Industry at ECRD 2024: A conversation with Tamar Thompson, Alexion, AstraZeneca Rare Disease
EURORDIS-Rare Diseases Europe
Working across borders and diseases to improve the lives of all people living with rare diseases.
The 12th European Conference on Rare Diseases and Orphan Products (ECRD 2024) took place in Brussels and online on 15-16 May. We spoke with Tamar Thompson , Vice President, Head of Corporate Affairs of Alexion, AstraZeneca Rare Disease – a top contributor to the conference.?
In our interview, Tamar delves into strategies aimed at improving treatment accessibility for rare disease patients and shortening diagnostic timelines. She also explores how industry, in collaboration with the broader rare disease community, can drive innovative policy changes with potential to significantly impact patients’ lives.?
Why are meetings like the ECRD important for Alexion??
Tamar: At Alexion, we are focused every day on delivering transformative outcomes for patients with rare diseases. But we certainly cannot do this alone. It is critical to collaborate and actively participate in dialogue with a broad network of stakeholders to recognise and address the myriad challenges facing the rare disease community. Bringing everyone together at meetings like ECRD is a key part of those efforts to develop workable policy solutions. ?
The 2024 ECRD took place at a pivotal time in Europe, where ongoing legislative updates promise to have a real impact on people living with rare diseases. ?Key exchanges on the proposed new measures with patient advocates and representatives of the EU and Member States on critical areas of progress include improving diagnosis and an EU Action Plan on rare diseases.?
How can industry collaborate across the stakeholder ecosystem to ensure equitable access to innovative medicines for patients??
Tamar: Addressing health equity challenges will require policymakers and healthcare stakeholders to consider the unique circumstances inherent to rare disease drug development, reimbursement and diagnosis, among other issues, and craft sound policy solutions. Such solutions also should encourage and facilitate patient access to effective screening and diagnostic tools and treatments; encourage the use of digital technology; and be supported by a healthcare infrastructure that encourages appropriate data collection and medical research, the use of real-world evidence and collaboration across communities. ?
These steps go hand-in-hand with the need to enhance outreach and education, especially among healthcare providers, because raising awareness of rare diseases in underserved communities can make a difference in care. Again, this is why ECRD and the work of EURORDIS is so important: to convene and engage with the entire ecosystem, including healthcare professionals and rare disease experts, to expand awareness and understanding of these conditions and the unique challenges they place on patients and their families.?
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How will digital technologies and developments like AI be used to accelerate diagnosis of rare diseases? How else can these innovations be leveraged to expedite drug discovery and development, and improve the rare disease patient experience??
Tamar: It can take five years or more to obtain an accurate rare disease diagnosis. New technologies can play a key role in shortening this ‘diagnostic odyssey’ in a number of ways:?
For example, next generation sequencing can help shorten diagnosis timelines to a matter of weeks when patients are referred at an early stage. This spares patients fromcountless invasive tests, procedures and hospital stays, and can help prevent the potentially catastrophic outcomes of delayed diagnosis and treatment. Since many rare diseases can be severe and progressive, this time saved is critically important.?
Digital tools are making decentralised clinical trials possible, a patient-centric approach that allows for real-time interactions with participants from their own homes. This approach is enabling remote patient data collection and increased use of real-world data, supporting efforts to improve clinical trial accessibility, diversity and retention.?
Telemedicine can help people living in rural, marginalised or underserved communities gain access to the right doctors and specialists who can diagnose, treat and monitor patients remotely. For example, European Reference Networks can connect patients from one country with experts from other countries via telemedicine and reduce some of the patient’s ongoing monitoring and travel challenges. The idea is for the data to travel, rather than the patient. ?
Many digital innovations are extremely promising, but often are being made available in a disparate approach by country and even by state or region. Challenges remain, including timely diagnosis and ensuring health systems have the capabilities to cater to complex challenges posed by rare disease treatment and management. ?
We need to continue working together to drive policy changes that will improve access to diagnostic tools and remote-based care, as well as advance the discovery of new treatments to benefit people living with rare diseases.?
Alexion Pharmaceuticals, Inc. is a top supporter of EURORDIS-Rare Disease Europe’s European Conference on Rare Diseases and Orphan Products (ECRD 2024).