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]

After more than three decades in the pharmaceutical industry, I know one thing: science transforms lives, but policy determines whether innovation thrives or stalls. That reality shapes outcomes for patients — and for Europe’s competitiveness. Today, Europeans stand at a defining moment. The choices we make now will determine whether Europe remains a global leader in life sciences or we watch that leadership slip away. It’s worth reminding ourselves of the true value of Europe’s life sciences industry and the power we have as a united bloc to protect it as a European good. Europe has an illustrious track record in medical discovery, from the first antibiotics to the discovery of DNA and today’s advanced biologics. Still today, our region remains an engine of medical breakthroughs, powered by an extraordinary ecosystem of innovators in the form of start-ups, small and medium-sized enterprises, academic labs, and university hospitals. This strength benefits patients through access to clinical trials and cutting-edge treatments. It also makes life sciences a strategic pillar of Europe’s economy. The economic stakes Life sciences is not just another industry for Europe. It’s a growth engine, a source of resilience and a driver of scientific sovereignty. The EU is already home to some of the world’s most talented scientists, thriving academic institutions and research clusters, and a social model built on universal access to healthcare. These assets are powerful, yet they only translate into future success if supported by a legislative environment that rewards innovation. > Life sciences is not just another industry for Europe. It’s a growth engine, a > source of resilience and a driver of scientific sovereignty. This is also an industry that supports 2.3 million jobs and contributes over €200 billion to the EU economy each year — more than any other sector. EU pharmaceutical research and development spending grew from €27.8 billion in 2010 to €46.2 billion in 2022, an average annual increase of 4.4 percent. A success story, yes — but one under pressure. While Europe debates, others act Over the past two decades, Europe has lost a quarter of its share of global investment to other regions. This year — for the first time — China overtook both the United States and Europe in the number of new molecules discovered. China has doubled its share of industry sponsored clinical trials, while Europe’s share has halved, leaving 60,000 European patients without the opportunity to participate in trials of the next generation of treatments. Why does this matter? Because every clinical trial site that moves elsewhere means a patient in Europe waits longer for the next treatment — and an ecosystem slowly loses competitiveness. Policy determines whether innovation can take root. The United States and Asia are streamlining regulation, accelerating approvals and attracting capital at unprecedented scale. While Europe debates these matters, others act. A world moving faster And now, global dynamics are shifting in unprecedented ways. The United States’ administration’s renewed push for a Most Favored Nation drug pricing policy — designed to tie domestic prices to the lowest paid in developed markets — combined with the potential removal of long-standing tariff exemptions for medicines exported from Europe, marks a historic turning point. A fundamental reordering of the pharmaceutical landscape is underway. The message is clear: innovation competitiveness is now a geopolitical priority. Europe must treat it as such. A once-in-a-generation reset The timing couldn’t be better. As we speak, Europe is rewriting the pharmaceutical legislation that will define the next 20 years of innovation. This is a rare opportunity, but only if reforms strengthen, rather than weaken, Europe’s ability to compete in life sciences. To lead globally, Europe must make choices and act decisively. A triple A framework — attract, accelerate, access — makes the priorities clear: * Attract global investment by ensuring strong intellectual property protection, predictable regulation and competitive incentives — the foundations of a world-class innovation ecosystem. * Accelerate the path from science to patients. Europe’s regulatory system must match the speed of scientific progress, ensuring that breakthroughs reach patients sooner. * Ensure equitable and timely access for all European patients. No innovation should remain inaccessible because of administrative delays or fragmented decision-making across 27 systems. These priorities reinforce each other, creating a virtuous cycle that strengthens competitiveness, improves health outcomes and drives sustainable growth. > Europe has everything required to shape the future of medicine: world-class > science, exceptional talent, a 500-million-strong market and one of the most > sophisticated pharmaceutical manufacturing bases in the world. Despite flat or declining public investment in new medicines across most member states over the past 20 years, the research-based pharmaceutical industry has stepped up, doubling its contributions to public pharmaceutical expenditure from 12 percent to 24 percent between 2018 and 2023. In effect, we have financed our own innovation. No other sector has done this at such scale. But this model is not sustainable. Pharmaceutical innovation must be treated not as a cost to contain, but as a strategic investment in Europe’s future. The choice before us Europe has everything required to shape the future of medicine: world-class science, exceptional talent, a 500-million-strong market and one of the most sophisticated pharmaceutical manufacturing bases in the world. What we need now is an ambition equal to those assets. If we choose innovation, we secure Europe’s jobs, research and competitiveness — and ensure European patients benefit first from the next generation of medical breakthroughs. A wrong call will be felt for decades. The next chapter for Europe is being written now. Let us choose the path that keeps Europe leading, competing and innovating: for our economies, our societies and, above all, our patients. Choose Europe. -------------------------------------------------------------------------------- Disclaimer POLITICAL ADVERTISEMENT * The sponsor is European Federation of Pharmaceutical Industries and Associations (EFPIA) * The ultimate controlling entity is European Federation of Pharmaceutical Industries and Associations (EFPIA) * The political advertisement is linked to the Critical Medicines Act. More information here.

