What a managed access programme is, how it differs from trials and expanded access, and the roles of sponsors, physicians and regulators in the EU and US.
A managed access programme gives patients with a serious condition and no suitable authorised treatment access to a medicine that is not authorised in their country, outside a clinical trial and on the request of their physician. It exists to treat, not to prove that a medicine works, although it can collect real-world data. In the EU the routes are named patient supply under Article 5(1) of Directive 2001/83/EC and compassionate use programmes under Article 83 of Regulation (EC) No 726/2004; in the US the equivalent is FDA expanded access.
Every year, patients with serious or life-threatening conditions exhaust the authorised treatment options available to them, leaving them and their physicians searching for alternatives that have not yet received full regulatory approval. In the United States alone, the FDA receives more than 1,000 requests for all types of expanded access each year (Jarow et al., 2017). For these patients, a managed access programme can be the only lawful route to a medicine that is not yet authorised in their country, rather than waiting until it reaches the market. Understanding the definition, types and roles involved in these programmes is essential for pharmaceutical companies, clinicians and patient advocates who operate within an increasingly complex global regulatory environment. The mechanisms governing pre-approval access to medicines differ significantly between the United States and the European Union, and the operational demands placed on every stakeholder continue to grow as regulators push for greater transparency, equity and data integrity. This article examines how managed access programmes function, who participates in them and where the field is heading in 2026 and beyond; for the wider context, see managed access and early access explained.
A managed access programme is a structured framework through which patients gain access to investigational or unauthorised medicines outside of a standard clinical trial. These programmes exist because the traditional drug approval process, while rigorous and necessary, takes many years. For the innovative medicines the FDA approved between 2010 and 2020, the clinical development phase alone, from the first study in humans to marketing authorisation, took 9.1 years for a typical medicine, and an orphan designation was associated with about 552 more days (Brown et al., 2022). For patients who have no other therapeutic options, that timeline is simply untenable. The core purpose of any such programme is to balance urgency with safety: providing access to medicines that are not yet authorised while still collecting data, monitoring adverse events and maintaining a degree of regulatory oversight that protects both the patient and the integrity of the drug development process. The EMA's guideline on compassionate use describes these programmes as "intended to facilitate the availability to patients of new treatment options under development" (European Medicines Agency, 2007).
Managed access programmes are designed to treat patients, not to prove whether a medicine works.
Clinical trials are designed primarily to generate efficacy and safety data for regulatory submission. They follow strict protocols, randomise patient populations and often include placebo arms. Managed access programmes, by contrast, are not designed to prove whether a medicine works; they are designed to treat individual patients or defined patient cohorts who cannot participate in existing trials. Enrolment criteria tend to be broader, the documentation requirements are different, and the primary goal shifts from scientific hypothesis testing to treatment. A clinical trial asks, "Does this medicine work?" A managed access programme asks, "Should this patient, who has no satisfactory alternative, be treated with a medicine that is not yet authorised?" The EMA guideline draws the same line: clinical trials are "practically the only means of obtaining reliable and interpretable efficacy and safety data for a medicinal product", compassionate use "is not a substitute for properly conducted trials", and patients should always be considered for inclusion in a trial before they are offered a compassionate use programme (European Medicines Agency, 2007).
For a side-by-side view of the three routes, read the key differences between managed access, expanded access and clinical trials.
The terms "expanded access" and "compassionate use" are often used interchangeably, but they carry distinct regulatory connotations depending on jurisdiction, and the lack of a common definition has led to access programmes being misclassified, for example registered as clinical trials (Iudicello et al., 2016). In the United States, the FDA uses "expanded access" as the official term for programmes that allow patients to receive investigational drugs outside of clinical trials, and notes that it is "sometimes called compassionate use" (U.S. Food and Drug Administration, 2026a). In Europe, "compassionate use" is the more common regulatory label, though the European Medicines Agency also recognises use on a "named patient" basis for individual access requests; that route rests on Article 5 of Directive 2001/83/EC and national law, not on Article 83 (European Medicines Agency, 2007). The EMA puts it plainly: compassionate use "should not be confused with named-patient basis treatments, which see doctors obtain medicines directly from manufacturers before authorisation" (European Medicines Agency, n.d.-b). Both terms describe similar mechanisms, yet the procedural requirements, reporting obligations and approval timelines can vary substantially. Precision in terminology matters, because the wrong label in a regulatory submission can delay or derail patient access entirely.
