Unlicensed medicines: the routes in the UK and Europe (specials, imports, named-patient and compassionate use)

What “unlicensed” means

A medicine is unlicensed in a country when no marketing authorisation covers it there: it may be authorised elsewhere, in development, discontinued, or made to order. Using a licensed medicine outside its licensed indications is a different thing (off-label use, a prescribing matter) and is not covered here. The law’s starting point is that an unlicensed medicine may not be sold; the routes below are the exceptions, and they are where a surprising amount of trade happens.

The EU framework

Named patient (Article 5(1)). A member state may exclude from the authorisation requirement a medicine supplied in response to a bona fide unsolicited order, formulated to the specifications of an authorised healthcare professional, for an individual patient under that professional’s direct responsibility. Every member state has implemented a version; the mechanics (who may import, what paperwork, whether a pharmacist or the agency decides) are national.

Emergency exemptions (Article 5(2)). A member state may temporarily authorise distribution of an unauthorised medicine in response to a suspected or confirmed spread of pathogens, toxins or chemical or nuclear agents.

Compassionate use (Regulation 726/2004, Article 83). For groups of patients with a chronically or seriously debilitating or life-threatening disease and no satisfactory authorised alternative, a member state may make available a medicine that is under centralised assessment or in clinical trials. The EMA’s committee gives opinions that member states may adopt; the programmes themselves are national and the company supplies the product, usually free.

The UK

Specials. The Human Medicines Regulations exempt from the licensing requirement a medicine supplied to meet the special needs of an individual patient on the order of a doctor, dentist or other authorised prescriber, where no licensed equivalent is suitable. Such products are either manufactured under a manufacturer’s “specials” licence, in the UK, to the prescriber’s specification (a liquid formulation of a tablet, a strength that does not exist), or imported.

Imports. An importer holding a wholesale dealer’s licence (for products from the approved list of countries, which includes the EEA) or a manufacturer’s specials licence (for others) must notify the MHRA of its intention to import a specific unlicensed product before each importation. The MHRA may object within a set period (28 days in the standing guidance) and may ask for justification; the quantity per notification is limited (the guidance’s usual ceiling is 25 units or courses) and larger quantities need a case. The MHRA does not allow the route for reasons of cost or convenience where a licensed UK equivalent exists, and it keeps a list of products it has refused.

Early Access to Medicines Scheme (EAMS). A separate route for promising medicines before licensing, with a scientific opinion from the MHRA and supply by the company.

Price. Specials are reimbursed in England under the Drug Tariff’s specials part at listed prices for common lines and at invoice price otherwise; imported unlicensed products are reimbursed on the invoice. The route has a history of high prices and of NHS efforts to contain them, which is why the specials tariff exists.

How the large member states run it

France. The 2021 reform replaced the old temporary authorisations with accès précoce (early access to medicines expected to be innovative, with reimbursement and a later price settlement) and accès compassionnel (individual or cohort access outside trials). Imports for individual patients run through the agency’s authorisation.

Germany. Pharmacies may import an unauthorised medicine for an individual patient under section 73(3) of the Medicines Act where no equivalent is authorised in Germany and the prescriber requests it; cohort access runs through hardship programmes notified to the authority.

Italy. The agency’s Law 648/96 list reimburses specified unlicensed or off-label uses at national level; individual imports for named patients are authorised by the agency, and shortages are commonly bridged by authorised imports of foreign equivalents.

Spain. Royal Decree 1015/2009 covers compassionate use, off-label use and the import of unauthorised medicines through the agency’s platform, with hospital pharmacies as the applicants.

The Netherlands. Pharmacists may supply an unregistered medicine on a prescriber’s declaration where no registered alternative exists, with the inspectorate informed, and compassionate-use programmes run with the medicines board.

What it means, by role

For a parallel trader or importer. Unlicensed import is a licensed activity with a different licence: a wholesale dealer’s or specials licence plus notification, not a parallel import licence. It is the route by which discontinued products, products in shortage and products launched elsewhere reach UK patients, and the paperwork and quantity limits are the business model. The MHRA’s refusal list and the approved-country list are the first checks.

For a launch planner or licensor. Early-access and compassionate routes are the pre-launch channel: they build clinical experience, and in France and Italy they carry reimbursement. They also reveal demand to competitors and can set a reference for the later price.

For a buyer. Specials and imports are the expensive end of the supply chain; a product that has moved from unlicensed import to a parallel import licence or a full licence usually drops in price, which the licence registers show.

MPA Search records the licensed position: product licences, parallel import licences and the European authorisations behind them. Unlicensed routes sit outside those registers, which is precisely why knowing where the licensed position ends is the first step.