Human factors validation services for a cell and gene therapy product

Our client, a biotechnology company developing an encapsulated cell-based gene therapy designed for implantation in the eye to treat a progressive retinal degenerative disease, required a comprehensive human factors program to support a regulatory submission in the United States.

The therapy, a capsule implanted into the vitreous of the human eye, contains living cells that enable the continuous release of a therapeutic protein. Because the product involves surgical implantation and specialised handling procedures, careful evaluation of usability and risk mitigation was required.

Human factors | Cell and gene therapies | Drug delivery | Surgical

Contextual research – understanding the unique challenges for an encapsulated cell-based gene therapy

Working on a novel encapsulated cell-based gene therapy, we needed to understand the unique challenges and tasks presented for surgeons and associated surgical staff.

Unlike standard ophthalmic procedures, such as intraocular drug injections, this implant required surgical staff to follow a different approach to handling and administration, which needed to be considered in the design of the human factors study and in the IFU.

“Design sprint” formative human factors study

As part of our formative human factors study, we carried out a “design sprint” of the client’s IFU, watching how the surgeons interacted with the implant and instructional material. Iterative usability tests were conducted to help refine the IFU. This approach offered the client insights into how eye surgeons and surgical teams engaged with the implant and where clearer instructions were needed.

Making ‘on-the-spot’ edits to the IFU between participants relies on a strong understanding of effective information design and common human factors challenges. In doing so, we are able to rapidly test different solutions to present the most accessible instructions for clinicians based on our experience, for review by the client’s development team.
Claire Young, Senior Human Factors Consultant, Team Consulting

Commercial IFU and labelling design

The client placed a strong emphasis on developing an IFU that would clearly and effectively guide clinical staff through all required use-steps. To support this objective, we focused on systematically incorporating user feedback throughout the development process to refine and strengthen the IFU.

Drawing on insights from our human factors studies and the expertise of the client’s team, we helped the client develop the IFU, including defining the IFU format and hierarchy, based on applicable regulatory guidance and human-factors design principles. We then expanded this design to apply to incorporate the product labelling, creating a professional, bespoke appearance in line with the client’s branding and regulatory requirements.

We created a checklist of key information that needed to be conveyed to the surgical staff, ensuring the information is presented in alignment with regulatory expectations for the product.
Derek Dumolo, Senior Human Factors Consultant, Team Consulting

Use-Related Risk Analysis (URRA) development

To inform the human factors validation study design, we developed a comprehensive URRA. As the product is surgically implanted, this involved clearly differentiating the risks between a standard clinical procedure with surgical risks.

Labelling and IFU review

Regulatory feedback during the development process was incorporated into the IFU and labelling, ensuring alignment with the biological license application (BLA) requirements and timelines.