Hosted by MEP Olivier Chastel, the Alpha-1 Foundation (USA) held its annual meeting at the European Parliament in Brussels on the morning of 20 November, bringing together policymakers, researchers, clinicians, and patient organizations to address the most urgent challenges facing the Alpha-1 community. The event also featured the participation of Agnes Cser of the European Economic and Social Committee (EESC).
The meeting took place in the framework of Alpha-1 Awareness Month, observed every November to raise understanding of Alpha-1 Antitrypsin Deficiency (AATD).
It brought together several key speakers in the Alpha-1 field, including Scott Santarella, CEO of the Alpha-1 Foundation; Dr Marc Miratvilles, clinical expert in Alpha-1, chair of the EARCO international registry, and Vice-President of the European Respiratory Society (ERS) and President-Elect for 2026-2027; and Prof Pavel Strnad, representing the ERN Liver – Alpha-1 Liver Network.
Alpha-1 Europe Alliance (A1EA) President, Fernanda Aspilche Ferro, powerfully relayed her experience living with AATD and Flaminia Macchia, A1EA Policy Advisor, voiced the Alliance’s Call to Action.
Opening policy framing: A changing EU landscape
MEP Olivier Chastel opened the session by situating Alpha-1 within the broader EU agenda on rare diseases, plasma supply, biotechnology reform, and the implementation of the new Substances of Human Origin (SoHO) Regulation.
He stressed the need for coordinated policy efforts, noting that Alpha-1 patients continue to face major inequalities in access to diagnosis and treatment across EU member states. Looking ahead to the upcoming Biotech Act, he underlined the importance of ensuring that the needs of rare-disease patients remain central in the evolving regulatory landscape.
Global collaboration in research
For his part, Scott Santarella offered a global overview of ongoing research efforts, noting that around 50% of Alpha-1 scientific output originates in Europe, evidence of the region’s pivotal role in advancing the field.
He reaffirmed the Alpha-1 Foundation’s commitment to supporting research worldwide and emphasized the importance of transatlantic collaboration to accelerate new therapies and improve patient outcomes.
From clinical evidence to better care
In his intervention, Dr Marc Miratvilles outlined the persistent challenges patients face across Europe, particularly under-diagnosis and significant delays in receiving an accurate diagnosis. He noted that although evidence-based guidelines exist, their implementation remains uneven. Strengthening clinician awareness and standardising diagnostic pathways, he explained, would be key to improving early detection and access to appropriate care.
Bringing in the liver perspective, Prof Pavel Strnad highlighted the dual nature of Alpha-1 as a condition affecting both the lungs and the liver. He stressed that AATD related liver disease continues to be under-recognized and called for increased research investment to address this gap. A comprehensive approach that considers both organ systems, he noted, is essential to improving outcomes across the full patient journey.
Presenting the European Call to Action
A central moment of the meeting was the presentation of Alpha-1 Across Europe: The Alpha-1 Europe Alliance’s Call to Action, introduced by Flaminia Macchia.
The document sets out six priority areas to guide European and national action over the next five years:
- Recognition of the specific needs of people living with Alpha-1 and integration of Alpha-1 into rare-disease and chronic-care strategies.
- Earlier and more consistent diagnosis.
- Fair and equitable access to treatment.
- Strengthened plasma supply and governance.
- Enhanced research into both lung and liver manifestations.
- Meaningful involvement of patient communities at all decision-making levels.
“This strategy outlines what needs to happen in AATD to ensure that no European citizen living with AATD is left behind because of where they live. We believe that Europe has the tools and expertise to make a real difference. What we need now is political will and shared commitment.”
She stressed that the moment to act is now, as upcoming EU policy initiatives create a unique window of opportunity for meaningful progress.
Rare disease policy: Ensuring no one is left behind
Agnes Cser presented the European Economic and Social Committee’s opinion “Leaving No One Behind: A European Commitment to Tackling Rare Diseases.”
She highlighted the need for stronger EU-level coordination, firm national commitments, and long-term strategies to ensure that rare disease communities, including those living with Alpha-1, receive adequate and equitable support across the Union. She also reiterated a strong call for the adoption by the European Commission of an EU Action Plan on Rare Diseases.
The patient voice: Living the reality of unequal access
The patient perspective was brought to the forefront by Fernanda Aspilche Ferro, who shared her personal experience of being diagnosed in 2019 and still lacking access to treatment in Belgium, where she lives.
“This means that European citizens like me, living with a rare condition, are left behind by systems that should, in principle, protect us equally,” she stated.
“My story is just one example, but I know it reflects the experience of many others living with Alpha-1 and other rare diseases across Europe. It shows how urgent it is to ensure equal access to diagnosis, to treatment, and to expert care, wherever you live.”
Her testimony captured the core issue at stake: the stark inequalities in access to life-saving therapies depending on where patients live, both between and within EU Member States. It underscored the urgent need for coordinated action to ensure that treatment availability and standards of care do not depend on national borders.
Closing reflections and shared priorities
The meeting concluded with an open exchange among speakers and participants, highlighting a shared commitment to improving outcomes for people living with Alpha-1 through coordinated political, clinical, and research efforts. With upcoming changes in the EU institutions and new health policy frameworks under negotiation, maintaining momentum will be essential to ensure sustained progress.
Learn more about the Call to Action
The Alpha-1 Europe Alliance’s Call to Action sets out a clear roadmap to strengthen diagnosis, care pathways, and equitable access to treatment across Europe.
To explore the priorities and learn how to support this initiative, visit:
