“My job is to be a guardian of patient safety.”
As Safety Science Director, she works across both early development and post-marketing, ensuring that risks are anticipated, analyzed, and managed so treatments can reach patients responsibly.
Her work currently includes a marketed medicine and another in early development, preparing for the first-in-human trial. “We have to define all the potential risks that we anticipate based on what’s been seen in non-clinical studies and other similar compounds,” she explains. “Once the first patient is treated, GPS is very closely involved in monitoring what happens—every reported adverse event, every side effect.”
That attention does not stop at trial data. “It’s analyzing and interpreting all safety data, whether it comes from clinical trials or the post-marketing side,” she says. “We work to identify any trends or signals that may impact the benefit–risk balance of the medicines. If we do identify something, then we decide what action is needed.”
Her role also extends to Ipsen’s Benefit Risk Decision Board. “It’s the highest level of safety governance,” she says. “Teams bring their data, and the board makes the ultimate decisions. For me, it’s a unique opportunity to understand the important safety decisions being taken for products across the Ipsen portfolio.”
One moment that stands out came during a recent European regulatory submission for an extension of indication. “Part of the strategy was to show that the safety profile was consistent with what was already known for the product,” she recalls. “It was a long journey, but I learned so much along the way, and it was so rewarding when the final decision was approval.”
For Nisha, that is the purpose of pharmacovigilance. “To understand, minimize, and communicate the risks or harmful reactions associated with a medicine, allowing an assessment on the benefit versus the risk to be made,” she says. “What we do is crucial to ensure patients can access safe treatments.”
An article by Pavlos Stampoldis, Medical Affairs Director, Oncology, Ipsen.
Renal cell carcinoma (RCC) has seen remarkable scientific progress over the past decade. Advances in immunotherapy, targeted agents, and combination strategies have reshaped the treatment landscape and transformed outcomes for many patients. Yet for people living with the most common form of kidney cancer, clinical progress alone does not tell the whole story. The International Kidney Cancer Coalition (IKCC) released their 2025 Global Patient Survey, finding that 85% of kidney cancer patients experienced emotional wellbeing challenges, from scan anxiety and strains on interpersonal relationships, to feelings of isolation and uncertainty about the future. Despite this, many never raise these issues with their care team.
For those working in this area every day, the challenge is clear. If the past decade was defined by expanding treatment options, the next must also be defined by how effectively we use then, ensuring patients can access the most appropriate and effective treatment earlier in their journey, while also supporting the people behind the diagnosis and integrating emotional wellbeing into quality RCC care.
RCC is no longer a ‘one size fits all’ disease: subtypes behave and respond differently, presenting unique clinical challenges. For many people, RCC is still diagnosed at an advanced stage, narrowing treatment windows and increasing the urgency of having the right treatment in place as early as possible. While advances in immunotherapy and targeted agents have transformed many patient journeys, outcomes still vary, reinforcing the importance of selecting and initiating the most effective treatment at the right time, rather than delaying optimal care. At the same time, the emotional burden of living with RCC, navigating treatment decisions, managing side effects, and coping with fear of recurrence, is often overlooked within health systems that are primarily structured around clinical endpoints.
Scientific momentum is strong; the challenge now is ensuring that progress translates into real-world impact – supporting clinicians to feel confident in treatment decisions and enabling patients to benefit from effective therapies as early as possible to improve both outcomes and outlooks.
Across global oncology discussions, several shared priorities are increasingly shaping the future of RCC care.
Understanding the Full Patient Experience:
First, we must move beyond clinical outcomes to understand the complete impact of RCC on people’s lives. Traditional evidence frameworks, while essential, rarely capture the emotional and psychological dimensions of the disease. Real-world evidence, patient-reported outcomes, and qualitative research can help close these gaps, offering deeper insight into how patients feel, cope, and what support they need at each stage of their journey.
Embedding Quality of Life into how we Generate Evidence:
Second, the rise of increasingly complex treatment strategies calls for new methodological approaches that place quality of life at their core. Adaptive trial designs, platform studies, and the earlier integration of patient-reported and quality-of-life endpoints offer opportunities to build a more complete picture of treatment value, one that reflects what matters most to patients, not just tumor response.
Keeping Systems in Step with Patient Needs:
Third, regulatory and health technology assessment (HTA) processes must evolve to recognize the full burden of RCC, including its emotional toll, while ensuring that patients can access the most appropriate treatments at the right time in their journey, without unnecessary delays or sequencing barriers. Emerging frameworks, such as joint clinical assessments in Europe, create potential for more aligned, timely evaluations, if implemented in ways that reflect the holistic realities of living with RCC and value emotional wellbeing as a meaningful outcome.
