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.

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

As trilogue discussions on the Critical Medicines Act (CMA) approach, its potential effects on medicine supply, patient access and Europe’s competitiveness are increasingly in focus. From an industry standpoint, several considerations are central to understanding how this act can best achieve its objectives and support a robust pharmaceutical ecosystem in Europe.  Keeping the CMA focused where it matters  Much of the debate around the CMA has centered on its promises to strengthen the availability and security of supply of critical medicines in the EU while improving accessibility to other medicines. These are goals that our industry fully supports.   The European Commission’s proposal is designed to focus on critical medicines, with a vulnerability assessment foreseen to identify which products are truly at risk of disruption and tailor solutions accordingly. Alongside critical medicines, the proposal also introduces a new definition of ‘medicinal products of common interest’. Under current wording, this would include any medicine unavailable in at least three member states, regardless of the underlying reason.   Such a broad definition risks turning a targeted framework for resilience into an all-encompassing mechanism covering almost every medicine on the market, blurring the distinction between supply and access challenges. These are fundamentally different issues that require fundamentally different policy tools.   > Applying the CMA’s tools across the entire medicines market would dilute > priorities, stretch healthcare budgets and create administrative burdens for > industry without delivering real benefits for patients.   The act will be far more effective if it remains focused on where the risks are greatest — in other words, by limiting the ‘medicinal products of common interest’ definition to cases of demonstrable market failure and directing measures toward genuinely critical medicines with a proven risk of supply disruption.  Fixing supply and access hurdles needs more than joint procurement   The CMA places joint procurement at the center of its strategy to address both supply and access challenges. While this approach can contribute to improving availability in certain circumstances, joint procurement will only deliver lasting results if it is designed to address the underlying causes of access delays and shortages, which vary across geographies and products.  For medicines where the main challenge lies in fragile supply chains, joint procurement can play a role, particularly when it enhances predictability and economic viability for suppliers. Experience from the Covid-19 pandemic has shown that coordinated purchasing can be effective in targeted situations. For medicines facing access delays, joint procurement could help improve availability in countries where genuine market failures exist. However, the value of joint procurement for countries where products are already available, or where access barriers can be better addressed by improving national pricing and reimbursement systems, is very questionable.  To ensure that joint procurement does not hinder access, several safeguards are essential. Tenders should reward quality and promote innovation, recognizing the value that innovative medicines bring to patients and society. Price confidentiality must be protected to prevent unintended spillovers, such as reference pricing effects. Once joint procurement agreements are concluded, to ensure commercial and supply predictability there should be no additional national renegotiations or expenditure control measures. Finally, allowing national procurement processes to run in parallel will be key to avoid delays and maintain flexibility.  Beyond these design safeguards, real progress will depend on tackling the broader root causes of shortages and access delays. For supply fragility, this means, among other actions, reducing strategic dependencies where necessary, improving transparency across supply chains and avoiding rigid national stockpiling rules. For access delays, progress will require addressing national pricing and reimbursement challenges, and a greater willingness from governments to reward the value that innovative medicines deliver.  Protectionism won’t make Europe stronger  Few elements of the CMA debate have attracted as much attention as the idea of prioritizing EU-made medicines. The rationale is straightforward: producing more within Europe is expected to reduce reliance on third countries, reinforce strategic autonomy and, ultimately, improve supply security. While this narrative is understandable, taking it at face value risks overlooking the realities of how medicines are manufactured and supplied today.  