Thirty-six million Europeans — including more than one million in the Nordics[1] — live with a rare disease.[2] For patients and their families, this is not just a medical challenge; it is a human rights issue. Diagnostic delays mean years of worsening health and needless suffering. Where treatments exist, access is far from guaranteed. Meanwhile, breakthroughs in genomics, AI and targeted therapies are transforming what is possible in health care. But without streamlined systems, innovations risk piling up at the gates of regulators, leaving patients waiting. Even the Nordics, which have some of the strongest health systems in the world, struggle to provide fair and consistent access for rare-disease patients. Expectations should be higher. THE BURDEN OF DELAY The toll of rare diseases is profound. People living with them report health-related quality-of-life scores 32 percent lower than those without. Economically, the annual cost per patient in Europe — including caregivers — is around €121,900.[3] > Across Europe, the average time for diagnosis is six to eight years, and > patients continue to face long waits and uneven access to medications. In Sweden, the figure is slightly lower at €118,000, but this is still six times higher than for patients without a rare disease. Most of this burden (65 percent) is direct medical costs, although non-medical expenses and lost productivity also weigh heavily. Caregivers, for instance, lose almost 10 times more work hours than peers supporting patients without a rare disease.[4] This burden can be reduced. European patients with access to an approved medicine face average annual costs of €107,000.[5] Yet delays remain the norm. Across Europe, the average time for diagnosis is six to eight years, and patients continue to face long waits and uneven access to medications. With health innovation accelerating, each new therapy risks compounding inequity unless access pathways are modernized. PROGRESS AND REMAINING BARRIERS Patients today have a better chance than ever of receiving a diagnosis — and in some cases, life-changing therapies. The Nordics in particular are leaders in integrated research and clinical models, building world-class diagnostics and centers of excellence. > Without reform, patients risk being left behind. But advances are not reaching everyone who needs them. Systemic barriers persist: * Disparities across Europe: Less than 10 percent of rare-disease patients have access to an approved treatment.[6] According to the Patients W.A.I.T. Indicator (2025), there are stark differences in access to new orphan medicines (or drugs that target rare diseases).[7] Of the 66 orphan medicines approved between 2020 and 2023, the average number available across Europe was 28. Among the Nordics, only Denmark exceeded this with 34. * Fragmented decision-making: Lengthy health technology assessments, regional variation and shifting political priorities often delay or restrict access. Across Europe, patients wait a median of 531 days from marketing authorization to actual availability. For many orphan drugs, the wait is even longer. In some countries, such as Norway and Poland, reimbursement decisions take more than two years, leaving patients without treatment while the burden of disease grows.[8] * Funding gaps: Despite more therapies on the market and greater technology to develop them, orphan medicines account for just 6.6 percent of pharmaceutical budgets and 1.2 percent of health budgets in Europe. Nordic countries — Sweden, Norway and Finland — spend a smaller share than peers such as France or Belgium. This reflects policy choices, not financial capacity.[9] If Europe struggles with access today, it risks being overwhelmed tomorrow. Rare-disease patients — already facing some of the longest delays — cannot afford for systems to fall farther behind. EASING THE BOTTLENECKS Policymakers, clinicians and patient advocates across the Nordics agree: the science is moving faster than the systems built to deliver it. Without reform, patients risk being left behind just as innovation is finally catching up to their needs. So what’s required? * Governance and reforms: Across the Nordics, rare-disease policy remains fragmented and time-limited. National strategies often expire before implementation, and responsibilities are divided among ministries, agencies and regional authorities. Experts stress that governments must move beyond pilot projects to create permanent frameworks — with ring-fenced funding, transparent accountability and clear leadership within ministries of health — to ensure sustained progress. * Patient organizations: Patient groups remain a driving force behind awareness, diagnosis and access, yet most operate on short-term or volunteer-based funding. Advocates argue that stable, structural support — including inclusion in formal policy processes and predictable financing — is critical to ensure patient perspectives shape decision-making on access, research and care pathways. * Health care pathways: Ann Nordgren, chair of the Rare Disease Fund and professor at Karolinska Institutet, notes that although Sweden has built a strong foundation — including Centers for Rare Diseases, Advanced Therapy (ATMP) and Precision Medicine Centers, and membership in all European Reference Networks — front-line capacity remains underfunded. “Government and hospital managements are not providing  resources to enable health care professionals to work hands-on with diagnostics, care and education,” she explains. “This is a big problem.” She adds that comprehensive rare-disease centers, where paid patient representatives collaborate directly with clinicians and researchers, would help bridge the gap between care and lived experience. * Research and diagnostics: Nordgren also points to the need for better long-term investment in genomic medicine and data infrastructure. Sweden is a leader in diagnostics through Genomic Medicine Sweden and SciLifeLab, but funding for advanced genomic testing, especially for adults, remains limited. “Many rare diseases still lack sufficient funding for basic and translational research,” she says, leading to delays in identifying genetic causes and developing targeted therapies. She argues for a national health care data platform integrating electronic records, omics (biological) data and patient-reported outcomes — built with semantic standards such as openEHR and SNOMED CT — to enable secure sharing, AI-driven discovery and patient access to their own data DELIVERING BREAKTHROUGHS Breakthroughs are coming. The question is whether Europe will be ready to deliver them equitably and at speed, or whether patients will continue to wait while therapies sit on the shelf. There is reason for optimism. The Nordic region has the talent, infrastructure and tradition of fairness to set the European benchmark on rare-disease care. But leadership requires urgency, and collaboration across the EU will be essential to ensure solutions are shared and implemented across borders. The need for action is clear: * Establish long-term governance and funding for rare-disease infrastructure. * Provide stable, structural support for patient organizations. * Create clearer, better-coordinated care pathways. * Invest more in research, diagnostics and equitable access to innovative treatments. Early access is not only fair — it is cost-saving. Patients treated earlier incur lower indirect and non-medical costs over time.[10] Inaction, by contrast, compounds the burden for patients, families and health systems alike. Science will forge ahead. The task now is to sustain momentum and reform systems so that no rare-disease patient in the Nordics, or anywhere in Europe, is left waiting. -------------------------------------------------------------------------------- [1] https://nordicrarediseasesummit.org/wp-content/uploads/2025/02/25.02-Nordic-Roadmap-for-Rare-Diseases.pdf [2] https://nordicrarediseasesummit.org/wp-content/uploads/2025/02/25.02-Nordic-Roadmap-for-Rare-Diseases.pdf [3] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [4] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [5] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf [6] https://www.theparliamentmagazine.eu/partner/article/a-competitive-and-innovationled-europe-starts-with-rare-diseases? [7] https://www.iqvia.com/-/media/iqvia/pdfs/library/publications/efpia-patients-wait-indicator-2024.pdf [8] https://www.iqvia.com/-/media/iqvia/pdfs/library/publications/efpia-patients-wait-indicator-2024.pdf [9] https://copenhageneconomics.com/wp-content/uploads/2025/09/Copenhagen-Economics_Spending-on-OMPs-across-Europe.pdf [10] https://media.crai.com/wp-content/uploads/2024/10/28114611/CRA-Alexion-Quantifying-the-Burden-of-RD-in-Europe-Full-report-October2024.pdf Disclaimer POLITICAL ADVERTISEMENT * The sponsor is Alexion Pharmaceuticals * The entity ultimately controlling the sponsor: AstraZeneca plc * The political advertisement is linked to policy advocacy around rare disease governance, funding, and equitable access to diagnosis and treatment across Europe More information here.