The patient experience must be central to every aspect of care. Living with RCC often means navigating profound uncertainty: treatment choices, side-effect burden, and long-term quality of life. Patients consistently remind us that value is not only clinical — it is also practical, emotional, and deeply personal. Yet far too many people never discuss these struggles with family, friends, or their healthcare teams. As this year’s World Kidney Cancer Day campaign highlights, empowering patients to talk more openly about their emotional wellbeing and ensuring they can access support when they need it is a critical next step for the RCC community.
Progress in RCC depends on partnership. Researchers, clinicians, patient advocates, policymakers and industry each hold part of the solution. Within this evolving landscape, there is a clear opportunity for industry to play a responsible role — supporting clinicians with the evidence, tools and confidence needed to make informed decisions, and working in partnership to help ensure that the right patients receive the right treatment at the right time.
A more joined-up, future-proofed approach would include:
- Care pathways that integrate emotional and psychological support from diagnosis onwards
- Evidence frameworks that capture patient-reported wellbeing alongside clinical endpoints
- Access to psycho-oncology services that reduces geographic disparities in support availability
- Partnership models that embed the patient voice, including emotional needs, from discovery through delivery
The goal is simple: to ensure that every person with RCC, no matter where they live, is supported not only in their treatment but in their emotional wellbeing throughout the entire journey.
Engagement in these debates is not optional. By stepping forward constructively, responsibly, and with humility, organizations can help shape a system that matches the urgency felt by patients and clinicians alike.
The future of RCC care will not be defined by a single breakthrough. It will be defined by how effectively the community ensure existing advances are used to their full potential — reaching patients earlier, improving outcomes, and addressing both the clinical and emotional dimensions of the disease.
If we collectively commit to smarter evidence, stronger partnerships, and a more holistic view of what good care looks like, the next chapter of RCC does not need to resemble the last.
People living with RCC deserve a faster, fairer, more compassionate system — one that enables timely access to the most effective treatments, supports clinicians in making confident decisions, and recognizes patients as whole people, not just diagnoses. Across the oncology ecosystem, we each have a role to play in making that vision a reality.
Mariam Soukouna didn’t expect to be moved. She was attending an internal finance and procurement seminar the kind of event focused on strategy, performance, and operational priorities. But that day, the team welcomed a guest who had nothing to do with any of that: the mother of a child living with a rare disease.
She spoke about her son, about the years of living with a condition for which no treatment existed, and about what Ipsen’s work represented for their family. A potential future. Something to hold on to.
.
For Mariam, it was a heavy testimony to receive. Far removed from slides and dashboards, it was a direct and unfiltered reminder that behind every decision, every contract, every negotiation, there is a patient.
Working in procurement means operating at a distance from patients and healthcare professionals. That distance is structural, not intentional. But it can make it easy to lose sight of the purpose behind the process. That seminar closed the gap.
Since then, Mariam carries that testimony into her daily work. Not as a one-off reminder, but as something that continues to fuel her commitment. Because understanding what your work ultimately serves changes how you show up to do it.
Progressive Familial Intrahepatic Cholestasis (PFIC) can be a complex condition. Finding trusted information about PFIC shouldn’t be. PFIC Colors is designed to help families make sense of these conditions and find the information and support they need, fast.
Progressive familial intrahepatic cholestasis (PFIC) is a group of rare inherited liver conditions that can affect people in different ways.1,2 Symptoms, how the disease progresses and the treatments needed can all vary depending on the different types of PFIC.1
This complexity means PFIC can be difficult for families to understand, especially in the early stages after diagnosis. Families facing a PFIC diagnosis often describe the early days as overwhelming, confusing, and isolating. The condition’s complexity and variable presentation from person to person can make it difficult for families to understand and find information that reflects their lived experience.
It can be a lonely and confusing time, filled with questions and uncertainty. In these moments, having access to trustworthy, community-developed resources can help bring clarity and light to what may otherwise feel like a daunting situation. This is exactly what families can find on the PFIC Network website.
.
Understanding PFIC through color
To help people navigate their way to factual, accurate and supportive information, Ipsen has joined with PFIC Network to develop an engaging and positive campaign that reaches out through digital channels, including social media, to find people who are searching for information on PFIC. Engagement is encouraged with the content using the positive, inspiring and simple concept of talking about PFIC as a disease of many colors.
This color‑inspired initiative is designed to help make sense of PFIC’s complexity by guiding families to the full spectrum of resources available on the PFIC Network website. It aims to bring color back into people’s lives through clearer, more accessible information and meaningful connection with a community that understands their lived experience.
