Pedro Batista

Please find below the reply to your questions:

1. The substance preferred term must always be public. For substances that arent in the public domain, this name can be a company code. However, note that a company code will not be sufficient to support certain procedures at EMA (e.g. Orphan Designation, Paediatric Investigation Plan/Waiver, Marketing authorization, etc.) when the substance is part of an RPI. 
2. If a substance name is registered as a restricted synonym,  it will not be visible in any EMA public database (e.g. XEVMPD, EUTCT, IRIS, eAF, etc.).
3. Privacy settings can be adjusted after registration by submitting a change request from the requestor or by EMA if the substance name becomes available in the public domain (e.g. INN is issued by WHO or the product gets marketing authorisation). EMA may also change privacy settings, for examples when rINNs are published by WHO. 

1. Only sheet 1 needs to be filled with all substance names available

2. For an ATMP, the following information should be provided:

• Source (i.e. Autologous, Allogeneic or Xenogeneic);
• Tissue of origin (if applicable, e.g. bone-marrow derived, adipose-derived);
• Target cell type (e.g. mesenchymal stem cells, fibroblasts, chondrocytes);
• Any relevant modifications (e.g. genetically modified, ex vivo expanded).

3. The request can be submitted by any user in EMA Service Desk. No special role is required.

You can provide the requested information concatenated into one line. For example: "Allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded". 

Regarding the sheet 2, please disregard "nature of the active substance" in the Substance Request Form. That information will be filled directly in IRIS, when you are creating your RPI. That list is supposed to match the ATMP classification.

Please note that the following substance information should be provided for cell therapies:

• Source (i.e. Autologous, Allogeneic or Xenogeneic);
• Tissue of origin (if applicable, e.g. bone-marrow derived, adipose-derived);
• Target cell type (e.g. mesenchymal stem cells, fibroblasts, chondrocytes);
• Any relevant modifications (e.g. genetically modified, ex vivo expanded).

In addition, the substance company code should also be provided.