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:
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
With so many organisations writing standards for cell, gene and tissue therapies its easy to lose track of them all. This is an initial draft map (Dec 2020), please feel free to send details of any standards not yet included.
Note: for pharmacopeia’s, only texts specific to ATMP are included.
NEW in Beta!! Elements of the CoA when selecting materials.
This decision tree is designed to aid evaluation of the certificate of analysis (CoA) of a material to ensure the range of methods included are appropriate.
This is a beta version, so please do provide feedback at info at advbiols.com
Coming soon: decision tree for identifying and mitigating risks associated with raw materials.
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.
Links to external documents are included and indicated by the following symbol Simply click to open the document in a new tab.
Some tools contain notes, hover or click on note symbol to see note.
We value your feedback; should you have comments or suggestions for inclusions and amendments please let us know. Contact: info at advbiols.com
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 (http://www.biolatris.com).