Biotechnology is central to modern medicine and Europe’s long-term competitiveness. From cancer and cardiovascular disease to rare conditions, it is driving transformative advances for patients across Europe and beyond . 1         Yet innovation in Europe is increasingly shaped by regulatory fragmentation, procedural complexity and uneven implementation across  m ember s tates. As scientific progress accelerates, policy frameworks must evolve in parallel, supporting the full lifecycle of innovation from research and clinical development to manufacturing and patient access.  The proposed EU Biotech Act seeks to address these challenges. By streamlining regulatory procedures, strengthening coordination  and supporting scale-up and manufacturing, it aims to reinforce Europe’s position in a highly competitive global biotechnology landscape .2       Its success, however, will depend less on ambition than on delivery. Consistent implementation, proportionate oversight and continued global openness will determine whether the  a ct translates into faster patient access, sustained investment and long-term resilience.  Q: Why is biotechnology increasingly seen as a strategic pillar for Europe’s competitiveness, resilience and long-term growth?  Gilles Marrache, SVP and regional general manager, Europe, Latin America, Middle East, Africa and Canada, Amgen:  Biotechnology sits at the intersection of health, industrial policy and economic competitiveness. The sector is one of Europe’s strongest strategic assets and a leading contributor to  research and development  growth . 3    At the same time, Europe’s position is under increasing pressure. Over the past two decades, the EU has lost approximately 25  percent of its global share of pharmaceutical investment to other regions, such as the  United States  and China.   The choices made today will shape Europe’s long-term strength in the sector, influencing not only competitiveness and growth, but also how quickly patients can benefit from new treatments.  > Europe stands at a pivotal moment in biotechnology. Our life sciences legacy > is strong, but maintaining global competitiveness requires evolution .” 4   > >  Gilles Marrache, SVP and regional general manager, Europe, Latin America, > Middle East, Africa and Canada, Amgen. Q: What does the EU Biotech Act aim to do  and why is it considered an important step forward for patients and Europe’s innovation ecosystem?  Marrache: The EU Biotech Act represents a timely opportunity to better support biotechnology products from the laboratory to the market. By streamlining medicines’ pathways and improving conditions for scale-up and investment, it can help strengthen Europe’s innovation ecosystem and accelerate patient access to breakthrough therapies. These measures will help anchor biotechnology as a strategic priority for Europe’s future  —  and one that can deliver earlier patient benefit  —  so long as we can make it work in practice.  Q: How does the EU Biotech Act address regulatory fragmentation, and where will effective delivery and coordination be most decisive? Marrache: Regulatory fragmentation has long challenged biotechnology development in Europe, particularly for multinational clinical trials and innovative products. The Biotech Act introduces faster, more coordinated trials, expanded regulatory sandboxes and new investment and industrial capacity instruments.   The proposed EU Health Biotechnology Support Network and a  u nion-level regulatory status repository would strengthen transparency and predictability. Together, these measures would support earlier regulatory dialogue, help de-risk development   and promote more consistent implementation across  m ember  s tates.   They also create an opportunity to address complexities surrounding combination products  —  spanning medicines, devices and diagnostics  —  where overlapping requirements and parallel assessments have added delays.5 This builds on related efforts, such as the COMBINE programme,6 which seeks to streamline the navigation of the In Vitro Diagnostic Regulation , 7 Clinical Trials Regulation8 and the Medical Device Regulation9 through a single, coordinated assessment process. Continued clarity and coordination will be essential to reduce duplication and accelerate development timelines .10 Q: What conditions will be most critical to support biotech scale-up, manufacturing  and long-term investment in Europe?  Marrache: Europe must strike the right balance between strategic autonomy and openness to global collaboration. Any new instruments under the Biotech Act mechanisms should remain open and supportive of all types of biotech investments, recogni z ing that biotech manufacturing operates through globally integrated and highly speciali z ed value chains.   Q: How can Europe ensure faster and more predictable pathways from scientific discovery to patient access, while maintaining high standards of safety and quality?   Marrache: Faster and more predictable patient access depends on strengthening end-to-end pathways across the lifecycle.  The Biotech Act will help ensure continuity of scientific and regulatory experti z e, from clinical development through post-authori z ation. It will also support stronger alignment with downstream processes, such as health technology assessments, which  are  critical to success.   Moreover, reducing unnecessary delays or duplication in approval processes can set clearer expectations, more predictable development timelines and earlier planning for scale-up.    Gilles Marrache, SVP and regional general manager, Europe, Latin America, Middle East, Africa and Canada, Amgen. Via Amgen. Finally, embedding a limited number of practical tools (procedural, digital or governance-based) and ensuring they are integrated within existing  European Medicines Agency and EU regulatory structures can help achieve faster patient access . 11 Q: What role can stronger regulatory coordination, data use and public - private collaboration play in strengthening Europe’s global position in biotechnology?  