An NGO leading the fight for drug price transparency has been forced into signing secrecy pacts with manufacturers, revealing the full might of Big Pharma in keeping its prices hidden. Documents seen by POLITICO reveal that Doctors Without Borders, also known as MSF, signed a confidentiality clause with German pharma company Bayer in a contract to buy contraceptives for distribution in lower-income countries. The deal prevented MSF from disclosing the price it paid for the medicines. A Bayer spokesperson said the company would not comment on the content of agreements with third parties. But it’s not a one-off: A top MSF official admitted the NGO had “reluctantly” signed NDAs with pharma companies on more than one occasion. The news has shocked former staff at MSF who led the NGO’s world-renowned and successful campaign to expose Big Pharma’s drug prices. Tido von Schoen-Angerer, a pediatrician who from 2006 to 2012 led the MSF Access Campaign, said he was “a bit speechless” that MSF would sign nondisclosure agreements (NDAs) because its approach in the past was to “never sign such agreements when it came to supply and cost.” MSF has made transparency a key demand in its campaigning on access to medicines, often disclosing the price it pays for some drugs, including insulin pens, as well as the costs of its clinical trials. But while MSF’s actions have drawn surprise, others can understand the pressure the NGO is under when negotiating prices with big players in the sector, arguing it’s a sign of the leverage pharma companies hold in such talks. MSF, signed a confidentiality clause with German pharma company Bayer in a contract to buy contraceptives for distribution in lower-income countries. | Najeeb Almahboobi/EPA Ellen ‘t Hoen, another former head of the Access Campaign, argued the blame should be on the drug companies. “This is a symptom of the hostage-like situation single source suppliers — e.g. drug companies that hold patents and control the market — create with their pricing policies. It would be a difficult position to take for MSF to withhold medicines from the patients they care for. In other words, the resistance has its medical ethical limits.” Drug companies often prefer to keep the price of medicines secret to prevent other countries from demanding the lowest available price. The European Federation of Pharmaceutical Industries and Associations has advocated for confidential tiered pricing that it says would ensure lower-income countries would pay less than higher income ones. Transparency campaigners say NDAs allow pharma companies to inflate prices and assert extra conditions on buyers. “MSF is firmly opposed to pricing secrecy, as we believe transparency is essential to improve access to affordable medicines,” Maria Guevara, international medical secretary at MSF International, told POLITICO. “However, sometimes companies force us into a position where we must sign NDAs or confidentiality clauses to be able to obtain critical medicines and tools to treat our patients.” Guevara said that MSF “systematically resists” demands from suppliers, “though unfortunately we do not always win.” “It is the option of last resort; we try our best efforts not to have to sign one,” Guevara added. In 2023, the Access Campaign announced the NGO had refused to sign a contract to buy HIV medication from ViiV because of the pharma company’s “last-minute” demand for terms that are “not acceptable in MSF purchase agreements,” including an NDA. “We refused to sign the [ViiV] agreement with these terms, as it would undermine drug pricing transparency, limit civil society activism for lower drug prices, and restrict supply to [low- and middle-income countries],” Guevara told POLITICO. ViiV declined to comment on the story when contacted. The company has disclosed its non-profit price for the drug — currently £20.70 per vial excluding distribution costs — to buyers, but this price doesn’t apply to middle-income countries. MSF has called for ViiV to publish all of its prices and extend the access terms to all countries. Access Campaign staff have urged MSF management to adopt a policy on NDAs. An email sent by members of the campaign to management last year noted MSF did “not currently have an agreed approach internally to guide the strategic decision making and practices concerning NDA.” When asked what MSF’s current policy was, Guevara said: “We focus our resistance [to drug price secrecy clauses]on products where we know access is a major issue.” This story has been updated with ViiV’s position.

