Collaborating With The Rare Disease Patient Community Ipsen Global

Leo Migdal

-Dec 5, 2025, 3:17 PM

collaborating with the rare disease patient community ipsen global

We transform our science to make a meaningful difference to the lives of people living with rare diseases. We collaborate with the rare disease patient community to gather evidence and quality-of-life data that ensures our scientific discoveries, clinical studies and support solutions accurately address their needs. One example of this is our collaboration with the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) and 14 national fibrodysplasia ossificans progressiva (FOP) patient organizations to co-create the first ever survey investigating the true impact... We work together with the rare disease patient community around the world on a range of different projects, across all stages from research and development, clinical study information, and patient support programs. We collaborated with PBCers in the USA, the Canadian PBC foundation, and the UK PBC foundation, on a co-created symposium at the American Association for the Study of Liver Diseases (AASLD), entitled ‘Living with... The symposium raised awareness of the unique needs of people living with PBC among healthcare professionals, emphasizing the importance of looking ‘beyond the chart’ to understand the needs of people living with PBC and...

By integrating insights from both patients and experts, we ensure our initiatives are grounded in real-world experiences and cutting-edge research. Listening to patients and their families is vital for understanding the unique challenges faced by those impacted by rare diseases. By prioritizing these voices, we ensure our initiatives are impactful and tailored to meet the needs of the rare disease community. Needs Assessment: we conducted interviews with 45 rare disease patient organizations across the U.S. and Europe to explore the financial impact for caregivers of children with rare diseases. The report will cover the impact of being a caregiver on work and school, the insurance and out of pocket costs, and the impact on quality of life.

By collaborating with rare disease experts around the world , we ensure that our efforts are grounded in the latest scientific research and clinical expertise. Insights from professionals, including researchers, healthcare providers, and policy expert guide our initiatives, inform best practices, and drive innovation in diagnosis and treatment Webinars with Science/AAAS: bimonthly webinars about rare diseases bring together leading authorities in the field and rare patient voices.. The full webinars are available for free and transcripts are edited and published under the name “Rare Disease Gazette”. By Jennifer Schranz, M.D., Senior Vice President, Global Head, Rare Diseases, Ipsen Ipsen is a global, mid-sized biopharmaceutical company focused on building and expanding a high-value, sustainable pipeline in oncology, rare disease (RD), and neuroscience.

For example, in March 2023, the company completed the acquisition of Albireo (NASDAQ: ALBO), a biotech focused on bile-acid modulators to treat pediatric and adult cholestatic liver disease with an approved RD product, Bylvay. Ipsen has executed an internal and external innovation strategy that prioritizes best-in-class technology platforms and products that can differentiate from existing treatment options or fill unmet patient needs. We spoke with Ipsen’s Senior Vice President and Global Head of Rare Diseases, Dr. Jennifer Schranz, about what she considers best practices in navigating the challenging and fast-changing rare disease space. At Ipsen, we undertake a strategy refresh every two years, which includes a rigorous examination of the rare disease regulatory and policy environment and our evolving portfolio. We set the bar high for internal programs and the acquisition of external assets.

Programs we invest in—or sustain investment in—must be potential game changers based on transformational or disruptive technologies. First-in-class or best-in-class products make the biggest impact for patients and fare best in the current market. We define unmet need with a cross-functional group that includes scientists, clinicians, regulatory, market access, patient affairs, and other experts. We focus on conditions with no or few approved therapies available where there is a well-defined mechanism of action and preclinical data. We hone in on epidemiology to determine the possibility of developing and delivering an innovative treatment in this area. Is it rare or ultra-rare?

Is the natural history of the disease well understood? Are there patient registries? Are there established patient advocacy groups that will provide a patient journey and advise on clinical endpoints? Will we need to conduct a natural history (NH) study or rely on disease registries to inform clinical development? For example, first-in-class products often require an NH study to ensure we have captured endpoints that are both clinically meaningful and relevant to patients, and acceptable to regulatory authorities. We also examine the current standard of care, the landscape of existing and future competitor treatments, and whether there are well-established regulatory and reimbursement pathways.

