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[Clinical trials with advanced therapy medicinal products]
M Schüssler-Lenz1, C K Schneider
1Abteilung Medizinische Biotechnologie, Paul-Ehrlich-Institut, 63225, Langen. schmr@pei.de
Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz
|December 17, 2009
Summary
Advanced therapies require specific quality, safety, and efficacy data, with evolving European regulations. New guidelines address challenges like traceability in clinical trials for these innovative treatments.
Area of Science:
- Biotechnology
- Regenerative Medicine
- Clinical Research
Context:
- Advanced therapies, including tissue-engineered products, faced inconsistent European regulation.
- Lack of clinical study data for some advanced therapies, like autologous chondrocyte products, highlighted regulatory gaps.
- The Committee for Advanced Therapies (CAT) was established by the European Medicines Agency (EMEA) to address scientific and regulatory complexities.
Purpose:
- To outline the basic assessment principles for advanced therapies as biotechnological medicinal products.
- To highlight the specific data requirements for quality, safety, and efficacy of advanced therapies.
- To discuss the evolving regulatory landscape in Europe and the need for standardized guidelines, such as Good Clinical Practice for advanced therapies.
Summary:
- Assessment of advanced therapies follows general principles for biotechnological products but requires highly specific quality, safety, and efficacy data.
- Regulatory inconsistencies across Europe previously affected advanced therapies, with some products lacking clinical study data.
- New guidelines, including draft Good Clinical Practice for clinical trials with advanced therapies, introduce specific requirements like traceability.
Impact:
- Improved regulatory harmonization for advanced therapies across Europe.
- Enhanced clinical trial methodologies and data collection for novel treatments.
- Facilitation of the development and approval of safe and effective advanced therapies through standardized regulatory frameworks.
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