Disclaimer POLITICAL ADVERTISEMENT * This is sponsored content from AstraZeneca. * The advertisement is linked to public policy debates on the future of cancer care in the EU. More information here. Europe has made huge strides in the fight against cancer.[1] Survival rates have climbed, detection has improved and the continent has become home to some of the world’s most respected research hubs.[2],[3] None of that progress came easy — it was built on years of political attention and cooperation across borders. However, as we look to 2026 and beyond, that progress stands at a crossroads. Budget pressures and tougher global competition threaten to push cancer and health care down the EU agenda. Europe’s Beating Cancer Plan — a flagship initiative aimed at expanding screening, improving early detection and boosting collaboration — is set to expire in 2027, with no clear plan to secure or extend its gains.[4],[5] “My [hope is that we can continue] the work started with Europe’s Beating Cancer Plan and make it sustainable… [and] build on the lessons learned, [for other disease areas] ” says Antonella Cardone, CEO of Cancer Patients Europe. A new era in cancer treatment Concern about the lapsing initiative is compounded by two significant shifts in health care: declining investment and increasing scientific advancement. Firstly, Europe has seen the increased adoption of cost-containment policies by some member states. Under-investment in Europe in cancer medicines has been a challenge — specifically with late and uneven funding, and at lower levels than international peers such as the US — potentially leaving patients with slower and more limited access to life-saving therapies.[6],[7],[8] Meanwhile, the U.S., which pays on average double for medicines per capita than the EU,[9] is actively working to rebalance its relationship with pharmaceuticals to secure better pricing (“fair market value”) through policies across consecutive administrations.[10] All the while, China is rapidly scaling investment in biotech and clinical research, determined to capture the trials, talent, and capital that once flowed naturally to Europe.[11] The rebalancing of health and life-science investment can have significant consequences. If Europe does not stay attractive for life-sciences investment, the impact will extend beyond cancer patient outcomes. Jobs, tax revenues, advanced manufacturing, and Europe’s leadership in strategic industries are all at stake.[12] Secondly, medical science has never looked more promising.[7] Artificial intelligence is accelerating drug discovery, clinical trials, and diagnostics, and the number of approved medicines for patients across Europe has jumped from an average of one per year between 1995 and 2000 to 14 per year between 2021 and 2024.[13],[14],[15], [7] Digital health tools and innovative medtech startups are multiplying, increasing competitiveness and lowering costs — guiding care toward a future that is more personalized and precise.[16],[17] Europe stands at the threshold of a new era in cancer treatment. But if policymakers ease up now, progress could stall — and other regions, especially the U.S. and China, are more than ready to widen the innovation gap. Recognizing the strategic investment Health spending is generally treated as a budget item to be contained. Yet investment in cancer care has been one of Europe’s smartest economic bets.[18],[19] The sector anchors millions of high-skilled jobs (it employs around 29 million people in the EU[11]) and attracts global life sciences investment. According to the European Commission, the sector contributes nearly €1.5 trillion to the EU economy.[12] Studies from the Institute of Health Economics confirm that money put into research directly translates into better survival outcomes.[20] The same report shows that although the overall spend on cancer is increasing, the cost per patient has actually decreased since 1995, suggesting that innovative treatments are increasing efficiency.[20] Those gains matter not only to patients and families, but to Europe’s long-term stability: healthier populations mean fewer costs down the line, stronger productivity, and more sustainable public finances.