At the recent IPOPI (International Patient Organisation for Primary Immunodeficiencies) conference, the spotlight was on the critical challenges Europe faces in ensuring essential medicines for the most vulnerable patients. At the heart of the debate was the Critical Medicines Act (CMA), the European Commission’s proposed legislation that could be a game-changer for rare diseases, including Primary Immunodeficiencies (PID).
PIDs include nearly 600 rare and chronic genetic diseases, often debilitating or life-threatening if not treated properly. Their management is complex and requires a multidisciplinary approach, as many patients live with a combination of immunological, autoimmune, inflammatory, or oncological problems, making constant and safe access to life-saving drugs a non-negotiable necessity.
The fragility of the system and the urgency of plasma-derived drugs
For most patients with PIDs, replacement therapy with immunoglobulins (IG) derived from human plasma is a fundamental pillar. Yet the sector is fragile: the European Medicines Agency (EMA) confirms that shortages of immunoglobulins will persist until at least June 2026.
Life-saving immunoglobulins for patients with primary immunodeficiencies depend on fragile supplies
Production depends almost entirely on plasma donations, a finite resource. Approximately 30% of the plasma used in Europe is imported from US donors, a dependency that exposes patients to vulnerability in the event of supply chain disruptions. The 2017-2018 crisis in Romania, with patients left without treatment for over a year and tragic consequences—serious infections and even loss of life—is a warning.
Curative innovation: gene therapies
In addition to plasma-derived medicines, the CMA must look to gene therapies. Daniela Ene, a Romanian patient with PID, highlighted her immense difficulties in accessing treatment: despite the availability of a life-saving gene therapy, she struggled for three and a half years to convince the national authorities to find a legal basis for paying for her €1-2 million treatment. “Time is life, and wasting time means wasting life,” she recalled.
Joint purchases between Member States, provided for by the Critical Medicines Act, can be a key tool for timely access
Fabio Candotti, President of the European Society for Immunodeficiencies (ESID), reiterated that high prices and inadequate reimbursement policies are the “crucial crux of the problem.” The CMA, with tools such as joint purchasing between Member States, can facilitate faster and more coordinated access.
Beyond the lowest price: the need for resilient purchasing criteria
One of the main factors behind shortages, especially in smaller markets, is procurement driven solely by the lowest price, not by quality or suitability for the individual patient. For biological therapies, there is no “one size fits all”: each patient may have different needs in terms of composition, mode of administration, and compatibility.
The experts brought together by IPOPI called on the CMA to introduce procurement criteria that reward reliability, resilience, and continuity of supply. According to Rafał Ramotowski of the Plasma Protein Therapeutics Association (PPTA), the correct approach should balance these factors, avoiding an exclusive focus on the cheapest offer.
Stockpiling: patients first
The issue of stockpiling, i.e., the accumulation of large national stocks of medicines, was a central point of the debate. Ramotowski emphasized: “The guiding principle should be ‘Patients first, stockpiling second,’” to prevent national stockpiles from exacerbating shortages elsewhere. Leni von Bonsdorff, from the International Plasma and Fractionation Association (IPFA), agreed on the priority of strengthening EU production to reduce dependence on imports and increase the resilience of the healthcare system.
The perspective of small Member States
During the event, a panel of MEPs took the floor: Peter Agius (EPP, Malta) highlighted the difficulties faced by small countries: “Our healthcare systems are only as strong as their supply chains.” In Malta, medicines arrive years late.
Without alignment between the EU and national reimbursement systems, access to medicines remains uncertain
Agius drew a parallel between rare patients and small states, both of which are penalized by lower bargaining power, and proposed making the joint procurement provisions of the Critical Medicines Act more flexible by allowing a smaller number of countries to participate.
The economic and legal perspective
Czech MEP Ondřej Dostál (NI), a lawyer specializing in health policy and patients’ rights, drew attention to the link between European procurement and national reimbursement systems. Even if the EU were to negotiate favourable supplies for innovative medicines, the challenge remains economic: “The main problem is how EU agreements translate into national reimbursements.” Without alignment, solidarity risks remaining theoretical. A mechanism is needed to link joint procurement to national budgets, allowing for joint negotiation while maintaining national competence over pricing and reimbursement.
The Critical Medicines Act and the European Parliament’s roadmap
MEP Tomislav Sokol (EPP, Croatia), rapporteur for the CMA, provided an overview of the status of the dossier. According to Sokol, the CMA has two objectives: to strengthen the strategic autonomy of European production and to ensure equal access to medicines for all EU citizens. Sokol outlined the way forward: abandoning the ‘lowest price’ criterion, incentivizing European production with strategic projects, promoting joint procurement, and coordinating stocks at EU level.
The vote in the SANT Committee and the European Parliament plenary will determine the future of the CMA.
The vote in the European Parliament’s Committee on Public Health (SANT) is expected in early December, with the plenary session in January 2026.
Martine Pergent, President of IPOPI, concluded: the CMA is perhaps the last big chance to protect access to essential medicines. Policymakers must seize it.
