By the PharmaTradz BD Team. Published October 2026.
An early access program is a legal route that lets a doctor treat a patient with a medicine that is not yet approved, or not yet available, in the patient's country. It is meant for serious or life-threatening conditions where no satisfactory approved treatment exists and the patient cannot join a clinical trial. Regulators call these routes by different names, such as expanded access in the US, compassionate use in the EU and EAMS in the UK, but they share the same idea: controlled supply, one patient or one group at a time, outside the normal market.
A managed access program is the industry name for a structured programme that a manufacturer runs, often through a specialist supplier, to handle these requests across many countries under one set of rules.
The terms, and how industry uses them
The words overlap, and people use them loosely. It helps to separate the legal terms from the business ones.
Expanded access is the US legal term, set out in the FDA's rules at 21 CFR 312 Subpart I. Compassionate use is the EU legal term for programmes for groups of patients, under Article 83 of Regulation (EC) No 726/2004, though many people use it for any pre-approval supply. Named-patient supply means a medicine ordered by a doctor for one identified patient, under the doctor's own responsibility. In the EU this rests on Article 5(1) of Directive 2001/83/EC.
Early access program (EAP) is the umbrella term companies use for all of these. Managed access program (MAP) usually means a company-run programme with a written protocol, eligibility rules, a single ordering process and a partner that handles import and delivery. Be careful in the UK: there, NICE uses "managed access" to mean something different, a funding arrangement in which the NHS pays for a medicine while more evidence is collected, for example through the Cancer Drugs Fund.
United States: expanded access and Right to Try
The FDA recognises three categories of expanded access:
- Individual patient access (21 CFR 312.310), including emergency use. The doctor must judge that the risk from the drug is not greater than the risk from the disease, and the FDA must be satisfied the patient cannot get the drug under another IND (investigational new drug application) or protocol. Doctors can use the short Form FDA 3926.
- Intermediate-size patient populations (312.315), for groups larger than one but well below a full treatment programme.
- Treatment IND or treatment protocol (312.320), for widespread use of a drug that is still being developed for marketing.
In an emergency, the FDA can authorise use by telephone or other rapid means. The written submission must follow within 15 working days, and if there was no time for ethics committee (IRB) review, the IRB must be told within 5 working days. Individual treatment is generally limited to one course unless the FDA agrees to more. Manufacturers must also publish their expanded access policy, but the FDA cannot force a company to supply.
The Right to Try Act, signed on 30 May 2018, adds a second route. It covers patients with a life-threatening condition who have exhausted approved options and cannot join a trial, and drugs that have completed a Phase 1 trial and remain in active development. Supply runs from manufacturer to doctor without FDA authorisation of each use, but the patient must give written informed consent, charges are limited to direct costs, and the manufacturer must send the FDA an annual summary of doses supplied, patients treated and serious adverse events.
European Union: compassionate use and named-patient supply
EU compassionate use programmes are run by each Member State under its own rules. The medicine must be in clinical trials or under review for a marketing authorisation, and the disease must be life-threatening, long-lasting or seriously debilitating, with no satisfactory authorised treatment. Under Article 83, a national authority can ask the EMA's human medicines committee (CHMP) for an opinion on how a medicine should be used. These opinions are recommendations, not binding law.
Named-patient supply under Article 5(1) is the more common route for single patients. A Member State may exempt a medicine supplied to meet a genuine, unsolicited order from a healthcare professional for an individual patient under that professional's direct responsibility. The EMA does not need to be told. Each country then sets its own permit, import and reporting rules.
EU medicines law is being reformed. Political agreement on a new directive and regulation was reached in December 2025, and formal adoption was still being completed in autumn 2026. Until the new rules apply, expected from around 2028, the current articles remain the legal basis.
United Kingdom: EAMS and unlicensed specials
The Early Access to Medicines Scheme (EAMS) opened in April 2014 and now sits in regulation 167C of the Human Medicines Regulations 2012. It has two steps. The MHRA first grants a Promising Innovative Medicine (PIM) designation, then a scientific opinion that supports prescribing before a licence. A scientific opinion lasts one year and can be renewed. The company supplies the medicine and must run a risk management plan, with serious adverse reactions reported to the MHRA within 15 days.
Outside EAMS, UK patients receive unlicensed medicines as specials under regulation 167, which allows supply to meet a special clinical need when no licensed medicine is available. An importer must notify the MHRA of each import with evidence of clinical need, and must not import if the MHRA objects within 28 days. Our guide to unlicensed medicines in the UK explains the licences and paperwork in detail.
France and other countries
France reformed its system on 1 July 2021. The AAP (autorisation d'accès précoce, early access authorisation) is granted by the HAS (the national health technology body) at a company's request, with a binding opinion from the ANSM (the medicines regulator) if the medicine is not yet authorised. It covers a group of patients under a treatment and data collection protocol. The AAC (autorisation d'accès compassionnel) is granted by the ANSM to a doctor for a named patient, usually for a medicine not being developed for that use, and lasts one year, renewable. These replaced the older ATU schemes.