In the United States, the equivalent of managed access is FDA expanded access (U.S. Food and Drug Administration, 2026a). Each request needs an Investigational New Drug (IND) application or amendment, informed consent documentation and the manufacturer's agreement to supply the drug, and brings together the FDA, the manufacturer, an institutional review board and the treating physician (Jarow et al., 2017).
The United States maintains one of the most formalised systems for pre-approval access, with the FDA serving as the central regulatory authority. Its regulations first described a pathway for access to unapproved drugs in 1987 and were revised in 2009 (Jarow et al., 2017). The agency receives more than 1,000 expanded access requests a year, and one review covered almost 11,000 requests over a ten-year period (Jarow et al., 2016, 2017). Approval rates have stayed at or above 99 per cent: 99.7 per cent of the applications received by the FDA's Center for Drug Evaluation and Research from 2005 to 2014 were allowed to proceed (Jarow et al., 2016), and the FDA states today that it allows over 99 per cent of single patient expanded access requests to proceed (U.S. Food and Drug Administration, 2025a). That high approval rate, however, does not mean the process is simple; it requires coordination among the FDA, the drug manufacturer, an institutional review board and the treating physician. The FDA cannot require a manufacturer to provide its drug, and a manufacturer may have valid reasons to decline, such as an ongoing clinical trial the patient can join or limited drug supply (Jarow et al., 2017).
The FDA recognises three primary categories of expanded access under its Investigational New Drug (IND) regulations, set out in 21 CFR part 312, subpart I (21 C.F.R. part 312, subpart I). Individual patient access, which includes emergency use, covers a single patient; in an emergency, the FDA may authorise treatment by telephone before a written request is made, and the written submission must follow within 15 working days (21 C.F.R. § 312.310). Intermediate-size patient populations allow access for a defined group, for example when a disease is so rare that a trial cannot recruit, or when patients cannot join an ongoing trial because they do not meet its criteria, enrolment is closed or the trial site is too far away (21 C.F.R. § 312.315). Treatment IND or treatment protocol programmes serve larger populations, typically when the drug is being studied in a controlled trial designed to support a marketing application, or all its trials are complete, the sponsor is actively pursuing marketing approval and the FDA judges the clinical evidence sufficient to support the use, ordinarily from phase 3 trials (21 C.F.R. § 312.320). Each pathway requires the submission of an IND application or amendment, informed consent documentation, review by an institutional review board and agreement from the manufacturer to supply the drug (21 C.F.R. § 312.305; Jarow et al., 2017). The FDA developed Form FDA 3926 specifically to simplify individual patient requests, and estimates the physician's paperwork at roughly 45 minutes per request (Jarow et al., 2017; U.S. Food and Drug Administration, 2024a, 2025b).
The federal Right to Try Act, signed into law on 30 May 2018 (U.S. Food and Drug Administration, 2018), created an alternative pathway that bypasses FDA review for certain eligible patients: the FDA states that it "does not review or approve requests for Right to Try Act use" (U.S. Food and Drug Administration, 2024c). Under this legislation, a patient who has been diagnosed with a life-threatening disease, has exhausted approved options and cannot take part in a clinical trial may request an investigational drug directly from the manufacturer, provided a Phase 1 trial of the drug has been completed, the drug is not approved for any use and its development has not been discontinued (U.S. Food and Drug Administration, 2024c). Unlike expanded access, the federal pathway requires neither FDA authorisation nor institutional review board review (Chapman et al., 2020). The law was intended to reduce bureaucratic delays, but its practical use has been limited. In a survey of 238 community oncologists and haematologists, 46 per cent had tried to obtain an investigational drug for a patient through expanded access and 14 per cent through Right to Try (Zettler et al., 2021); among paediatric haematologists and oncologists, 40 of 46 respondents had used expanded access for their most recent request and 4 had used Right to Try (Chapman et al., 2021). Physicians and manufacturers who choose expanded access keep institutional review board review, adverse event reporting to the sponsor and FDA oversight in place (21 C.F.R. § 312.305). As of 2026, the Right to Try pathway remains in force alongside expanded access (U.S. Food and Drug Administration, 2024c).
In the EU, the legal basis for supplying one patient is Article 5(1) of Directive 2001/83/EC; for a group of patients it is Article 83 of Regulation (EC) No 726/2004.