This article is presented by EFPIA with the support of AbbVie I made a trip back to Europe recently, where I spent the vast majority of my pharmaceutical career, to share my perspectives on competitiveness at the European Health Summit. Now that I work in a role responsible for supporting patient access to medicine globally, I view Europe, and how it compares internationally, through a new lens, and I have been reflecting further on why the choices made today will have such a critical impact on where medicines are developed tomorrow. Today, many patients around the world benefit from medicines built on European science and breakthroughs of the last 20 years. Europeans, like me, can be proud of this contribution. As I look forward, my concern is that we may not be able to make the same claim in the next 20 years. It’s clear that Europe has a choice. Investing in sustainable medicines growth and other enabling policies will, I believe, bring significant benefits. Not doing so risks diminishing global influence. > Today, many patients around the world benefit from medicines built on European > science and breakthroughs of the last 20 years I reflect on three important points: 1) investment in healthcare benefits individuals, healthcare and society, but the scale of this benefit remains underappreciated; 2) connected to this, the underpinning science for future innovation is increasingly happening elsewhere; and 3) this means the choices we make today must address both of these trends. First, let’s use the example of migraine. As I have heard a patient say, “Migraine will not kill you but neither [will they] let you live.”[1] Individuals can face being under a migraine attack for more than half of every month, unable to leave home, maintain a job and engage in society.[2] It is the second biggest cause of disability globally and the first among young women.[3] It affects the quality of life of millions of Europeans.[4] From 2011-21 the economic burden of migraine in Europe due to the loss of working days ranged from €35-557 billion, depending on the country, representing 1-2 percent of gross domestic product (GDP).[5]   Overall socioeconomic burden of migraine as percentage of the country’s GDP in 2021 Source: WifOR, The socioeconomic burden of migraine. The case of 6 European Countries.5 Access to effective therapies could radically improve individuals’ lives and their ability to return to work.[6] Yet, despite the staggering economic and personal impacts, in some member states the latest medicines are either not reimbursed or only available after several treatment failures.[7] Imagine if Europe shifted its perspective on these conditions, investing to improve not only health but unlocking the potential for workforce and economic productivity? Moving to my second point, against this backdrop of underinvestment, where are scientific advances now happening in our sector? In recent years it is impressive to see China has become the second-largest drug developer in the world,[8] and within five years it may lead the innovative antibodies therapeutics sector,[9] which is particularly promising for complex areas like oncology. Cancer is projected to become the leading cause of death in Europe by 2035,[10] yet the continent’s share of the number of oncology trials dropped from 41 percent in 2013 to 21 percent in 2023.10 Today, antibody-drug conjugates are bringing new hope in hard-to-treat tumor types,[11] like ovarian,[12] lung[13] and colorectal[14] cancer, and we hope to see more of these advances in the future. Unfortunately, Europe is no longer at the forefront of the development of these innovations. This geographical shift could impact high-quality jobs, the vitality of Europe’s biotech sector and, most importantly, patients’ outcomes. [15] > This is why I encourage choices to be made that clearly signal the value > Europe attaches to medicines This is why I encourage choices to be made that clearly signal the value Europe attaches to medicines. This can be done by removing national cost-containment measures, like clawbacks, that are increasingly eroding the ability of companies to invest in European R&D. To provide a sense of their impact, between 2012 and 2023, clawbacks and price controls reduced manufacturer revenues by over €1.2 billion across five major EU markets, corresponding to a loss of 4.7 percent in countries like Spain.[16] Moreover, we should address health technology assessment approaches in Europe, or mandatory discount policies, which are simply not adequately accounting for the wider societal value of medicines, such as in the migraine example, and promoting a short-term approach to investment. By broadening horizons and choosing a long-term investment strategy for medicines and the life science sector, Europe will not only enable this strategic industry to drive global competitiveness but, more importantly, bring hope to Europeans suffering from health conditions. AbbVie SA/NV – BE-ABBV-250177 (V1.0) – December 2025 -------------------------------------------------------------------------------- [1] The Parliament Magazine, https://www.theparliamentmagazine.eu/partner/article/unmet-medical-needs-and-migraine-assessing-the-added-value-for-patients-and-society, Last accessed December 2025. [2] The Migraine Trust; https://migrainetrust.org/understand-migraine/types-of-migraine/chronic-migraine/, Last accessed December 2025. [3] Steiner TJ, et al; Lifting The Burden: the Global Campaign against Headache. Migraine remains second among the world’s causes of disability, and first among young women: findings from GBD2019. J Headache Pain. 2020 Dec 2;21(1):137 [4] Coppola G, Brown JD, Mercadante AR, Drakeley S, Sternbach N, Jenkins A, Blakeman KH, Gendolla A. The epidemiology and unmet need of migraine in five european countries: results from the national health and wellness survey. BMC Public Health. 2025 Jan 21;25(1):254. doi: 10.1186/s12889-024-21244-8. [5] WifOR. Calculating the Socioeconomic Burden of Migraine: The Case of 6 European Countries. Available at: [https://www.wifor.com/en/download/the-socioeconomic-burden-of-migraine-the-case-of-6-eu­ropean-countries/?wpdmdl=358249&refresh=687823f915e751752703993]. Accessed June 2025. [6] Seddik AH, Schiener C, Ostwald DA, Schramm S, Huels J, Katsarava Z. Social Impact of Prophylactic Migraine Treatments in Germany: A State-Transition and Open Cohort Approach. Value Health. 