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:
Federal Institute for Vaccines and Biomedicines
- or by email to: firstname.lastname@example.org
- 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:
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 email@example.com or by phone on +49 (0)6103-77-1012. A fee will apply for the consultation.