The importance of clarity, connection and community
Emily Ventura, co-founder and executive director of PFIC Network and mother to Cedar, who lives with PFIC, has experienced firsthand how challenging the early stages of diagnosis can be.
Emily shares more about this initiative in the video below, reflecting on why bringing clarity, connection and community to PFIC families is so important.
“When my daughter was diagnosed [with PFIC], back in 2012 we were not given any resources or contacts with any community for support, none existed yet. That left us feeling lonely, isolated and fearful of her diagnosis.”
As Emily connected with other parents who were going through similar experiences, it became clear that many people were navigating PFIC without guidance or support. These shared realities led to the creation of PFIC Network, now a global community offering the full spectrum of trusted information and connection for people living with PFIC and those who care for them.
.
Explore the Colors of PFIC
The PFIC Colors approach is designed to help families navigate PFIC in a clearer, more accessible way. By organizing information into six colour‑coded topic areas and grounding every section in lived experience, the PFIC Network helps families feel more confident and in control as they navigate their PFIC journey.
If you wish to find out more about PFIC, click on the individual topic boxes below to visit the dedicated section of the PFIC Network’s website to learn more.
Diagnosing PFIC
PFIC is usually diagnosed in infants and young children, but also in teens and adults too. At first, you may feel scared, isolated and unsure where to turn – these feelings are normal.
The Itch (Pruritus)
Symptoms can have a huge impact on day-to-day life but can be managed. One of the most common is extreme itch (pruritus).
Types of PFIC
There are currently 13 known types of PFIC, each caused by changes in a different gene. While may symptoms overlap across PFIC types, each type has distinct features that set it apart. More types are being discovered regularly, helping us understand PFIC better and, ultimately, improve care.
PFIC in Adults
PFIC is typically diagnosed in children, but it can affect adults too, sometimes after years of unexplained liver symptoms. It may present differently than in children, which can make diagnosis challenging. Understanding adult experiences with PFIC can help you find the right support and care.
Genetics
PFIC is inherited, meaning it’s passed down through families. Even with the same type of PFIC, people can have very different experiences depending on the specific changes – or mutations – in the gene involved. New mutations are being discovered all the time, helping shape how PFIC is diagnosed, understood, and managed.
Treatment
There are different treatment options to consider, from medications to surgery and liver transplant. Talking about these with your doctor can help you understand the best one for you or your child.
Visit PFIC.org for the full spectrum of information about PFIC.
This campaign was developed together by Ipsen and PFIC Network.
.
References
[1] Baker A, et al. 2019. Systematic review of progressive familial intrahepatic cholestasis. Clin Resh Hepatol Gastroenterol. 43(1):20-36.
[2] Mighiu C, et al. 2022. Impact of progressive familial intrahepatic cholestasis on caregivers: caregiver-reported outcomes from the multinational PICTURE study. Orphanet J Rare Dis. 17(32):1-12.
By Ivan Diaz-Padilla, Senior Vice President and Global Head, Oncology Therapeutic Area Unit, R&D
Acute myeloid leukaemia (AML) remains a difficult blood cancer to treat. Despite progress, relapse is still common and long-term disease control remains out of reach for many, especially older patients or those who cannot receive intensive chemotherapy. As we learn more about AML, we are seeing that if we want to change this, we need to think differently.
From evasion to engagement
Research is revealing a dynamic and complex AML picture, one in which the immune system plays a central role. AML is not simply a disease of cancer cells; it is shaped by a continuous interaction with the body’s immune system. This evolving understanding is shifting how we think about treatment and raises a fundamental question: could engaging and supporting the body’s own immune system open new treatment possibilities?
Using the immune system to treat cancer is not a new idea. But in AML, it has proved challenging, revealing the true extent to which AML not only hides from the immune system but actively weakens it. This has shown we cannot rely on broad immune activation alone. We need strategies that are more precise, more targeted and designed to overcome these barriers AML creates.
Emerging treatment strategies
At Ipsen, we are advancing this thinking by exploring next-generation approaches to mobilize immune responses, including selective activation of specific T cell subsets, with greater precision and control. This marks a shift towards selective immuno-modulation and with it, a more deliberate way to harness the immune system.
Combining these new immune-targeted therapies with established treatments may offer a path to improving efficacy while maintaining tolerability, and to address multiple overlapping pathways impacted by AML.
Looking ahead
The need for new approaches in AML is clear. What is also clear is that the next wave of progress will come from a deeper understanding of the disease as a system.