2021 Oct;24(10):1446-1453. doi: 10.1016/j.jval.2021.04.1281 [7] Moisset X, Demarquay G, et al., Migraine treatment: Position paper of the French Headache Society. Rev Neurol (Paris). 2024 Dec;180(10):1087-1099. doi: 10.1016/j.neurol.2024.09.008. [8] The Economist, https://www.economist.com/china/2025/11/23/chinese-pharma-is-on-the-cusp-of-going-global, Last accessed December 2025. [9] Crescioli S, Reichert JM. Innovative antibody therapeutic development in China compared with the USA and Europe. Nat Rev Drug Discov. Published online November 7, 2025. [10] Manzano A., Svedman C., Hofmarcher T., Wilking N.. Comparator Report on Cancer in Europe 2025 – Disease Burden, Costs and Access to Medicines and Molecular Diagnostics. EFPIA, 2025. [IHE REPORT 2025:2, page 20] [11] Armstrong GB, Graham H, Cheung A, Montaseri H, Burley GA, Karagiannis SN, Rattray Z. Antibody-drug conjugates as multimodal therapies against hard-to-treat cancers. Adv Drug Deliv Rev. 2025 Sep;224:115648. doi: 10.1016/j.addr.2025.115648. Epub 2025 Jul 11. PMID: 40653109.. [12] Narayana, R.V.L., Gupta, R. Exploring the therapeutic use and outcome of antibody-drug conjugates in ovarian cancer treatment. Oncogene 44, 2343–2356 (2025). https://doi.org/10.1038/s41388-025-03448-3 [13] Coleman, N., Yap, T.A., Heymach, J.V. et al. Antibody-drug conjugates in lung cancer: dawn of a new era?. npj Precis. Onc. 7, 5 (2023). https://doi.org/10.1038/s41698-022-00338-9 [14] Wang Y, Lu K, Xu Y, Xu S, Chu H, Fang X. Antibody-drug conjugates as immuno-oncology agents in colorectal cancer: targets, payloads, and therapeutic synergies. Front Immunol. 2025 Nov 3;16:1678907. doi: 10.3389/fimmu.2025.1678907. PMID: 41256852; PMCID: PMC12620403. [15] EFPIA, Improving EU Clinical Trials: Proposals to Overcome Current Challenges and Strengthen the Ecosystem, efpias-list-of-proposals-clinical-trials-15-apr-2025.pdf, Last accessed December 2025. [16] The EU General Pharmaceutical Legislation & Clawbacks, © Vital Transformation BVBA, 2024.

As Europe redefines its life sciences and biotech agenda, one truth stands out: the strength of our innovation lies in its interconnection between human and animal health, science and society, and policy and practice. This spirit of collaboration guided the recent “Innovation for Animal Health: Advancing Europe’s Life Sciences Agenda” policy breakfast in Brussels, where leading voices from EU politics, science and industry came together to discuss how Europe can turn its scientific excellence into a truly competitive and connected life sciences ecosystem. Jeannette Ferran Astorga / Via Zoetis Europe’s role in life sciences will depend on its ability to see innovation holistically. At Zoetis we firmly believe that animal health innovation must be part of that equation, as this strengthens resilience, drives sustainability, and connects directly to the wellbeing of people. Innovation without barriers Some of humanity’s greatest challenges continue to emerge at the intersection of human, animal and environmental health, sometimes with severe economic impact. The recent outbreaks of diseases like avian influenza, African swine fever and bluetongue virus act as reminders of this. By enhancing the health and welfare of animals, the animal health industry and veterinarians are strengthening farmers’ livelihoods, supporting thriving communities and safeguarding global food security. This is also contributing to protecting wildlife and ecosystems. Meanwhile, companion animals are members of approximately half of European households. Here, we have seen how dogs and cats have become part of the family, with owners now investing a lot more to keep their pets healthy and able to live to an old age. Because of the deepening bonds with our pets and their increased longevity, the demand for new treatment alternatives is rising continuously, stimulating new research and innovative solutions making their way into veterinary practices. Zoonotic diseases that can be transferred between animals and humans, like rabies, Lyme disease, Covid-19 and constantly new emerging infectious diseases, make the rapid development of veterinary solutions a necessity. Throughout the world, life sciences are an engine of growth and a foundation of health, resilience and sustainability. Europe’s next chapter in this field will also be written by those who can bridge human and animal health, transforming science into solutions that deliver both economic and societal value. The same breakthroughs that protect our pets and livestock underpin the EU’s ambitions on antimicrobial resistance, food security and sustainable agriculture. Ensuring these innovations can reach the market efficiently is therefore not a niche issue, it is central to Europe’s strategic growth and competitiveness. This was echoed at the policy event by Dr. Wiebke Jansen, Policy Lead at the Federation of Veterinarians of Europe (FVE) when she noted that ‘innovation is not abstract. As soon as a product is available, it changes the lives of animals, their veterinarians and the communities we serve. With the many unmet needs we still face in animal health, having access to new innovation is an extremely relevant question from the veterinary perspective.’ Enabling innovation through smart regulation To realize the promise of Europe’s life sciences and biotech agenda, the EU must ensure that regulation keeps pace with scientific discovery. The European Commission’s Omnibus Simplification Package offers a valuable opportunity to create a more innovation-friendly environment, one where time and resources can be focused on developing solutions for animal and human health, not on navigating overlapping reporting requirements or dealing with an ever increasing regulatory burden. > In animal health, biotechnology is already transforming what’s possible — for > example, monoclonal antibodies that help control certain chronic conditions or > diseases with unprecedented precision. Reviewing legislative frameworks, developing the Union Product Database as a true one-stop hub or introducing digital tools such as electronic product information (e-leaflets) in all member states, for instance, would help scientists and regulators alike to work more efficiently, thereby enhancing the availability of animal health solutions. This is not about loosening standards; it is about creating the right conditions for innovation to thrive responsibly and efficiently. Science that serves society Europe’s leadership in life sciences depends on its ability to turn cutting-edge research into real-world impact, for example through bringing new products to patients faster. In animal health, biotechnology is already transforming what’s possible — for example, monoclonal antibodies that help control certain chronic conditions or diseases with unprecedented precision. Relieving itching caused by atopic dermatitis or alleviating the pain associated with osteoarthritis significantly increases the quality of life of cats and dogs — and their owners. In addition, diagnostics and next-generation vaccines prevent outbreaks before they start or spread further. Maintaining a proportionate, benefit–risk for veterinary medicines allows innovation to progress safely while ensuring accelerated access to new treatments. Supporting science-based decision-making and investing in the European Medicines Agency’s capacity to deliver efficient, predictable processes will help Europe remain a trusted partner in global health innovation. Continuum of Care / Via Zoetis A One Health vision for the next decade Europe is not short of ambition. The EU Biotech Act and the Life Sciences Strategy both aim to turn innovation into a driver of growth and wellbeing. But to truly unlock their potential, they must include animal health in their vision. The experience of the veterinary medicines sector shows that innovation does not stop at species’ borders; advances in immunology, monoclonal antibodies and the use of artificial intelligence benefit both animals and humans. A One Health perspective, where veterinary and human health research reinforce each other, will help Europe to play a positive role in an increasingly competitive global landscape. The next five years will be decisive. By fostering proportionate, science-based adaptive regulation, investing in digital and institutional capacity, and embracing a One Health approach to innovation, Europe can become a genuine world leader in life sciences — for people and the animals that are essential to our lives. -------------------------------------------------------------------------------- Disclaimer POLITICAL ADVERTISEMENT * The sponsor is Zoetis Belgium S.A. * The political advertisement is linked to policy advocacy on the EU End-of-Life Vehicles Regulation (ELVR), circular plastics, chemical recycling, and industrial competitiveness in Europe. More information here.

With multiple legislative processes underway, we are now in an important moment for Europe’s ambition to boost access and be a global leader in innovation. An agile, modernized regulatory system — coupled with supportive intellectual property and access policies — can attract research and development and advanced manufacturing to Europe. This will contribute to the earlier availability of new cures for European patients and a healthier innovative ecosystem. Unfortunately, today we see that Europe is falling behind global competition. Over the last decade, there has been a 10 percent decrease in clinical trials in the European Union, which has led to 60,000 fewer European patients participating in trials.[1] Europe’s fragmented system for clinical trial approvals is a leading cause of this decline, impacting early access to innovative treatments. As scientific breakthroughs can deliver better health outcomes for patients, governments need to keep pace with this speed of innovation. > Draghi report on EU competitiveness importantly identified pharmaceutical > innovation as a strategic sector for growth in Europe. That said, the report > also noted that what is missing is a simple and strong execution plan behind > it, with simplified regulation and coherent and predictable policies that > could drive the European goals of increased competitiveness and strategic > autonomy. Europe’s marketing authorisation process now exceeds 14 months (444 days), causing patients to wait nearly three months longer than in the US (356 days) and over five months longer than in Japan (290 days) for access to innovative medicines.[2] Such delays, combined with complex and lengthy country-level market access systems, mean patients in Europe are waiting an average of 20 months longer than people living in the United States to benefit from scientific innovation.[3] Last year’s Draghi report on EU competitiveness importantly identified pharmaceutical innovation as a strategic sector for growth in Europe. That said, the report also noted that what is missing is a simple and strong execution plan behind it, with simplified regulation and coherent and predictable policies that could drive the European goals of increased competitiveness and strategic autonomy. Ongoing discussions on the revision of the General Pharmaceutical Legislation and the In Vitro Diagnostic Regulation (IVDR), the Critical Medicines Act and the upcoming Biotech Act (Part 1) mark crucial opportunities for Europe to become a global leader for innovation. However, to make this vision a reality, the EU must address structural challenges that undermine innovation and patient access to novel, lifesaving medicines. > To reverse the worrying decline in European clinical trial activity, the EU > should implement a maximum two-month approval process for clinical trial > applications (CTAs), encompassing the reviews of both regulators and ethics > committees consistent with other global leaders. The successful implementation of structural, future-proof policy changes can ensure timely access to innovative medicines for EU citizens, and this can be achieved through five key policy recommendations: Facilitate and accelerate clinical trial applications