Marrache: To unlock biotechnology’s full potential, consistent implementation is essential. Fragmented approaches to secondary data use, divergent  m ember   state interpretations and uncertainty for data holders still limit access to high-quality datasets at scale. The Biotech Act introduces key building blocks to address this.   These include Biotechnology Data Quality Accelerators to improve interoperability, trusted testing environments for advanced innovation, and alignment with the EU AI Act ,12  European Health Data Space13 and wider EU data initiatives. It also foresees AI-specific provisions and clinical trial guidance to provide greater operational clarity.  Crucially, these structures must simplify rather than add further layers of complexity.   Addressing remaining barriers will reduce legal uncertainty for AI deployment, support innovation and strengthen Europe’s competitiveness.  > These reforms will create a moderni z ed biotech ecosystem, healthier > societies, sustainable healthcare systems and faster patient access to the > latest breakthroughs in Europe .” 14 > > Gilles Marrache, SVP and regional general manager, Europe, Latin America, > Middle East, Africa and Canada, Amgen.  Q: As technologies evolve and global competition intensifies, how can policymakers ensure the Biotech Act remains flexible and future-proof?  Marrache:  To remain future-proof, the Biotech Act must be designed to evolve alongside scientific progress, market dynamics and patient needs. Clear objectives, risk-based requirements, regular review mechanisms and timely updates to guidance will enhance regulatory agility without creating unnecessary rigidity or administrative burden.  Continuous stakeholder dialogue combined with horizon scanning will be essential to sustaining innovation, resilience and timely patient access over the long term. Preserving regulatory openness and international cooperation will be critical in avoiding fragmentation and maintaining Europe’s credibility as a global biotech hub.  Q: Looking ahead, what two or three priorities should policymakers focus on to ensure the EU Biotech Act delivers meaningful impact in practice?  Marrache: Looking ahead, policymakers should focus on three priorities for the Biotech Act:    First, implementation must deliver real regulatory efficiency, predictability and coordination in practice. Second, Europe must sustain an open and investment-friendly framework that reflects the global nature of biotechnology.  And third, policymakers should ensure a clear and coherent legal framework across the lifecycle of innovative medicines, providing certainty for the use of  artificial intelligence   —  as a key driver of innovation in health biotechnology.  In practical terms, the EU Biotech Act will be judged not by the number of new instruments it creates, but by whether it reduces complexity, increases predictability and shortens the path from scientific discovery to patient benefit. An open, innovation-friendly framework that is competitive at the global level will help sustain investment, strengthen resilient supply chains and deliver better outcomes for patients across Europe and beyond. -------------------------------------------------------------------------------- References 1. Amgen Europe, The EU Biotech Act Unlocking Europe’s Potential, May 2025. Retrieved from https://www.amgen.eu/media/press-releases/2025/05/The_EU_Biotech_Act_Unlocking_Europes_Potential 2. European Commission, Proposal for a Regulation to establish measures to strengthen the Union’s biotechnology and biomanufacturing sectors, December 2025. Retrieved from https://health.ec.europa.eu/publications/proposal-regulation-establish-measures-strengthen-unions-biotechnology-and-biomanufacturing-sectors_en 3. EFPIA, The pharmaceutical sector: A catalyst to foster Europe’s competitiveness, February 2026. Retrieved from https://www.efpia.eu/media/zkhfr3kp/10-actions-for-competitiveness-growth-and-security.pdf 4. The Parliament, Investing in healthy societies by boosting biotech competitiveness, November 2024. Retrieved from https://www.theparliamentmagazine.eu/partner/article/investing-in-healthy-societies-by-boosting-biotech-competitiveness#_ftn4 5. Amgen Europe, The EU Biotech Act Unlocking Europe’s Potential, May 2025. Retrieved from https://www.amgen.eu/docs/BiotechPP_final_digital_version_May_2025.pdf   6. European Commission, combine programme, June 2023. Retrieved from https://health.ec.europa.eu/medical-devices-topics-interest/combine-programme_en  7. European Commission. Medical Devices – In Vitro Diagnostics, March 2026. Retrieved from https://health.ec.europa.eu/medical-devices-vitro-diagnostics_en 8. European Commission, Clinical trials – Regulation EU No 536/2014, January 2022. Retrieved from https://health.ec.europa.eu/medicinal-products/clinical-trials/clinical-trials-regulation-eu-no-5362014_en 9. European Commission, Simpler and more effective rules for medical devices – Commission proposal for a targeted revision of the medical devices regulations, December 2025. Retrieved from https://health.ec.europa.eu/medical-devices-sector/new-regulations_en#mdr 10. Amgen Europe, The EU Biotech Act Unlocking Europe’s Potential, May 2025. Retrieved from https://www.amgen.eu/docs/BiotechPP_final_digital_version_May_2025.pdf   11. AmCham, EU position on the Commission Proposal for an EU Biotech Act 12. European Commission, AI Act | Shaping Europe’s digital future, June 2024. Retrieved from https://digital-strategy.ec.europa.eu/en/policies/regulatory-framework-ai 13. European Commission, European Health Data Space, March 2025. Retrieved from https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space-regulation-ehds_en 14. The Parliament, Why Europe needs a Biotech Act, October 2025. Retrieved from https://www.theparliamentmagazine.eu/partner/article/why-europe-needs-a-biotech-act -------------------------------------------------------------------------------- Disclaimer POLITICAL ADVERTISEMENT * The sponsor is Amgen Inc * The ultimate controlling entity is Amgen Inc * The political advertisement is linked to advocacy on the EU Biotech Act. More information here.

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.