| Verified against primary record | |
| European legal basis | A member state may make an unauthorised product available to a group of patients; programmes are set up and run nationally and committee opinions are not binding[1] |
|---|---|
| Group, not individual | The European route targets a group of patients, defined as more than one, and expressly does not cover named-patient supply, which remains national[2] |
| United States conditions | Serious or life-threatening disease, no satisfactory alternative, trial enrolment not possible, benefit justifies risk, and no interference with trials[3] |
| Independently reported | |
| Cross-border supply | The European guideline states that conditions for distribution do not cover the strategy for supplying the product across member states[2] |
| Bands apply only to the rows beneath them. No source was located establishing that a patient may travel to another country to obtain compassionate-use access; access is tied to the programme’s jurisdiction. | |
Compassionate use, also called expanded access, is the supply of a medicine that has not been authorised to patients with a serious condition who cannot take part in a trial. It is a domestic arrangement everywhere it exists, which is why it is a poor fit for the kind of travel it is sometimes used to justify.
The European route
The European framework rests on a single article of a 2004 regulation. The medicines agency issues recommendations but these do not create a legal framework; member states coordinate and run the programmes and set their own rules and procedures; programmes are established only for life-threatening, long-lasting or seriously debilitating illnesses that no authorised medicine can treat satisfactorily; patients must be unable to enter clinical trials and trials should be considered first; the medicine must be in clinical trials or under marketing-authorisation review; and committee opinions are not legally binding, although member states should take note of them.[1]
The implementing guideline draws a distinction that is routinely missed. The route allows a member state to make an unauthorised centrally eligible product available to patients with a chronically or seriously debilitating or life-threatening disease who cannot be treated satisfactorily by an authorised product, and the programme must target a group of patients, defined in the guideline as more than one individual patient. It expressly does not cover supply on a named-patient basis, which remains governed by national rules, and it is distinct from off-label use of authorised products. Implementation is each member state’s competence; the competent authority notifies the agency and may request an opinion; opinions are not binding but must be taken into account, apply to later notifications from other member states concerning the same programme, and are entered in a public register.[2]
On the question this entry exists to answer, the guideline is explicit in the negative: while it aims to increase transparency between member states about treatment availability and to support a common approach, the conditions for distribution do not cover the strategy for supplying the product across member states, such as quantities or which member states are chosen.[2] Access therefore follows the programme’s jurisdiction rather than the patient’s travel.
The United States route
Five conditions apply: the patient has a serious or immediately life-threatening disease or condition; no comparable or satisfactory alternative therapy exists to diagnose, monitor or treat it; enrolment in a clinical trial is not possible; the possible benefit justifies the possible risks; and providing the product will not interfere with the investigational trials. The patient-facing material states that the product has not been approved or cleared, that the agency has not found it safe and effective for that use, that its effectiveness is uncertain and that it may cause unexpected serious side effects; that all costs of the product and associated medical services must be considered and third-party payers may not cover them; and that meeting the criteria does not guarantee access, since the company, the physician or the review board may decline.[3]
The regulation states the same general criteria in binding form and divides access into three categories by population: individual patients including emergency use, intermediate-size patient populations, and treatment protocols.[4] Charging follows the rules set out in the entry on clinical trials: prior written authorisation, direct cost recovery only, with monitoring and reporting costs recoverable for the larger categories, and an accountant’s review of the calculation; authorisation lasts a year unless a shorter period is set, and the sponsor must give assurance that charging will not interfere with marketing approval.[5]
That last point is the one to carry. Compassionate use in American law is cost-recovery only, pre-authorised and audited. An offering abroad described as compassionate use and priced at a market rate is not operating under this framework, whatever it is called.
The British scheme
The United Kingdom operates an early access scheme giving patients with life-threatening or seriously debilitating conditions access to medicines that lack a marketing authorisation where there is a clear unmet medical need. It has two evaluation steps, a promising innovative medicine designation and then a scientific opinion on the benefit and risk balance based on the data available at submission, valid for up to a year and renewable; the scheme is voluntary; the scientific opinion does not replace the normal licensing procedures; and the opinion holder, usually a pharmaceutical company, makes the medicine available.[6] The published overview does not state who pays for the medicine or whether patients may be charged, and neither is asserted here.
