How named patient programmes work in Europe: the Article 5(1) legal basis, the physician's role, import and customs, national rules and the US comparison.
A named patient programme supplies a medicine that is not authorised in the patient's country to one patient, on the bona fide unsolicited order of the treating physician, who takes clinical responsibility. In the EU the legal basis is Article 5(1) of Directive 2001/83/EC, applied through national rules that differ on documents, import permits and approval times. A specialist distributor usually handles sourcing, import and customs under good distribution practice.
Across the European Union, some patients face a difficult reality: the medicine their physician considers necessary exists, but it is not authorised in their country, or it is authorised elsewhere and not available at home. Named patient supply is the regulated route that answers this situation one patient at a time: it can give access to a medicine before its marketing authorisation, or allow the import of a medicine authorised in another country (Rosenberg et al., 2025). Named patient requests are not publicly listed, and the lack of centralised data limits any count of how often the route is used (Rosenberg et al., 2025). Named patient programmes serve as the bridge between pharmaceutical development and individual patient need, offering a regulated pathway for physicians to prescribe unlicensed or unavailable medicines on a case-by-case basis. These programmes operate under a patchwork of national regulations, each with distinct requirements for documentation, import, and oversight. Understanding how named patient programmes work in Europe requires a close examination of the legal frameworks, operational logistics, and compliance considerations that shape access to medicines before or outside standard commercial availability. For pharmaceutical manufacturers, healthcare providers, and the patients who depend on them, these programmes are far more than a regulatory formality.
Named patient supply refers to the provision of a medicinal product to an individual patient, under the direct responsibility of a treating physician, when no suitable authorised alternative exists within that patient's country. The product in question may already hold marketing authorisation in another jurisdiction, or it may still be undergoing clinical trials. What distinguishes this pathway from standard pharmaceutical distribution is its individual, patient-specific nature: each request is tied to a single patient with a documented, unmet medical need.
The underlying rationale is straightforward. A patient should not lose access to a medicine that their physician considers necessary solely because the regulatory process has not yet concluded in their territory, or because the medicine has not been launched there. Named patient supply exists to close that gap, as an individual exemption that answers clinical need case by case rather than waiting for administrative timelines. It is distinct from a compassionate use programme for a group of patients: the European Medicines Agency's guideline states that using an unauthorised medicine for compassionate use on a named patient basis, under Article 5 of Directive 2001/83/EC, does not fall under the scope of Article 83 of Regulation (EC) No 726/2004 (European Medicines Agency, Committee for Medicinal Products for Human Use, 2007). Anticancer and orphan medicines are among the products reached this way (Rosenberg et al., 2025), alongside medicines for other serious conditions, such as infectious diseases, that are not yet commercialised locally.
Related: managed access programmes explained
The legal basis for named patient programmes across Europe stems primarily from Article 5(1) of Directive 2001/83/EC, which permits Member States to exclude certain medicinal products from the requirement for marketing authorisation when supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised healthcare professional, and intended for use by an individual patient under the professional's direct personal responsibility (Directive 2001/83/EC).
The reform of EU pharmaceutical law keeps this route. In the new directive that the Council adopted as its position at first reading on 28 September 2026, Article 4(1) lets a Member State exempt medicinal products supplied in response to a bona fide unsolicited order, or an anticipated one, to meet the special needs of individual patients under the direct personal responsibility of an authorised healthcare professional, and asks Member States to encourage healthcare professionals and patients to report safety data to the competent authority (Council of the European Union, 2026). On 29 September 2026 the Commission sent the European Parliament its communication on the Council's position, the step that precedes the Parliament's second reading (European Commission, 2026). Once the directive enters into force, Member States would have 24 months to transpose it (Council of the European Union, 2026).
However, the current directive is a framework, not a uniform rulebook. Each EU Member State applies it through national legislation with significant variation. In France, the Autorisation Temporaire d'Utilisation (ATU) was replaced on 1 July 2021 by early access authorisation (autorisation d'accès précoce, granted by HAS) (Haute Autorité de Santé, n.d.) and compassionate access authorisation (autorisation d'accès compassionnel, granted by ANSM) (Agence nationale de sécurité du médicament et des produits de santé, n.d.); Décret n° 2026-448 harmonised both procedures from 1 September 2026 (Décret n° 2026-448). In Germany, Section 73(3) of the German Medicinal Products Act (AMG) permits individual imports: a pharmacy may bring in a finished medicine that is not authorised in Germany when it is ordered for an individual person in a small quantity, when it may lawfully be marketed in the country it comes from, and when no medicine with the same active substance and a comparable strength is available in Germany for the indication; for medicines from outside the EU and the European Economic Area, the order and dispensing need a medical prescription (AMG, 1976, § 73(3)). Germany's separate hardship ordinance for compassionate use programmes expressly excludes such individual use under a physician's direct responsibility (AMHV, 2010, § 1(2)).