[20] Fixing Europe’s access gap Cancer medicines bring transformative value — to patients, to society and to the wider economy. [21] However, even as oncology therapies advance, patients across Europe are not benefiting equally. EFPIA’s 2024 Patients W.A.I.T. indicator shows that, on average, just 46 percent of innovative medicines approved between 2020 and 2023 were available to patients in 2024.[22] On average, it takes 578 days for a new oncology medicine to reach European patients, and only 29 percent of drugs are fully available in all member states.[23] This is not caused by a lack of breakthrough medicines, but by national policy mechanisms that undervalue innovation. OECD and the Institute for Health Economics data show that divergent HTA requirements, rigid cost-effectiveness thresholds, price-volume clawbacks, ad hoc taxes on pharmaceutical revenues and slow national reimbursement decisions collectively suppress timely access to new cancer medicines across the EU.[24] These disparities cut against Europe’s long-standing reputation as a collection of societies that values equitable, high-quality care for all of its citizens. It risks eroding one of the EU’s defining strengths: the commitment to fairness and collective progress. Cancer policy solutions for the EU Although this is ultimately a matter for member states, embedding cancer as a permanent EU priority — backed by funding, coordination, and accountability — could give national systems the incentives and strategic direction to buck these trends. These actions will reassure pharmaceutical companies that Europe is serious about attracting clinical trials and the launch of new medicines, ensuring that its citizens, societies and economies enjoy the benefits this brings. Europe’s Beating Cancer Plan delivered progress, but its expiry presents a pivotal moment. 2026 and beyond bring a significant opportunity for the EU to build on this by ensuring that member states implement National Cancer Control Plans and have clear targets and accountability on their national performance, including on investment and access. To do this, EU policymakers should consider three actions as an immediate priority with lasting impact: * Embed cancer and investment within EU governance. Build it into the European Semester on health with mandatory indicators, regular reviews, and accountability frameworks to ensure continuity. This model worked well during Covid-19 and should be adapted for non-communicable diseases starting with cancer as a pilot. * Secure stable and sufficient funding. The Multiannual Financial Framework must ensure adequate funding for health and cancer to encourage coordinated initiatives across member states. * Strengthen EU-level coordination. Ensure that pan-EU structures such as the Comprehensive Cancer Centres and Cancer Mission Hubs are adequately funded and empowered. These are the building blocks of a lasting European commitment to cancer. With action, Europe can secure a sustainable foundation for patients, resilience and continued scientific excellence. -------------------------------------------------------------------------------- [1] European Commission, OECD/European Observatory on Health Systems and Policies. 2023. State of Health in the EU: Synthesis Report 2023. Available at: https://health.ec.europa.eu/system/files/2023-12/state_2023_synthesis-report_en.pdf [Accessed December 2025] [2] Efpia. 2025. Cancer care 2025: an overview of cancer outcomes data across Europe. Available at: https://www.efpia.eu/news-events/the-efpia-view/statements-press-releases/ihe-cancer-comparator-report-2025/ [Accessed December 2025] [3] Cancer Core Europe. 2024. Cancer Core Europe: Advancing Cancer Care Through Collaboration. Available at: https://www.cancercoreeurope.eu/cce-advancing-cancer-care-collaboration/ [Accessed December 2025] [4] European Commission. 2021. Europe’s Beating Cancer Plan. Available at:https://health.ec.europa.eu/system/files/2022-02/eu_cancer-plan_en_0.pdf [Accessed December 2025] [5] European Parliament. 