To reverse the worrying decline in European clinical trial activity, the EU should implement a maximum two-month approval process for clinical trial applications (CTAs), encompassing the reviews of both regulators and ethics committees consistent with other global leaders. It is equally important to increase collaboration among EU member states to remove unique and specific national CTA requirements and questions, and to also introduce opportunities for an informal dialogue with regulators to expediently address smaller challenges that can be quickly fixed. Legislative overlaps and fragmentation between the Clinical Trials Regulation (CTR) and the IVDR should also be addressed to avoid delays in clinical trials that utilize companion diagnostics. Expand expedited pathways Despite their potential, the EU’s expedited pathways (such as the European Medicines Agency’s PRIME scheme for unmet medical needs, Conditional Marketing Authorisation and Accelerated Assessment) are underutilised, limiting rapid patient access to important medicines. Similar expedited pathways are widely used by other regulators around the world, like the United States and Japan. Expanding the use of expedited pathways in the EU to new indications and aligning eligibility criteria with global standards would ensure that the EU has more competitive regulatory pathways and earlier patient access to life-saving medicines. Shorten scientific advice and approval timelines Shortening the EU’s scientific advice procedure is critical to optimise the development of innovative products, ensure timely and efficient resource management for both applicants and regulators, and maintain the EU’s influence in global scientific and clinical research. By evolving to a more integrated and agile dialogue, the EU can provide comprehensive, consistent guidance throughout the product lifecycle and remain competitive with other regions. Given their growing number, scientific advice should be available for medicines used with all types of medical devices and in vitro diagnostics (including combinations diagnostics) to address the complexities of working across these regulatory frameworks. > An agile, modernized regulatory system — coupled with supportive intellectual > property and access policies — can attract research and development and > advanced manufacturing to Europe. Regarding the current lengthy approval times, the proposed reduction of EMA’s standard assessment timelines from 210 to 180 days — as suggested in the revision of the pharmaceutical legislation — would allow regulators to accelerate their scientific assessments. Furthermore, the European Commission can streamline its decision phase (currently requiring up to 67 days) by conducting its activities in parallel with the scientific assessment. Strengthen the EU Medicines Regulatory Network and embrace regulatory sandboxes Achieving greater speed and agility within a regulatory system requires an appropriately resourced, sustainable regulatory infrastructure. We support transparent regulatory budgets across the network, backed by consistent investments in expertise, funding and infrastructure to support continuous capacity and capability advancements. Collaborative regulatory pathways (such as the EMA OPEN framework) could be further expanded to encourage simultaneous approvals and supply chain resilience across geographies. Additionally, regulatory sandboxes would be beneficial to pilot and adapt frameworks for disruptive future innovations, while ensuring appropriate guardrails to enable the safe development and implementation of these innovations. Enhance patient engagement Effective regulatory decision-making requires both inclusivity and adaptability. Limited patient and expert input can hinder effective regulatory decision-making, while rapid pharmaceutical innovation requires adaptable frameworks. Expert and patient perspectives are crucial for informed benefit-risk and clinical meaningfulness determinations. Disclaimer POLITICAL ADVERTISEMENT * The sponsor is Eli Lilly & Company * The advertisement is linked to General Pharmaceutical Legislation (GPL), In Vitro Diagnostic Regulation (IVDR), Critical Medicines Act (CMA), Biotech Act (Part 1), Clinical Trials Regulation (CTR), EU Medicines Regulatory Network More information here. -------------------------------------------------------------------------------- [1] IQVIA, Assessing the clinical trial ecosystem in Europe, Final Report, October 2024: efpia_ve_iqvia_assessing-the-clinical-trial-ct-ecosystem.pdf. [2] Lara J, Kermad A, Bujar M, McAuslane N. 2025. R&D Briefing 101: New drug approvals in six major authorities 2015-2024: Trends in an evolving regulatory landscape. Centre for Innovation in Regulatory Science. London, UK: https://cirsci.org/wp-content/uploads/dlm_uploads/2025/08/CIRS-RD-Briefing-101-v1.1.pdf. [3] The Patients W.A.I.T. Indicator 2024 Survey. https://www.efpia.eu/media/oeganukm/efpia-patients-wait-indicator-2024-final-110425.pdf

The European Commission is set to unveil the Biotech Act I, an EU cardiovascular health plan and a simplification of the bloc’s medical devices and in vitro diagnostics rules on Dec. 16, according to the latest Commission agenda published Monday. The first part of the Biotech Act will focus on the pharmaceutical industry and is being produced without a dedicated impact assessment. The second part — covering other biotech sectors — is expected in the third quarter of 2026. The upcoming cardiovascular health plan — inspired by the bloc’s Beating Cancer Plan — will cover prevention, early detection and screening, treatment and management, and rehabilitation. Meanwhile, simplification of the bloc’s medical devices and in vitro diagnostics rules comes after the regulations drove up assessment costs, caused certification delays, and led to product withdrawals from the market. Europe’s Health Commissioner Olivér Várhelyi has previously said the sector needs a “major overhaul.” Additionally, the Commission’s agenda includes a “drugs package” comprising new rules on drug precursors and an EU Drugs Strategy and European action plan against drug trafficking — both scheduled for Dec. 3.