Other countries organise the route differently. When Rosenberg et al. (2025) compared seven high-income European countries in 2023, the national competent authority handled named patient applications in France, Norway and the United Kingdom, the national inspectorate in the Netherlands and cantonal inspectorates in Switzerland, while in Belgium, which had no national procedure, individual pharmacies handled them; the Netherlands was the only country where a manufacturer could apply directly. Spain regulates the availability of medicines in special situations, including medicines authorised abroad, by Royal Decree 1015/2009 (Real Decreto 1015/2009). Italy keeps the Law 648/1996 list, through which the national health service reimburses medicines with no valid therapeutic alternative, including medicines authorised abroad but not in Italy (Agenzia Italiana del Farmaco, n.d.). Portugal grants an exceptional use authorisation for medicines without a marketing authorisation (INFARMED, n.d.), and Austria's medicines agency distinguishes compassionate use programmes from named patient use (Bundesamt für Sicherheit im Gesundheitswesen, n.d.). The United Kingdom and Switzerland, both outside the European Economic Area, run their own national procedures (Rosenberg et al., 2025).
This fragmentation means that a named patient import approved in one country may require an entirely different set of documentation, timelines, and regulatory interactions in another. Pharmaceutical companies and specialist distributors must therefore maintain granular, country-specific expertise to ensure compliance.
That country-specific expertise is the work. Ideogen holds it across Europe, MENA, CIS and 94 markets worldwide: sourcing from a market where the medicine is lawfully marketed, the import permit or notification, customs, GDP-compliant delivery, and the documentation file each authority expects. Talk to our experts about bridging access to unmet need in orphan and rare disease, where earlier access changes patient outcomes, at [email protected].
The treating physician requests the medicine for a named patient, the request is checked against national rules, and a specialist distributor sources, imports and delivers it under good distribution practice.
The treating physician stands at the centre of every named patient request.
It is the physician who determines that no authorised alternative can adequately address the patient's condition, initiates the request, and assumes clinical responsibility for the treatment. This is not a passive role: the prescriber must document the medical justification, confirm that the patient has been informed of the unlicensed status of the medicine, and in many jurisdictions, obtain informed consent, which can itself raise practical and ethical difficulties (Borysowski & Górski, 2019).
Patient eligibility criteria vary by country but share common elements:
In some countries, hospital pharmacies or national health authorities must also approve the request before the medicine can be sourced and dispensed (Rosenberg et al., 2025). The process is deliberately rigorous to prevent misuse and to ensure that named patient supply remains an exception rather than a routine distribution channel.
Once a physician's request has been validated, the operational complexity of sourcing, importing, and delivering the medicine begins. Specialist pharmaceutical distributors typically manage this process, acting as intermediaries between the manufacturer, the regulatory authority, and the prescribing institution.
The logistics chain involves several critical steps. The distributor must first confirm product availability from the manufacturer or an authorised supplier in a country where the medicine holds marketing authorisation. Good distribution practice (GDP) must be maintained throughout the supply chain: under the EU GDP guidelines, wholesale distributors obtain medicines only from holders of a wholesale distribution or manufacturing authorisation, a distributor that receives medicines from a third country to place them on the EU market must hold a manufacturing authorisation, and the supplying wholesaler must keep temperature conditions within acceptable limits during transport (Guidelines 2013/C 343/01). Import permits or notifications must be filed with the relevant national authority, and their scope varies: most countries grant named patient access for an individual patient, but Norway can also grant it to a clinic or hospital, the Netherlands per indication and Belgium per patient group to a single prescriber (Rosenberg et al., 2025).
Customs clearance for pharmaceutical products adds another layer of complexity. Shipments must be accompanied by certificates of analysis, proof of origin, and regulatory authorisation documents. Delays at customs can be clinically consequential, particularly for time-sensitive treatments. Experienced distributors maintain established relationships with customs authorities and employ pre-clearance strategies to minimise disruption. The time from physician request to patient delivery depends on the country, the product, and the regulatory pathway involved; because named patient requests are rarely published and centralised data are lacking, these timelines cannot readily be compared across countries (Rosenberg et al., 2025).
Related: specialty supply chain for unlicensed medicines
Europe runs named patient supply through national rules under Article 5(1) of Directive 2001/83/EC, so documents and timelines differ by country (Directive 2001/83/EC). The US runs expanded access through one federal authority, the FDA, under 21 CFR Part 312, and supply depends on the manufacturer agreeing to provide the product (21 C.F.R. pt. 312, subpt. I, 2026; U.S. Food and Drug Administration [FDA], 2025).