2025. Europe’s Beating Cancer Plan: Implementation findings. https://www.europarl.europa.eu/RegData/etudes/STUD/2025/765809/EPRS_STU(2025)765809_EN.pdf [Accessed December 2025] [6] Hofmarcher, T., et al. 2024. Access to Oncology Medicines in EU and OECD Countries (OECD Health Working Papers, No.170). OECD Publishing. Available at: https://www.oecd.org/content/dam/oecd/en/publications/reports/2024/09/access-to-oncology-medicines-in-eu-and-oecd-countries_6cf189fe/c263c014-en.pdf [Accessed December 2025] [7] Manzano, A., et al. 2025. Comparator Report on Cancer in Europe 2025 – Disease Burden, Costs and Access to Medicines and Molecular Diagnostics (IHE). Available at: https://ihe.se/app/uploads/2025/03/IHE-REPORT-2025_2_.pdf [Accessed December 2025] [8] Efpia. [no date]. Europe’s choice. Available at: https://www.efpia.eu/europes-choice/ [Accessed December 2025] [9] OECD. 2024. Prescription Drug Expenditure per Capita. https://data-explorer.oecd.org/vis?lc=en&pg=0&snb=1&vw=tb&df[ds]=dsDisseminateFinalDMZ&df[id]=DSD_SHA%40DF_SHA&df[ag]=OECD.ELS.HD&df[vs]=&pd=2015%2C&dq=.A.EXP_HEALTH.USD_PPP_PS%2BPT_EXP_HLTH._T..HC51%2BHC3.._T…&to[TIME_PERIOD]=false&lb=bt [Accessed December 2025] [10] The White House. 2025. Delivering most favored-nation prescription drug pricing to American patients. Available at: https://www.whitehouse.gov/presidential-actions/2025/05/delivering-most-favored-nation-prescription-drug-pricing-to-american-patients/ [Accessed December 2025] [11] Eleanor Olcott, Haohsiang Ko and William Sandlund. 2025. The relentless rise of China’s Biotechs. Financial Times. Available at: https://www.ft.com/content/c0a1b15b-84ee-4549-85eb-ed3341112ce5 [Accessed December 2025] [12] European Commission, Directorate-General for Communication. 2025. Making Europe a Global Leader in Life Sciences. Available at: https://commission.europa.eu/news-and-media/news/making-europe-global-leader-life-sciences-2025-07-02_en [Accessed December 2025] [13] Financial Times. 2025. How AI is reshaping drug discovery. Available at: https://www.ft.com/content/8c8f3c10-9c26-4e27-bc1a-b7c3defb3d95 [Accessed December 2025] [14] Seedblink. 2025. Europe’s HealthTech investment landscape in 2025: A deep dive. https://seedblink.com/blog/2025-05-30-europes-healthtech-investment-landscape-in-2025-a-deep-dive [15] European Commission. [No date]. Artificial Intelligence in healthcare. Available at: https://health.ec.europa.eu/ehealth-digital-health-and-care/artificial-intelligence-healthcare_en [Accessed December 2025] [16] Codina, O. 2025. Code meets care: 20 European HealthTech startups to watch in 2025 and beyond. EU-Startups. Available at: https://www.eu-startups.com/2025/06/code-meets-care-20-european-healthtech-startups-to-watch-in-2025-and-beyond [Accessed December 2025] [17] Protogiros et al. 2025. Achieving digital transformation in cancer care across Europe: Practical recommendations from the TRANSiTION project. Journal of Cancer Policy. Available at: https://www.sciencedirect.com/science/article/pii/S2213538325000281 [Accessed December 2025] [18] R-Health Consult. [no date]. The case for investing in a healthier future for the European Union. EFPIA. Available at: https://www.efpia.eu/media/xpkbiap5/the-case-for-investing-in-a-healthier-future-for-the-european-union.pdf [Accessed December 2025] [19] Pousette A., Hofmarcher T. 2024.Tackling inequalities in cancer care in the European Union. Available at: https://ihe.se/en/rapport/tackling-inequalities-in-cancer-care-in-the-european-union-2/ [Accessed December 2025] [20] Efpia. 2025. Comparator Report Cancer in Europe 2025. Available at: https://www.efpia.eu/media/0fbdi3hh/infographic-comparator-report-cancer-in-europe.pdf [Accessed December 2025] [21] Garau, E. et al. 2025. The Transformative Value of Cancer Medicines in Europe. Dolon Ltd. Available at: https://dolon.com/wp-content/uploads/2025/09/EOP_Investment-Value-of-Oncology-Medicines-White-Paper_2025-09-19-vF.pdf?x16809 [Accessed December 2025] [22] IQVIA. 2025. EFPIA Patients W.A.I.T. Indicator 2024 Survey. Available at: https://www.efpia.eu/media/oeganukm/efpia-patients-wait-indicator-2024-final-110425.pdf [Accessed December 2025] [23] Visentin M. 2025. Improving equitable access to medicines in Europe must remain a priority. The Parliament. Available at: https://www.theparliamentmagazine.eu/partner/article/improving-equitable-access-to-medicines-in-europe-must-remain-a-priority [Accessed December 2025] [24] Hofmarcher, T. et al. 2025. Access to novel cancer medicines in Europe: inequities across countries and their drivers. ESMO Open. Available at: https://www.esmoopen.com/action/showPdf?pii=S2059-7029%2825%2901679-5 [Accessed December 2025]

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.