Today, as the world reaches a critical juncture in the fight against HIV/AIDS, tuberculosis (TB) and malaria, the EU must choose: match scientific breakthroughs with political will and investment or retreat, putting two decades of hard-won progress at risk. Having saved over 70 million lives, the Global Fund to Fight AIDS, Tuberculosis, and Malaria (the Global Fund) has proven what smart, sustained investment can achieve.  But the impact of its work — the lives protected, the life expectancy prolonged, the systems strengthened, the innovations deployed — is now under threat due to declining international funding.  > The real question is no longer whether the EU can afford to invest in the > Global Fund, but whether it can afford to let these hard-won gains unravel. The real question is no longer whether the EU can afford to invest in the Global Fund, but whether it can afford to let these hard-won gains unravel. Declining international funding, climate change, conflict and drug resistance are reversing decades of progress. HIV prevention is hampered by rising criminalization and attacks on key populations, with 1.3 million new infections in 2024 — far above targets. TB remains the deadliest infectious disease, worsened by spreading multidrug resistance, even in Europe. Malaria faces growing resistance to insecticides and drugs, as well as the impacts of extreme weather. Without urgent action and sustained investment, these threats could result in a dangerous resurgence of all three diseases. The stakes could not be higher  The Global Fund’s latest results reveal extraordinary progress. In 2024 alone: * 25.6 million people received lifesaving antiretroviral therapy, yet 630,000 still died of AIDS-related causes; * 7.4 million people were treated for TB, with innovations like AI-powered diagnostics reaching frontline workers in Ukraine; and * malaria deaths, primarily among African children under five, have been halved over two decades, with 2.2 billion mosquito nets distributed and ten countries eliminating malaria since 2020. Yet one child still dies every minute from this treatable disease.  What makes this moment unprecedented is not just the scale of the challenge, but the scale of the opportunity. Thanks to extraordinary scientific breakthroughs, we now have the tools to turn the tide:  * lenacapavir, a long-acting antiretroviral, offers new hope for the possibility of HIV-free generations; * dual active ingredient mosquito nets combine physical protection with intelligent vector control, transforming malaria prevention; and  * AI-driven TB screening and diagnostics are revolutionizing early detection and treatment, even in the most fragile settings. Some of these breakthroughs reflect Europe’s continued research and development and the private sector’s leadership in global health. BASF’s dual-active-ingredient mosquito nets, recently distributed by the millions in Nigeria, are redefining malaria prevention by combining physical protection with intelligent vector control. Delft Imaging’s ultra-portable digital X-ray devices are enabling TB screening in remote and fragile settings, while Siemens Healthineers is helping deploy cutting-edge AI software to support TB triage and diagnosis.  But they must be deployed widely and equitably to reach those who need them most. That is precisely what the Global Fund enables: equitable access to cutting-edge solutions, delivered through community-led systems that reach those most often left behind. A defining moment for EU Leadership The EU has a unique chance to turn this crisis into an opportunity. The upcoming G20 summit and the Global Fund’s replenishment are pivotal moments.  President Ursula von der Leyen and Commissioner Síkela can send a clear, unequivocal signal: Europe will not stop at “almost”. It will lead until the world is free of AIDS, tuberculosis and malaria.  The Global Fund is a unique partnership that combines financial resources with technical expertise, community engagement and inclusive governance. It reaches those often left behind — those criminalized, marginalized or excluded from health systems.  > Even in Ukraine, amid the devastation of war, the Global Fund partnership has > ensured continuity of HIV and TB services — proof that smart investments > deliver impact, even in crisis. Its model of country ownership and transparency aligns with Africa’s agenda for health sovereignty and with the EU’s commitment to equity and human rights. Even in Ukraine, amid the devastation of war, the Global Fund partnership has ensured continuity of HIV and TB services — proof that smart investments deliver impact, even in crisis. The cost of inaction Some may point to constraints in the Multiannual Financial Framework. But history shows that the EU has consistently stepped up, even in difficult fiscal times. The instruments exist. What’s needed now is leadership to use them. Failure to act would unravel decades of progress. Resurgent epidemics would claim lives, destabilize economies and undermine global health security. The cost of inaction far exceeds the price of investment. For the EU, the risks are strategic as well as moral. Stepping back now would erode the EU’s credibility as champion of human rights and global responsibility. It would send the wrong message, at precisely the wrong time.  Ukraine demonstrates what is at stake: with Global Fund support, millions continue to receive HIV and TB services despite war. Cutting funding now would risk lives not only in Africa and Asia, but also in Europe’s own neighborhood. A call to action Ultimately, this isn’t a question of affordability, but one of foresight. Can the EU afford for the Global Fund not to be fully financed? The answer, for us, is a resounding no. We therefore urge the European Commission to announce a bold, multi-year financial commitment to the Global Fund at the G20.  This pledge would reaffirm the EU’s values and inspire other Team Europe partners to follow suit. It would also support ongoing reforms to further enhance the Global Fund’s efficiency, transparency and inclusivity. > Ultimately, this isn’t a question of affordability, but one of foresight. Can > the EU afford for the Global Fund not to be fully financed? The answer, for > us, is a resounding no. This is more than a funding decision. It is a moment to define the kind of world we choose to build: one where preventable diseases no longer claim lives, where health equity is a reality and where solidarity triumphs over short-termism. Now is the time to reaffirm Europe’s leadership. To prove that when it comes to global health, we will never stop until the fight is won.

