After years of discussion, negotiation, and political debate, the European Union has reached a milestone moment for medicines policy. The revision of EU pharmaceutical legislation—first launched by the European Commission in 2020 and formally proposed in 2023—has now reached a political agreement between EU institutions. This reform is widely recognised as the most far-reaching overhaul of Europe’s pharmaceutical framework in more than two decades.
The need for reform has been driven by profound changes in the healthcare landscape since the early 2000s: rapid scientific innovation, growing unmet medical needs, persistent medicine shortages, unequal access across countries, and increasing pressure on health systems. At the same time, recent crises, including the COVID-19 pandemic, exposed vulnerabilities in medicine supply chains and highlighted the importance of stronger EU-level coordination.
Against this backdrop, the European Medicines Agency (EMA) has welcomed the agreement as a critical step toward modernising how medicines are developed, assessed, approved, and supplied across Europe. While much of the public debate has focused on regulation, incentives, and the impact on industry, the ultimate purpose of the reform is to improve outcomes for patients.
Here is what the reform could mean in practical terms for people who rely on medicines every day.
One of the central aims of the new legislation is to reduce the time it takes for medicines to reach patients. Regulatory procedures will be being streamlined, and assessment timelines for new medicines at EU level are expected to shorten. For patients, this means that innovative treatments—particularly those addressing serious or life-threatening conditions—could become available sooner, without compromising safety or scientific standards.
Medicine shortages have become an increasingly visible problem across Europe, directly affecting patients’ ability to continue treatment.
The new legislation strengthens obligations on pharmaceutical companies to:
In addition, a new EU-level focus on “critical medicines” aims to prioritise regulatory and policy action where shortages would have the greatest patient impact.
Patients in different EU member states often experience significant differences in when, and whether, new medicines become available. The reform seeks to reduce these disparities by creating clearer expectations around availability and by aligning incentives with broader EU access. Over time, this could help ensure that patients in smaller or less well-resourced countries are not left behind when new treatments are introduced.
The agreement places greater emphasis on medicines that address unmet medical needs, including treatments for rare diseases, complex conditions, and patient populations that have historically been underserved. New regulatory tools and support mechanisms are intended to encourage the development of innovative therapies, including advanced and personalised medicines that do not always fit neatly into existing regulatory frameworks.
Digitalisation is a key feature of the reform. Product information, such as package leaflets and safety updates, will increasingly be available in electronic formats.
For patients, this should mean:
Importantly, the reforms do not lower the bar for medicine approval. Scientific evaluation, benefit–risk assessment, and post-marketing safety monitoring remain central to the EU system. Patients can expect that medicines approved under the new framework will continue to meet robust standards for quality, safety, and effectiveness.
The legislation also includes measures aimed at protecting long-term patient interests, such as:
These steps are designed to safeguard the effectiveness of treatments not just today, but for future generations.
While the political agreement is an important milestone, the legislation still needs to be formally adopted and implemented. Many of the patient-relevant benefits will depend on how the new rules are applied in practice at EU and national levels.
For patients, the promise of the reform is clear: quicker access to medicines, fewer shortages, fairer availability across Europe, and continued trust in the safety of treatments.
The real test will be whether these ambitions translate into tangible improvements in patients’ everyday experience of care.
Prepared by
Dr. Marina Leroy,
FH Europe Foundation Scientific Communications Manager
The High-Level Meeting on European Research and Innovation for Rare Diseases (HLM Rare 2025) convened senior leaders from science, healthcare, patient organisations, industry, and EU institutions with a clear objective: to move Europe’s rare disease ecosystem from fragmented progress to coordinated delivery.
Led by the Brains for Brain Foundation, the meeting brought together stakeholders spanning clinical research, European Reference Networks (ERNs; learn more here), patient advocacy groups, regulators, and policymakers. The ambition was explicit and shared—to accelerate diagnosis, improve access to therapies, and align investment and policy through a pan-European Declaration that could reshape rare disease research and care across all 27 EU Member States.
The programme was structured around three strategic pillars, each addressed over a dedicated day.
Day 1: Research and innovation. Discussions centred on closing gaps in Europe’s clinical research landscape and strengthening its global competitiveness. Leaders from organisations including the EMA, HaDEA, and EU institutions explored how to streamline clinical development, improve trial readiness, and better connect scientific discovery with patient benefit.
Day 2: Infrastructure and skills. Attention shifted to the foundations needed to sustain innovation at scale, with a strong focus on the ERNs. Sessions addressed data and artificial intelligence, newborn screening, registries, and workforce development. A highlight was a high-level panel featuring Vytenis Andriukaitis, Nathalie Moll, and Professor Maurizio Scarpa, which examined how cross-sector collaboration can underpin a modern, integrated rare disease system in Europe.
