosed with NETs six years ago.
NETs are a relatively uncommon type of cancer that develops in cells of the neuroendocrine system.¹ These cells are found throughout the body, with NETs commonly developing in areas such as the lungs, gastrointestinal tract and pancreas.² Many NETs grow slowly, although their behavior and symptoms can vary considerably.³ Whatever the type or stage, receiving a cancer diagnosis can have a profound impact on a person’s life.
In this film, Nadia shares her experience in her own words, from the first symptoms that led her to seek medical advice and the moment she received her diagnosis, to how life changed in the years that followed.
Living with NETs is part of Nadia’s story, but it does not define it. She reflects openly on the challenges she has faced, the impact on her and her family, and what matters most to her today. Her story also carries a message of hope: that with appropriate care and support, it is possible to continue living a full and meaningful life.
At Ipsen, hearing directly from people like Nadia helps us better understand the realities of living with cancer. Their experiences and priorities reinforce why we continue to pursue scientific progress focused on what matters most to patients.
Watch Nadia’s story here.
“Analytics is more than a service; it provides knowledge that helps shape strategies and innovation. We might get a request for hundreds of tests, but without these tests, we wouldn’t know if anything needs to change.”
As Director of Analytical Development, her team supports manufacturing, clinical operations, and regulatory submissions and is responsible for developing and validating the methods that define Ipsen’s Quality Control Strategy for its Biologic portfolio of medicines. Those methods determine how purity is measured, how stability is assigned, and how consistency is confirmed.
“That medicine is safe for you to take because my team has developed tests that measure and control quality to ensure a safe and consistent patient experience,” she explains. “We apply criteria to say that every batch is the same.”
Unique to working with one of Ipsen’s emerging medicines within the neuroscience space is the challenge of developing methods with high sensitivity; therefore, several novel and innovative methods have been developed to support the quality control for these products. “As a team, we’ve got that little taster now of what it’s like to be implementing novel and innovative methods as part of our control strategy, and it’s exciting,” she says.
Ipsen’s expanding portfolio in biologics brings new challenges. “We’re going to be at the forefront of developing methods,” she says. “We’re going to be trailblazing the way with new analytics to support new products.”
That work demands more than instruments. It requires collective intelligence. “Every week we all come together as a team,” she says. “If someone’s got a problem, they share it. As a collective, we try and solve it.”
Some team members have decades of experience. Others are just beginning their careers. “There’s no one individual that doesn’t contribute as a team member.”
While the methods her team develops may present raw data in many different forms, Elizabeth’s team takes this information and interprets it to deliver meaningful insights that drive the drug development process forwards. That knowledge her team generates doesn’t just sit in a report; it travels with the medicine, from development to approval, helping to secure safe and effective medicines for our patients.
Rare liver diseases are often complex, progressive and frequently under-recognized, creating significant challenges for people affected by them. At Ipsen, we are focused on changing that narrative. By combining scientific leadership with a deep understanding of the needs of those living with these conditions, we are working to reshape the future of hepatology and help raise standards of care worldwide.
We are currently witnessing a major shift in how we approach the treatment of PBC. For years, clinical guidelines have focused on lowering levels of ALP (an enzyme that serves as a marker of disease activity in PBC) as a measure of how well treatment is working.4,5 Today, that ambition is evolving.
There is growing consensus among experts that aiming for normalization of ALP levels, rather than simply lowering them, is not only achievable but should be the standard target for preserving long-term liver health.1,6 However, managing these biochemical aspects is only half the picture. We are committed to supporting a more holistic approach, where clinicians treat not just the ALP levels, but also the burdensome symptoms like fatigue and pruritus that can profoundly affect a person’s quality of life.7,8
One of the most persistent barriers in rare disease management is the assumption that genetic conditions, such as PFIC and ALGS, only affect children.
This perception often leaves adolescents and adults who have unexplained cholestasis (abnormal flow of bile from the liver) without a clear diagnosis. Expanding awareness, using genetic testing where appropriate, and developing better identification tools could help more people reach an accurate diagnosis and appropriate specialist care.3,4
Our approach goes beyond research; it is rooted in partnership. We are constantly generating real-world evidence, supporting independent medical education, and engaging with patient advocacy groups to ensure the experiences of people living with rare liver diseases directly inform clinical decision-making.
Explore daily insights from The Liver Meeting 2026 from Hugo Gomes da Silva, Senior Vice President, Global Medical Affairs (Rare Disease) at Ipsen, on the congress’ key moments and reflections on the data driving the future of liver care.
Recorded during AASLD The Liver Meeting 2026:
Hear from leading voices in hepatology as they discuss the evolving landscape of rare liver disease – recorded live at The Liver Meeting 2026. Avaiblable soon.
At The Liver Meeting 2026 (AASLD), Ipsen brought together hepatologists, patient advocates and the global PBC community to explore how care can be improved for people living with PBC. This advisory board marked a key milestone in both the global and local effort to identify barriers to optimal care and turn collective insights into meaningful action.