Imagine a Europe at the cutting-edge of research, development and manufacturing. Where patients have access to the latest vaccines and treatments, first. A Europe with confidence in its resilience, health and economic security. The General Pharmaceutical Legislation (GPL) is one important step in realizing that vision. Get it right and we can begin to rebuild Europe’s life science eco-system. Get it wrong, and we risk accelerating the loss of research, development and manufacturing to other regions of the world.  It’s hard to miss the irony that almost nine months from Mario Draghi urging Europe to catch up with other regions by implementing a coordinated industrial policy or face a ‘slow agony’, our industry is nervously anticipating decisions that could, at best, maintain the same incentives framework that has seen our share of global investment decline over the last 20 years. The GPL offers some hope to up Europe’s game with a future-proof regulatory framework and new tools to tackle antimicrobial resistance. This does not sound like the recipe for European success … yet. Fierce competition from the US and China Whatever the geopolitical situation today, Draghi’s conclusions echoed numerous warnings highlighting Europe’s decades-long decline in life sciences. Since the legislation was last addressed in 2004, Europe has lost a quarter of its share of global R&D investment. And its share of global trials has halved, resulting in 60,000 fewer clinical trial places available for Europeans. Concurrently, spending on pharmaceutical R&D in China has grown almost five times faster than in Europe. China has doubled its number of commercial clinical trials since 2018; it now accounts for 18 percent of the global share. Their recent adoption of a proposal to introduce Regulatory Data Protection (RDP) signals China’s efforts to attract investment in R&D for innovative medicines. A step likely to be even more effective if Europe were to move in the opposite direction. Just 30 years ago, one in two new medicines originated in Europe, now it is just one in five. This is hardly a surprise given that the US went from spending €2bn more than Europe to spending €25bn more today over the same period. > Just 30 years ago, one in two new medicines originated in Europe, now it is > just one in five. Proposals to shape the future Since the framework was first introduced over 20 years ago, science has taken giant leaps forward. We are in a golden era of innovation, with much more to come. Immunotherapies empower the body to attack and destroy cancer cells, HIV can be managed, Hep C can be cured, and there is a good chance that cervical cancer could be eradicated thanks to the HPV vaccination. From prevention to care to cure, medicine is evolving rapidly. We strongly welcome, support and need the proposals to future proof the regulatory approval system. The regulatory sandbox is a big step in helping bring future breakthrough innovations of today and tomorrow to EU patients. Meanwhile, the proposal to use electronic-only patient information for medicinal products directly administered by healthcare professionals will reduce the administrative burden. > We strongly welcome, support and need the proposals to future proof the > regulatory approval system. Addressing another health emergency Similarly, since the legal framework was last addressed there has been widespread recognition that antimicrobial resistance represents one of the world’s most pressing health emergencies. Yet despite the urgent need, private investment in antimicrobial R&D is limited. To prevent bacteria developing resistance to existing treatments, the aim with any new antibiotic is to use it as little as possible — not an attractive proposition for investors. Included as a proposal in the GPL, the transferable exclusivity voucher is designed to address this challenge by rewarding the discovery of novel antibiotics without requiring upfront public investment. The GPL provides an opportunity for Europe to take the lead in delivering new antibiotics. Not only would this bring considerable cost-savings to every member state, it would also significantly boost R&D efforts in antibiotics, putting European researchers at its center. Proposals that accelerate negative trends In the context of recent shifts in US policy, trade tensions, concerns over national security, supply chain resilience, increasing levels of protectionism and the drive to address Europe’s competitiveness crisis, the initial proposals to erode European intellectual property (IP) in life sciences are now completely out of step. It takes two to launch a new medicine Many of the decisions that impact when patients get access to a new medicine are outside a company’s control, in fact, two-thirds of the delays to access medicines occur after companies have filed for pricing and reimbursement. For example, in Greece, companies can only file for reimbursement once a medicine is reimbursed in at least five of 11 designated EU countries, delaying filing due to the external reference pricing system. This structural condition adds to other delays, including those linked to limited healthcare funding and launch decisions. As a consequence, measures like obligating companies to launch in all 27 member states will completely fail to address inequalities in access to medicines while proving to be highly damaging to the sector’s presence in the EU. The proposal to reduce the Regulatory Data Protection (RDP) and Orphan Market Exclusivity would have a similar impact; the US already leads Europe on every investor metric from availability of capital to speed of approval and rewards for innovation. RDP for small molecules is the only appreciable benefit that the EU has over the US. The proposed expansion of the Bolar exemption — a legal provision that permits the narrow use of a patented medicine to support a generic’s marketing authorization application before IP expiration — would further erode Europe’s IP framework. It would likely lead to more litigation, reduce legal certainty and predictability, and negatively impact patients, including potentially introducing co-payment. Instead, the EU should create a clear notification system to give both generic and innovative companies transparent, reliable ‘day one’ certainty and safeguard IP protection. “If Europe truly wants to have research, development and manufacturing in the region, as well as delivering the best care for its citizens, it has to align the outcomes of discussions on the GPL with its ambition to be a world leader in medical innovation, maintain resilient supply chains and compete economically.” Nathalie Moll, director general, EFPIA. High stakes, high risk The stakes are high, and the risks tangible and immediate — but so are the opportunities. In recent weeks, global pharmaceutical companies, some of them headquartered in Europe, have announced hundreds of billions of dollars in investment into US manufacturing. This is likely just the beginning. > The stakes are high, and the risks tangible and immediate — but so are the > opportunities. A survey of 18 EFPIA members in April this year identified as much as 85 percent of capital expenditure investments (around €50.6 billion) and as much as 50 percent of R&D expenditure (around €52.6 billion) as being potentially at risk. Over the next three months alone, surveyed companies estimated that a total of €16.5 billion, or 10 percent of the total investment plans, are at risk. From intent to action There have been important positive statements of intent, reflected in the announcement of the Biotech Act, Life Sciences Strategy and Competitiveness Compass. Turning this intent into rapid and radical policy change will help, and the first tangible and visible signal will be the operating environment for companies in Europe created by the GPL. Investors and innovators are watching closely to see which side the GPL lands on.