We believe this means embracing the complexity of immune biology and translating it into targeted, scalable innovation. We do not underestimate the challenge. Progress will require scientific rigour, collaboration and persistence. But the opportunity is real: to move beyond short-term responses and towards more durable outcomes for the patients who need it most.
Philippe Lopes‑Fernandes, Chief Business Officer
Science has changed – the best deals evolve with it
A decade ago, medicine development looked very different. Today, some of the most exciting science is coming from small, focused teams moving at speed – teams working under real operational constraints, with little room for anything that slows them down.
Many conventional deal structures weren’t built for this reality. They prioritize stability at signing: rigid frameworks based on assumptions that may not survive first contact with the data. Science is iterative and unpredictable. When a deal structure and the way partners work together cannot move with it, friction builds and can ultimately get in the way of work.
What I look for in any partnership goes beyond the structure of the deal itself. It is shared purpose in action, ongoing connection between the parties before and long after signing, and an alignment of judgment that holds even when the science moves faster than any contract could anticipate.
The challenge is building something sturdy enough to hold, yet flexible enough to adapt. The best deal structures are not off the shelf. They are built for the partnership in front of you, and that’s where creativity, innovation and an ongoing willingness to evolve come in.
Building creative deal structures
No two partners are the same, so no two deals should be. Our starting point is always genuine understanding: not just what a partner is bringing to the table, but what would actually make the collaboration work for them. Where can our complementary strengths most effectively accelerate the science? Where are the critical decision points? What does success look like at each stage of the science?
The deal structure is the output of that conversation, not a template applied to it. That’s what makes it creative: fit-for-purpose design built around bold, transformative science.
The proof is in the pipeline
Since 2020, Ipsen has built more than 35 programs through partnership. In 2025 alone, three deals I’m particularly proud of – each structured differently, each shaped around the specific science and the specific partner – are unified by one purpose: bold science with the potential to change treatment paradigms.
The future belongs to deal structures that are flexible, personal, and fit for how medicine development is actually done today: lean teams, rapid cycles and real constraints. That’s the standard we hold ourselves to and our pipeline reflects it.
By mobilizing the right partners across our value chain and engaging our employees to move forward together
For a company like Ipsen, environmental impact doesn’t stop at the boundaries of its operations. In fact, that’s only a small part of the picture.
Most of our footprint lies elsewhere across our value chain. It is shaped by the partners we work with, the materials we source, the way our medicines are developed and delivered. Today, around 90% of our emissions fall under Scope 3, reflecting the complexity of the pharmaceutical ecosystem.
This reality is reshaping how we approach sustainability.
In recent years, Ipsen has made significant progress in reducing emissions from its own operations. Scope 1 and 2 emissions have been cut by more than half compared to 2019, driven by improvements in energy efficiency, electrification, and more sustainable operational choices In parallel, we have also taken action on key parts of our Scope 3 footprint, including business travel, fleet, packaging, and employee commuting, areas where change can be implemented more directly. These results demonstrate that progress is already underway, and that growth and environmental responsibility can move forward together.
The next phase is different. It is less about what we can do alone, and more about what we can do together.
We are now expanding our approach to Scope 3, moving from ambition and frameworks to execution with our suppliers and partners. Reducing these emissions requires a shift in how an entire ecosystem operates. It means partnering with our key suppliers to better understand their emissions and to identify where meaningful reductions can be achieved. It means moving beyond estimates to more precise data, enabling targeted and measurable action. And it means embedding sustainability into core business processes across our purchasing process, from strategy to operations.
In a highly regulated industry like pharmaceuticals, change does not happen overnight. Supply chains are complex, and even seemingly simple evolutions such as packaging require time, validation, and regulatory approval. But these constraints do not limit ambition. They define the path forward.
At Ipsen, we believe that progress depends on shared responsibility. It is built through long-term partnerships, continuous improvement, and a willingness to tackle the most complex parts of the challenge.
On World Environment Day, the message is clear: we want to reduce our impact and we act where it matters most. Building on what we have already achieved, we are accelerating action across our value chain.
By Christelle Huguet, EVP & Head of R&D, Ipsen
In biopharmaceutical research, not all challenges are equal. Some of the most meaningful opportunities lie in areas defined by complex biology, limited evidence, and patients who have waited far too long for effective treatment options.
Why we focus where others hesitate
At Ipsen, we have made a deliberate choice to focus on these areas of highest unmet need. They demand deeper scientific insight, greater adaptability, and long-term commitment, but they also offer the greatest potential to deliver truly transformational impact for patients.
