Product Development

Most cell and gene therapy products are regulated as medicinal products in the EU and the US; as such their development must result in data that confirm quality, safety and efficacy.  
One of the key objectives of
 Product development, aside from demonstration of clinical efficacy, is to fully understand the characteristics of the product and the process used to make it. Characterisation is central to product development, this figure provides an overview of the scope of characterisation activities and how they interrelate.

Effective development processes provide all the tools necessary to implement changes, and these tools are a result of thorough characterisation.  Implementation of changes to the manufacture of medicinal products requires that the product before and after the change is comparable.  Comparability is a central concept in the regulation of medicinal products; this tool provides an overview of the various parameters that might be required for a comparability exercise. 


Regulatory Tools (last update)

Product development (Jul-11)

Importance of Characterisation (Aug-08) 

Comparability  (Oct-11)

Regulatory Considerations for Technology Transfer  (Feb-12)

Use of reference materials  (Nov-11)

Agency Advice (EU/US) (Nov-11)


Regulatory Guidance (last update)

There are a number of specific guidelines covering the development of ATMP's, however, many more general biological/biotech guidance is also indirectly relevant since they provide the general principle described in ICH documents are harmonised across ICH member countries


ICH guidelines (Jan-19) Minor update to include new guidelines

EMA guidelines (Jan-19) MAJOR UPDATE, now includes gene therapy

FDA guidelines (Jan-19) MAJOR UPDATE, now includes gene therapy

Guidelines related to Reference Materials  (Mar-15)

Comparability guidelines  (Oct-11)

Pharmacopoeial chapters with relevance to cell-based medicinal products:

PhEur (Feb-15)

USP (Aug-11)

Note: These figures do not include hyperlinks since the content requires subscription.
Now with increased detail including excerpts from ICH guidelines and authors comments and suggestions 

Instructions: Many of these tools are interactive and can be expanded or collapsed by clicking on the (+).  Alternatively you can expand or collapse levels by right clicking on the diagram and selecting the required level.  

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Some tools contain notes, hover or click on note symbol   to see note.


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These tools were developed with the support of a grant from the Technology Strategy Board as part of the VALUE consortium (Regenerative Medicine Value Systems: Navigating the Uncertainties) lead by Biolatris Ltd (