Regulatory Aspects of Advanced Therapy Medicinal Products in the EU
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Advanced therapy medicinal products are human cells and tissues or products with a genetic mode of action; they generate huge expectations but are also associated to new significant threats including tumorigenicity, cell (de)differentiation, and patient integration.
The ATMP Regulation 1394/2007 aims to facilitate the patient access to these products and to foster the competitiveness of European pharmaceutical companies in the field, while guaranteeing the highest level of health protection for patients. ATMPs are pharmaceuticals with high complexity linked to their development, manufacturing, or administration process.
The Regulation addresses the following points:
- Definition: ATMPs are gene therapy, somatic cell therapy, or tissue-engineered medicinal products.
- For Marketing Authorizations, the centralized procedure is mandatory. Because of the complexity of ATMPs, a Committee for Advanced Therapies (CAT) has been created. The CAT’s main responsibilities are:
- The evaluation of MA applications by providing opinions to the Committee for Medicinal Products for Human Use (CHMP); the CHMP may adopt or refuse the CAT opinion.
- The optional scientific certification of quality and non-clinical data of a proposed ATMP-compound in development.
- The optional scientific recommendation on ATMP-classification, prior to their clinical development.
The Tissues and Cells Directive (2004/23/EC) applies to donation, procurement and testing of human tissues and cells.
The Regulation defines the pre- and post-authorization requirements: GMP and GCP standards, product follow-up on efficacy and safety, risk management plan, and traceability.
The Regulation also provides incentives for applicants by offering scientific advice at various development steps at substantially reduced fees, mainly to small- and medium-sized enterprises and hospitals.
Two exemptions are foreseen to the ATMP Regulation:
- Products, which were legally on the Community market on December 30, 2008 (when the Regulation became applicable), should be compliant to the Regulation requirements no later than December 30, 2012. These products will be withdrawn from the market afterward if no centralized MA application has been submitted and granted.
- Exemptions to the Regulation are also defined under the hospital exemption rule:
The first exemption relates to a transition period allowing existing products to evolve toward ATMP-compliance. The hospital exemption allows hospitals and medical practitioners to provide ATMP-classified products to patients, e.g., in case of high unmet medical need because there is no authorized ATMP alternative available. Examples are some tumor vaccines, made by hospitals for treating cancer patients having no treatment alternatives.
The hospital exemption is limited to non-routine products, custom-made for individual patients.
ATMPs with different development tracks and quality control procedures may co-exist
on the Community market satisfying different standards on efficacy, safety and quality; for hospital exemptions the Regulation requires traceability, quality, and pharmaco-vigilance standards to be “equivalent” to standards for centrally authorized products. Manufacture of ATMPs under hospital exemption has to be authorized by the appropriate member state to ensure appropriate quality. There are no efficacy criteria mentioned.
Why should you Attend:
Advanced-therapy medicinal products (ATMPs) are medicines for human use that are based on gene therapy, somatic-cell therapy or tissue engineering. They offer groundbreaking new opportunities for the treatment of disease and injury.
As this is a new area, early contacts with the regulatory authorities are particularly important. There are a number of procedures available during the development of these products. These procedures are explained in this webinar.
Companies can apply to the European Medicines Agency to determine whether a medicine they are developing is an advanced-therapy medicinal product (ATMP). The procedure allows them to receive certification that a medicine, based on genes, cells or tissues, meets the scientific criteria that define ATMPs.
The European Medicines Agency's Committee for Advanced Therapies (CAT) provides a certification procedure for advanced-therapy medicinal products (ATMPs) under development by micro, small and medium-sized enterprises (SMEs). This is an opportunity for SMEs to get an assessment of the data they have generated and check that they are on the right track for successful development.
The certification procedure involves the scientific evaluation of quality data and, when available, non-clinical data that SMEs have generated at any stage of the ATMP development process. It aims to identify any potential issues early on, so that these can be addressed prior to the submission of a marketing authorization application.
After the assessment, the CAT may recommend issuing a certification confirming the extent to which the available data comply with the standards that apply for evaluating a marketing authorization application. Following the CAT recommendation, the Agency issues a certification.
Areas Covered in the Session:
Who Will Benefit:
- Committee for Advanced Therapies (CAT)
- Stem cells
- Hospital exemption
- Relevant guidelines
- Scientific recommendation on classification
- Certification procedure
- Senior management
- Project Managers
- Medical writers
- CRAs and CRCs
- QA / Compliance personnel
- Clinical Research Scientists
- QA / QC Auditors and Staff