Around 30-40% of patients do not achieve an adequate response to first-line treatment,9 while symptoms such as fatigue, affecting up to 80% of people with PBC, remain under-recognized despite their significant impact on daily life.10
Together, participants examined how earlier diagnosis, better symptom recognition, higher treatment goals and more consistent care pathways could help improve outcomes for people living with PBC. The discussion reinforced a shared ambition across the global PBC community: to translate growing knowledge and emerging evidence into meaningful improvements in patient care.
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Our presence at The Liver Meeting represents our long-standing dedication to the scientific community and our commitment to transforming lives in rare liver disease.
Explore our library of expert sessions, digital posters, and scientific insights from previous meetings.
References
By Sandra Silvestri, EVP and Chief Medical Officer, Ipsen
When we talk about medical innovation, it is easy to focus on the medicine itself. The breakthrough discovery. The clinical trial. The regulatory milestone.
But some of the most important lessons begin long before a medicine reaches a patient and continue for years beyond.
These lessons centre on conversations.
A physician describing the difficult decisions they face when existing treatments fall short. A patient sharing the uncertainty of living with a rare disease. A family explaining how the impact of illness reaches far beyond what can be captured in clinical data. These moments remind us that developing a medicine is only part of the journey.
Throughout my career, I have seen how treatment paradigms evolve through scientific discovery and evolutions in clinical experience. Yet across rare diseases and complex cancers, many patients continue to face limited options, while physicians navigate often impossible trade-offs in pursuit of the best possible outcomes.
This is why deep disease knowledge matters.
Sometimes this points us to building on existing standards of care and helping more patients benefit from scientific progress. In other cases, it means pursuing entirely new approaches to care where patients and physicians simply have nothing to turn to.
While the scientific path may differ, the objective is the same: not simply to fit within today’s treatment paradigms, but to redefine them. However, translating innovation into clinical practice can take time.
Physicians often work within treatment paradigms built on years of experience and established standards of care, particularly in complex diseases where treatment decisions carry significant consequences. Meaningful change therefore requires more than scientific advancement alone. It requires ongoing education, dialogue and partnership with the healthcare community.
By remaining curious and learning alongside physicians, patients and other stakeholders, we can help accelerate paradigm shifts that can ultimately have a transformational impact for patients.
For people living with rare cholestatic liver diseases, progress starts with asking the right questions. Why does cholestasis remain unexplained in some adults? Could a condition traditionally associated with childhood be behind it? Are we setting ambitious enough treatment goals in primary biliary cholangitis (PBC)? And are symptoms such as fatigue being given the attention they deserve?
These are questions where greater understanding could help support better diagnosis, management and care.
At Ipsen, we believe progress comes from working in partnership with the rare liver disease community, including leading clinical experts and people affected by these conditions, to explore what we know, what we still need to learn and where care can continue to evolve.
When cholestasis remains unexplained
Progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS) have traditionally been associated with paediatric care. Yet genetic cholestatic diseases can also be relevant when investigating unexplained cholestasis in adults.
This creates an important challenge: when the cause of cholestasis is unclear, should clinicians look further?
Recognising the clues that could prompt further investigation and the contribution of genetics in unexplained cholestasis in adults are becoming increasingly important. So too is building awareness among adult hepatologists of conditions they may previously have expected to encounter primarily in children.
In conversation leading specialists explore the changing understanding of unexplained cholestasis, the clues that can help clinicians diagnose the specific condition and why finding an explanation matters for patients and their care.
Raising our ambition in PBC
For people living with PBC, another set of questions is shaping how we think about optimal care.
PBC is a chronic, progressive disease requiring ongoing monitoring and management. Biochemical markers, including alkaline phosphatase (ALP), provide important information about disease control. As understanding evolves, the conversation is increasingly moving beyond simply achieving a treatment response towards asking a more ambitious question: can we aim for normal ALP levels and potentially improve long-term outcomes?
But optimal PBC care cannot focus on biomarkers alone.
Fatigue is one of the most commonly reported symptoms of PBC and can have a profound effect on everyday life. Yet it can be difficult to recognise, explain and manage. Listening to how patients describe fatigue, understanding its impact and considering symptoms alongside disease control are therefore essential to delivering truly patient-centred care.
Our second expert conversation explores this whole-person approach, from striving for stronger disease control to recognising fatigue as an important part of the burden of PBC.
Finding answers together
There are still questions to answer across rare cholestatic liver disease. That is precisely why continued research, education, dialogue and partnership matter.
At Ipsen, our ambition is to work alongside the rare liver disease community to keep asking those questions and help turn greater understanding into meaningful progress for patients.
In just four years, the share of electric vehicles in Ipsen’s global company car fleet has grown from 2% in 2022 to 62% in 2026. Meanwhile, fleet-related greenhouse gas emissions fell by 57% between 2022 and 2025.