Rick Pazdur, the FDA’s top drug regulator, told staff Tuesday he submitted his resignation to the agency, an abrupt departure weeks after he was convinced by Commissioner Marty Makary to take the post to help bring stability to an agency reeling from months of upheaval, according to four people familiar with the decision granted anonymity to discuss the move. The decision — which comes days after top vaccine regulator Vinay Prasad said the agency would ratchet up regulatory requirements for new vaccines — is almost certain to raise new questions about Makary’s leadership of the FDA. Pazdur in recent weeks clashed with Makary over the Commissioner’s National Priority Voucher program, according to media reports. That program — which aims to speed final review of drugs that address health priorities, pose a transformative innovative impact, address an unmet medical need, help onshoring efforts or increase affordability — was also criticized by Pazdur’s predecessor, George Tidmarsh. FDA experts have worried the involvement of political appointees in the process of choosing which firms receive a voucher could raise questions about the program’s integrity. STAT first reported the news of Pazdur’s decision to retire. It is unclear if the decision is final — one person familiar with the decision said the longtime cancer drug regulator has 30 days to change his decision. “We respect Dr. Pazdur’s decision to retire and honor his 26 years of distinguished service at the FDA,” an FDA spokesperson said in a statement. “As the founding director of the Oncology Center of Excellence, he leaves a legacy of cross-center regulatory innovation that strengthened the agency and advanced care for countless patients. His leadership, vision, and dedication will continue to shape the FDA for years to come.” The White House and Pazdur did not immediately respond to requests for comment. Pazdur, a 26-year agency veteran, initially rebuffed efforts by Makary to convince him to assume leadership of the FDA’s Center for Drug Evaluation and Research — but ultimately agreed to take the job after being assured he would be given autonomy in the role free from political influence and the ability to rehire staff.

Since a young age growing up in Italy, the workings of the human body have fascinated me — a curiosity that brought me to medical research. I’ve always wanted to help people. And through discovering new medicines, we can potentially help millions of patients. What drives me — and so many other scientists in Europe and around the globe — is a desire to find new therapies that will improve people’s lives, especially in areas where there is currently an unmet need. As a personal example, my passion for psychiatric diseases stems from witnessing the profound suffering they cause not just to the patient, but also within families. The global rise of mental health illnesses deeply concerns me and demands urgent attention. Focusing on what matters to patients In current European policy discussions, the term ‘unmet medical need’ is often defined by a simple question: Will the treatment keep the person alive for longer? Survival is obviously an important marker, but it is not the only one. The burden of the disease and the patient’s quality of life must be considered too. For example, a skin disease might not be life-threatening or affect a person’s ability to function autonomously, but it can have an enormous impact on their mental state and productivity. There are many layers to the burden of a disease on a personal and emotional level. At the company I work for, Boehringer Ingelheim, we look at an unmet need in its totality to help us make decisions about where to focus our research and development (R&D). We work with patients at every possible step of the R&D process, helping us to incorporate their feedback, and to focus on what the actual unmet patient need is. > We work with patients at every possible step of the R&D process, helping us to > incorporate their feedback, and to focus on what the actual unmet patient need > is. We can’t make true progress on unmet needs unless we understand from the patients themselves what they need or what they think is missing from their treatment options. This is why I am concerned about the European Commission’s proposals in the EU pharma package regarding unmet medical need. As currently written, the definition is too narrow and fails to reflect the diverse challenges faced by patients. This limited scope will not only leave many health conditions unaddressed, it also risks stifling innovation by discouraging R&D efforts aimed at tackling the broader, more complex needs of patients. For example, an injection that can be done at home rather than in a hospital or a pill replacing an injection could make a world of difference for patients. For people with scleroderma, who often suffer from swelling and limited mobility in their fingers, a tool that assists