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

It could have been the ominous cold open to a classic Bond film. The Russian and Chinese leaders caught on a hot mic at a Beijing military parade, casually musing about cheating death. “With the development of biotechnology, human organs can be continuously transplanted and people can live younger and younger, and even achieve immortality,” Russian leader Vladimir Putin told Chinese ruler Xi Jinping, his tone half clinical, half conspiratorial. “Predictions are that this century, there is a chance of living to 150,” Xi replied. But this wasn’t a scriptwriter’s villainous fantasy. It was a jaw-droppingly real exchange between two of the world’s most powerful, heavily armed leaders. While it may have sounded absurd, behind palace walls, the obsession with longevity is more than idle chatter. The Russian and Chinese leaders were caught on a hot mic at a Beijing military parade, casually musing about cheating death. | Pool photo by Alexander Kazakov/AFP via Getty Images In 2024, the Kremlin ordered scientists to fast-track anti-aging research on cellular degeneration, cognitive decline and the immune system. Meanwhile, China has also been pouring resources into exploring nanotechnology-delivered hydrogen therapy and compounds such as betaine and lithocholic acid, hoping to slow down aging and extend healthy lifespans. But even as the world’s autocrats fantasize about replacing body parts like car tires, the science remains far less accommodating. James Markmann, executive council president at the American Society of Transplant Surgeons, called Xi and Putin’s idea of living to 150 through transplants “unfounded.” “There is currently no evidence suggesting that living to 150 years of age is possible through organ transplantation,” Markmann said. “While there is much interest in related research and some progress in intervening in the aging process, there is no evidence that a 150-year lifespan can currently be achieved.” While organ transplantation can and does save lives, there’s no data that it can also slow or reset an individual’s biological clock, Markmann said. Replacing a single organ, or even several, may improve health temporarily, but it cannot halt the overall aging process of the body. “The concerning idea here is that there is a surplus of organs available that can consistently be replenished for a single individual to prolong their life; this is simply not the case,” Markmann said. THE OLDEST OBSESSION The Xi-Putin exchange didn’t happen in a vacuum. History is littered with rulers who believed they could outsmart death. Qin Shi Huang, China’s first emperor, swallowed mercury pills in pursuit of eternal life, a habit that eventually killed him. Egyptian pharaohs mummified themselves for eternity, Cleopatra dabbled in youth potions and medieval alchemists peddled elixirs. By the 20th century, Russia’s last czar, Nicholas II, and Empress Alexandra were consulting Rasputin and other mystics for advice on health and longevity. Today, the same quest has migrated to Silicon Valley, where the mega-rich pour fortunes into cryonics, anti-aging biotech and “biohacking” in the hope of buying more time. According to Elizabeth Wishnick, an expert on Sino-Russian relations and senior research scientist at the Center for Naval Analyses (CNA), a non-partisan research and analysis organization, this fixation is typical of the world’s wealthiest and most powerful. “They want to go into outer space, they want to go underwater … the human body for them is just another frontier,” she told POLITICO. “It’s logical for people who don’t feel limits to try to extend those boundaries.” But there’s a stark contrast close to home. Life expectancy in Russia remains just over 73 years, while in China, it hovers around 79 years, with access to healthcare being deeply unequal. In Wishnick’s view, Xi and Putin “would do better to focus on that, but instead their focus seems to be on their own longevity, not the health of their societies.” UNFINISHED BUSINESS There’s also a significant cultural dimension agitating Xi and Putin. Robert Jay Lifton, the American scholar who coined the term “symbolic immortality,” argued that humans invent religions, nations and political legacies as ways of cheating death. Xi’s mantra of “national rejuvenation” and Putin’s mission to restore a “great Russia” fit neatly into that framework — even if they can’t physically live forever. “Both of them are really hostage to their own propaganda,” said Wishnick. “They truly believe they are the only leaders who can do the job. They’re concerned about their legacy and how they’ll be remembered in history.” That, she said, helps explain their obsession with reclaiming “lost” territories — Taiwan for Beijing; Ukraine for Moscow — as if completing unfinished maps might also complete their historical destinies. Qin Shi Huang’s attempt at immortality, the Terracotta Army, still stands today. | Forrest Anderson/Getty Images They’ve made creeping moves toward that goal domestically. Xi has upended China’s tradition of leadership turnover to maintain his dominance, while Putin has dismantled elections and eliminated rivals until only he remains. “It’s not surprising they would look to science as a way of extending that,” Wishnick added. While the scientific limitations persist, immortality will — at least for the time being — remain tied to public consciousness and memory. See, for example, Qin Shi Huang’s Terracotta Army, which still stands, or Russia’s expansionist czar, Peter the Great, an 18th-century leader who inspires Putin even today. But even in a world of nanotech and organ swaps, immortality has a catch: you still have to live with yourself. And for the world’s Bond villains, that might be the cruelest sentence of all.