America’s most celebrated global health program is on life support, former U.S. government officials and global health advocates say. President Donald Trump’s decision to suddenly halt and then terminate most U.S. foreign aid, and GOP concerns that organizations receiving government grants to combat HIV and AIDS were performing abortions, have key congressional Republicans broaching what was once unthinkable: ending PEPFAR, the program President George W. Bush created to combat HIV and AIDS in the developing world. Bush has long championed it and the 25 million lives it’s saved as the best example of his “compassionate conservatism.” But Trump has lumped the President’s Emergency Plan for AIDS Relief in with other foreign aid programs he sees as indicative of the way Washington has put the needs of foreigners over Americans and the seismic shift in GOP attitudes since Trump took over the party. The Bush Institute, an arm of the center that promotes Bush’s legacy, is pleading with the administration and Republicans to keep the program alive, making the case that it’s good for America. “PEPFAR is a strategic investment in our own national security,” Hannah Johnson, a senior program manager for global policy at the institute, wrote earlier this month, arguing that “it engenders goodwill toward the United States at a time when Russia and China are competing for greater influence, in ways that are not beneficial in the long-term for the African continent.” She called on the administration to continue the program — “whether through USAID, the CDC, the Pentagon, or the State Department. It is a matter of life and death.” Since late February, the Trump administration has terminated hundreds of millions of dollars in PEPFAR grants and contracts amid its rapid effort to align foreign aid with its “America First” policy, according to a list obtained by POLITICO. Next week, the 2003 law that established PEPFAR is set to expire with no indication it’ll be renewed anytime soon. Congress did appropriate funding to cover PEPFAR’s expenses through September earlier this month. Its programs can continue even if the law authorizing it expires, but only if Trump wants to spend the money. PEPFAR’s budget is between $6 and $7 billion per year. Trump has halted most programs overseen by the U.S. Agency for International Development, which handled a majority of PEPFAR’s projects, but so far hasn’t touched the Centers for Disease Control and Prevention’s, which run nearly $2 billion a year. The cuts the administration has made have alarmed public health advocates. A sudden end to PEPFAR could kill six million people in the next four years, reverse decades of progress and lead to growing HIV epidemics across the world, over 500 AIDS physicians and researchers warned in a letter to Secretary of State Marco Rubio. “Over time, these policy decisions may be proven illegal in U.S. courts but the human suffering and loss of lives happening now cannot be reversed by any court order,” they wrote, asking Rubio to restart all PEPFAR projects. The State Department did not respond to a request for comment on the letter. ABORTION POLITICS PEPFAR enjoyed bipartisan support until two years ago, when congressional Republicans accused then-President Joe Biden of indirectly funding abortion abroad by providing PEPFAR funds to groups that support or provide abortions. After allowing the law that authorizes the program to expire in 2023, Congress ended up reupping it for one year last March. Every previous renewal was for five. Then in January, the Biden administration acknowledged that a routine check on grant compliance in the southeast African country of Mozambique found that four nurses in a small province whose salaries were funded by PEPFAR provided abortions, which is legal in the country. Mozambique refunded the money — $4,100 — but Senate Foreign Relations Chair Jim Risch (R-Idaho), whose panel oversees PEPFAR and would lead any effort to renew it, said it called into doubt his support for the program. “This violation means that the future of the PEPFAR program is certainly in jeopardy,” he said in a statement at the time. Advocates of PEPFAR have in the past turned to Rep. Michael McCaul, an 11-term Texan representing a swath of suburbia from Austin to Houston and friend of Bush’s who’d helped convince fellow Republicans in 2024 to reup PEPFAR for a year despite their misgivings. But in January, GOP term limits for committee chairs forced McCaul to give up his post atop the House Foreign Affairs Committee. McCaul’s replacement, Florida Republican Brian Mast, told POLITICO earlier this year that he wants to rethink the U.S. investment in PEPFAR. “If Americans are spending billions of dollars for multiple decades funding extremely expensive HIV medication for 20 million Africans, there should be a conversation about that,” he said. “At what point do some or all countries start to handle that on their own?” The bottom dropped out for PEPFAR shortly after Trump’s inauguration in January when, as one of his first actions, he closed the agency that sponsors most foreign aid, USAID, and then terminated billions in State Department funding. The cuts included grants and contracts supporting HIV prevention for teenage girls and gay men, who are at high risk of acquiring HIV in some countries in Africa; efforts to control the spread of HIV in Nepal, Uganda