What a travelling patient actually faces
No source was located stating that a patient may travel to another country to obtain compassionate-use access. For a patient seeking to bring a medicine home rather than travel to it, the operative question in the United States is not compassionate use but personal importation, and that is enforcement discretion rather than a right. Current law generally does not permit individuals to import or reimport prescription drugs for their own use, and instead directs the responsible secretary to use discretion case by case to permit personal importation clearly for personal use where that use does not appear to pose an unreasonable risk. Under the agency’s policy an individual is generally allowed to bring in a 90-day supply of an unapproved drug for personal use where no effective treatment is available domestically, the drug treats a serious medical condition, and the drug is not commercialised to residents; the policy is not intended as a route to lower-priced drugs.[7]
No regulator statement was found on charging patients for compassionate use in a cross-border setting. The charging rules that exist are domestic, and the European guideline’s fee provisions concern a company’s fee for an opinion rather than any charge to a patient.
See also
- Clinical Trials Abroad, the route patients must be unable to use before this one opens
- Experimental Treatment, and the difference between an unauthorised product and an illegally marketed one
- Off-Label Treatment, which concerns authorised products used outside their terms
- Medical Travel for Unavailable Treatments, the situation that prompts these requests
References
- European Medicines Agency. Compassionate use. Page last modified 26 October 2020. Verified against primary record: the page was opened and the legal basis, the national competence, the eligibility conditions and the non-binding status of committee opinions read. Retrieved 10 October 2026.
- European Medicines Agency, Committee for Medicinal Products for Human Use. Guideline on compassionate use of medicinal products pursuant to Article 83 of Regulation (EC) No 726/2004. EMEA/27170/2006, adopted 19 July 2007. Verified against primary record: the guideline was opened and the group requirement, the exclusion of named-patient supply, the implementation arrangements and the statement on cross-member-state supply strategy read. Retrieved 10 October 2026.
- United States Food and Drug Administration. Patient fact sheet: expanded access. No date on the fetched content. Verified against primary record: the fact sheet was opened and the five conditions and the statements on uncertainty, cost and refusal read. Retrieved 10 October 2026.
- United States Code of Federal Regulations, 21 CFR 312.305, requirements for all expanded access uses, and 21 CFR part 312 subpart I. Read at the Legal Information Institute reproduction. Verified against primary record only to the extent that this reproduction was opened and the three general criteria and the subpart’s category structure read; the text should be confirmed on the official register before being relied on. Retrieved 10 October 2026.
- United States Code of Federal Regulations, 21 CFR 312.8, charging for investigational drugs. Read at the Legal Information Institute reproduction. Verified against primary record only to that extent; see the caution in the previous reference. Retrieved 10 October 2026.
- Medicines and Healthcare products Regulatory Agency. Early Access to Medicines Scheme: overview. First published 10 September 2024, last updated 2 March 2026. Verified against primary record: the overview was opened and the two evaluation steps, the validity period and the statement that the opinion does not replace licensing read. The page does not state who pays for the medicine. Retrieved 10 October 2026.
- Sarata AK. Prescription Drug Importation. Congressional Research Service In Focus IF11056, version 6, updated 8 December 2022. Verified against primary record: the report was opened and the general prohibition, the discretionary personal-importation policy and its conditions read. Read via a third-party repository, since these reports are not hosted publicly by the service itself. Retrieved 10 October 2026.
Sourcing note: two of the United States regulations were read in a reproduction of the regulatory text rather than on the official register, which is recorded in their references. A companion 1998 agency guidance on charging for investigational products was withdrawn in October 2023 and is not cited. The finding that no instrument supports travelling to another country for compassionate-use access is a negative result from searching the European and United States frameworks and the British scheme, and is stated as such.