The United States operates its own framework for providing patients with access to unapproved medicines, known as Expanded Access or Compassionate Use, regulated by the FDA under 21 CFR Part 312 (21 C.F.R. pt. 312, subpt. I, 2026; FDA, 2026). The FDA distinguishes between three categories: individual patient access (emergency and non-emergency), intermediate-size patient populations, and treatment INDs or treatment protocols for larger groups (21 C.F.R. §§ 312.310 to 312.320, 2026). The FDA considers expanded access appropriate when, among other conditions, the patient has a serious or immediately life-threatening disease or condition, there is no comparable or satisfactory alternative therapy, enrolment in a clinical trial is not possible, and supply will not interfere with clinical trials that could support approval (FDA, 2026).
An individual patient request may be submitted by the sponsor or by a licensed physician (21 C.F.R. § 312.310(b), 2026), and a physician must first make sure that the manufacturer is willing to provide the investigational drug before submitting it (FDA, 2025). The FDA reviews these requests quickly: in an emergency the request can be made by telephone, and the FDA official can authorise shipment and use over the telephone before the written submission follows (FDA, 2025; 21 C.F.R. § 312.310(d), 2026). For a non-emergency request, treatment may begin 30 days after the FDA receives the application, or earlier if the FDA says it may proceed, and the agency allows over 99 per cent of single patient expanded access requests to proceed (FDA, 2025). The Right to Try Act, signed into law on 30 May 2018 (FDA, 2018), also created an alternative pathway for certain eligible patients that the FDA does not review or approve, and it does not oblige a sponsor to provide the drug (FDA, 2024).
Unlike the European model, the US system is centralised under a single federal authority, which simplifies the regulatory interaction but introduces its own challenges around manufacturer willingness and liability considerations.
The structural differences between named patient programmes in Europe and the US expanded access system produce distinct practical outcomes. In Europe, the fragmented regulatory environment means that timelines, documentation requirements, and approval processes differ from country to country. Even the validity of an approval varies: when Rosenberg et al. (2025) collected their data, a named patient approval was valid for one year in Belgium, France, the Netherlands and Norway. This variability demands specialised knowledge and infrastructure from distributors operating across multiple markets.
In the United States, the centralised FDA pathway sets common rules for every request but depends on manufacturer cooperation: the physician must first confirm that the manufacturer will provide the drug (FDA, 2025), so a company that declines to supply a product under expanded access effectively closes the pathway for that patient, regardless of FDA willingness to authorise it. European rules, by contrast, can allow import from other markets where the product is lawfully marketed: Germany's import rule applies to medicines that may lawfully be marketed in the country they come from (AMG, 1976, § 73(3)), and Switzerland's to medicines authorised in a country with comparable medicines control (AMBV, 2018, Art. 49). Where several such markets exist, this gives more flexibility in sourcing.
Cost structures also diverge. In the seven European countries studied by Rosenberg et al. (2025), charging and reimbursement for named patient supply were often not regulated, and where reimbursement was possible it was usually granted only on an exceptional basis (Rosenberg et al., 2025). Compassionate use is usually covered by pharmaceutical companies, while some early access programmes are reimbursed by third-party payers (Tarantola et al., 2023); Italy's Law 648/1996 list is one example of public reimbursement for medicines without a valid therapeutic alternative (Agenzia Italiana del Farmaco, n.d.). In the United States, a sponsor needs prior written authorisation from the FDA to charge for an investigational drug and may recover only the direct costs of making it available, plus certain monitoring and administrative costs for intermediate-size and treatment use (21 C.F.R. § 312.8, 2026).
Manufacturers run named patient programmes to answer treating physicians' unsolicited requests for patients who have no suitable authorised alternative, to keep supplying trial participants after a trial ends, and to collect safety and outcome data while doing so; the programmes cannot be used to promote a medicine before its authorisation (Directive 2001/83/EC).
For pharmaceutical manufacturers, named patient programmes have a strategic dimension that sits alongside their humanitarian purpose, and it has firm limits. The programmes provide a route for early access: a medicine reaches individual patients in a territory before formal marketing authorisation is granted there. They cannot, however, be used to build demand before launch. Article 5(1) applies only to a bona fide unsolicited order, and Article 87(1) of the same Directive requires Member States to prohibit any advertising of a medicinal product that has no marketing authorisation (Directive 2001/83/EC); Rosenberg et al. (2025) likewise stress that unauthorised medicines should not be overly advertised. A manufacturer can therefore respond to physicians' requests, but it cannot solicit them.
Equally significant is the opportunity to collect real-world evidence. Data gathered from named patient use, including observations on outcomes, tolerability and dosing across diverse patient populations, can complement clinical trial data and inform regulatory submissions. Regulators do use such data: a review of FDA and EMA approval documents identified 39 approvals in which data from expanded access programmes informed the evaluation of clinical efficacy, 13 of them as the main evidence, and 12 of those 13 concerned medicines with orphan designation (Polak et al., 2020).
