Compassionate Use: how it works and where it is recognized

Last reviewed 2026-05-16 by Reserve Meds clinical and regulatory team.

What Compassionate Use is

Origin and authorising body. EMA guidance under Article 83 of Regulation (EC) No 726/2004 set the European framework; FDA expanded access under 21 CFR 312 Subpart I sets the US parallel; national pharmacy agencies elsewhere have adopted similar architectures under their own statutes. The authorising body for each individual case is the destination-country regulator at the national level (EMA at the EU level, MHRA in the UK, FDA in the US, EDE in the UAE, SFDA in Saudi Arabia, and the relevant pharmacy or medicines agency in each other jurisdiction), acting under that jurisdiction's pharmacy or medicines legislation.

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Legal basis. Article 83 of Regulation (EC) No 726/2004 defines compassionate use at the European level as the provision of a medicinal product to patients with a chronically or seriously debilitating or life-threatening disease who cannot be treated satisfactorily by an authorised medicinal product, where the product in question is the subject of an application for a marketing authorisation or is undergoing clinical trials. National implementations sit on top of the EMA framework. Outside the EU, parallel statutes (FDA expanded access under 21 CFR 312 Subpart I, UK Early Access to Medicines Scheme, Health Canada Special Access Programme, Australia's Special Access Scheme) carry the same architectural idea.

Compassionate Use is the pathway for supplying an unapproved medicine to a seriously ill patient outside a clinical trial when no satisfactory authorised alternative exists. Unlike a routine Named-Patient import (which is typically for a drug already approved somewhere and registered for that indication elsewhere), Compassionate Use frequently covers products that are still in development or that lack any registration in the destination country. The framework is gravity-loaded toward serious, life-threatening, or chronically debilitating disease: regulators apply tighter eligibility scrutiny than under a generic Named-Patient route, and most programmes require evidence that the medicine is in active development with a credible regulatory programme behind it.

Most jurisdictions distinguish between single-patient compassionate use (an individual prescription) and cohort compassionate use (a group of patients treated under a single protocol). Cohort programmes function more like an extended-access trial: the manufacturer submits a protocol, the regulator approves the cohort, and enrolling physicians use that approval as the legal basis for supply. Single-patient compassionate use is closer in operational shape to a Named-Patient import but with stricter eligibility documentation. In the United States, the FDA expanded access programme under 21 CFR 312 Subpart I distinguishes individual patient INDs, intermediate-size patient populations, and treatment INDs. In the EU and UK, compassionate use is governed at the Member-State level with EMA guidance setting the framework. In the Gulf, compassionate-use cases typically channel through the same EDE, SFDA, or equivalent permit infrastructure as named-patient cases, with the regulator applying tighter clinical-justification scrutiny.

This page consolidates Reserve Meds's operational view of the Compassionate Use framework across the ten destination countries where we currently coordinate cross-border supply. Earlier in our content build we maintained separate per-country sub-pages for each Compassionate Use-in-country combination; those sub-pages remain available for archival reference but are no longer separately indexed, and the operational intelligence they carried has been folded into the country-compatibility table below.

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Who can use Compassionate Use

Compassionate Use is used by treating physicians on behalf of patients with serious, life-threatening, or chronically debilitating disease for whom no satisfactory authorised treatment is available and who cannot enrol in an active clinical trial. Typical scenarios include late-line oncology where the patient has exhausted approved therapies, rare diseases with no approved options in the destination country, paediatric indications where the FDA or EMA approval has not extended to the patient's age band, and patients with contraindications to all locally approved alternatives. The treating physician must be licensed in the destination country, must have clinical responsibility, and must document a credible benefit-risk assessment specific to the patient.

In practical terms, a Reserve Meds case opens when the treating physician has identified the clinical need, has reached a decision to prescribe a specific medicine, and has determined that the locally available route is unsuitable or unavailable. We then layer the regulatory and supply-chain coordination on top of that clinical decision; we do not originate the prescribing question and we do not advise on the clinical choice. The decision sits with the physician throughout.

Drug categories typically covered under Compassionate Use

Investigational oncology agents undergoing late-phase development, post-approval extensions of FDA or EMA-approved oncology agents to indications not yet locally registered, rare-disease therapies including enzyme replacement and gene therapies in development, advanced therapy medicinal products in registration limbo, antiviral and antibacterial agents in development for resistant or rare pathogens, and paediatric formulations of adult-approved medicines.

The medicine itself must be source-country approved or in active regulatory development under a recognised reference authority (typically the US FDA, the European Medicines Agency, the UK MHRA, the Japan PMDA, or Health Canada). Most destination regulators will not authorise a Compassionate Use import for a medicine that lacks a recognised reference authorisation anywhere in the world, because the regulator's risk assessment leans on the source-country review as a substitute for its own.

