- Authorisation of ATMP
- Classification as ATMP
- Modular Authorisation
- Submission Paths and Costs
- Follow-up Authorisation
- Questions and Answers
Authorisation of ATMP
Authorisation of ATMP
Non-routinely produced ATMPs in accordance withSection 4b AMG (Arzneimittelgesetz [German Medicines Act])
In Germany, the Paul-Ehrlich-Institut (PEI) is responsible for the authorisation of non-routinely manufactured advanced therapy medicinal products (ATMP) in accordance with 4b (3) AMG. In order to facilitate the submission process for applicants, we provide customised information through forms, process descriptions and FAQ.
Updated: 26.11.2019
Classification as ATMP
Classification as ATMP
Decision tree for the classification of a drug as ATMP
Source: PEI
Updated: 21.11.2019
Modular Authorisation
Modular Authorisation
Modular application for authorisation
The authorisation forms are based on the modular CTD format (common technical document), which was designed for classical drug approval. However, they are much shorter and geared to the legal requirements for approval in accordance with Section 4b AMG.
In order to simplify the classification of a medicine as an advanced therapy medicinal product (ATMP) and the necessary handling of the relevant legislation, modules 1 and 3-5 were preceded by module 0. It is intended to enable applicants to classify their own medicinal product as an ATMP. In addition, the overview graphic presented as a decision tree serves as a guide for orientation.
By way of derogation, applications based on other application templates in CTD format - e.g. templates for the application for clinical trial, for authorisation pursuant to Section 21a AMG for stem cell preparations or for authorisation are accepted.
Updated: 21.11.2019
Submission Paths and Costs
Submission Paths and Costs
- The submission of documents is usually done electronically.
- In some cases defined by law, the submission of signed documents is still required. Corresponding documents can be submitted via De-Mail (pei@pei.de-mail.de) or by post to the PEI:
Paul-Ehrlich-Institut
Federal Institute for Vaccines and Biomedicines
Paul-Ehrlich-Str. 51-59
63225 Langen
Germany
Costs
The fees for the authorisation of ATMP in accordance with 4b AMG are based on the updated cost ordinance for official acts of the PEI (PEI-KostVO). This constitutes a framework fee, which can be set at € 4,250 to € 17,000, depending on the scope of the procedure.
Updated: 21.11.2019
Follow-up Authorisation
Follow-up Authorisation
Updated: 21.11.2019
Questions and Answers
Questions and Answers
Application procedure pursuant to Section 4b (3) AMG - hospital exception
In which form can the application be submitted?
The application may be submitted electronically (as Adobe Acrobat PDF files together with the corresponding Microsoft Word files) together with the required documentation, as well as on paper in one of the following ways:
- by post in one copy to:
Paul-Ehrlich- Institut
Federal Institute for Vaccines and Biomedicines
Paul-Ehrlich-Str. 51-59
63225 Langen
GERMANY - or by email to: amg-ev@pei.de
- or by fax to: +49 6103 77 1234
It should be noted, however, that pages with signatures must be submitted in the original.
The Applicant can not provide information on all areas requested in Modules 1-5 or these details are not available or not available to the extent requested. Can he still apply?
Yes, you can still apply. Module 1, which is used for medicinesegistration, is a template and modules 3-5 are to be considered as guides to the application. If information on certain areas is not available at the time of initial filing, this will not automatically lead to the rejection of the authorisation. Nevertheless, all information available to the applicant should be indicated at the appropriate place in the respective modules. As part of the formal review, the PEI will ask for all necessary documents.
Who can be an applicant?
The Applicant may be a person or legal entity who intends to give the medicine to others. This can be e.g. a company, a university research institution or even a doctor.
Does the system for monitoring (pharmacovigilance system) have to be fully implemented if the applicant treats only a small number of patients a year?
In addition to the capture of the data on the medicine, module 1 queries are made on systematic monitoring to ensure the safety of the ATMP. The points to be specified under point 1.5.1 should be considered as orientation by the applicant. Under certain circumstances, when only a few patients are treated, a database for the detection of suspected adverse reactions can be waived. The existing system for the registration and archiving of suspected side effects should then be described in detail.
The applicant does not yet have a permit for authorised extraction of fabric and manufacture. Can he still apply?
Yes, an application can first be submitted without authorisation for authorised collection and production. However, these will subsequently be requested as part of the formal application check. Therefore, the applicant should have already initiated an appropriate procedure for obtaining the permits from the competent Land authority. However, the authorisations should be available at the latest when the application is approved.
Does the applicant have to be registered in the commercial register?
The entry in the commercial register is not a prerequisite for the application. If there is an entry, a copy should be submitted (see module 1, point 1.2.1).
Is the opinion from the Ethics Committee required for the application pursuant to Section 4b (3) AMG?
No, an opinion from the Ethics Committee is not needed.
Does the applicant have to provide his own data on pharmacodynamics, pharmacokinetics and toxicology (module 4, non-hospital)?
In principle, the applicant should base his non-clinical information mainly on his own data. In addition, he may also refer to literature data. In order for the latter to contribute to the non-clinical evaluation of the medicinal product, it must be specific to the active substance and mechanism of action of the ATMP to be authorised.
Does the applicant have to provide his own clinical data, e.g. submit clinical observations from a clinical trial or therapeutic trials?
In principle, the applicant may provide all information from processed medical cognitive material, such as studies by the manufacturer, data from publications or subsequent assessments of clinical results of the produced ATMP. If possible, these should be specific to the ATMP and its mechanism of action, so that these literature data contribute to the evaluation of the medicine.
Finally, the non-clinical and clinical data provided by the applicant must allow a risk-benefit assessment.
Where can the applicant find scientific guidance on ATMP?
Useful scientific guidelines on quality, non-hospital and clinic of ATMP can be found at the following link of the European Medicines Agency EMA:
http://www.emea.europa.eu/htms/human/advanced_therapies/guidelines.htm
Pursuant to Section 4b (3) AMG, the holder of the Section 4b approval is required to report, at certain intervals, to the Paul-Ehrlich-Institut on the extent of manufacture and findings gained. Is there a general timeframe for these reports, or is this set for each individual application?
A general time frame is not planned. In individual cases, this will result from the evaluation of the risk, the expected amount of data and the resulting insights.
How much does a license according to Section 4b (3) AMG cost and when is this fee to be paid?
The costs for a license pursuant to Section 4b (3) AMG are based on the Statutory Cost Regulation of the PEI. This is a framework fee, which can be set at between € 4,250 and € 17,000, depending on the extent of the application. The fee has to be paid upon the decision, i.e. at the end of the procedure.
Your question about the approval of ATMP according to Section 4b (3) AMG is not included in the Q&A list, whom can you contact the PEI?
The PEI offers all applicants the opportunity for a personal consultation. Here, regulatory, procedural, as well as scientific issues can be addressed. Inquiries for meetings and the procedure can be made by email to innovation@pei.de or by phone on +49 (0)6103-77-1012. A fee will apply for the consultation.
Updated: 21.11.2019
