Explore the unique challenges and opportunities in early-phase clinical trials for cellular and gene therapy (CGT) products. Learn about regulatory considerations, trial design, manufacturing barriers, and more with BioBoston Consulting.
Following recommendations are of utmost importance for quality and regulatory consulting firms operating in the life science industry, which should work on their understanding and implementation for CGT products.
I. A New Age: The Range of Products for CGT
The guidance describes the regulatory scope of Office of Cellular, Tissue and Gene Therapies (OCTGT), identifies the unique attributes and challenges associated with CGT products. This is largely centered on safety, tolerability, and feasibility evaluations in early-phase studies while ruling out other products under diverse regulatory frameworks.
II. The Context: Risk and Return
As background, the authors highlight features that set CGT products apart and describe risks encountered in early-phase testing that are without precedent. These illustrative examples demonstrate why special consideration is warranted in designing trials for these classes of rare diseases, providing context for the recommendations made in this guidance.
III. Characteristics of CGT products: A guide for designing a trial
In this section, the unique features of CGT products are broken down in detail with their implications on trial design. Each product is examined, from extended biological activity to the possibility of immunogenicity. BioBoston Consulting is here and ready to help navigate sponsors through the maze of considerations during this squeeze period.
IV. Barriers to Manufacturing: How Close Are We to the Practical Limits?
This segment of the guidance, which goes deeper into manufacturing details, outlines both the scientific and logistical obstacles. The guidance entails practical limits on product doses, concentrations, and volumes, which are carefully elucidated to facilitate sponsors’ consideration of these aspects seamlessly.
Conclusion
BioBoston Consulting are shining a light on the strides in early-phase clinical trials development for CGT products.
Excelling in quality and regulatory consulting, we make sure that sponsors and investigators face challenges head on so that they can work towards revolutionizing the field of cellular and gene therapies.
Reach out to BioBoston Consulting today or check our website for more information on how we can assist your organization.