Day 3: Policy and funding. The final day focused on enablers—regulatory pathways for earlier patient access, the case for an EU-wide Action Plan for Rare Diseases, and how future EU budgets could be aligned to support these goals. Central to these discussions was the launch of work towards a European Declaration designed to guide priorities, incentives, and resource allocation across Member States.
For individuals and families living with Homozygous Familial Hypercholesterolaemia (HoFH) and Familial Chylomicronaemia Syndrome (FCS), the outcomes of HLM Rare 2025 are particularly relevant. These rare inherited lipid disorders are often marked by delayed diagnosis, limited specialist expertise, and uneven access to advanced therapies.
The meeting’s emphasis on faster diagnosis, data-driven innovation, strengthened ERNs, and more predictable access pathways directly aligns with the unmet needs of the HoFH and FCS community. A more coordinated European approach offers the prospect of earlier identification, more consistent standards of care, and improved access to specialist treatment across borders.
A strong and unified patient voice
Patient organisations played a prominent role throughout the meeting. Retina International and EURORDIS, represented by Avril Daly, stressed the importance of a unified stakeholder community to drive an EU Action Plan or Mission, emphasising that scientific advances must translate rapidly into real-world benefits for patients.
ERN BOND highlighted the significance of the intensive three-day programme, noting that the emerging Declaration represents a concrete step towards aligning research, infrastructure, and access. The International MPS Network reinforced this message, underlining the Declaration’s potential to create clearer innovation pathways and improve access to therapies. Collectively, these contributions reflected a shared commitment to collaboration, acceleration, and patient-centred outcomes.
One of the clearest messages from both the formal agenda and side discussions was that public–private collaboration is no longer theoretical—it is already delivering results and should now be scaled.
Examples cited included EFPIA’s Rare Disease Moonshot, which demonstrates how structured public–private partnerships can address research gaps, accelerate clinical development, and create system-level solutions through aligned incentives and shared infrastructure. Similarly, Together4RD showcased ERN–industry pilots that have moved from concept to implementation. A notable example was the collaboration between ERN EuroBloodNet and Takeda on an AI-supported diagnostic pathway for thrombotic thrombocytopenic purpura (TTP), alongside further pilots on registries and diagnostics discussed at WODC Europe.
The consensus was clear: these “moonshot-style” initiatives are already in motion and generating value. With robust governance, transparency, and data-sharing frameworks, they could be expanded across ERNs to deliver consistent, Europe-wide impact.
Common practices as the backbone of scale
Another consistent theme was the need for common practices and shared platforms as the foundation for progress in rare disease care. Interoperability and harmonisation were repeatedly identified as prerequisites for effective cross-border care and for multinational clinical trials coordinated through the ERNs.
Discussions highlighted the importance of dedicated operating platforms for registries, consent, data standards, and evidence generation. Sessions on the European Health Data Space (EHDS), data-driven innovation, and real-world evidence reinforced the case for interoperable data infrastructures. Leaders from ERNs and collaborative initiatives such as ERDERA and Together4RD described ongoing work to develop templates for partnerships, data reuse, ethics, and conflict-of-interest management—helping shift Europe from isolated pilots to portfolio-level collaboration.
Skills are as strategic as technology
Day 2 also underscored that infrastructure alone is insufficient without a sustainable skills pipeline.
Donata Meroni, Head of Unit B3 ‘Health monitoring and cooperation, Health networks’ at the European Commission, emphasised workforce development and capacity building, pointing to the potential of ERN Academy-style models and structured upskilling for rare disease professionals. Professor Marta Mosca, Coordinator of ERN ReCONNET, illustrated how guidelines, registries, and multidisciplinary care models can be translated into scalable capacity across Member States.
The conclusion was unambiguous: a formalised ERN Academy, harmonised training pathways, and protected, funded time for clinicians are prerequisites for any sustainable EU-wide operating model.
Connecting stakeholders for impact
The fireside chat with Vytenis Andriukaitis, Nathalie Moll, and Professor Maurizio Scarpa—under the theme “Connecting stakeholders for impact: building the infrastructure of a modern rare disease Europe”—distilled many of the meeting’s core messages. The panellists argued for moving decisively beyond fragmented initiatives towards structured public–private partnerships that align goals, data, and infrastructure from the outset, supported by common practices and shared governance.