in removing a pill from its packaging could significantly enhance their independence and ease of medication management, improving both comfort and quality of life. By narrowing the concept of unmet medical need to strict metrics, we risk drifting away from real patient need. Advances in science will guide our decisions on where to invest in R&D At Boehringer Ingelheim, we look for areas of unmet patient need, how they have changed and progressed and where we might enter or leave an area of research, either because the science has advanced, or because there are already treatments in the pipeline. We use the World Health Organization’s International Statistical Classification of Diseases and Related Health Problems — which categorizes thousands of disease areas, from cancer to infectious diseases. We look at where the breakthrough science matches our capabilities and expertise and what patients need the most. For Boehringer Ingelheim, this includes, for example, oncology, inflammatory diseases and serious mental health illnesses, as well as research into the broader cardio-renal metabolic diseases. The challenges of research into unmet patient need — and why we keep going My hope as a research scientist is that we can solve some of the areas of unmet patient need we are working on today and progress toward a future where science empowers us to treat even more diseases. Scientific research is never linear, and looking for a first-in-class treatment can take us into uncharted territory. Our work relies on hypotheses based on the scientific knowledge we have at the time. We’re constantly learning from failure, and we apply this knowledge to future research. In clinical research, serendipity often plays a part. We follow the science, which sometimes leads us to finding treatments for diseases that were not originally targeted.  We must learn from the science and readjust accordingly to bring new treatment options forward.  The more we progress, the more we also want Europe to remain in the global innovation race and at the forefront of pharmaceutical R&D. > It will be vital that Europe takes the right political decisions to ensure > that tomorrow’s technologies and treatments are also researched and developed > in Europe, and swiftly reach patients wherever they live.   Once the global powerhouse of pharmaceutical innovation, Europe has sadly seen a significant decline in its global share of R&D investments over the last two decades, with troubling consequences: 25 years ago, one in every two new treatments originated from Europe. Today, it is fewer than one in five. In clinical trials, Europe’s share of clinical trials has dramatically reduced from 22% in 2013, to 18% in 2018 and to 12% in 2023. These trends underscore the urgent need for action and a more supportive environment, as recently stipulated by the EU’s political leadership, who now acknowledge the pharmaceutical industry as a strategic sector for the future of Europe. It will be vital that Europe takes the right political decisions to ensure that tomorrow’s technologies and treatments are also researched and developed in Europe, and swiftly reach patients wherever they live.   As an optimist by nature, a passionate scientist and committed European, I am confident that nurturing scientific progress and a patient-first mindset in Europe can pave the way for more transformative therapies and help millions of people lead healthier and happier lives.

This is a paid editorial funded by Sanofi. Pragmatism is simple. Pragmatism is powerful. We are all working toward common goals in healthcare, and pragmatism can be a driver to turn ideas into a reality. When talking about helping those living with cancer, we continuously need to evolve and find better ways of approaching clinical trials. As the oncology treatment landscape continues to advance, the bar to making meaningful improvements versus standard of care continues to be raised. As cancer prognoses improve and we aim to catch and treat it earlier, time to achieve significant overall survival (OS)* is increasing, if ever reached. As a result, in instances where regulatory/reimbursement processes rely on OS, patient access to innovative treatments is delayed or compromised.1 Faced with this, we need to evolve the way we choose and interpret clinical and biological endpoints to accelerate access to potential life-saving treatments and advance health through personalized medicine. > At Sanofi, we strive to modernize the treatment of cancer by working to bring > new therapies for difficult-to-treat cancers” At Sanofi, we strive to modernize the treatment of cancer by working to bring new therapies for difficult-to-treat cancers and that starts with focusing our efforts to make a difference where we can. To that end, we work closely with leading cancer institutions and cooperative groups, as well as biotech companies and public-private initiatives, like the Paris-Saclay Cancer Cluster, to move the needle on oncology R&D. We’ve centered our efforts on select hematologic malignancies and select solid tumors with critical unmet needs, including multiple myeloma (MM), acute myeloid leukemia (AML), certain types of lymphomas, as well as gastrointestinal and lung cancers.   