Under Article 5(1) of Directive 2001/83/EC, a member state may, to fulfil special needs, exclude from the Directive's provisions medicines supplied in response to a bona fide unsolicited order, formulated by an authorised healthcare professional for use by an individual patient under his or her direct personal responsibility (Directive 2001/83/EC). Under Article 83 of Regulation (EC) No 726/2004, member states may make a medicine that falls within the scope of the centralised procedure available for compassionate use to a group of patients with a chronically or seriously debilitating or life-threatening disease who cannot be treated satisfactorily by an authorised medicine, provided the medicine is the subject of a marketing authorisation application or is undergoing clinical trials (Regulation (EC) No 726/2004).
The European regulatory environment for managed access is more fragmented than its American counterpart, reflecting the EU's structure of centralised agencies operating alongside sovereign member states. The European Medicines Agency provides overarching guidance, but individual countries retain significant authority over how compassionate use and named patient programmes are implemented within their borders: national programmes, whether on a named patient basis or for cohorts of patients, "are governed by individual Member States (MS) legislation" (European Medicines Agency, 2007), and compassionate use programmes "are coordinated and implemented by Member States, which set their own rules and procedures" (European Medicines Agency, n.d.-b).
The EMA's Committee for Medicinal Products for Human Use (CHMP) can issue opinions on compassionate use for groups of patients, but these opinions are not binding on member states, which must nevertheless take account of any available opinions (European Medicines Agency, 2007; Regulation (EC) No 726/2004). A member state that intends to use Article 83 must notify the EMA, and the CHMP may then adopt an opinion on the conditions for use, the conditions for distribution and the patients targeted (European Medicines Agency, 2007). Each country establishes its own legal framework, application procedures and approval timelines. France operates its Autorisations d'Accès Compassionnel system, under which medicines without a marketing authorisation in France may be used, by way of derogation, for serious or rare diseases that have no appropriate treatment (Agence nationale de sécurité du médicament et des produits de santé, 2026). In Germany, compassionate use programmes run under Section 21(2) no. 3 of the German Medicinal Products Act (AMG) with the Arzneimittel-Härtefall-Verordnung (AMHV) (AMHV, 2010). Individual named patient imports run under Section 73(3) AMG, which allows a pharmacy to bring in small quantities of a finished medicine that is not authorised in Germany on an individual order, provided it may be lawfully marketed in the country it comes from and no medicine with the same active substance and a comparable strength is available in Germany for that indication (§ 73(3) AMG). The Netherlands has its own named patient framework: the treating physician records in a physician's declaration (artsenverklaring) that the patient cannot be treated with the medicines available, and a pharmacy, wholesaler or manufacturer then asks the Health and Youth Care Inspectorate for an exemption under Article 40(3)(c) of the Dutch Medicines Act (Inspectie Gezondheidszorg en Jeugd, n.d.). A comparison of seven European countries found variable national rules and inconsistent reimbursement for compassionate use programmes and named patient supply (Rosenberg et al., 2025). This patchwork creates real challenges for pharmaceutical manufacturers attempting to run a managed access programme across multiple EU countries simultaneously, because a single medicine may require different documentation, different pricing arrangements and different reporting structures in each jurisdiction.
To go further, country by country, read how named patient programmes work in Europe.
The EU Clinical Trials Regulation, Regulation (EU) No 536/2014, has applied since 31 January 2022 (Commission Decision (EU) 2021/1240; Regulation (EU) No 536/2014). Trials authorised under the former Clinical Trials Directive could continue under it for three years, and since 31 January 2025, when that transition period ended, any such trial still running has had to comply with the Regulation and be recorded in the Clinical Trials Information System (CTIS) (European Medicines Agency, n.d.-a; Regulation (EU) No 536/2014, Art. 98). The Regulation has indirect but meaningful effects on managed access programmes across Europe. By centralising clinical trial authorisation through a single online application in CTIS and increasing transparency around ongoing studies, it makes it easier for physicians and patients to identify whether a relevant trial exists before pursuing compassionate use (European Medicines Agency, n.d.-a), which matters because patients should always be considered for a clinical trial first (European Medicines Agency, 2007). The Regulation itself applies to clinical trials and not to non-interventional studies (Regulation (EU) No 536/2014, Art. 1); compassionate use and named patient supply remain governed by Article 83 of Regulation (EC) No 726/2004 and by national law (European Medicines Agency, 2007). Data collection in access programmes is therefore set by those rules. Where the CHMP has adopted an opinion on a compassionate use programme, the pharmacovigilance rules for centrally authorised medicines apply to it (European Medicines Agency, 2007), and national schemes add their own requirements: of four countries compared in one analysis, France had the most structured early access programmes, and they provide for data collection (Tarantola et al., 2023).
