What is the difference between a Named Patient Programme and Compassionate Use?

Named Patient Programmes and Compassionate Use are both regulated pathways for providing access to unlicensed medicines, but they differ in scope and regulatory framework. A Named Patient Programme (NPP) provides access to an unlicensed medicine for a specific, individual patient at the request of a treating physician. It is typically used for medicines already licensed in another country but not yet available in the patient's market. A Compassionate Use Programme (CUP) is designed to provide access to a group (cohort) of patients with a serious, chronic, or life-threatening condition that cannot be adequately treated with any authorised medicine. Under EU regulation, a CUP typically requires that a marketing authorisation application has been submitted, or that supporting clinical trials are ongoing. The most appropriate pathway depends on the medicine, the patient population, the clinical circumstances, and the local regulatory framework.

Who initiates a Named Patient Programme?

A Named Patient Programme is initiated by a licensed treating physician on behalf of an individual patient, supported by medical justification and the patient's informed consent. However, three parties are typically involved in every NPP: The physician – identifies the medical need and submits the request. The manufacturer or sponsor – must agree to supply the medicine and provide product documentation. The local regulatory authority – may need to approve the import or issue a permit, depending on the country. In some jurisdictions, hospital pharmacies or national health services also play a coordinating role. COREX supports sponsors in managing the operational, documentation, and supply aspects across all involved parties.

What information is required to start a Named Patient Programme?

Requirements vary by country and product, but a typical Named Patient Programme submission includes: Physician's prescription and medical justification, explaining why the specific medicine is needed and why licensed alternatives are unsuitable. Patient information: diagnosis, treatment history, and dosing requirements (subject to data protection regulations). Product details: manufacturer, batch information, and country of origin. Patient informed consent, confirming the patient understands the unlicensed status of the medicine. Sponsor or manufacturer agreement, confirming willingness to supply the requested product. Import licence or regulatory notification – where required by the destination country's authority. COREX conducts a country-specific feasibility review at project intake, so sponsors know exactly which documentation is needed before shipment.

What type of medicinal product pack can be supplied under a Named Patient Programme?

The type of pack used in a Named Patient Programme depends on the medicine's regulatory status and the sponsor's supply strategy. Commercial packs are most commonly used, sourced from a country where the medicine is licensed and marketed. Additional import labelling or a supplementary information leaflet may be required to comply with the destination country's regulations. Investigational Medicinal Product (IMP) packs may also be used in specific cases – for example, when a medicine is still in clinical development and no commercial pack exists yet, or when a hybrid access framework is used. Their use depends on both the sponsor's supply approach and the local regulatory framework.

How do I choose between a Named Patient Programme and a Managed Access Programme?

The choice between a Named Patient Programme and a Managed Access Programme depends primarily on the scope and structure of the access you need to provide. Choose a Named Patient Programme (NPP) when you need to supply a medicine to specific, individual patients on a case-by-case basis – typically triggered by physician requests. NPP is ideal for low-volume, patient-led access. Choose a Managed Access Programme (MAP) when you want to provide structured, sponsor-led access to a medicine across multiple patients, countries, or sites – often in the pre-launch phase or where a coordinated global framework is required. Other factors include the regulatory status of the medicine, the anticipated patient volume, the target countries, and the sponsor's commercial and operational strategy. COREX operates across 45+ countries and can help identify the most appropriate pathway based on your specific programme goals.

COREX support services for rare diseases

How it works

Our regulatory team coordinates with manufacturers and health authorities to secure import permits, manage compliant storage, and arrange direct delivery to patients within approved timelines.

Challenge:

Individual patients with serious conditions need access to medicines not yet approved locally.

Solution

A Named Patient Programme enables doctors to request unauthorised medicines for individual patients when no approved alternative exists locally, the product is authorised elsewhere or in late-stage trials, the patient has a serious or rare condition, and the clinical need is urgent and well justified.

How COREX Supports NPP Access

Sourcing & Importation of Unlicensed Medicines

Regulatory Consultation & Country-Specific Compliance

Secure Cold Chain Storage & Distribution

Documentation Management & Product Traceability

Collaboration with Healthcare Professionals (HCPs) and Ministries of Health (MoH)

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Why NPP Access Matters

For patients facing life-threatening conditions with no other
treatment options, time is critical. NPPs provide a legal and ethical
pathway to receive potentially life-saving therapy — when
clinical trials are not accessible or marketing authorisation
is not yet granted.