Europe already has one of the world’s strongest pharmaceutical manufacturing footprints and, unlike some other pharma manufacturing regions, Europe exports 71 percent of its pharmaceutical production. This output depends on global supply networks for active substances, raw materials and specialized technologies. Introducing local-content requirements or preferential treatment for EU-made products would disrupt those networks, fragment supply chains and drive up costs, with limited evidence that such measures would enhance resilience. Local-content requirements could also affect Europe’s trade relationships and weaken, rather than strengthen, its industrial base in the long term, while distorting competition within the single market and undermining the competitiveness of both European and international companies operating in Europe. The likely outcome would be less diversity and greater concentration in supply chains: the opposite of what a resilient system requires.  If procurement criteria referencing resilience or strategic autonomy are used, they should be proportionate and tied to clearly demonstrated dependencies or supply risks. Protectionist approaches, however well-intentioned, cannot substitute for the broader policy environment needed to keep Europe attractive for investment in research and development and manufacturing. A competitive European ecosystem depends first and foremost on predictable intellectual-property rules, timely regulatory processes, access to capital, and a strong scientific and technical skills base.  The EU institutions still have time to steer the CMA on course  The CMA offers a real chance to get things right. The European Parliament’s proposal for more consistent contingency stock rules could help if it stays focused on medicines genuinely at risk of shortage. The act can also make reporting more efficient by using existing systems rather than creating new ones. Policymakers should also be aware that wider regulatory initiatives directly affect Europe’s ability to manufacture and supply medicines. A more coherent policy framework will be essential to strengthen resilience.  Europe’s goal must be to build an environment where pharmaceutical innovation and production can thrive. Europe’s choice is clear: supply security cannot be achieved by weakening the industry that ensures it. The CMA will only work if it tackles the right problems with the right tools and keeps competitiveness at its core.   > Europe’s goal must be to build an environment where pharmaceutical innovation > and production can thrive. Our industry remains ready to engage with EU and national policymakers to make that happen. A high-level forum on the CMA involving all stakeholders could help guide the act’s implementation in a way that improves supply security and speeds up access for patients, while reinforcing Europe’s position as a global player in life sciences.  Disclaimer POLITICAL ADVERTISEMENT * The sponsor is European Federation of Pharmaceutical Industries and Associations (EFPIA) * The political advertisement is linked to the Critical Medicines Act More information here.

LONDON — The American drugmaker Eli Lilly wants to see more changes to Britain’s medicine market before it pivots on its abandoned £279 million investment in a biotech incubator project. The U.K. government has drawn up proposals to increase the amount the state-funded National Health Service is allowed to pay pharmaceutical firms for drugs after intense discussions with officials from Donald Trump’s administration. The U.S. president has demanded lower drug prices for Americans, and suggested other developed countries should pay more. The British plans under consideration could increase the threshold at which the NHS pays firms for medicines by up to 25 percent. But for the U.S. pharmaceutical company — which shelved its planned facility meant to support early-stage life sciences businesses with lab space, mentorship and potential financial backing — the proposal alone is not enough. “I don’t think we have heard enough to say that we are willing to get the Lilly Gateway Lab started,” Patrik Jonsson, president of Lilly’s international business, which covers all markets outside the U.S., told POLITICO. “I think once we see the right signs from the U.K. government, we’re more than happy to restart those discussions, and we could move quite quickly,” Jonsson said. However, “we need to see some significant and sustainable change here.” The comments will be a blow to British negotiators, who are in advanced talks to agree their drug-pricing deal with the U.S. administration as part of wider trade negotiations. Officials are hoping to wrap up the pharma talks ahead of the U.K.’s budget in late November. Ministers last week granted a two-week extension to the deadline by which pharma firms must tell the government if they intend to leave the NHS’s voluntary drug pricing scheme. If Washington and London strike a deal — effectively committing the NHS to higher drug spending — Chancellor Rachel Reeves will face pressure to spell out how much the increase will cost taxpayers. ‘WE NEED THE RIGHT CONDITIONS’ Drugmakers have long called for changes to the U.K.’s tightly-controlled drug prices. Britain limits the annual cost for a year of good-quality life (QALY) for a patient at £30,000 for most drugs. Industry also pays an annual rebate