Not all cancers are created equal > Cancer treatment is not a one-size-fits-all approach – and neither are the > endpoints that are crucial to ensure that any treatments are truly beneficial > to patients” Cancer treatment is not a one-size-fits-all approach – and neither are the endpoints that are crucial to ensure that any treatments are truly beneficial to patients. It is vital that we consider the myriad factors – such as cancer type, stage and the individual preferred outcomes for patients – when determining clinical trial endpoints. By using certain oncology-relevant endpoints, there is an opportunity for earlier measurement of medicine efficacy. Practically speaking, this enables shorter clinical trial durations, potentially leading to quicker approval of treatments that may benefit patients.   Minimal residual disease (MRD)** is an example of an endpoint that can provide earlier readouts.2,3 Both Sanofi and the MM community see this as an opportunity to continue to generate data that demonstrate the correlation between certain patient-relevant endpoints, such as MRD, depth of response and longer-term clinical outcomes.4,5,6,7 To see change, we need to be agile and pragmatic There is a need – at a regulatory and heath technology assessment (HTA) level – to define and accelerate the qualification of patient-relevant endpoints beyond OS. This will support research into treatments that do more than prolong survival, as they may enhance quality of life and other important efficacy outcomes that really matter to patients. Access to newer, potentially more effective treatments can be accelerated if the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) best practice is considered. The FDA ODAC recently decided there is available data to support the use of MRD as an intermediate endpoint for accelerated approval in MM clinical trials, in both newly diagnosed and relapsed/refractory disease settings.8 > “Despite the available body of evidence, the HTA agencies still consider OS > the ‘gold standard’ endpoint in oncology, creating a misalignment with science > and patients’ interests” Despite the available body of evidence, the HTA agencies still consider OS the ‘gold standard’ endpoint in oncology,9 creating a misalignment with science and patients’ interests to get earlier access to treatments. At a national level, assessment frameworks must also evolve and place greater value on quality-of-life benefits that really matter to patients. These are the type of patient-centered policy changes that the Europe’s Beating Cancer Plan should champion in its next phase. To stay relevant, Europe must consider pursuing the development of guidelines from regulatory, clinical and HTA perspectives to enable the use of biological and patient-centered endpoints. We must first standardize and address key uncertainties in aspects such as key methods, frequencies and sensitivity thresholds at which these new endpoints should be measured10 – and that’s not going to happen without a catalyst. It is very clear that Europe has made huge progress and built strong foundations in its fight against cancer. Now is the time to build on it and think more broadly. Considering biological and additional patient-relevant endpoints is crucial, as we look into measuring outcomes beyond OS. MAT-GLB-2407554-v1.0-11/2024 | November 2024 ____________________ Definitions: * OS: Duration of patient survival from the time of treatment initiation. ** MRD: MRD refers to the small number of cancerous cells that may survive in the body after treatment. The number of surviving cells in MRD is too small for traditional tests (like biopsies or blood tests) to detect. So, a positive MRD test result indicates that cancer cells are present in the body – even at a minute level. References: 1. European Federation of Pharmaceutical Industries and Associations, Oncology Platform. September, 2023. White Paper – Improving the understanding, acceptance and use of oncology–relevant endpoints in HTA body / payer decision-making. Accessed on 6 November, 2024: https://www.efpia.eu/media/t2nlhr0k/improving-the-understanding-acceptance-and-use-of-oncology-relevant-endpoints.pdf 2. Anderson KC, Auclair D, Kelloff GJ, et al. The Role of Minimal Residual Disease Testing in Myeloma Treatment Selection and Drug Development: Current Value and Future Applications. Clin Cancer Res. 2017;23(15):3980-3993. doi:10.1158/1078-0432.CCR-16-2895 3. Avet-Loiseau H, Ludwig H, Landgren O, et al. Minimal Residual Disease Status as a Surrogate Endpoint for Progression-free Survival in Newly Diagnosed Multiple Myeloma Studies: A Meta-analysis. Clin Lymphoma Myeloma Leuk. 2020;20(1):e30-e37. doi:10.1016/j.clml.2019.09.622 4. Perrot A, Lauwers-Cances V, Corre J, et al. Minimal residual disease negativity using deep sequencing is a major prognostic factor in multiple myeloma. Blood. 