and Ukraine, and clinical trials researching a vaccine and other HIV prevention measures. A State Department spokesperson said a list of the programs obtained by POLITICO “is inaccurate and unverified” but didn’t provide more details. Payments for some of the PEPFAR projects still intact, such as a major contract to supply and deliver HIV drugs, aren’t flowing to the organizations running them, keeping crucial lifelines effectively frozen, according to a person familiar with the USAID programs allowed to speak anonymously for fear of reprisal from the administration. Eight countries already face significant disruptions to HIV drugs and are expected to run out in the coming months, the World Health Organization, an arm of the United Nations, said Monday, listing Kenya, Lesotho, and Ukraine among them. The Trump administration has argued in court that it needed to verify most payments manually to ensure there’s no fraud involved but a federal judge ordered it to pay a large batch of backlogged invoices for foreign aid programs. In many cases, those payments are still pending: There are about 10,000 payments that need to be processed, the State Department said in a court document on March 19. The administration has kept a few hundred USAID employees out of more than 10,000. Trump is folding what’s left of the agency into the State Department. Just over a dozen PEPFAR specialists from USAID’s global health bureau will be hired at the State Department office managing the program, according to a State Department memo obtained by POLITICO. Mast told POLITICO that he’s considering reupping the law undergirding PEPFAR in September when the State Department will also come up for reauthorization. Mast suggested he’ll prioritize shifting responsibility for HIV and AIDS prevention and care to the countries that have relied on PEPFAR: “There’s countries — and their leaders — that have just taken it for granted that the United States is just going to pay for their HIV medication forever.” Some of those countries have worked with China on mineral extraction and other things, Mast said, suggesting America’s PEPFAR investment didn’t serve the U.S. as many tout it. He said those countries could borrow money to provide HIV-prevention services that were funded by PEPFAR to their citizens. The Trump administration’s shock-and-awe approach in freezing and then cutting most of foreign aid, and, with it, many PEPFAR programs, has left global health advocates and some on Capitol Hill wondering what’s left of the program that until recently was fiercely supported by most Republicans and Democrats. “It’s hard to understand how PEPFAR, as we know it, can continue at this moment,” said a House Democratic aide granted anonymity to speak candidly. TRUMP’S PLANS PEPFAR is an ecosystem of services that goes beyond providing medication and includes testing and reaching out to vulnerable groups, such as teenage girls, the aide said. The program “is not going to be as successful if we chip away at pieces along the way and strip it down to something that is just, perhaps a straight provision of medication,” the aide added. Pete Marocco, the foreign assistance director at the State Department who has led the foreign aid cuts and USAID’s dismantling, told lawmakers from the House and Senate foreign affairs committees in meetings earlier this month that around $4 billion from PEPFAR’s annual funding wasn’t spent on lifesaving treatment and went to advocacy instead, according to two people with knowledge of the conversation speaking anonymously because they aren’t allowed to publicly comment on private meetings. Marrocco also said the program only needed about $2 billion to provide lifesaving treatment, according to the two people. Marocco didn’t provide a list of terminated or retained programs to lawmakers, Rep. Sydney Kamlager-Dove (D-Calif.) and Sen. Jeanne Shaheen (D-N.H.) said after meeting him. The State Department said it doesn’t comment on its officials’ communications and briefings with Congress. While uncertainty about what’s been eliminated and what remains persists, the cuts will damage the foreign aid system, including the programs that the administration may want to keep, said Andrew Natsios, a Republican who ran USAID in the Bush administration. Dr. Atul Gawande, who ran USAID’s global health programs in the Biden administration, said the funding freeze and terminations are putting the whole program at risk. “This is the end of PEPFAR as we know it, and if certain issues aren’t addressed, it’s just the plain end of PEPFAR,” he told reporters in a call in late February. Some global health advocates and lawmakers are holding out hope Gawande is wrong. “PEPFAR, unlike the health programs that are based at USAID, is based at the State Department; does still have a team there overseeing the program; was given, at least on paper, the ability to continue some care and treatment,” said Jen Kates, senior vice president and director of the Global Health & HIV Policy Program at KFF, a health policy think-tank. Sen. Chris Coons (D-Del.), a member of the Senate Foreign Relations Committee, acknowledged that it will be difficult for the program to recover from the blows it has suffered over the past few weeks. “But I’m determined that it’s not the end for PEPFAR. It is too important, too valuable, too effective a program for us to give up on,” he said.

