Read the Conversation
Meeting Highlights:
- Commitment to Rare Diseases – Alexion’s mission is to improve the care of patients with rare diseases, aligning company and personal goals. The company has a strong global footprint with a focus on pioneering complementary biology.
- Strategic Priorities for 2025 – The key objectives include staying mission-focused, preparing for multiple product launches, enhancing diagnostic capabilities, and shaping healthcare policy to improve access to rare disease treatments.
- Challenges in Diagnostics – Diagnosing rare diseases can take up to five years in Germany, despite it being relatively advanced. Efforts to improve this include medical education, AI-driven diagnostic tools, and establishing Centers of Excellence.
- Healthcare Policy & Collaboration – Germany has a strong rare disease framework, but ongoing discussions on AMNOG reform must ensure continued accessibility. Initiatives like “change4RARE” facilitate multi-stakeholder collaboration to improve policy and access.“Magic takes place when people from different perspectives get together to work towards the same goal.”
- Talent & Culture at Alexion – The company attracts and retains top talent through its strong mission-driven culture, collaborative work environment, and professionalism, particularly in Munich’s biotech hub. Having a great talent for rare diseases and building a real and genuine company.
EF: Could you tell us about the mission that drives Alexion?
AP: Our company mission guides everything we do at Alexion. Whether you are in leadership, a sales representative, or a receptionist, everyone inside and outside the office plays a role in advancing our mission—to improve care for people living with rare diseases. This mission is the foundation of all our efforts.
My personal mission aligns with that; my focus is on making an impact. That means ensuring our strategy and actions bring this mission to life and truly improve patients' lives with rare diseases.
EF: What are your top priorities now, and what can we expect from you in 2025?
AP: We have been leaders in rare diseases for 30 years and are pioneers in the field of complement biology. The complement system is a key part of the immune system, and this foundation has shaped our work.
Globally, we have around 5,200 employees. In Germany, we have a strong presence with over 200 employees. Our focus is exclusively on rare diseases, and we provide treatments for seven of them. In Germany, we have six approved therapies and are actively involved in over 20 clinical trials across 120 centers.
Looking ahead, we have bold ambitions to expand our presence. Before discussing Germany specifically, I would like to touch on our global priorities. We aim to maintain and strengthen our leadership in rare diseases by 2030. Our goal is to bring at least five new drugs to market by then. Currently, Alexion operates in 70 countries, separate from AstraZeneca’s footprint, and we plan to expand to more than 100 countries by 2030. That gives a broad overview of Alexion’s global vision.
Regarding our priorities for Germany, it is essential to have a clear and focused set of priorities rather than too many. While we have a substantial presence, we are still a relatively small company compared to larger players like Amgen or BMS, which have thousands of employees. With a team of just 200 people, maintaining focus is key. Our first priority is aimed at staying true to our mission and ensuring that everything we do aligns with Alexion’s purpose.
The second priority is preparing for multiple parallel launches over the next three to four years. Our pipeline includes several products across various indications, all focused exclusively on rare diseases. This will be a demanding period, and we must be ready.
The third priority is improving diagnostics. As you may know, diagnosing rare diseases remains a major challenge in Germany and globally. It can take up to five years for a patient to receive a diagnosis, and even when they do, more than 90% of rare diseases currently have no available treatment. Addressing this issue will be an ongoing priority for us.
The fourth priority is influencing healthcare policy, particularly concerning rare and orphan diseases. Rare diseases come with unique challenges that must be reflected in policy decisions. We are actively working to create a more favorable environment for rare disease treatment access. This is a crucial priority, as it directly impacts the availability of treatments and our mission to improve patients' lives.
EF: What is Alexion doing to enhance diagnostics and the patient journey for those with rare diseases?
AP: The majority of our work in the rare disease space is focused on supporting diagnosis. It is remarkable that, even in Germany, a country with one of the highest diagnostic rates, patients still face an average of nearly five years before receiving a diagnosis. While Germany is ahead of many European and non-European countries in this regard, there is still room for improvement. In countries with fewer resources and less access, patients often go undiagnosed for even longer, and awareness of these diseases is significantly lower.
Improving diagnostic rates accounts for about 80% of our efforts. A major part of this involves education - raising awareness that certain rare diseases exist, how they can be diagnosed, and what testing methods are available, whether genetic or biology-based. We also contribute to developing diagnostic tools, methodologies, and algorithms, including integrating artificial intelligence to analyze data more effectively.
Beyond education and tools, we support the medical community in establishing Centers of Excellence—specialized institutions, often university-based, that focus on specific rare diseases such as aHUS, PNH, or gMG. These centers help advance expertise and improve patient outcomes.
Our efforts also extend to collaboration with multiple stakeholders, including patient organizations, policymakers, and payers, to shape healthcare policies that facilitate earlier and more accurate diagnoses. Since diagnosing rare diseases is inherently complex, it requires coordinated efforts across industries, scientific experts, and policymakers.
In summary, our approach covers multiple areas: education, diagnostic tools (including AI-driven solutions), Centers of Excellence, and healthcare policy shaping. These elements work together to create a more effective and accessible diagnostic landscape for rare diseases.