High unmet need diseases often fall outside traditional development models, requiring new ways of thinking and working. Patient populations may be small or heterogeneous. Endpoints may be harder to define. Biology may still be unfolding. Yet this is precisely where innovation can be most powerful and where progress can change lives.
Turning scientific ambition into meaningful progress
Taking on these challenges means embracing uncertainty. It pushes us to ask better questions, to work more collaboratively, and to design smarter development programs.
We build deep understanding of disease mechanisms and continuously adapt as new insights emerge. This allows us to develop programs that are both scientifically rigorous and closely aligned with patient needs. By applying this discipline, we can take thoughtful, intentional risks, prioritizing programs with first‑ or best‑in‑class potential and creating new possibilities where few existed before.
Raising expectations through impact
By focusing on the hardest problems, we seek not only to deliver new therapies, but also to help redefine how progress is measured in complex and rare populations. In doing so, we aim to raise expectations, demonstrating that rigorous, patient‑centred innovation is possible even in the most challenging settings.
Choosing the hardest problems is not the simplest path. But for patients with the greatest unmet needs, it is the path that matters most and one we are proud to take in pursuit of scientific progress.
By Sandra Silvestri, for Ipsen.com
In today’s rapidly evolving treatment landscape the approval of a medicine is no longer the finish line; it’s just the beginning. Real world evidence (RWE), post-hoc analyses and structured Phase IV studies all offer new opportunities for scientific learning. At Ipsen, we’re committed to pursuing the unanswered questions, generating robust evidence that goes beyond medicine development, which provides broader long-term evidence-based perspectives for the communities we serve.
The expanding role of RWE
Clinical trials answer essential scientific questions, but by design, they can never fully capture the reality of people’s everyday lives—the diversity of experiences, the complexity of conditions, and the unpredictability of care journeys.
RWE helps bridge that gap. It reflects what really matters to people: how well a medicine works over time, how tolerable it is day-to-day, and how it fits into the realities of living with a condition. By investing in robust RWE, we are deepening our understanding of patients’ needs, capturing the genuine impact of our medicines and ensuring they continue to deliver meaningful benefits for the people who rely on them.
Post-hoc analyses and Phase IV trials: deepening insight where it matters most
Post hoc analyses and Phase IV studies, conducted after a medicine is approved, play an equally important role. Directed by the diversity of patients and the often-varied care pathways, we explore assumptions to generate insights in the context of real-world challenges. How do our medicines impact patients with certain comorbidities or receiving other medications? What other markers may be clinically relevant to investigate? How has the treatment landscape and in turn, treatment guidelines evolved? By taking a focused approach, we’re designing analyses to answer the most pressing topics for clinicians and patients, enriching understanding and supporting more individualized care.
A future defined by continuous learning
Real world insights combined with the depth of Phase IV trials and post-hoc analyses, offer a powerful, multidimensional understanding of a medicine’s impact. For Ipsen, data generation post approval is not an add on, it is a commitment. A commitment to truly understand the impact of our medicines, not just for the many but for the unique individuals who deserve answers.
The future of patient care will depend on this continuous learning mindset. Approval is a milestone worth celebrating, but it is not the end. Every real-world insight, every long-term outcome, every post-approval analysis brings us closer to optimizing care for the people who need it most. Our goal is clear: to ensure that scientific progress does not stop at approval but accelerates through it so that every patient receives not only an innovative medicine, but one that evolves with evidence, precision and purpose.
A diagnosis is supposed to be the end of the uncertainty. For some families, it is also the moment they realize there is nothing more medicine can offer. Loubna Ouriaghli, Head of Government Affairs & Policy at Ipsen, witnessed that moment with her own mother who was diagnosed with a rare disease. She saw the symptoms appear one by one, the years it took to finally have an answer and the silence that followed when that answer came with no treatment.
That feeling of powerlessness became a turning point. Rather than resigning herself to it, Loubna chose to transform it into commitment with her expertise, her position, her skills so that other families, other children like her, would not have to face the same situation. As Loubna is growing a family, this personal perspective is further deepening her commitment to improving outcomes for future generations.
.
Since then, she approaches her role with a clarity that goes beyond professional responsibility. Government Affairs and Policy may not seem like an obvious place for that kind of purpose. It is the work of negotiations, frameworks, and political relationships. But it is also precisely where the gap between a treatment existing and a patient actually receiving it gets decided. Rare disease patients don’t just need treatments to exist, they need those treatments to be recognized, reimbursed, and made accessible. For someone with Loubna’s background, that work carries a weight that goes beyond the professional.
She knows the road is long, and that no single person can change the system alone. But for someone who has seen what is at stake, that is not a reason to step back. It is reason enough to act.