When United States President Donald Trump announced he would tariff Denmark if the country didn’t relinquish control of Greenland, Danish Prime Minister Mette Frederiksen turned to a coterie of CEOs for advice. But rather than the fist-pumping tech bros or mixed martial arts fighters that made up Trump’s circle at his inauguration, Frederiksen went instead to a rather more reserved character: a softly spoken 58-year old, Lars Fruergaard Jørgensen. In person the Dane is quiet, polite and understated. But he inspires enough fear in fast food giants that they call him up in a panic about the threat he poses to their businesses. He’s the CEO of Danish pharmaceutical firm Novo Nordisk — best known as the maker of Ozempic and Wegovy, the blockbuster diabetes and weight-loss drugs-turned-cultural phenomena that have single-handedly prevented Denmark from falling into a recession. But how did the self-confessed introvert who “needs his quiet time” become the man to advise Denmark on how to handle the American president? THE AMBIVALENT BOFFIN In the helix-shaped headquarters half an hour from central Copenhagen, Jørgensen was at pains to explain to POLITICO his rise to the top of Novo Nordisk — until recently, Europe’s most valuable firm — was never his intention. As a young graduate he applied to four companies — and accepted the first offer he got. Joining Novo Nordisk as an economist in 1991, Jørgensen said he had “no ambition, no clue.”  From the company’s offices overlooking the city, Jørgensen spoke in December about his anxiety from that time.“In the early years of my career I did not talk a lot about that I grew up on a farm, that I’m the first one to go to high school and business school … because I felt I was less good,” he said. “Others came from academic families and I felt they were better.” He had no great desire to one day run the company — “I think nobody saw that early on” — but Novo put all the levers in place for a young Jørgensen to learn and succeed. He said he just made the most of them. “[They] kind of threw me into stuff I had no clue about. You learn the art of quickly trying to assess: ‘OK, what is actually the problem? What’s going on? What am I supposed to do?’” It took him a while to feel fully comfortable; being more open about his farming roots was a big part of it, he said. “I think that has made me more authentic and also it resonates with many other people who have the same upbringing.” Jørgensen didn’t fully shed that insecurity until he was made CEO, he said, a role which has thrust him blinking into the spotlight — and now sees him having to reassure a panicked prime minister. Frederiksen summoned Danish business leaders, including Jørgensen, for crisis talks in January after Trump refused to rule out military or economic action to take control of Greenland, a semi-autonomous territory of Denmark. Danish Prime Minister Mette Frederiksen turned to a coterie of CEOs for advice. | Johannes Simon/Getty Images The United States president has also suggested he would impose tariffs on Danish goods if Copenhagen turns down his offer to buy the Arctic island — no empty threat given he’s already following through with tariffs on goods from Canada, China and Mexico. NOVO’S RISE It’s difficult to overstate the recent success of Novo Nordisk, which boasted a turnover of €38.9 billion in 2024 — of which €13.5 billion was profit — and accounts for nearly half of Denmark’s gross domestic product growth. The Scandinavian company has long been a leader in diabetes medicine but hit the jackpot with its GLP-1 class of drug for diabetes that also caused weight loss. Although Ozempic has become synonymous with a shrinking waistband, it’s Novo’s drug Wegovy, which is licensed for weight loss and clinical trials showed patients lost on average 15 to 16 percent of their body weight after just over a year. Both drugs contain semaglutide, which mimics a hormone released after eating, tricking the brain into feeling full. Wegovy’s approval — in 2021 in the U.S. and 2022 in Europe — was a game changer for the company.  Celebrities including Oprah Winfrey and Trump’s “efficiency czar” Elon Musk have boosted its profile (the latter describing himself as an “Ozempic Santa”), while Senator Bernie Sanders — who hauled Jørgensen in front of his U.S. Senate health committee last year — said semaglutide “may end up being one of the bestselling pharmaceutical products in the history of humanity.” One in eight people have taken Ozempic, Wegovy or one of its competitors in the U.S. Scientists are already questioning whether their rapid adoption is causing obesity rates to fall in America. That stratospheric rise in profile has come during Jørgensen’s reign as CEO, a position he has held since 2017, when he became only the fifth person to lead the company in its 100-year history. It also makes Novo a rare European success story at a time when businesses and investors pivot to the U.S. and China. Despite that success, Jørgensen doesn’t have a reputation as a ball-breaker, nor a political animal in his home country. “He’s seen more as a civil servant than a CEO,” one Danish diplomat, granted anonymity like others in this article to speak candidly, said.  