Recent media reports highlight the need for public authorities at all levels to enforce their legal responsibilities to ensure that patients only have access to those treatments that comply with relevant quality standards and for which there is appropriate traceability of materials, treatment protocols and patient follow-up measures.
The European Medicines Agency stresses that the protection of patients is at the core of those rules. In addition to applying the same safety and efficacy rules as for all medicinal products, the quality and manufacturing of these products is set out in 'Good Manufacturing Practices' (GMP) requirements. These are globally-recognised standards for quality assurance in the production and control of medicines.
Security and control of medicines derived from stem cell manipulation is tightly controlled by the EU advanced therapy legislation. Cell therapies are defined as medicinal products when there is more than minimal manipulation of any cell type destined for clinical application or where the intended use of the cells is different to their normal function in the body. Any use of such cell-based medicines is subject to authorisation and controls, including their manufacture.
Permitting manufacturers to avoid compliance with quality standards, for example by inappropriate re-classification of the treatment beyond the mandate of competent authorities for control of medicines, could risk exposing patients to cross-contamination and inadequate characterisation of the cell preparations, resulting in short and long-term risks for individual patients.
Similar concerns were previously raised by the Agency’s Committee for Advanced Therapies (CAT) in a public statement of 16 April 2010 and in a paper published in The Lancet of 14 August 2010 on the use of unregulated stem-cell based medicinal products.