to the NHS at 23 percent of their U.K. sales. These measures have contained the medicine bill for the U.K.’s publicly-funded health care system. While Jonsson acknowledged the U.K. is “well positioned to be a source of innovation” thanks to a “small but really impressive group of scientists,” he said the country needs to demonstrate sustained changes. The British plans under consideration could increase the threshold at which the NHS pays firms for medicines by up to 25 percent. | Anna Barclay/Getty Images “At the end of the day if you want us to research, develop and produce medicines in your country you need to put the right conditions in place so that your citizens can get access to those patients at least who need it most,” Jonsson said. An editorial in the Lancet medical journal last week said “the argument that paying more for medicines leads to more innovation is unfounded.” “If the U.K. Government wants to attract pharma investment, it should follow the evidence. Rather than handing over more money for medicines, it should invest in creating fertile conditions for attracting world-leading scientists, boosting public infrastructure for research and development, and facilitating clinical trials,” the article states. “Although the tangible outcomes of applied research might appeal to politicians, investing massively in a second-to-none basic science sector will allow scientific innovation to flourish.” Jonsson was speaking to POLITICO as the company announced a €2.6 billion new manufacturing facility in the Netherlands to produce oral medicines, including its first GLP-1 weight-loss pill. A Department of Health and Social Care spokesperson said: “We will always prioritise the needs of NHS patients. Investment in patient access to innovative medicines is critical to our NHS. “We are now in advanced discussions with the US Administration to secure the best outcome for the UK, reflecting our strong relationship and the opportunities from close partnership with our pharmaceutical industry,” the spokesperson added.

One-sixth of confirmed bacterial infections are resistant to antibiotics and rates of drug-resistance are rapidly growing, presenting a growing threat to public health. Over five years — from 2018 to 2023 — antibiotic resistance rose in over 40 percent of the monitored antibiotics with an average annual rise of 5 percent to 15 percent, found the World Health Organization report, published Monday. The Global Antibiotic Resistance Surveillance Report tracked 22 key antibiotics used to treat infections caused by the eight most common bacterial pathogens, including urinary tract infections and gonorrhea. Antimicrobial resistance (AMR) is one of the biggest public health threats of the 21st century and could make it much harder to treat everyday infections, according to the WHO. It’s mainly caused by the overuse and misuse of medicines, allowing pathogens to become resistant, and is fuelled by poor sanitation and lack of clean water. Bacterial AMR killed 1.27 million people in 2019 and contributed to another 4.95 million deaths that year. “Antimicrobial resistance is outpacing advances in modern medicine, threatening the health of families worldwide,” said Tedros Adhanom Ghebreyesus, WHO director general. “As countries strengthen their AMR surveillance systems, we must use antibiotics responsibly, and make sure everyone has access to the right medicines, quality-assured diagnostics, and vaccines,” he added. The median level of antibiotic resistance in Europe was 10.2 percent, the second-lowest rate of any region and below the global average of 17.2 percent.  The greatest threat comes from gram-negative bacteria, which include E. coli and K. pneumoniae, and are among the most severe bacterial infections. More than 40 percent of E. coli and 55 percent of K. pneumoniae globally are resistant to the first-choice treatment, the report found. Other concerns include the bacteria that causes gonorrhea, which has developed resistance to every drug available for first-line treatment. One drug, ciprofloxacin, is no longer suitable due to widespread resistance, while another, azithromycin, has been removed from routine therapy. Resistance to azithromycin is highest in the European region at almost 26 percent. As bacteria become resistant to first-line antibiotics, doctors must increasingly rely on last-resort treatments, which are more expensive and harder to access, especially in low- and middle-income countries. Development of new antibiotics has slowed to a halt due to the lack of a profitable market and incentives for private companies to invest in them. The European Commission has proposed an exclusivity “voucher” to incentivize companies that develop new antibiotics as part of its pharma package. The European Parliament also wants to introduce market entry rewards for new drugs and an EU subscription payment model — proposals that are currently under debate to upgrade EU pharma rules. Other models, such as the Global Antibiotic Research and Development Partnership, seek to develop new antibiotics on a non-profit basis.