2018;132(23):2456-2464. doi:10.1182/blood-2018-06-858613 5. Landgren O, Prior TJ, Masterson T, et al. EVIDENCE meta-analysis: evaluating minimal residual disease as an intermediate clinical end point for multiple myeloma. Blood. 2024;144(4):359-367. doi:10.1182/blood.2024024371 6. Meseha M, Hoffman J, Kazandjian D, Landgren O, Diamond B. Minimal Residual Disease-Adapted Therapy in Multiple Myeloma: Current Evidence and Opinions. Curr Oncol Rep. 2024;26(6):679-690. doi:10.1007/s11912-024-01537-2 7. Munshi NC, Avet-Loiseau H, Anderson KC, et al. A large meta-analysis establishes the role of MRD negativity in long-term survival outcomes in patients with multiple myeloma. Blood Adv. 2020;4(23):5988-5999. doi:10.1182/bloodadvances.2020002827 8. FDA. April 12, 2024. Final Summary Minutes of the Oncologic Drugs Advisory Committee Meeting April 12, 2024. Accessed on 6 November, 2024: https://www.fda.gov/media/180108/download 9. Holstein SA, Suman VJ, McCarthy PL. Should Overall Survival Remain an Endpoint for Multiple Myeloma Trials? Curr Hematol Malig Rep. 2019;14(1):31. doi:10.1007/s11899-019-0495-9 10. Myeloma Patients Europe. January, 2023. Patient and haematologist perspectives on minimal residual disease testing in myeloma. Accessed on 6 November, 2024: https://www.mpeurope.org/wp-content/uploads/2023/01/MRD-in-myeloma-Report.pdf

Without doubt, when we reflect on the many achievements of the UK healthcare system and our NHS, there is much to be proud of – but today we are faced with a paradox. We are living in a time where breakthrough science has the potential to transform treatment options for some of the toughest health conditions faced by patients in the UK. Yet the question is whether the system is currently set up to match the pace of innovation that science provides, so patients can fully benefit? At Johnson & Johnson, our teams are continuously working to get ahead of the most complex diseases affecting patients and their families, but we know that these treatments only matter if patients can access them when they need them. > The question is whether the system is currently set up to match the pace of > innovation that science provides, so patients can fully benefit? The last decade has seen a series of changes to the UK access environment for new medicines that have made it increasingly challenging for the NHS to deliver the innovative care that patients need. Right now, just 56% of all new medicines approved by the European Medicines Agency (EMA) are available to patients in England and only 54% in Scotland. This compares with 88% in Germany and 77% in Italy.[i]     Without bold reform, access to medicines will continue to stagnate in the UK, risking our position as a leading destination to do life sciences. We are already witnessing the impact. The government’s Office for Life Sciences charts that foreign direct investment in the UK life sciences sector more than halved between 2021 and 2023.[ii] Now with a new government in place, we have an unparalleled opportunity to strengthen collaboration across the life sciences community, achieve our shared ambitions for the sector and truly deliver the best possible care for every patient in the UK. The good news is that we have a strong recent precedent to draw upon. The UK’s COVID-19 vaccination programme showed what’s possible when political leaders, the pharmaceutical industry and health systems work together. By doing so, we were able to bring innovation directly into the hands of healthcare providers, deploy the nation’s resources more effectively and, most importantly of all, transform outcomes for patients. The challenge now is to build on the lessons learned during the pandemic. Even the most pioneering drugs and therapies are only valuable if patients can actually be treated with them. Going forward, it is a certainty that UK policymakers must prioritise a shared vision and joint action to ensure the NHS can deliver “the best that modern science can offer”. [iii] > Even the most pioneering drugs and therapies are only valuable if patients can > actually be treated with them. Bodies that assess new medicines for adoption by health systems, such as The National Institute for Health and Care Excellence (NICE) for NHS England, must strike a tricky balance. Appraisal frameworks need to be robust and inclusive while keeping pace with exciting scientific developments and evolving treatment pathways. However, in my opinion, a recent review of NICE’s methods[iv] was a missed opportunity to incorporate more insight from the life sciences sector and introduce greater flexibility into the system. For one, severity modifiers were introduced. These enable adjustments to the thresholds at which medicines for particularly debilitating conditions are assessed. While these modifiers could benefit a wider range of patients, their rigid, formulaic criteria may unintentionally limit access to treatments for those with the most severe conditions. To highlight another example, an increasing number of innovative medicines are effective across multiple rare diseases or cancers.