Epilepsy affects 50 million people globally and 6 million in the EU.1 Despite this, it is a chronically underfunded and underserved condition in need of strategic investment. The latest report from Headway1 — a survey dedicated to tracking and analysing epilepsy care in the EU — underscores the urgent funding gap across the EU in epilepsy care. At the launch of the latest report in Brussels, members of the European Parliament, advocacy and patient organizations, key industry leaders, and I discussed the current picture painted by the report, and the decisions we must make to support the European epilepsy community.  Overcoming barriers to epilepsy care  Epilepsy continues to be one of the most significant neurological conditions across Europe. As the fourth most common neurological disorder,2 it takes a startling toll on people’s health. People with epilepsy tend to have more physical problems (such as fractures and bruising from injuries related to seizures), as well as higher rates of psychological conditions, including anxiety and depression.3 Defined as a chronic non-communicable neurological disease, epilepsy is characterized by unprovoked seizures often associated with neurobiological, cognitive and social consequences.4   Despite the size of the patient population, the condition is often hidden and therefore heavily stigmatized, with such stigma contributing to a crisis of care. Nearly 40 percent of people living with epilepsy in Europe remain untreated, a figure that rises as high as 90 percent in underserved areas.5 Moreover, individuals with epilepsy have more than a twofold increased risk of premature death compared with the general population, and their life expectancy is reduced by approximately 10-12 years.6   > Individuals with epilepsy have more than a twofold increased risk of premature > death compared with the general population, and their life expectancy is > reduced by approximately 10-12 years. Epilepsy is not currently recognized in some countries as a brain disorder, and while new treatments have been coming to the EU, the scarce investment in brain health impacts access to care, which is already unequal — subject to geographic lottery, socioeconomic status and gender. Additionally, the stigma associated with epilepsy, alongside limited seizure control, significantly hinders social and economic inclusion, resulting in individuals with epilepsy feeling isolated, engaged in lower employment rates and without long-term financial security.   Addressing these barriers is not just a healthcare imperative, but a societal one  via Angelini Pharma Embracing brain capital  Central to the Headway report is the concept of ‘brain capital’. This framework underscores that investing in brain health, including epilepsy, is a robust economic strategy. Avoidable epilepsy-related costs are estimated to reach €49.2 billion annually within the EU27 and the U.K., which is approximately 0.28 percent of the combined GDP of the EU and the U.K.. These figures include €20.1 billion in direct costs and €29 billion in indirect costs.7   > Avoidable epilepsy-related costs are estimated to reach €49.2 billion annually > within the EU27 and the U.K., which is approximately 0.28 percent of the > combined GDP of the EU and the U.K. The Headway report outlines three return-on-investment models that address both the human and financial costs:  1. Closing the treatment gap by ensuring timely access to appropriate care could yield a return on investment of €1.9 for every €1 invested.8,9,10  2. Addressing psychiatric comorbidities, such as anxiety and depression, by integrating mental health support into standard epilepsy care can offer a return of €1.5 per €1 spent.11,12 This intervention is critical, as mental health disorders often exacerbate the challenges faced by individuals with epilepsy.  3. Preventing avoidable cases through public health strategies such as stroke prevention and improved perinatal care could present a return of €1.7 per €1 spent.13   If national health systems across the EU and the U.K. invest €1 in each of these targeted actions and allocate a larger portion of their total national healthcare budgets to brain health services such as diagnostics testing, hospitalizations and antiseizure medications, to name a few, it’s obvious that it repays itself. It also yields an additional €0.50-€0.90 in reduced healthcare spending and increased productivity of patients and caregivers. In a climate of tight healthcare budgets and growing demand, these findings provide an evidence-based roadmap to better care and stronger systems.  A unified approach to a healthier future  The Headway report is a clear wake-up call for European policymakers to prioritize epilepsy as part of the broader brain health agenda. By investing in epilepsy care and engaging the public, countries will not only improve individual health outcomes but also realize substantial economic and societal benefits in both the short and long term. Moreover, they can lead the way in global best practice by scaling up proven solutions such as deploying epilepsy-specialist nurses and modernizing clinical trial regulations, especially for complex studies, to promote person-centered care and improve outcomes.  > By investing in epilepsy care and engaging the public, countries will not only > improve individual health outcomes but also realize substantial economic and > societal benefits. Countries should establish dedicated additional funding for epilepsy and brain health research within the forthcoming EU Brain Health Partnership and Horizon Europe. Additionally, strengthening cross-border networks like EpiCARE and aligning with the World Health Organization’s IGAP framework will support EU member states and the U.K. in implementing effective national responses, improve access to highly specialized care and shared expertise, and knowledge from the inclusion of patient-reported indicators and real-world evidence. Epilepsy should be included as a distinct priority in the EU’s and member states’ mental health strategies with tailored indicators and goals for the best possible outcomes.  > The Headway report lay the foundation for a clear path to a more resilient and > inclusive society, one that ensures a future where every individual living > with epilepsy has the opportunity to thrive. The EU27 and the U.K. stand at a crossroads. The research we’ve done, the insights we’ve discussed in Brussels and the findings outlined clearly in the Headway report lay the foundation for a clear path to a more resilient and inclusive society, one that ensures a future where every individual living with epilepsy has the opportunity to thrive. The need now is for committed action. It is crucial that policymakers, medical and healthcare professionals, and those living with epilepsy come together to effect change, improve access to treatment and turn our vision into reality.  > -------------------------------------------------------------------------------- 1. Szaflaraski M (2014), “Social determinants of health in epilepsy” 2. TEHA on GBD 2021 Nervous System Disorders Collaborators (2024), “Global, regional, and national burden of disorders affecting the nervous system, 1990-2021: a systemic analysis for the Global Burden of Disease Study 2021,” 2025 3. World Health Organisation. Epilepsy. Signs and Symptoms. Available online here: https://www.who.int/news-room/fact-sheets/detail/epilepsy. (Accessed August 2025] 4. Fisher RS, et al. Epilepsia 2014;55: 475-482 5. IBE, ILAE, WHO (2011), “Epilepsy in the WHO European Region.” and European Parliament (2011), “Proceedings of the workshop ‘Treating and living with Epilepsy’” 6. Thurman DJ et al. (2014), “The burden of premature mortality of epilepsy in high-income countries: A systematic review from the Mortality Task Force of the International League Against Epilepsy”. Epilepsia. 7. TEHA on Begley C et al. (2022), “The global cost of epilepsy: A systematic review and extrapolation”, Strzelczyk et al. (2015), “Costs of epilepsy and cost‐driving factors in children, adolescents, and their caregivers in Germany”, and Willems LM et al. (2021), “Multicenter, cross-sectional study of the costs of illness andcost-driving factors in adult patients with epilepsy”, 2025 8. Kwon C et al. (2022), “The worldwide epilepsy treatment gap: A systematic review and recommendations for revised definitions – A report from the ILAE Epidemiology Commission”. Epilepsia. 9. De Zélicourt M et al. (2014), “Management of focal epilepsy in adults treated with polytherapy in France: The direct cost of drug resistance (ESPERA study)”. Seizure. 10. Willems LM et al. (2022), “Multicenter, cross-sectional study of the costs of illness and cost-driving factors in adult patients with epilepsy”. Epilepsia 11. Dewhurst E et al. (2015), “A prospective service evaluation of acceptance and commitment therapy for patients with refractory epilepsy”. Epilepsy & Behavior. 12. TEHA Group elaboration on OECD data and Fleishman JA et al. (2006), “Using the SF-12 health status measure to improve predictions of medical expenditures”. Medical Care 13. The European House of Ambrosetti and Angelini Pharma. (2025) Brain Health in Uncertain Times: A strategic investment for Europe’s future