EF: How do you evaluate rare disease policy in Germany compared to other markets, and what collaborative efforts are you making to help shape it?
AP: As someone with an international perspective, I can offer a broader view. While participating in the change4RARE initiative, I could compare Germany’s rare disease framework with other healthcare systems in which I have worked.
Germany has a strong healthcare system, and its rare disease framework is generally positive. Of course, there is always room for improvement, and that is what we aim to achieve. Compared to other countries, rare diseases are well addressed here, with several advantages that support access and treatment.
One key factor is that once a medicine receives EMA approval, it becomes immediately available to patients, with only minimal administrative steps required. This makes Germany one of the top countries in Europe and globally for access to medicines.
Additionally, Germany’s system includes an orphan drug designation, facilitating market access through the AMNOG process. This designation provides certain advantages, helping ensure that treatments for rare diseases reach patients more efficiently.
Our goal is to maintain these favorable conditions while identifying areas for improvement. Right now, there is ongoing discussion around a potential AMNOG reform, with various perspectives on how it should evolve. We prioritize ensuring that any changes continue supporting facilitated access to rare disease treatments rather than making the process more complex. We want to enhance the system, not make it more restrictive, so patients can continue benefiting from innovative therapies.
The change4RARE initiative serves as a platform, or think tank, where stakeholders from various backgrounds come together to discuss critical healthcare-related topics. The discussions can cover multiple areas, including digitalization, the healthcare system, and rare diseases. The focus is on identifying major challenges, improvement opportunities, and potential solutions that can contribute to strengthening Germany’s healthcare landscape.
What makes this initiative impactful is the collaboration between diverse stakeholders - key opinion leaders, medical experts, patient organizations, policymakers, representatives from the statutory health insurance funds, and industry members. The magic occurs when individuals with different perspectives work together toward a shared goal, fostering meaningful progress in rare disease care and beyond.
The old way of thinking about the healthcare system often involved different groups pursuing separate interests, where one side wins and the other loses. This approach is outdated. The challenges within the healthcare system need to be addressed by focusing on a common goal and collaborating towards that shared vision.
To reach this common goal—improving the healthcare system, ensuring better medication access, or reducing wait times—everyone involved must recognize the importance of aligning their interests. While stakeholders may have different priorities, finding common ground and working together is crucial.
This collaborative approach is the only way forward rather than an "us versus them" mindset. It’s about the intersection, cooperation, and mutual understanding essential to making meaningful progress.
EF: How do you view the potential of AI in the field of rare diseases, and what are the challenges to its implementation?
AP: I am not an AI expert, but I can see the potential for AI in rare diseases. We have studied its use cases, and the possibilities are significant. One challenge, particularly in countries like Germany, is digitalization. For AI to be truly effective, sufficient and consolidated data sources must exist. Once that is addressed, AI applications could be incredibly impactful.
For example, AI can help with predictive algorithms, where you combine various variables to assess potential diagnoses. This is particularly challenging in rare diseases due to limited data and inconsistent information. However, AI algorithms have the potential to improve over time by self-adjusting and refining their predictions. This could make a substantial difference in diagnostics, even with smaller patient populations.
AI can also play a major role in research and development. It can help streamline the R&D process by processing vast amounts of data more efficiently, potentially benefiting rare diseases and other areas.
Another application is in medical education. AI can assist in processing and summarizing complex medical or genetic information. This could make it easier to educate physicians, healthcare professionals, and patient organizations about rare diseases, ultimately improving knowledge sharing and collaboration.
Additionally, we recently partnered with an American company, Envision, to develop an AI algorithm for analyzing echocardiograms. This collaboration aims to accelerate the detection of amyloidosis with cardiac involvement, enabling earlier and faster diagnoses. This is just one example, and there are many other ways AI could revolutionize the field.
EF: Given that rare diseases are a highly specialized field, how do you attract, acquire, and develop talent within the company?
AP: Talent acquisition is a significant topic in the pharmaceutical industry, but it becomes even more crucial in biotech and rare diseases. As a hub, Munich offers great opportunities due to its large talent pool and the presence of many companies. It has become a major biotech hub in Germany and across Europe.
Regarding why people join and stay at Alexion, the core motivation is the mission around rare diseases. We refer to it internally as the "stickiness of the mission." The desire to find solutions for patients with rare diseases draws people in and keeps them engaged. This sense of purpose is essential for attracting talent. Of course, other factors such as location, job type, and compensation also matter, but the main draw is the resonance with our mission.
Additionally, collaboration is another key aspect. Our employees often speak highly of the collaborative environment at Alexion. People describe it as a company where colleagues are approachable, helpful, and supportive, which is a major selling point for attracting talent.
Finally, the professionalism of the people at Alexion sets us apart. With a smaller team of about 200–220 people, we have the luxury of selecting the best candidates on the market. Our reputation is strong, and people know that Alexion is a top company in rare diseases. This reputation positions us well when it comes to attracting talent. Ultimately, a combination of our mission, collaborative culture, professionalism, and leadership enables us to bring in and retain the right caliber of talent.
