The Cell and Gene Therapies industry is growing at a tremendous speed, building both on innovative solutions and a solid investment by life science companies who wish to diversify their market offer.

The results have been impressive and Patients who had exhausted treatment options are getting exciting new opportunities.

To support the development of new products, the U.S. Food and Drug Administration (FDA) has issued a set of recommendations based on:

  • SISPQ (safety, identity, strength, purity, and quality);
  • Critical Quality Attributes (CQAs);
  • Application of risk management processes.

Implementing Quality by Design

You should leverage a risk-based approach for every aspect of a product, throughout its lifecycle. And Cell & Gene Therapy products are not an exception.

We recommend that you implement a Quality by Design (QbD) strategy based on large data-collection to support risk-based decisions.

Why? Because we believe it’s the approach best positioned to mitigate any significant risk posed by complex manufacturing processes. As a result, you will follow the ICH Q9 principles while ensuring the level of effort and documentation adequate to the level of risk.

Setting up the Quality Target Product Profile

The Quality Target Product Profile contains a list of predefined quality characteristics. You evaluate these different attributes through both risk assessment and considering their potential impact on product safety and efficacy.

Among these attributes, we find Critical Quality Attributes (CQAs), Critical Material Attributes (CMAs) and Critical Process Parameters (CPPs).

You can then use a risk-based analysis as well as Design of Experiment Studies to help you understand the impact of CMAs and CPPs in the CQAs.


article icon cell and gene therapies

If you’d like to have a deeper understanding of this topic with further details on:

  • FDA’s Guidelines for Cell and Gene Therapies
  • Process development for CGT products
  • CQA evaluation through Design of Experiments Studies (DoE)
  • Process characterization studies

We suggest that you download this article of ours: Risk Management Processes for Cell and Gene Therapies.


Our Recommendations

The development of cell and gene therapies calls for a standard application of risk management principles. These principles, aligned with the current guidelines for product development, provide a strong reference that supports all the decisions made, with a solid science-based rationale.

irisk risk management platform

4TE’s expertise in pharmaceutical risk management is consistently clear about one thing. A strong risk and knowledge management culture is key for a successful product development.

We recommend that you should have a platform where all the information is centralized, shared and used as a foundation for new developments.

In our work, we deliver iRISK™ to our clients, a Risk Management Platform for the Life Sciences industry. iRISK™ allows you to identify, quantify and prioritise risks, enabling the formulation of the risk mitigation strategy throughout the processes and products lifecycle.

Check our QRM Digitalisation Services. If you think we may be able to help you out, don’t hesitate to book a call, we’ll do our absolute best to help.

Related topics

If QRM is a topic of interest to you, we recommend you checkout this article of ours: QRM over Lifecycle Management.