Finally, we prioritize disease-modifying treatments over symptomatic ones to truly impact the course of the disease. It’s an exhaustive process; it takes discipline and passion to do regularly. But we take it seriously because making wise advancement decisions is critical to the patients whose lives we hope to improve and our sustainability as a company. At Ipsen, our focus and areas of strength in rare diseases include; musculoskeletal (bone), metabolic, endocrine, gastrointestinal (liver) and neuromuscular disorders. Still, any company can apply these concepts to a strategy refresh in their area of focus. Rare diseases impact one in 10 people.

Globally, that is 350-400 million people living with one of over 7,000 identified rare diseases. We are Ipsen in rare disease. We are ordinary people with the extraordinary ambition to improve the health and prolong the lives of people living with rare diseases. At Ipsen, we are dedicated to developing new and transformative treatment options for people living with rare diseases. We use our scientific expertise to develop medicine that have life changing potential for people living with a rare disease, because we believe in being there for those who often get left behind.

We focus our research and innovation in the areas of greatest unmet need and where we can make a meaningful impact for patients. Our priority is advancing medicines and scientific understanding in rare liver disease, rare bone disease and endocrine growth disorders. People living with a rare disease face daily barriers, including delayed diagnoses and delayed or inadequate treatment. The chronic, progressive, degenerative, and frequently life shortening nature of many rare diseases can have a profound impact on quality of life. We’re committed to supporting patients from diagnosis to treatment and partnering with the community to deliver better outcomes for everyone.

Ahead of #RareDiseaseDay, our EVP and Chief Corporate Affairs Officer Josep Catlla shares his reflections on the challenges faced by the rare disease community and his vision for a future shaped by collaboration, innovation,... Let’s keep working together to make the unimaginable possible. #MoreThanYouCanImagine Tomorrow is #RareDiseaseDay—a time to reflect, connect, and take action. This year’s theme, “More Than You Can Imagine,” is a powerful reminder of what we can achieve when we work together. Here are some thoughts on the incredible resilience of the rare disease community.

My respect and admiration to all of them. Their stories inspire us at Ipsen to keep pushing boundaries, because our commitment to rare diseases is… Rare. #MoreThanYouCanImagine https://lnkd.in/eG7rXTZu Received 2024 Feb 2; Accepted 2024 Jun 16; Collection date 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit... The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material.

If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from... To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. People with rare lysosomal storage diseases face challenges in their care that arise from disease complexity and heterogeneity, compounded by many healthcare professionals being unfamiliar with these diseases. These challenges can result in long diagnostic journeys and inadequate care. Over 30 years ago, the Rare Disease Registries for Gaucher, Fabry, Mucopolysaccharidosis type I and Pompe diseases were established to address knowledge gaps in disease natural history, clinical manifestations of disease and treatment outcomes.

Evidence generated from the real-world data collected in these registries supports multiple stakeholders, including patients, healthcare providers, drug developers, researchers and regulators. To maximise the impact of real-world evidence from these registries, engagement and collaboration with the patient communities is essential. To this end, the Rare Disease Registries Patient Council was established in 2019 as a partnership between the Rare Disease Registries and global and local patient advocacy groups to share perspectives on how registry... The Patient Council has resulted in a number of patient initiatives including patient representation at Rare Disease Registries advisory boards; development of plain language summaries of registry publications to increase availability of real-world evidence... The Patient Council is building on the foundations of industry–patient advocacy group collaboration to fully integrate patient communities in decision-making and co-create solutions for the rare disease community. The online version contains supplementary material available at 10.1186/s13023-024-03262-2.

Keywords: Fabry disease, Gaucher disease, Pompe disease, MPS I, Lysosomal storage diseases, Patient communities, Registries, Real-world data, Real-world evidence 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... 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... 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.

Collaborating With The Rare Disease Patient Community Ipsen Global

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