Supporting patient access also matters for a manufacturer's relationships with healthcare institutions and patient organisations, and transparency is part of it: information on named patient supply is often undisclosed, which can leave some physicians unaware of the medicines available through the route, and Rosenberg et al. (2025) recommend public lists of the medicines supplied this way.
Next step: compassionate use for sponsors
By supplying the medicine to an individual patient while the regulatory process unfolds, including patients who took part in a clinical trial and whose physician considers that the treatment should continue after the trial ends.
The period between the completion of pivotal clinical trials and access through normal channels can span months or even years. Under the centralised procedure alone, the Committee for Medicinal Products for Human Use has 210 days from a valid application to give its opinion, after which the Commission prepares and adopts its decision (Regulation (EC) No 726/2004, Arts. 6(3) and 10), and national reimbursement decisions after authorisation can take more than a year (Rosenberg et al., 2025). Across the EU27, the medicines centrally authorised between 2021 and 2024 took 597 days on average from authorisation to reach patients (Newton et al., 2026). For patients with serious or life-threatening conditions, this gap is not merely an administrative matter.
Named patient supply bridges this gap by enabling continued or initial access to a medicine while the regulatory process unfolds. In oncology, for example, a patient who received a medicine in a clinical trial may need continued access after the trial concludes but before commercial availability. The Declaration of Helsinki requires post-trial provisions to be arranged before a clinical trial begins (World Medical Association, 2024), and the EMA's approach to expanded access includes patients treated in a clinical trial who wish to continue the treatment (Iudicello et al., 2016). Without an access route, that patient's treatment would stop when the trial ends.
This bridging function also matters to the manufacturer: it maintains treatment continuity for trial participants, supports the collection of long-term outcome data, and means that trial participants are not left without treatment during a regulatory transition.
Operating a named patient programme across multiple European jurisdictions demands rigorous adherence to compliance and safety standards that vary in specificity but converge on core principles. Product quality must be assured through verified supply chains, with medicines sourced only from authorised manufacturers or licensed wholesalers (Guidelines 2013/C 343/01). Pharmacovigilance obligations apply. The EU guideline on good pharmacovigilance practices states that its guidance on reports of suspected adverse reactions may also apply to named patient use under Article 5(1), subject to local requirements: when an organisation or a healthcare professional supplying a medicine on a named patient basis becomes aware of an adverse event, reports are submitted as individual case safety reports, as solicited reports where the Member State requires active collection of adverse events and as spontaneous reports where it does not (European Medicines Agency & Heads of Medicines Agencies, 2017). The reform text keeps this direction, asking Member States to encourage safety reporting on named patient use (Council of the European Union, 2026).
Documentation requirements are exacting. Each patient request typically generates a file that includes the physician's prescription or medical justification, import authorisation or notification, proof of GDP-compliant handling, and records of patient consent. These files must be maintained and made available for regulatory inspection. Non-compliance can result in product seizure, legal penalties, and reputational damage for both the distributor and the manufacturer.
The intersection of data protection regulations, particularly the General Data Protection Regulation (GDPR), with named patient supply adds further complexity. Data concerning health are a special category of personal data, which may be processed only where one of the conditions in Article 9(2) of the GDPR applies (Regulation (EU) 2016/679). Patient-identifiable information crosses organisational and sometimes national boundaries during the request and fulfilment process, requiring careful attention to data handling protocols. Organisations that manage named patient programmes at scale invest significantly in quality management systems, regulatory intelligence, and trained personnel to ensure that every transaction meets the applicable legal and ethical standards.
Article 5(1) of Directive 2001/83/EC lets Member States exclude from the authorisation requirement medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised healthcare professional and for use by an individual patient under their direct personal responsibility (Directive 2001/83/EC).
The treating physician, who documents the medical justification, confirms that no authorised alternative is suitable and takes clinical responsibility.
No. Article 5(1) is a framework; each Member State sets its own documents, import rules and timelines (Rosenberg et al., 2025).
Usually a specialist distributor working for the manufacturer: confirming availability in a market where the medicine is lawfully marketed, filing the import permit or notification, clearing customs, delivering under good distribution practice and keeping the documentation file each national authority expects. Ideogen does this for manufacturer partners across Europe, MENA, CIS and 94 markets worldwide. Talk to our experts about bridging access to unmet need in orphan and rare disease, where earlier access changes patient outcomes, at [email protected].
Ultimately, named patient programmes in Europe represent a sophisticated intersection of clinical need, regulatory flexibility, and pharmaceutical logistics. They exist because medicine does not always move at the pace of bureaucracy, and patients cannot always wait. For manufacturers responding to physicians' requests, and for physicians seeking an option for a patient with no authorised alternative, these programmes remain an indispensable tool in the global healthcare system. The organisations that operate them well do so with precision, compliance, and an unwavering focus on the patient at the centre of every request.
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