In conversation with the Alexion team working to help rare diseases patients

At the World Orphan Drug Congress 2023, we caught up with some of the Alexion – AstraZeneca Rare Disease team to hear more about rare diseases and how the EU can best support patients.
The Parliament Partner Content

By The Parliament Partner Content

The Parliament Partner Content team works with organisations from across the world to bring their stories to the eyes of policy makers and industry stakeholders across Europe.

11 Dec 2023

At the World Orhpan Drug Congress 2023, we took the time to catch-up with some of the team from Alexion - Astra Zeneca Rare Diseases to hear about how policymakers are supporting the rare disease community.

  • Soraya Bekkali (SB), Senior Vice President, International Commercial Operations.  
  • Cecile De Coster (CDC), Executive Director, Global Regulatory Affairs – Development Strategy 
  • Wendy Erler (WE), Vice President, Global Head Patient Experience and Insights.  
  • Josep Maria Espinalt (JME), VP Alexion Shared Markets  

How reassured are you by the recent focus on rare diseases by policymakers? 

SB: I’m cautiously confident. It is universally acknowledged that there is an unmet need and a health inequity. That needs to be addressed at the highest level, but how this policy attention translates into concrete action is what we hope to see at every step of the process. We hope to partner with policymakers to make sure this happens. Europe owns the legislation and member states own the implementation within their healthcare system so we need to engage on both levels. Decisions made today will have consequences over the next 20 years so we can’t afford for this to remain talk, we need actions.  

WE: It is reassuring that there is high level interest. It means there is possibility that the unique needs of rare disease patients, unique research pathways and access barriers can be better addressed by policy. To achieve health equity for rare disease patients, more needs to be done to align at the National level and ensure policy can be implemented holistically in the member states. Today, much of the burden for advocacy and raising burden of disease awareness rests with the patient communities. We can do more to support them in tangible ways. Today, processes are disjointed and policymakers still work largely independently instead of in partnership. 

CDC: It has been positive seeing interest from the recent European Presidencies to make improvements to the Orphan Drug plan. It has also been a positive to see more reimbursement bodies at the table.  

JME: We saw a commitment from European policymakers but efforts seem to have stalled. The problem may be at a national level.  

Several European Council presidencies have focused on rare diseases, but do you see it as a problem or a positive that they have each faced it from different perspectives?  

SB: It reflects the diversity of perspectives and shows the magnitude of the challenge to get a consensus at a European level. It is an acknowledgement of the challenge to get to an alignment on the diversity of priorities.  

CDC: It can be good to look into different perspectives at the same time. If you only look at one challenge you may miss a perspective. I want to make sure we understand how to fix priorities based on all available perspectives.  

Alexion interviews

What do you believe are the key ways to improve access to medicines across Europe? 

SB: We must be mindful not to disincentivise investment into early research, that’s where innovation comes from. We also recommend not to require access to all 27 markets. Some countries may not see the need for some drugs and it could be an unintended consequence giving hope to patients.  

CDC: Science goes quickly so we need flexibility to keep up with the evolving landscape. It’s also great to put emphasis on patients being at the centre of legislation so we know what is important for them.  

JME: The EU’s work isn’t finalised when EMA has approved a drug. The Commission needs a higher commitment to help countries enable access to their markets for the benefit of rare disease patients. Beyond Europe we need to encourage collaboration between healthcare and political institutions, patients and industry.  

How can we ensure patients are heard at all levels and their input is formally integrated?  

SB: FDA Advisory committees are great to ensure patients are heard. Similarly, we should have patients as part of the EMA discussions, particularly as we now have expert patient groups on a country and European level with an informed understanding of processes. It’s their lives at stake, not ours. Their voice needs to be heard. 

WE: All patient groups want a seat at the table but when you ask ‘what does that mean’ it’s an active, consistent partnership with the drug developer and access to information in a timely and transparent way. We have to engage at a local level, with local patients about a local disease experience as healthcare is local and then take those learnings up to a national level. Patients need access to research and treatment data so they can be well informed to speak about the potential impact of a medicine, not just sharing their current lives experience, but what would a medicine actually mean to them and why. The answer to this is often building infrastructure and providing patients with tools.  

CDC: I was very pleased to hear about initiatives to have patients contributing to reimbursement processes and to see they can be engaged early on.  

JME: Patients are well organised in some markets and not others. They need to be heard at a national level and governments have a responsibility to help patients get organised.  

What are your hopes for the next European Commission and European Parliament mandate? 

SB: We would like to see an integrated, holistic action plan, not only on a European level but one which resonates with, and support national plans for rare diseases.  

WE: We need the ambition that rare diseases are on the main stage of everyone’s collective attention as it’s a worldwide healthcare crisis. Patients are at the forefront of changing healthcare so they need to have a seat at the table.  

CDC: I would ask the Commission to continue being engaged and to continue to prioritise patients so we can make the future sustainable to bring innovative and life transforming treatments to patients. European patients and European innovators must remain at the centre of our strategy. 

JME: We can’t take a step back, we need incentives to innovate. I hope we achieve better alignment amongst member state governments to reduce access inequity.  

What has stood out to you at the World Orphan Drug Congress? 

SB: People are putting their hearts and souls into debates and it’s great to see, it’s not only political jargon being used, we’re seeing real conversations.  

WE: Hearing the collective energy about the need to drive change from a policy perspective and taking rare diseases forward as a shared responsibility has given me hope.  

CDC: I was very inspired about was the focus and recognition of the need to address the price challenge associated with life transforming therapies as people were sitting at the table and willing to talk.  

JME: It’s a good thing to see patients are more organised with greater visibility. We have a long way to go to ensure this is across the board. Another good thing I learned is that China is paying more attention to rare diseases because we can get more data from such a large country with great innovation capacity.  

In partnership with


This article was produced in partnership with Alexion - Astra Zeneca Rare Diseases.

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