And he remained diplomatic when POLITICO asked him at the end of last year about Trump’s controversial picks for top jobs, such as vaccine-skeptic Robert F. Kennedy Jr. for health secretary. “Let’s just see how they play out,” he said. That kind of response is typical of Denmark’s approach to diplomacy, said Lars Sandahl Sørensen, head of the Confederation of Danish Industry and former deputy CEO of Scandinavian Airlines, who has known Jørgensen for years. It’s difficult to overstate the recent success of Novo Nordisk, which boasted a turnover of €38.9 billion in 2024. | Claus Rasmussen and Ritzau Scanpix/Getty Images “Standing up and shouting contests is not our style,” Sørensen told POLITICO over the phone last year. Nor is it to be “very flamboyant or show off, or show strength,” he said. “I understand that’s part of other cultures and that works there, but here it would work negatively.” Jørgensen is also unfazed by the success of Mounjaro, a drug produced by Novo’s U.S. rival Eli Lilly, which has demonstrated greater weight loss compared with Wegovy. “I welcome competition,” he said. “We have a 100-year history of competing with Eli Lilly and we tend to take turns about who has a product that’s ahead.” CRISIS MODE Trump’s threat of tariffs against Denmark is perhaps the biggest headache the CEO has had to deal with in his time as head of the organization. Last year, 58 percent of Novo’s sales came from the U.S. — 79 percent of Wegovy and 70 percent Ozempic sales. Asked about tariffs at a press conference in February, Jørgensen said Novo was “not immune, but we are confident our business is in a good position to meet the demands of the new [U.S.] administration.” The other major crisis was the Covid-19 pandemic; though not a vaccine-maker the company still had to navigate disrupted supply chains. His instinct was to let other people take over. “If I have to solve the problems, that’s a problem,” Jørgensen told Bloomberg last year. The company set up a crisis response team but Jørgensen didn’t initially attend and let others lead it. “I knew if I put myself into that I’d become a bottleneck,” he said. His attitude to leadership is one of consensus. “I was born with a big nose and big ears and I use the attributes of that each day to collect opinions from the company. Then it’s my role to combine it into an opinion together with my team,” he said. That goes as far as taking advice on running the company from his two environmentally conscious kids, who encouraged him to reevaluate Novo’s policy on flights. But that approach means that when the spotlight is on him, he’s out of his comfort zone. His reticence hasn’t always gone down well with policymakers.Senator Sanders said his committee “reached out time and time again” to schedule Novo Nordisk’s voluntary appearance at a hearing, without success. It wasn’t until he threatened the Dane with a subpoena that he turned up in person. Donald Trump’s threat of tariffs against Denmark is perhaps the biggest headache the CEO has had to deal with in his time as head of the organization. | Tierney L. Cross/Getty Images When he did, the Senate report into Novo’s pricing accused the company of “greed, greed, greed.” Jørgensen nonetheless was accommodating, promising to sit down with pharmacy benefit managers — middlemen who negotiate medicine rebates with manufacturers on behalf of insurers — to “collaborate on anything that helps patients get access and affordability.” SOFT POWER Jørgensen is not someone who schmoozes in Brussels either, preferring to spend his time in Denmark, ideally in the garden or on the tennis court, he said. Despite being current president of Europe’s pharma lobby EFPIA, he goes to the European Union capital only two or three times a year in that capacity, his press secretary told POLITICO. Nonetheless, Novo’s presence looms large over discussions on a major overhaul of European laws for the pharmaceutical industry — because of the ferocious loyalty Denmark and its lawmakers show to Novo in Brussels.  The country is the most vocal supporter of the pharma industry in the EU, where countries are currently negotiating their position on the legislative reform. One European Parliament official said that Novo is practically “in the room” during these talks, such is the extent Denmark mirrors the pharma position. Assistants for Danish members of the European Parliament had tried to insert amendments into the Parliament’s text to make it more favorable to the industry, the official said, adding that the company’s lobbyists would be “welcoming the new MEPs off the train in Strasbourg,” the seat of the Parliament. Nevertheless, those who have met Jørgensen in such a capacity say he is thoughtful and respectful. “Polite and quiet, like he had a genuine interest in what his staff and I were saying rather than in hearing himself talk,” said another Parliament official. “He appeared very knowledgable.” That’s a very Danish approach to leadership, pointed out Sørensen. “We like to be fact-based. We like to be honest. We like to be trustworthy,” he said. It’s a style that will surely go down well with the U.S. president, who calls himself “perhaps the most honest human being” God ever created. Right? This article has been updated to clarify the effect of GLP-1s on weight.

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.

LONDON — The British government indefinitely banned the use of puberty blockers by people under 18 years of age Wednesday after expert advice flagged an “unacceptable safety risk” in their use. Puberty blockers stopped being routinely prescribed to under-18s with gender dysphoria in the U.K. in March and were temporarily banned in May this year by the previous Conservative government. This prevented their prescription by European or private prescribers. National Health Service provision was restricted to clinical trials. The Department for Health and Social Care has now received advice from the government-ordered Commission on Human Medicines and on Wednesday backed its recommendation to restrict their use and supply indefinitely while further work is done to ensure young people’s safety. An earlier U.K. review into gender identity services found that studies about treatment for gender dysphoria were unreliable and there was a lack of attention paid to patients seeking to halt or reverse the gender transition process. New legislation will make the ban indefinite and be reviewed in 2027. Announcing the move, Health and Social Care Secretary Wes Streeting said: “We need to act with caution and care when it comes to this vulnerable group of young people, and follow the expert advice.” Streeting pledged to open more gender identity services in the U.K. The policy will apply across the U.K. and the government will set up a trial into the clinical use of puberty blockers next year.

