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[Compassionate use mechanisms in Europe]
1Regulatory Affairs Department, Nihon Schering K.K.
Gan to Kagaku Ryoho. Cancer & Chemotherapy
|February 16, 2007
Summary
European compassionate use mechanisms allow access to unlicensed medicines for special patient groups. These systems, either nominative or cohort-based, differ nationally but aim to provide critical treatments when no alternatives exist.
Area of Science:
- Pharmaceutical policy
- Regulatory affairs
- Patient access to medicines
Context:
- European Union (EU) regulations establish general principles for compassionate use mechanisms.
- National systems for accessing unlicensed medicines vary across EU member states.
- Two primary categories exist: nominative (individual patient) and cohort (multiple patients).
Purpose:
- To outline the framework and variations of compassionate use mechanisms in Europe.
- To highlight the criteria for accessing unlicensed medicines for patients with serious or life-threatening conditions.
- To propose the need for a suitable compassionate use system in Japan.
Summary:
- Compassionate Use Mechanisms in Europe facilitate access to unlicensed medicines for patients with unmet medical needs.
- Nominative systems operate on a per-patient basis, with all 25 EU countries having some form of this.
- Cohort systems serve multiple patients in specific hospitals, existing in 10 EU countries, some of which also have nominative systems.
Impact:
- Informs the development of regulatory frameworks for patient access to novel therapies.
- Provides a comparative overview of EU approaches to unlicensed medicine access.
- Suggests improvements for Japan's current 'Private Import System' by advocating for a structured compassionate use program.
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