Compassionate Use Programmes for Medicinal Products in Germany (BfArM)

Legal Basis

Compassionate use in Germany is governed by § 41 of the German Medicinal Products Act (Arzneimittelgesetz – AMG), which transposes Article 83 of Regulation (EC) No 726/2004 into national law. It enables the use of unauthorised medicinal products or authorised products outside their approved indications for patients with life-threatening, seriously debilitating, or chronically serious illnesses when no satisfactory alternative therapy is available. Compassionate Use - BfArM

Eligibility Criteria

• Patient must suffer from a life-threatening, seriously debilitating, or chronically serious disease.
• No satisfactory method of diagnosis, prevention, or treatment is authorised in the EU, or the patient cannot be included in a clinical trial.
• Use is justified on the basis of available data on efficacy and safety.
• The medicinal product is either:
  • Not yet authorised in the EU, or
  • Authorised but used outside the approved indication (off-label use under compassionate programme).

Application and Notification Procedure

• The treating physician initiates the request and submits a notification to BfArM (not a formal authorisation).
• Required documents include:
  • Patient’s medical history and justification for use.
  • Available data on the medicinal product (e.g., clinical trial results, SmPC if authorised).
  • Ethics committee positive opinion (mandatory).
  • Informed consent from the patient.
• BfArM reviews the notification and may request additional information or impose conditions.
• No formal approval is issued; the programme proceeds if BfArM raises no objections.

Responsibilities of Involved Parties

• Treating Physician: Responsible for patient selection, treatment, monitoring, and reporting of adverse reactions.
• Sponsor/Manufacturer: Provides the medicinal product free of charge and supports with data and pharmacovigilance.
• BfArM: Oversees the programme, collects safety data, and may suspend or terminate it if risks outweigh benefits.
• Ethics Committee: Must approve the compassionate use plan.

Pharmacovigilance and Reporting

• All serious adverse reactions must be reported to BfArM and the manufacturer in accordance with pharmacovigilance rules.
• Annual summary reports on safety and efficacy may be required.
• The programme can be terminated at any time if new safety concerns arise.

Practical Considerations

Compassionate use is distinct from clinical trials and named-patient programmes. It is not intended for collecting systematic data but for individual patient benefit. BfArM provides detailed guidance, forms, and contact information for notifications.

This mechanism ensures controlled access to potentially life-saving treatments for patients with unmet medical needs in Germany while maintaining high safety standards. Compassionate Use - BfArM