Behind these results is Fleet for Future, an initiative that is reshaping how Ipsen manages its company vehicles around the world.
Reducing greenhouse gas emissions across its operations and value chain is a priority of Ipsen’s Sustainability Strategy, under its Climate Action focus area. Through its Reduction of our emissions and preservation of vital resources program, Ipsen is working to reduce emissions across its operations and value chain. Initiatives range from improving energy efficiency and increasing renewable electricity use to working with suppliers and reducing emissions associated with transportation, including business travel and employee commuting.
Introduced in 2022, Fleet for Future takes a global approach while recognizing that each market faces different conditions. Where charging infrastructure is available, electric vehicles are prioritized. Where it is still developing, non-plug-in hybrids offer a transitional solution. The initiative also encourages smaller, lighter vehicles suited to actual needs, alongside more energy-efficient driving practices.
This approach has helped increase the share of electric vehicles in Ipsen’s global fleet from 2% in 2022 to 62% in 2026. The next milestone is 70% battery-electric vehicles by 2030.
In September 2026, Cyril Viéville, Global Car Fleet & Mobility Leader at Ipsen, was named Fleet Manager of the Year 2026 by the French publication Le Journal des Flottes.
The award recognizes the work behind this transformation and the contribution of teams across countries and functions.
With more than half of its global company car fleet now electric, Ipsen is continuing the transition, adapting to local conditions and working toward its 2030 target.
Ipsen delivered strong performance in the third quarter of 2026, with total sales growth of 24.6% at constant exchange rates, driven by continued momentum across Oncology, Rare Disease and Neuroscience. With an upgraded full-year outlook and continued investment in innovation, Ipsen remains focused on delivering sustainable growth and bringing innovative medicines to patients worldwide.
Watch the video to discover the key highlights and what lies ahead.
Author : Vanessa Barue
As the Ipsen team departs for #ESMO2026 in Madrid, one area I am particularly interested in is the growing role of real-world data in helping us understand cancer care beyond the clinical trial setting.
Clinical trials are, of course, fundamental to advancing cancer research. But they can never tell us everything. Real-world data can help build a richer picture of what happens when advances in treatment meet the complexity of everyday life for patients, different patient populations, different treatment journeys, and the decisions clinicians and patients face over time.
That feels particularly relevant as I look forward to discussions around renal cell carcinoma and neuroendocrine tumors, where evolving evidence continues to raise important questions about the need for more co-ordinated, patient centered treatment pathways and improved outcomes.
For me, there is a bigger question behind all of this: how do we close the gap between what we learn through research and what people living with cancer actually experience?
That is also why I am looking forward to the conversations beyond the scientific sessions. The ESMO Patient Advocacy Track, which Ipsen is sponsoring for the first time this year, will provide an important opportunity to hear perspectives on access, equity, innovation and the realities of living with cancer.
These conversations should not exist in isolation. Bringing together clinical research, real-world evidence and patients’ experiences can give us a much fuller understanding of where progress is being made and where important gaps remain.
That is the lens I will be taking with me to Madrid: not simply, “What are we learning?” but, “What else do we need to understand to turn scientific progress into meaningful impact for people affected by cancer?”
I look forward to listening, learning and exchanging perspectives at #ESMO2026.
If you are a healthcare professional attending ESMO 2026 and would like to learn more about how we transform Bold Science into Human Impact stop by and say hello.
Illness is something that touches all of us, directly or indirectly. Our parents, our children, our friends, our grandparents, no one is untouched. For Inès Quinty, this reality became deeply personal shortly after she joined the company.
When Personal and Professional Worlds Meet
Not long after starting her role, Inès learned that her mother had cancer.
“It was quite hard to receive that information and to digest,” she recalls.
At that moment, her professional environment and personal life suddenly intersected in a powerful way. Working every day on topics related to cancer took on an entirely new meaning.
“Working on cancer treatment makes definite sense to me.”
A Different Perspective on the Patient Journey
Going through this experience gave Inès a dual perspective: as someone working in the company, and as a family member supporting a patient.
This helped her better understand what patients and their families go through the complexity, the uncertainty, and the emotional weight behind each step. Access to treatment, the variety of options depending on the type and stage of cancer, and the difficulty of finding the right treatment for the right person all became much more tangible.
She also realized how overwhelming the journey can feel.
“Patients can sometimes feel lost.”
Finding Meaning in Contribution
“Working here makes me feel like I have an opportunity, at least, to support. I don’t know if I will change anything, but it relieves me to feel able to play my part.”
That sense of contribution of being able to do something, however small, brings her comfort and purpose.
Inès’s story is a reminder that purpose is often not something abstract. It is shaped by real experiences, real people, and real emotions.
For her, purpose comes from that connection: understanding what patients and families go through, and knowing that her work contributes, in some way, to supporting them.
And sometimes, that’s what makes all the difference.