In a managed access programme, the manufacturer bears the burden of drug supply, regulatory interaction and pharmacovigilance. The treating physician identifies eligible patients, submits the request, obtains informed consent and monitors treatment outcomes, while patient advocacy organisations help patients and families understand their options.
No managed access programme functions without coordinated effort among multiple parties, each carrying distinct responsibilities and facing unique pressures. The success or failure of a programme often hinges not on the medicine itself but on whether the operational infrastructure can deliver it to the right patient, in the right dose, at the right time.
One programme across several countries means a different legal basis, a different supply route and a different reporting structure in each of them. Ideogen does that work across Europe, MENA, CIS and 94 markets worldwide, in orphan and rare disease. If you want to bridge market access for unmet needs in orphan and rare disease reach out to [email protected].
The manufacturer bears the primary burden of drug supply, regulatory interaction and pharmacovigilance. Although an expanded access programme is regulated, the decision to set one up rests with the sponsor, and that decision should weigh ethical and practical considerations as well as the regulatory criteria (Klopfenstein et al., 2015). Deciding to establish a managed access programme is a strategic calculation: it can generate goodwill and provide early real-world data, but it also diverts drug supply from clinical trials, creates regulatory reporting obligations and exposes the company to liability risk. Supply chain logistics are particularly complex for biologics or cell therapies that require cold chain management, short shelf lives or patient-specific manufacturing. A manufacturer operating a programme across many countries may need a separate import licence, customs procedure and distribution route for each of them, often for a product that does not yet have a stable commercial supply chain.
For sponsors working through these choices, this is how Ideogen runs managed access programmes for partners.
Physicians serve as the gatekeepers of managed access, identifying eligible patients, submitting requests, obtaining informed consent and monitoring treatment outcomes. In US expanded access, the physician under whose direction the drug is given counts as an investigator: he or she reports adverse drug events to the sponsor, ensures informed consent and institutional review board review, and keeps case histories and drug disposition records (21 C.F.R. § 312.305). Institutional review board approval and informed consent must be obtained before treatment begins (U.S. Food and Drug Administration, 2025a). The administrative burden on clinicians is not trivial, particularly in countries where the paperwork requirements are extensive or where institutional review board approval is mandatory for each individual request.
Patient advocacy organisations play an increasingly visible role as well, raising awareness of available programmes, helping patients and families understand their options and sometimes pressing manufacturers and authorities to establish or expand access initiatives. EURORDIS-Rare Diseases Europe, for example, has called for compassionate use programmes built on equal access for all patients, transparency and a clear definition of the patients each programme is for, noting that not all countries have such programmes and that patients gain access at different times depending on where they live (EURORDIS-Rare Diseases Europe, 2017). Information about what is available has also become easier to find. In the United States, a company developing investigational drugs must make its policy for evaluating and responding to expanded access requests public, and posting it on the Reagan-Udall Foundation's Expanded Access Navigator can satisfy that requirement (U.S. Food and Drug Administration, 2024b). The Navigator offers a company directory of these policies and step-by-step guides for patients and physicians (Reagan-Udall Foundation for the FDA, n.d.), reducing one of the most persistent barriers: the simple lack of information about what is available.
Running a managed access programme is operationally demanding and ethically fraught. The tension between providing access and maintaining scientific rigour, between treating the patient in front of you and preserving the integrity of ongoing clinical trials, defines much of the daily decision-making involved. As access widens from single patients to larger groups, ethical issues gain prominence, including informed consent, funding, disparities in access and possible adverse effects on clinical drug development (Bunnik et al., 2017).
One of the most significant shifts in recent years has been the growing expectation that managed access programmes should generate usable real-world evidence. Regulators, payers and manufacturers all have an interest in capturing safety and outcome data from patients treated outside of clinical trials. However, the quality of this data varies enormously. Unlike clinical trials, managed access programmes rarely have standardised endpoints, centralised data management or independent monitoring. Some manufacturers have invested in digital platforms and electronic case report forms to improve data capture, but many programmes still rely on ad hoc physician reporting. The volume of published results is nonetheless growing: a review of peer-reviewed expanded access publications from 2000 to 2022 included 1,231 articles describing 507,481 patients (Polak et al., 2023). The challenge is real: imposing too much data collection burden on physicians can discourage participation, while collecting too little data wastes a valuable opportunity to understand how a medicine is used in diverse, real-world patient populations.