Country compatibility

The table below summarises how the Compassionate Use framework is recognised across the ten destination countries where Reserve Meds currently coordinates cross-border supply. The "Accepts Compassionate Use dossiers" column reflects whether the destination regulator typically reviews and grants permits on the strength of a Compassionate Use clinical justification packet, or whether the patient must use a different parallel framework on the receiving side. Timelines reflect routine cases with a complete documentation set; first-import scenarios, paediatric weight-banded presentations, advanced therapy medicinal products, and large multi-cycle quantities can extend the regulator review by several weeks.

Notes. "Indirect" means the destination regulator does not directly accept Compassionate Use authorisation as the legal basis for import but does recognise a Compassionate Use authorisation as supporting precedent within its own named-patient or compassionate-use review. "Not applicable" means the framework is not a patient-access pathway in the cross-border sense and is shown here for completeness. Regulator names: NHRA (Bahrain), EDA (Egypt), CDSCO (India), JFDA (Jordan), KDFC (Kuwait), MoPH (Lebanon), MoH-DGPADC (Oman), MoPH-DPA (Qatar), SFDA (Saudi Arabia), and EDE (United Arab Emirates, federal from 29 December 2025).

Documentation required

A Compassionate Use application is typically assembled in coordination with the treating physician, the dispensing facility, and the cross-border supply partner. The documentation set below covers the routine case; specific destination countries may require additional items (most frequently a hospital ethics committee notification for paediatric or complex cases, and an insurer pre-authorisation letter where the patient intends to seek reimbursement).

• Detailed clinical justification documenting the patient's diagnosis, disease severity, prior therapy history, current clinical status, and absence of satisfactory authorised alternatives.
• Treating physician licence verification and qualifying specialty credentials.
• Where applicable, manufacturer letter confirming willingness to supply the medicine and disclosing the regulatory status of the product (active marketing authorisation application, ongoing clinical trial, post-approval extension).
• Patient informed consent specific to compassionate use, acknowledging that the medicine is not approved for the indication or in the jurisdiction.
• Treatment protocol summary including dosing schedule, duration, monitoring plan, and adverse event reporting plan.
• Where required: institutional ethics committee or hospital ethics review approval.
• Destination-country regulator application form (varies by jurisdiction).
• Chain-of-custody and cold-chain plan for the supply route.

Typical timeline

Routine single-patient compassionate-use applications typically clear regulator review in 7 to 30 business days. Cohort applications take significantly longer because the protocol is reviewed alongside the clinical-trial-grade documentation set. UAE EDE and Bahrain NHRA tend to process single-patient applications most quickly in the Gulf cluster. EU national regulators (BfArM, ANSM, AIFA) typically process within 2 to 4 weeks for single-patient cases. The FDA processes single-patient expanded-access INDs within 30 days, with emergency requests handled by phone within 24 hours. End-to-end, including documentation assembly and shipment, most cases complete within 4 to 8 weeks.

Costs and reimbursement

Cost dynamics in compassionate use differ from routine Named-Patient imports. Some manufacturers provide the medicine free of charge during compassionate-use enrolment as part of a managed-access programme; others charge a cost-recovery price; in the United States, the FDA permits cost recovery for an investigational drug under 21 CFR 312.8. Where the manufacturer charges, the patient pays the manufacturer's stated cost-recovery price plus international logistics, customs, dispensing-pharmacy handling, and coordinator fees. Insurer reimbursement for compassionate-use imports is uncommon because the medicine is typically not formulary-listed. Patients should plan on a cash-pay basis unless the manufacturer's programme covers drug cost.

Where Reserve Meds fits in

Reserve Meds is a US-based concierge coordinator for cross-border specialty medicine access. For Compassionate Use cases, we coordinate the US-side sourcing through a DSCSA-compliant specialty channel, prepare the documentation packet your physician submits, coordinate validated cold-chain logistics with continuous temperature logging into the destination country, and assign a single named coordinator through the case. We do not replace your treating physician, we do not replace the destination-country regulator, and we do not replace your dispensing pharmacy. We coordinate the supply side so the physician can focus on patient care.

For each of the ten destination countries above, we publish a country-specific page documenting the operational realities (which dispensing facilities handle named-patient cases, how the cold chain is verified at the local airport, how the dispensing pharmacy releases the medicine, and what the typical insurer interaction looks like). Those country pages, combined with this pathway page and the specific drug pages, give the prescribing physician and the patient a coherent picture of what to expect before any commitment is made.

Our concierge fee is itemised separately on every firm quote alongside the drug acquisition cost, international logistics, customs, and dispensing pharmacy handling. The patient sees the full breakdown before deciding to proceed; we do not bundle costs and we do not charge an intake deposit. If the case cannot proceed for regulatory or supply reasons, we say so before any payment is taken.

Next step

If your treating physician has identified a clinical need that fits the Compassionate Use framework and you are weighing the cross-border route, the next step is a short intake. We confirm eligibility within 24 to 48 hours and send a documentation kit to your physician.

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Related

• All access pathways
• UAE access landscape
• Saudi Arabia: SFDA named-patient pathway
• When conventional access fails
• How Reserve Meds works