[v] Unfortunately, the current criteria NHS England relies upon does not straightforwardly evaluate the differential value that such multi-indication medicines provide. If a clear and accessible route were established to include indication-based pricing, it could make it easier for these critical new medicines to be recommended for use and for more patients to receive their full benefit. There’s real urgency when we’re talking about access to new medicines in the UK. Currently, just 25% of new oncology medicines approved by the EMA between 2019 and 2022 are fully available on the NHS in England.i This means that the most effective treatment may simply be unavailable for some patients, not due to its efficacy, but because of where they live. Timely access to the right treatment does two things, it keeps people healthy and prevents disease worsening so they can participate in society and a thriving economy. It also impacts patient outcomes and reduces the likelihood of co-morbidity. As highlighted in the September 2024 Lord Darzi report, by improving access to care and addressing long-term sickness the NHS plays a role in driving national prosperity.[vi] The recently announced Voluntary Scheme for Branded Medicine Pricing, Access and Growth (VPAG) Investment Programme is certainly a step in the right direction. Of the £400 million investment secured, 5% will focus on modernising the access environment.[vii] It will be critical for this investment to deliver meaningful change that ensures the UK avoids repeatedly falling behind when it comes to accessing the medicines of the future. Over and above individual policy and regulatory changes, the path to lasting improvements in the access landscape lies with all parts of the life sciences ecosystem working together to fuel a virtuous cycle of innovation. Only by actively working together – government, healthcare providers and industry – can we create an environment that fosters innovation and, more importantly, brings its benefits to patients. This year, ‎‎ ‎Johnson & Johnson proudly celebrated 100 years of operations in the UK. Our expertise has served as the foundation for decades of successful partnership with patients, healthcare providers, clinical researchers and the NHS. That’s why we wholeheartedly welcome the new government’s ambition to collaborate with the private sector on life sciences innovation. After all, healthy people build healthy societies and healthy economies. > …The path to lasting improvements in the access landscape lies with all parts > of the life sciences ecosystem working together to fuel a virtuous cycle of > innovation. Follow Johnson & Johnson Innovative Medicine UK on LinkedIn for updates on our business, our people and our community -------------------------------------------------------------------------------- [i] EFPIA (2024). EPFIA Patients W.A.I.T. Indicator 2023 Survey. Available here: https://efpia.eu/media/vtapbere/efpia-patient-wait-indicator-2024.pdf. Accessed September 2024. [ii] HM Government (2024). Life Sciences Competitiveness Indicators 2024: summary. Available here: https://www.gov.uk/government/publications/life-sciences-sector-data-2024/life-sciences-competitiveness-indicators-2024-summary. Accessed September 2024. [iii] The Labour Party (2024). Labour’s Manifesto: Build an NHS fit for the future. Available here: https://labour.org.uk/change/build-an-nhs-fit-for-the-future/. Accessed September 2024. [iv] NICE (2024). Public board meetings – NICE methods agenda board paper. Available here: https://www.nice.org.uk/get-involved/meetings-in-public/public-board-meetings/agenda-and-papers-march-2024. Accessed September 2024. [v] Mestre-Ferrandiz, J., Towse, A., Dellamano, R. and Pistollato, M. (2015) Multi-indication Pricing: Pros, Cons and Applicability to the UK. OHE Seminar Briefing. Available here: https://www.ohe.org/publications/multi-indication-pricing-pros-cons-and-applicability-uk/. Accessed September 2024. [vi] Lord Ari Darzi (2024). Independent Investigation of the National Health Service in England. Available here: https://assets.publishing.service.gov.uk/media/66e1b49e3b0c9e88544a0049/Lord-Darzi-Independent-Investigation-of-the-National-Health-Service-in-England.pdf. Accessed September 2024. [vii] HM Government (2024). UK secures £400 million investment to boost clinical trials [Press release]. Available here: https://www.gov.uk/government/news/uk-secures-400-million-investment-to-boost-clinical-trials. Accessed September 2024. CP-476396 | September 2024