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

This article is the product of a POLITICO Working Group presented by Sanofi. BRUSSELS — The EU wants to arrange purchases of the newest medicines for countries that currently don’t have access to them — but some in the pharmaceutical sector are not happy about it. The European Commission’s plan to use joint procurement to provide access to innovative drugs across the bloc risks overuse of this process and could drown out competition, according to David Elvira, global corporate public policy head at Sanofi. Draft legislation drawn up to fix Europe’s chronic drug shortages contains plans to expand the circumstances under which joint procurement could be used. That includes critical medicines with vulnerable supply chains, such as those with single-country dependencies, as well as novel therapies unavailable in three or more EU countries. The latter are known as medicines of common interest in the draft Critical Medicines Act. While joint procurement of widely used, critical, and often very cheap generic drugs was expected to be included in the legislation, the use of it for new, branded, and often expensive treatments — such as cell and gene therapies and cancer drugs — came as a surprise to many in the sector. The concern is that “we opened the window of the risk with this medical product of common interest,” said Elvira. “Because if the definition (of a medical product of common interest) is blurry, non-detailed, and we don’t know exactly what we want to solve with this, then we will start to have some overuse of the panacea of the joint procurement as the solution,” he said during a closed-door working group on the topic organized by POLITICO. One politician also warned against overusing the tool, noting that many root causes need to be addressed to prevent drug shortages. “Joint procurement is not a solution for all the (drug shortage) problems that we are facing,” Socialists and Democrats MEP Nicolás González Casares said. It can be useful to “give a boost for the market in order to obtain vaccines,” or to tackle shortages in some cases. But it “cannot solve all the problems,” he added. Joint procurement as an EU tool for improving access to medicines only really “found its destiny” during the Covid-19 pandemic, said Olivier Girard, head of medical countermeasures at the European Commission’s Health Emergency Preparedness and Response Authority (HERA). During that time, the bloc purchased lifesaving vaccines for European countries. Since then, it has become a more common tool in the EU’s public health arsenal, helping countries procure vaccines for bird flu, mpox and pandemic influenza. IMPROVING ACCESS IN SMALLER COUNTRIES For countries, particularly smaller ones with limited market size and weaker buying power, joint procurement could help to improve access to critical and innovative medicines. “Joint procurement for medicinal products are particularly in our interest as a small country,” said an official from an EU country with a small market size, granted anonymity to speak candidly. “And joint procurement is not just a part of the solution for shortages, but [also] access to innovation — equal access to innovation to all member states.” Thanks to the EU-wide rollout of Covid-19 vaccines, citizens and the capitals also know the EU can deliver in this area. “There is a strong case to support the development of the joint procurement,” Girard at HERA said. “We have built trust and we have demonstrated that we could deliver, so let us think how the model could evolve and be developed — only where there is added value for the member states.” But the pharmaceutical industry argues that any changes should be made cautiously and with clear guidelines for everyone involved. The sector wants safeguards to support companies’ participation and to prevent market monopolies, which it argues could develop if a drugmaker wins a contract for a large volume of medicines across the bloc. “If there is no potential competition in the way we are implementing these joint procurements for the future, and you are excluding players in the future, that will not be sustainable and that will create future concerns,” Elvira said. GENERIC DRUGS SECTOR ALSO SOUNDS ALARM Most critical medicines are generics, and the generics industry also has warnings for the negotiators. Adrian van den Hoven, director general of the sector lobby group Medicines for Europe, pointed to a few problems with the tool, including poor planning, countries’ lack of commitment to buy jointly procured medicines and different packaging requirements across countries. Legislators should also ensure that there is no conflict with national procurements, he said. “We have experience of member states doing both the European procurement, or the joint procurement, and the national procurement at the same time to see which one — we assume — delivers the better price,” he said. “But then that leaves the suppliers on the hook with a commitment to supply for both. And this is really something to avoid.” The solution? The Commission and countries should consider incentives such as multi-award contracts, a minimum binding commitment from countries to buy, and criteria that go beyond price, to ensure adequate forecasting for companies and to secure a more sustainable supply. Ultimately, it’s all about finding the right balance, said Claudia Louati, head of policy at the European Patients’ Forum. “[Joint procurement] works for member states, it works for companies, it works for the whole ecosystem,” Louati said. “Let’s not miss this opportunity, and let’s try to make it right… We don’t want it to just be in legislation and not actually be implemented and being used.” This article is the product of a POLITICO Working Group presented by Sanofi and was produced with full editorial independence by POLITICO reporters and editors. Learn more about editorial content presented by outside advertisers.