When drug supply is limited, someone must decide which patients receive treatment and which do not. This is among the most ethically sensitive aspects of any managed access programme, and fair patient selection is one of the main ethical challenges that reviews of compassionate use identify (Borysowski & Górski, 2019). Criteria for prioritisation can include disease severity, the expected balance of benefit and risk for the individual patient, absence of alternative treatments and geographic proximity to a treatment centre, but no single framework is universally accepted. One proposed ethics framework sets eight requirements for any treatment use of an investigational drug, among them a justified need, no threat to clinical development, an informed decision by the patient, fair access and independent review (Borysowski & Górski, 2020).
Disparities in access are well documented. Among one manufacturer's global compassionate use requests from 2018 to 2020, 87 per cent of 31,711 requests came from high-income countries and 3 per cent from lower-middle-income and low-income countries, and request activity was higher in countries whose compassionate use rules were publicly available and in countries with more clinical trial activity (Aliu et al., 2022). Published expanded access research shows the same imbalance, with Europe and the Americas accounting for 87.4 per cent of publications and Africa for 0.6 per cent (Polak et al., 2023). Even among high-income European countries, compassionate use programmes differ in availability and duration (Rosenberg et al., 2025), and physicians report unequal access to information about investigational drugs (Chapman et al., 2021). Addressing these inequities requires deliberate programme design, transparent eligibility criteria and, in some cases, partnerships with international health organisations that can extend a programme's geographic reach.
Expanded access is the US FDA term for access to an investigational medicine outside clinical trials. Managed access is the term used in Europe for programmes that supply a medicine before, or outside, local authorisation under national routes, such as named patient supply under Article 5(1) of Directive 2001/83/EC (Directive 2001/83/EC; U.S. Food and Drug Administration, 2026a).
The treating physician. The sponsor decides whether and how to supply, and the national rules decide the legal route and the documents required (European Medicines Agency, 2007; Jarow et al., 2017).
It can collect real-world safety and outcome data, but it is not designed to prove efficacy; that is the role of clinical trials (European Medicines Agency, 2007).
Yes. Ideogen builds and runs managed access and named patient programmes for partners across Europe, MENA, CIS and 94 markets worldwide, in orphan and rare disease. We assess the legal route, the supply model and the local requirements market by market, and we run the programme once it is live. Write to [email protected].
The trajectory of managed access is pointing towards greater standardisation, digital infrastructure and regulatory convergence, though the pace of change varies by region. Several trends are shaping the field in 2026 and will likely accelerate over the coming years.
Rules and guidance continue to move. In the United States, the FDA issued a revised version of its questions-and-answers guidance on expanded access in April 2026 (U.S. Food and Drug Administration, 2026b). In the EU, the European Commission proposed in 2023 to repeal Directive 2001/83/EC and Regulation (EC) No 726/2004, the two acts on which named patient supply and compassionate use rest today, and to replace them with a new directive and a new regulation (European Commission, 2023a, 2023b). Harmonised single applications already exist for multinational clinical trials in the EU through CTIS (European Medicines Agency, n.d.-a), while compassionate use is still coordinated and implemented by each member state under its own rules (European Medicines Agency, n.d.-b). Digital health platforms are making it easier for physicians to identify programmes, submit requests electronically and report outcomes in structured formats: the Expanded Access Navigator, for example, includes an electronic request route for non-emergency use (Reagan-Udall Foundation for the FDA, n.d.).
The integration of real-world evidence from managed access into regulatory decision-making is also gaining momentum. A review of FDA and EMA approval documents found 39 approvals in which data from expanded access programmes were used to inform the assessment of clinical efficacy; the yearly number rose from 1.25 in 1993 to 2013 to 4.6 in 2014 to 2018, and in 13 approvals these programmes formed the main evidence, almost all for medicines with an orphan designation (Polak et al., 2020). The authors conclude that sponsors and regulators increasingly include real-world data from expanded access in the assessment of a treatment (Polak et al., 2020). For pharmaceutical companies, this means that a well-designed managed access programme is no longer just a humanitarian obligation; when its data collection is planned from the start, it can also contribute to regulatory dossiers and to follow-up after authorisation.
Patients, physicians, regulators and manufacturers all share a common interest in ensuring that pre-approval access is safe, equitable and well documented. The programmes that succeed in the years ahead will be those that treat operational excellence and ethical rigour not as competing priorities but as inseparable ones. For any organisation considering the launch or expansion of a managed access programme, the time to invest in the right infrastructure, partnerships and governance frameworks is now.
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