We’re a Rare Disease Company in a People Business (original) (raw)
Wendy Erler
Published Feb 28, 2023
In rare disease, there are two sets of data that drive innovation: what the science tells us and what patients and their caregivers say. I’m oversimplifying a complex process, of course, but it comes down to this: to deliver the full potential of medicines for people living with rare diseases, we must rely on scientific rigor— and more - we must take a human-centered, collaborative approach if we want to truly transform their lives and not just their disease.
Everyone agrees that patient centricity is important, but, as a patient once told me, “You’re not patient centric until the patient says you are.” At Alexion, AstraZeneca Rare Disease, we see patients and their caregivers as the real experts in the rare disease experience. And just as we would never endeavor to develop a new therapy without the guidance of research, medical and regulatory experts, we cannot neglect the unique perspectives of those whom we ultimately seek to help. To do so, we set out to establish an organizational framework that would ensure patient and caregiver participation from the earliest stages of drug discovery all the way through commercialization. When we fully committed to this, we changed the paradigm of what it means to be patient centric. How did we get there? Alignment, accountability, and commitment to meaningful collaboration.
Becoming a patient-centric organization requires a fundamental alignment of priorities and a structure that backs it up. I joined Alexion in 2019 because I was believe in this ambition and wanted to both build the tools to carry it out and grow our dedicated global team. This team is focused on fostering long-term partnerships with patient communities centered around personal and authentic connections. Through proprietary frameworks, such as STAR (Solutions To Accelerate Results for patients) data and insights driven workshops, patients and caregivers lead conversations with internal and external stakeholders to share what’s important to them—which is often very different from what we assume is important to them. We then feed those insights to the broader organization to inform our work, pushing us to think differently about what we develop and how we deliver it.
For instance, in a protocol design meeting for a rare neurological disease, we gathered patients, key opinion leaders, a site coordinator, and our own research and development team in the same room to discuss and inform our plans. In reviewing the detailed educational materials we had prepared for the study, patients shared that the use of fewer colors, larger font, and less text made it easier for them to read important information. So, we redesigned our materials based on their feedback. I recognize this is a simple example, but it underscores a critical output of patient centricity in action: understanding the difference between what we think is valuable and what is actually meaningful to the patient community.
The shift from a product- to-patient centered organization requires a shift in mindset as well. The more we learn about the patient and caregiver experience, the more deeply we are able embed their perspectives in our decision-making. That is what led to the development of LEAP (Learn. Evolve. Activate for Patients), an immersive simulation experience that enables employees to walk the rare disease journey from the perspectives of diverse stakeholders, including patients and caregivers, healthcare professionals, and payers. This differentiated approach to learning fosters an empathetic connection to the people we serve and instills a greater sense of urgency in the work that we do by deeply connecting our colleagues to their purpose. On day one of new hire orientation, every new team member at Alexion, from associates to senior executives, goes through the LEAP experience. This is just one example of the tools we have developed to create a radically patient-centric culture, one where we are each held accountable to our commitment to the patient community and our role in the broader rare disease ecosystem.
We often refer to the world of rare disease as an ecosystem and each stakeholder plays a critical role in advancing diagnosis and care. Without the cooperation of every player, we will fall short of our mission to transform lives. While we make every effort to ensure what we learn from patients and caregivers ultimately benefits patients and caregivers, we cannot operate in a silo. Patient and caregiver voices are only loud enough for those who are in the room to hear them, so we must make it a priority to create space for all stakeholders—healthcare providers, regulators, payers, industry sponsors, and advocacy groups—to listen to and learn from them. Whether through consortia, advisory boards, or one of Alexion’s own workshops, engaging in authentic conversations with patients and caregivers enables us to develop meaningful relationships, better understand lived experiences, gain alignment on their needs, and collaborate on the development of products, programs, and policies that elicit meaningful and impactful results. We recognize that we are part of a community!
At a recent STAR workshop with internal and external stakeholders, we had the privilege of meeting and speaking with a woman living with a rare eye disease, which had caused her to lose most of her vision. As a result, she had to step back from her career in nursing management, a change that was as difficult and life-altering as her diagnosis. In speaking and engaging with our teams, she not only challenged us to think differently about the disease itself, but also the impact it has on the someone's entire existence. Today, she has become a vocal advocate in her patient community, using her nursing and management skills in a new way. Most importantly, she told us that she feels seen! Not only are engagements like these incredibly helpful in informing our clinical programs, but they are also deeply meaningful for those involved.
The last several years have been revolutionary for the role of the patient and caregiver in rare disease innovation. Once the last to be engaged at the end of a long, scientific process, they are now co-creators of their medicine from the start. We may be a rare disease company, but we are really in a people business, and I believe through continued partnership with all those in the rare disease community, we can truly transform people’s lives, not just their disease. It takes all of us.