LONDON — The U.K. government has drawn up proposals to increase the amount the National Health Service pays pharmaceutical firms for drugs, in a bid to steer U.S. President Donald Trump away from his threatened tariffs on the sector. Officials briefed the Trump administration on fresh proposals to adjust how the NHS prices medicines earlier this week, two industry figures told POLITICO. The core element of the plan includes raising the National Institute for Health and Care Excellence (NICE) threshold by 25 percent.  The NICE threshold measures whether a treatment offers good value for money. Under the current rules, if a drug costs the NHS between £20,000 and £30,000 for every extra year of good-quality life it delivers to a patient, it is considered good value. Increasing the threshold would make it easier for pricier drugs to reach patients, but would mean the NHS will pay more overall for medicines. The government is expected to brief U.K. pharmaceutical companies on the details later this week, the same two figures cited above said.  One of the figures said the government had long been resistant to changing the NICE threshold due to the cost to the Treasury for no direct extra benefit, but “we have kicked up enough of a stink and they have given in. This is the price you have to pay post-Trump for global pharma to continue to play in the U.K.” Donald Trump has threatened to impose tariffs of up to 100 percent on pharmaceutical imports. | Anna Moneymaker/Getty Images Trump has threatened to impose tariffs of up to 100 percent on pharmaceutical imports. The trade pact Britain and the U.S. signed in May left the door open to “preferential treatment” on tariffs — but only if the U.K. improved conditions for American pharma companies operating in Britain.  A U.K. government spokesperson said: “The pharmaceutical sector and the innovative medicines it produces are critical to our NHS, our economy and the Plan for Change. Through our Life Sciences Sector Plan, we’ve committed to working with industry to accelerate growth in spending on innovative medicines compared to the previous decade.” The spokesperson added: “We’ve secured a landmark economic partnership with the US that includes working together on pharmaceutical exports from the UK whilst improving conditions for pharmaceutical companies here. We’re now in advanced discussions with the US Administration to secure the best outcome for the UK, reflecting our strong relationship and the opportunities from close partnership with our pharmaceutical industry.” ‘HARD NEGOTIATORS’ NICE, a regulatory body within the NHS, measures the cost-effectiveness of new drugs by weighing their impact on patients’ life against its price. If a drug’s benefits don’t justify its cost, the NHS does not recommend it for use, forcing pharmaceuticals to negotiate price cuts until the drug is deemed cost-effective.  Although London has presented its proposal to Washington, it remains unclear how it’s been received across the Atlantic. Pharmaceutical companies have long been locked in talks with the government over NHS drug spending amid fears that more investment could flee Britain. Science Minister Vallance previously hinted the NHS would need to pay more if Britain wanted to stay attractive for investment, warning that Trump’s tariffs would make things worse if London doesn’t make “offers in this direction.”  But divisions persist inside government. Business and Trade Secretary Peter Kyle has indicated pharma companies are proving “hard negotiators” amid crunch talks tied to Trump’s deadline, saying they “know how to use the media and the press.” Starmer’s chief business adviser Varun Chandra flew to Washington earlier this month to try to head off Trump’s threatened tariffs, which were put on hold until the administration negotiates agreements with pharma giants.

Spanish Prime Minister Pedro Sánchez said he wants to enshrine the right to abortion in the Constitution, following the example of France, which last year became the first country in the world to take the historic step.  In a post on social media, Sánchez said he is planning to bring a proposal to Parliament to constitutionalize the right to voluntary termination of pregnancy. “With this government, there will be no step backward in social rights,” he said.  The post follows approval by Madrid’s city council of a measure which will make health centers inform women considering abortion about so-called “post-abortion trauma.” The measure was supported by the center-right Popular Party (PP) and the far-right Vox party.  “The PP has decided to merge with the far right. That’s their choice,” Sánchez wrote. “They can do that. But not at the expense of women’s freedoms and rights.” The prime minister said he plans to amend Spain’s abortion laws to prevent “misleading or scientifically inaccurate information about abortion from being provided.”  