Looking ahead: alignment with the Cypriot EU Presidency
Building on the momentum generated in Brussels, strengthening collaboration with leaders shaping Europe’s research and health agenda is now a strategic priority. In this context, our CEO had the opportunity to meet with Dr Konstantinos Kleovoulou and Professor Leonidas Phylactou, ahead of the upcoming Cypriot EU Presidency and our own High-Level Meeting under the auspices of Cyprus.
These discussions underscored the importance of continuity between EU-level policy ambition and national leadership in research and innovation. As Cyprus prepares to play a more prominent role in shaping Europe’s agenda, aligning priorities around rare diseases—particularly in areas such as research coordination, data infrastructures, and equitable access to innovation—will be essential to translating the European Declaration from intent into implementation.
From alignment to action
HLM Rare 2025 marked a shift in tone and intent for Europe’s rare disease community. The emerging European Declaration provides a focal point for translating shared ambition into coordinated action—directing resources, aligning incentives, and enabling equitable access to innovation across all Member States.
For rare disease communities, including those affected by HoFH and FCS, the message is clear: Europe is moving from vision to delivery. The challenge now is to sustain momentum and ensure that commitments made in Brussels—and reinforced through upcoming EU Presidencies—translate into measurable improvements in diagnosis, care, and outcomes for patients across Europe.
Prepared by
Elsie (Cindy) Evans,
FH Europe Foundation Ambassador Programme Manager
Recently, US health regulators (the Food and Drug Administration, FDA) green‑lit a new treatment for adults with familial chylomicronaemia syndrome (FCS), a rare and often devastating genetic disease. For people who face this condition every day, the decision represents more than just a new drug on the market. It offers real hope for a life with fewer crises, less fear, and more normality.
FCS is caused by inherited mutations (most commonly affecting the enzyme lipoprotein lipase, or LPL) that impair the body’s ability to break down certain fats carried in the blood called chylomicrons.
As a result, affected individuals often have dangerously high triglyceride levels, sometimes above 880–1,000 mg/dL (much higher than the normal range). This can lead to frequent, severe episodes of acute pancreatitis, chronic abdominal pain, digestive problems, fatigue, “brain fog,” and other complications like diabetes or liver issues.
The condition imposes a heavy daily burden: many patients must follow a very strict, extremely low-fat diet (often less than 15−20 g of fat per day), avoid alcohol and simple sugars, and live with a constant risk of sudden, life-threatening pancreatitis.
Because of its rarity and severity, FCS has historically lacked targeted treatments; a reality that has long left patients and physicians frustrated and anxious.
The newly approved therapy works by silencing a gene that produces a protein called apolipoprotein C-III (apoC-III). In people with FCS, apoC-III contributes to the accumulation of triglyceride-rich chylomicrons by slowing down their clearance from the blood. By reducing the production of apoC-III in the liver, the drug enables much faster breakdown and removal of these fat particles, helping to bring triglyceride levels down dramatically and sustainably.
In a pivotal clinical trial, patients receiving the drug saw a median reduction of around 80% in triglyceride levels, and, more importantly, a significant reduction in the risk of acute pancreatitis, a life-threatening complication.
For people living with FCS, these results could be life-changing. The prospect of fewer hospitalisations, fewer pancreatitis attacks, and a more manageable everyday life could lift a heavy burden. For many families, this approval brings hope for a future that feels closer to normal.
Beyond the numbers, the psychological and social impact may also be profound. Patients often describe FCS as invisible to others but ever-present in their daily lives, with diet restrictions, fear of flare-ups, and anxiety about long-term health. This new therapy could offer relief not only to the body, but also to the mind.
While this approval marks a major advance, it does not eliminate all challenges. The treatment is intended as an adjunct to diet, not a replacement for healthy eating and lifestyle adjustments.
Moreover, long-term real-world experience remains to be gathered: how the therapy performs over many years, how accessible and affordable it will be to patients worldwide, and how individuals will adapt their lives around it.
Still, for a community that has long endured uncertainty, severe restrictions, and life‑threatening crises, this represents a turning point. It is a message that science can, and does, deliver hope.
Read the official press release here.
(this announcement contains information originally published by Arrowhead Pharmaceuticals. We are sharing it for general awareness because it may be relevant to people affected by FCS. This link directs to external content created by Arrowhead Pharmaceuticals and sharing it does not imply endorsement of any product, treatment, or organisation.)
Prepared by
Dr. Marina Leroy,
FH Europe Foundation Scientific Communications Manager
November brought strong progress for FH Europe Foundation, with impactful advocacy, a successful Annual Network Meeting and growing recognition for prevention-focused projects.
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