No Patient Left Behind: the first 90 days into a Rare Disease Journey

"Leaving No Patient Behind”: Reflecting on 90 Days in a New Role in Rare Disease

The past 90 days have been an incredible journey for me as I transitioned from working in Oncology to Rare Disease, from a large organization to a smaller one, and from a place of large societal awareness (cancer) to a place where patients and their doctors have to struggle for societal awareness and resources. I've spent the past three months learning about the unique challenges faced by rare disease patients, and I'd like to acknowledge and applaud the wealth of rare disease patient insights, understanding and expertise we have within Alexion, AstraZeneca Rare Disease. These last weeks we have also started with my leadership team a strategy journey towards our vision: leave no patient behind in any part of our european countries.

Understanding Rare Diseases

As a trained GP (general practitioner medic) I previously mostly dealt with widespread diseases across the population, while during my latest years in Pharma I have worked mostly in oncology - which is now a very well established area. As such I was not “primed” to address the rare disease space head-on.

Rare Disease is an area in which individual conditions are often so rare that they affect fewer than 1 in 2000[1] individuals (and most of the time 1 in several tens or hundreds of thousands people) – even though taken altogether rare diseases affect more people than cancer and Alzheimer’s combined. These individual diseases - with complex names that most of us, doctors have heard of but mostly forgot them - frequently lack the attention, resources, and treatment options they deserve. I would say that the biggest challenge patients are facing in this space is that we – industry, HCPs, carers, society - don’t know what we don’t know: the diagnostic journey is often too long. The second challenge is that, unlike in the oncology space where global guidelines are in place and quickly updated with each innovation, a very limited number of rare disease management guidelines are available. Treatment management is very diverse, localized and fragmented: patients are cycled through many different physicians and hospitals before ultimately ending up in a specialized treatment center, of which there are few.

The past 90 days have been a deep dive into understanding these unique challenges and the profound impact they have on patients and their families.

Acknowledging Expertise Within Alexion

One of the most remarkable aspects of my journey into Rare Disease has been the incredible wealth of expertise within Alexion, AstraZeneca Rare Disease. I've had the privilege of engaging with numerous stakeholders across the CENE (Central and Northern Europe) region and more broadly the International region, and the dedication I’ve seen to rare disease patients is nothing short of inspiring.

I want to extend a heartfelt shout-out to some of the many individuals I've interacted with during this transition. Soraya Bekkali, MD , your coaching and empowerment have been critical, Wendy Erler your dedication to patients and families has touched my heart. I have been incredibly impressed on how Alexion, AstraZeneca Rare Disease is truly organized to listen, capture and actually act on patients insights in order to transform the lives of those affected by rare diseases. As for my team, spread up across multiple countries in Europe and here in Switzerland, i have just started to appreciate how amazing you all are!

Eager to Implement the Strategy

As I look forward to the next few years, I am full of enthusiasm and determination. Our vision, #NoPatientLeftBehind, encapsulates the essence of our mission in Rare Disease. Connecting the dots between the knowledge gained over the past 90 days and the rest of my experience in the pharmaceutical sector, and building on the support of the incredible experts within Alexion, I am eager to roll out a strategy that strives to improve health equity in Rare Disease in the Central and Northern Europe area.

Our strategy is built on a foundation of patient-centricity, unwavering dedication and authenticity in our purposeful interactions with all stakeholders. We will collaborate across disciplines, leverage cutting-edge technologies, and engage with patient communities to drive meaningful progress. We will push the boundaries of what is possible, not only in terms of continued research and development but especially in terms of full access for each patient.

In the world of Rare Disease, every patient's story is unique. The past 90 days have been a journey of discovery, learning, and appreciation for the particular challenges faced by rare disease patients. I'm deeply grateful for the support and expertise of my colleagues within Alexion, AstraZeneca Rare Disease.

[1] https://research-and-innovation.ec.europa.eu/research-area/health/rare-diseases_en