Dangerous viruses that cause severe paralytic illness are thriving in Gaza, where starving children living under an Israeli blockade can’t access the food or treatments they need to recover. For months, health officials have warned that the destruction of the Gaza Strip’s sanitation facilities by Israel could fuel the surge of infectious diseases, as seen in last year’s polio outbreak. Now, doctors are reporting a surge in cases of acute flaccid paralysis, a rare syndrome causing muscle weakness that can make it hard to breathe and swallow. Cases in Gaza include acute flaccid myelitis, which mostly affects children, and the better-known Guillain-Barré syndrome, said Ahmed al-Farra, head of pediatrics at Nasser Hospital in Khan Younis, Gaza.  Before October 2023, acute flaccid paralysis was “something strange and very rare,” with one or two cases turning up each year, al-Farra said. But in the last three months, according to al-Farra, doctors have seen nearly 100 cases. Lab samples sent to Jordan and Israel were positive for enterovirus, a group of bugs spread between people or through contaminated water, according to al-Farra. Enteroviruses, which also include poliovirus, are typically spread via the fecal-oral route. He said it was no surprise that one of the hot spots is Khan Younis, where there is raw sewage pooling in the streets.  International condemnation of Israel is growing and, in the strongest remarks from an EU official so far, European Commission Vice President Teresa Ribera told POLITICO’s Brussels Playbook earlier this month that Israel’s conduct in Gaza “looks very much” like genocide — a charge denied by Israel. A statement from the World Health Organization said there had been 32 cases of acute flaccid paralysis reported among under-15s as of July 31. The surge is partly down to better monitoring, the WHO said, but also reflects the collapse of water and sanitation infrastructure, malnutrition and restricted access to health care. In almost 70 percent of samples sent for testing, the cause was identified as a non-polio enterovirus, compared to 26 percent in previous years, the WHO said. Critical clinical assessment, its statement said, “is extremely difficult in Gaza at the moment due to the lack of diagnostic, lab and testing capacities.” Likewise, doctors say they have few options to treat patients. Staff at Al-Shifa Hospital in Gaza, mostly destroyed by Israel in early 2024, have diagnosed 22 cases of Guillain-Barré syndrome so far, said hospital director Muhammed Abu Salmiya. Three patients died, while 12 developed paralysis and were referred to rehab centers.  Treatment for acute flaccid myelitis and Guillain-Barré syndrome includes intravenous immunoglobulins — antibodies used to neutralize the infection — or plasma exchange, which involves filtering the blood. None of these are readily available in Gaza, where there is a chronic shortage of basic medical supplies due to the Israeli blockade. “Unfortunately, the treatment is not available to us,” Abu Salmiya said. For months, health officials have warned that the destruction of the Gaza Strip’s sanitation facilities by Israel could fuel the surge of infectious diseases, as seen in last year’s polio outbreak. | AFP via Getty Images Doctors in Gaza have appealed to colleagues volunteering with foreign groups to try and bring filters for plasma exchange with them, but these are often confiscated by Israeli authorities, al-Farra said. The Israeli government had not responded to POLITICO’s request for comment by the time of publication. Children in Gaza are especially vulnerable to the condition due to mass starvation and a lack of vitamins to support nerve regeneration, al-Farra explained. “No one has a good amount of vitamins B1, B6, B12,” al-Farra said. That’s because there are “no fruits, no vegetables, no meat, no eggs, no yogurt, no everything.” Israel has severely restricted the entry of food into Gaza since October 2023, leading to chronic malnutrition and mass starvation. Health officials in Gaza say 188 people have died from hunger since October 2023, most of them children. The Gaza Health Ministry, which is part of the Hamas-run government, maintains detailed casualty records that are seen as generally reliable by independent experts. The United Nations estimates at least 1,373 Palestinians have died seeking food, including 859 at sites run by the Israel- and United States-backed Gaza Humanitarian Foundation. In December 2024, Amnesty International cited Israel’s blockade and the destruction of sanitation facilities in its report concluding Israel was committing genocide in Gaza. Israeli Prime Minister Benjamin Netanyahu says his government intends to move forward with a full takeover of Gaza. The International Court of Justice has already ruled that Israel’s occupation of the territory is illegal.

BRUSSELS — While the EU’s latest U.S. retaliatory tariff proposal hits aircraft, vehicles and medical appliances hardest, health care, transport and agri-food lobbyists have secured a few wins. Having already agreed on an initial tariff package affecting around €21 billion in U.S. goods, set to come into force Aug. 6, the EU has been haggling over the details of a second retaliatory package for months. The second list, seen by POLITICO, would affect €72 billion worth of imports. That’s down from an initial proposal, published in May, that would have hit an estimated €95 billion worth of U.S. goods. Lobbying around the lists has been intense, as national and sectoral representatives scramble to get key goods they need from the U.S. scrubbed from the lineup of negotiating chips. We crunched the numbers on the goods most likely to get caught in the crossfire and which sectors may manage to escape unscathed. THE BIG PICTURE According to the latest EU plan, tariffs on industrial goods would hurt U.S. imports the most, to the tune of almost €66 billion. The remainder of the pain, €6 billion in affected goods, would come from tariffs on agricultural and food products. Aircraft products top the tariff impact charts by miles, with over €10 billion goods potentially affected.  Passenger vehicles and medical appliances round out the top three largest product categories hit in both the May and July versions of the tariff list.  BIGGEST WINNERS Diagnostic or laboratory reagents — i.e., chemicals used for medical testing — and gas turbines were set to be among the top 10 most affected product groups in the first version of the tariff list. They have been scrubbed from the latest version seen by POLITICO.  They are not the only products that managed to escape the fray.  Several goods related to health care appear to no longer be under threat, such as X-ray apparatus, thread for stitches, and materials used for surgery to separate tissue, as well as wheelchairs and scooters for people with disabilities. In the world of agriculture and food, soybean seeds also disappeared from the document. MAKING GAINS While unsuccessful in totally dodging tariffs, some key goods for large product categories have been removed from the firing line.  For instance, several data processing machines, i.e., computers, have been removed from the list. As have machines used to make semiconductors.  With U.S. President Donald Trump’s latest 30 percent tariff threat and the Aug. 1 deal deadline looming, European producers reliant on products still on the list will be bracing for impact or praying for a deal. Hanne Cokelaere contributed to this report.