Europe spends more on tackling mental health conditions that any other region in the world, but with rates of illness at high levels, it still needs to urgently invest more. That’s one of the warnings from the World Health Organization in two reports released Tuesday — one on governments’ mental health policies and another on mental wellbeing. Together, the reports, which collate rates of mental health conditions and policies from 2021 and 2024, respectively, point to countries playing catch-up as mental health disorders weigh down national health budgets and contribute significantly to disability. Governments in WHO’s Europe region spent $51.76 per capita in 2024 on mental health, far more than any other region globally. The next-highest-spending region is the Americas, with $6.86 per capita. European countries also spend a higher proportion of their overall budget on mental wellbeing, with 4.5 percent compared to the global median figure of 2.1 percent. The 2.1 percent figure is stuck at the same level as in 2017 and 2020, with no evidence of greater spending at any income level, WHO officials told reporters Monday. High-income countries in general greatly outspend lower- and middle income countries on mental health. In all WHO regions, the estimated prevalence of mental disorders has increased since 2001, with the greatest increases taking place in WHO’s Americas region (rising from 15.3 percent to 17.1 percent in 2021) and European region (from 14 percent to 15.4 percent). Globally, there were more than 1 billion people living with a mental health disorder, according to 2021 data. The prevalence of mental health disorders is generally evenly distributed by region, although Europe has lower rates of anxiety disorders than elsewhere in the world, with 3.7 percent of the population compared with 4.4 percent globally. Europe and Southeast Asia both have higher rates of intellectual development disorders than the global rate of 1.2 percent, with 1.8 and 2.7 percent. Underspending on mental health is costing people access to the services they need, the WHO told reporters Monday. Just 9 percent of people with depression, and 40 percent of people with psychosis, get treatment. “Those are the numbers to be worried about,” said Dévora Kestel, a director at the WHO’s department for non-communicable diseases and mental health.

The European Commission may be breaking the law by withholding funds from health NGOs without explanation, 12 MEPs have warned President Ursula von der Leyen in a letter obtained by POLITICO. European health NGOs say they signed partnership agreements with the Commission last year, outlining their planned activities with the understanding that they would receive operating grants for the following financial year. These payments cover daily overhead costs, such as salaries. But the Commission failed to issue a call for grants as expected, while officials informally told NGOs to expect no funding. “Withholding funds, especially without explanation, undermines trust, possibly breaches legal obligations, and weakens the fabric of European cooperation in health policy. The status quo is unacceptable and cannot be allowed to continue,” said the letter, sent July 17 and signed by MEPs from the Socialists and Democrats, Renew and the Greens. “Civil society and patient organisations signed agreements with the Commission in good faith, planned their activities accordingly, and have now been left without the operating support on which they rely,” the MEPs wrote. Several health NGOs have already made staff redundant due to the lack of expected funds. The Commission’s failure to agree on its 2026 work program and subsequent withholding of grants threatens Europe’s “democratic integrity,” the letter continued. “If these key stakeholders, the very voice of patients, citizens, and public health advocates, are weakened or silenced in Brussels, private commercial interests will fill the vacuum.” POLITICO contacted the Commission for a response to the letter. A Commission spokesperson responded on July 17 that the final EU4Health work program is expected to be “adopted and published very soon.” Several health NGOs have already made staff redundant due to the lack of expected funds. | Pool Photo by Andreas Arnold/EPA “The Commission values the contribution of civil society and many of the funded actions in the forthcoming work programme will involve NGOs’ participation, as well as certain actions which will be specifically directed to NGOs,” the spokesperson said. CORRECTION: This article has been updated to clarify when the letter was sent.