Elsewhere, Health Canada runs the Special Access Program, where a practitioner requests a drug for a patient with a serious or life-threatening condition, and the manufacturer decides whether to supply and on what terms. Australia's TGA runs the Special Access Scheme (Category A and C notifications and Category B applications) and the Authorised Prescriber scheme. Our global guide to named-patient import programmes covers many more countries.
| Country or region | Main route | Who asks | Who authorises |
|---|---|---|---|
| United States | Expanded access (individual, intermediate-size, treatment IND) | Doctor or sponsor | FDA, plus IRB |
| United States | Right to Try Act 2018 | Doctor, for a consenting patient | Manufacturer decides; no FDA authorisation of each use |
| European Union | Compassionate use programme (Art. 83) | Usually the manufacturer | National authority; CHMP opinion optional |
| European Union | Named-patient supply (Art. 5(1)) | Doctor | National rules in each Member State |
| United Kingdom | EAMS; unlicensed specials and imports | Company (EAMS); prescriber (specials) | MHRA |
| France | AAP (group); AAC (named patient) | Company (AAP); doctor (AAC) | HAS with ANSM (AAP); ANSM (AAC) |
| Canada | Special Access Program | Practitioner | Health Canada; manufacturer decides to supply |
Who pays
There is no single answer. In the US, a sponsor needs the FDA's prior written permission to charge for expanded access and may recover only direct costs, plus certain administrative costs for intermediate-size and treatment programmes. Right to Try supply is limited to direct costs. The UK EAMS was designed to be paid for by companies rather than the NHS. In Canada the manufacturer sets the payment terms. In many other countries the hospital or health insurer pays for a named-patient import at a price agreed with the supplier, and some companies give pre-approval medicines free of charge. Always confirm who pays before an order is placed.
How manufacturers run programmes, and where distributors fit
A manufacturer usually sets eligibility rules, a review process for each request and a supply limit. Many appoint a specialist supplier to run a managed access program on their behalf. That partner checks each request against the protocol, obtains import permits, ships under the right temperature conditions, keeps records and passes adverse event reports back to the manufacturer. The same partners often source comparator medicines for clinical trials, because the skills overlap: licensed supply chains, batch traceability and careful documentation.
For medicines already approved in another country, a licensed importer can often obtain stock through normal licensed channels for a named patient. For investigational medicines that are not approved anywhere, supply comes only from the manufacturer, through its programme.
The documents you will need
- Prescriber request: a signed letter or form naming the patient (or a patient code), diagnosis, dose, quantity and why no approved alternative is suitable.
- Import licence or permit: the national authorisation for the importer, and often a permit for each consignment.
- Informed consent: written consent showing the patient understands the medicine is unapproved, or not approved locally.
- Supplier documents: certificate of analysis, proof of the supply chain and storage records.
- Pharmacovigilance reporting: an agreed route for reporting adverse events to the manufacturer and, where required, the regulator.
A simple worked example
A hospital oncologist wants a medicine approved in the US and the EU but not yet registered in her country. She writes a named-patient request explaining that the local options have failed. The hospital pharmacy sends it to a licensed importer, which applies to the national regulator for an import permit with the prescription, the product details and the supplier's documents. While the permit is processed, the importer confirms the price, the cold-chain route and who pays. The permit arrives, the stock is shipped with a certificate of analysis and temperature records, and the pharmacy records receipt against the patient. Any side effect is reported back through the agreed channel. Total time depends on the regulator, but planning the paperwork in parallel saves weeks.
What hospitals and distributors should check
- Is the patient eligible under the local route, and is a clinical trial an option first?
- Is the medicine approved anywhere? If not, is there a manufacturer programme, and who runs it?
- Does the importer hold the right licence, and is a per-shipment permit needed?
- Can the supplier show an unbroken licensed supply chain back to the manufacturer?
- Who pays, and is the price fixed for repeat supplies?
- Are informed consent, adverse event reporting and record retention agreed in writing?
- How will continued supply work if the medicine is approved, or the programme closes?
Patients should never try to import unapproved medicines themselves. Supply should always go through a doctor, a licensed importer and the permits the law requires.
How PharmaTradz can help
PharmaTradz works with licensed buyers, including hospital pharmacies, importers and clinical research teams, to source named-patient and unlicensed medicines through licensed channels. See our named-patient import service for India, or request an import with the prescriber's details and we will check the route and documents with you. For other needs, send us an RFQ, and look up any unfamiliar term in our A-Z of pharma abbreviations.
Frequently Asked Questions(FAQs)
What is an early access program?
A legal route that lets a doctor treat a patient with a medicine that is not yet approved, or not yet available, in that country. It is meant for serious conditions where no satisfactory approved treatment exists and the patient cannot join a clinical trial.
What is a managed access program?
An industry term for a structured programme that a manufacturer runs, often through a specialist supplier, to handle requests for a pre-approval medicine under one protocol across many countries. In the UK, NICE also uses "managed access" for NHS funding arrangements while more evidence is collected, which is a different thing.
What is the difference between expanded access and compassionate use?
Expanded access is the US legal term under 21 CFR 312 Subpart I. Compassionate use is the EU legal term for programmes for groups of patients under Article 83 of Regulation 726/2004, though many people use it loosely for any pre-approval supply.
What is named-patient supply?
Supply of an unapproved or unlicensed medicine ordered by a doctor for one identified patient under the doctor's own responsibility. In the EU it rests on Article 5(1) of Directive 2001/83/EC, with permits and import rules set by each country.
Who pays for early access medicines?
It depends on the route and the country. Some manufacturers supply free of charge, US rules allow only limited cost recovery with FDA permission, and for many named-patient imports the hospital or insurer pays an agreed price.