Sometimes statistics don’t tell the full story. This is particularly true if you look at EFPIA’s recently published European Economic Impact report, which looks at the contribution of the pharmaceutical industry to Europe’s economy, as well as those of other regions. The report finds “a strong and growing sector”. It shows that research and development (R&D) expenditure in Europe has grown on average by 4.4 percent each year between 2010 and 2022, from €27.8 billion to €46.2 billion. Good news? Yes and no. If we look at the wider picture, Europe’s position is clearly becoming more and more precarious against a backdrop of falling competitiveness. While global research into medicines and vaccines is growing, Europe’s share of the pie is getting smaller as the distribution of R&D shifts elsewhere. > While global research into medicines and vaccines is growing, Europe’s share > of the pie is getting smaller as the distribution of R&D shifts elsewhere. R&D spending in Europe is consistently outpaced by the US and increasing competition from China. In the US, R&D investment grew by 5.4 percent between 2010 and 2022 (from €30.7 billion to €71.5 billion) and five times greater (20.7 percent) in China (from €1.4 billion to €14.8 billion). If we look at Europe first: In 2022, global R&D spending in the sector was €143.6 billion; Europe accounted for 32.2 percent of this. As a sector, we contribute more to the EU trade balance than any other. Added to this, 16 of the world’s top 50 life sciences universities are based here. * In 2022, the industry contributed €311 billion to the EU — 2.0 percentof its gross value-added (GVA). * The industry supports 2.3 million jobs in the EU — a 2.1 percent increase year on year between 2016 to 2022. * Driven by consistently high levels of research and innovation — and a GVA of €197,000 per worker — the pharmaceutical industry in Europe is three times as productive as the European economy as a whole. However, the report also found that in the US, for example, GVA per hour per worker is double that of the EU. China’s growth is one of a number of trends that is now shown to correlate with a relative decline in the number of new molecule entities (NME) discovered in Europe. The EU fell behind China for NME discovered in 2023. NMEs are drugs with an active ingredient, marketed for the first time, and vital to R&D activity. The report suggests that China’s life sciences boom is due to its dedication to improving its regulatory environment, increased funding streams and strategic investment in advanced technologies. This is clearly paying off, and while it might send shockwaves through Europe, lessons can be learned. As other regions ramp up their investments and streamline regulatory frameworks, Europe cannot afford to become a secondary or even tertiary player in global health innovation. > As other regions ramp up their investments and streamline regulatory > frameworks, Europe cannot afford to become a secondary or even tertiary player > in global health innovation. Europe’s fragmented regulatory environment exacerbates the gap, reducing the EU’s competitiveness as a location for clinical trials. Recent research showed that 60,000 fewer patients living in Europe had access to clinical trials since 2018, missing out on new research in immunisation, cancer, rare diseases and paediatrics. Europe’s global share of commercial trials — those sponsored and funded by a pharmaceutical company — is half of what it was a decade ago. Meanwhile, commercial trials in China rocketed, making up 18 percent of those taking place globally. For the most innovative therapeutics, advanced cell and gene therapies, China holds a 42 percent share, the biggest in the world. The impact of Europe’s decline reverberates beyond patient care. Skills and expertise are migrating — nearly three-quarters of European science graduates choose to remain in the US after completing their PhDs. Over time we stand to lose our ability to recruit and retain the next generation of scientists. > In Europe we have fantastic ideas, the skills, world-class academic > institutions and pockets of brilliance, but our processes are slow, and our > eco-system is very fragmented. In Europe we have fantastic ideas, the skills, world-class academic institutions and pockets of brilliance, but our processes are slow, and our eco-system is very fragmented. The scale and pace at which we are losing global share of research shows there is only a finite amount of time to turn things around. What needs to happen? Pharmaceutical companies support local economies, boost regional development, create highly skilled jobs, and bring funding to hospitals and research centres. In fact, the data shows that productivity per worker in the pharmaceutical sector is higher than in the economy as a whole. The recognition of the importance of the sector by EU leaders is a positive step. However, overcoming Europe’s widening competitiveness gap needs immediate and robust action now. Mario Draghi’s much anticipated report on how to boost Europe’s competitiveness makes for hopeful reading. The revision of the Pharmaceutical Legislation offers the opportunity to update a 20-year-old regulatory framework to create a unified and streamlined regulatory environment to foster innovation and competitiveness in Europe, on a par with the Food and Drug Administration, among other regulatory bodies. The industry has also outlined a detailed roadmap for policymakers through a Strategy for European Life Sciences, aligning closely with recommendations from the Draghi and Letta reports, which emphasize the urgent need to power up Europe’s innovation capacity. Together with insights from the EFPIA’s Clinical Trials Ecosystem Report, these recommendations form a comprehensive approach to addressing regulatory, investment and operational challenges, setting the foundation for Europe to secure its position as a global leader in health innovation. Europe has the potential. And now the ambition. It is time to turn things around.

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