Home Cellular health Breaking new ground in innovative clinical trial design (ICTD) for cell and gene therapies

Breaking new ground in innovative clinical trial design (ICTD) for cell and gene therapies

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Figure 5. Regulatory support (in terms of pre-consultation meetings) provided by regulatory agencies in selected countries.

The lack of a reference could create difficulties for sponsors seeking to conduct innovative clinical trials for possible approval. It is therefore imperative to have an individual consultation session to obtain an alignment with the authorities on their expectations when filing an application for regulatory approval.

3. Relative novelty and risk of ICTD

To date, little is known about the use of ICTDs for cell and gene therapy products – in the case of adaptive trials, there is a lack of clarity on when they are applicable, what they can or cannot accomplish, what their practical implications are and how their results should be interpreted or reported5. Studies using real-world data to provide information about a broader patient population may contain inherent biases in their data sources that could limit their value in establishing causality between drug exposure and outcomes.20.

Despite the potential benefits that ICTDs can bring to CGTPs, developers should still exercise due diligence when considering the suitability of this mode of test design for their IMPs. To maximize their chances of success in market access, it is recommended that industry players strive to understand and apply the recommendations of regulatory agencies, as well as partner with providers with a clinical trial experience for a better understanding of the ICTD landscape.

CGTPS offers hope for the treatment of serious illnesses with unmet medical needs – expertise is needed to navigate the regulatory space to ensure regulatory compliance and access to every market. To learn more about cell and gene therapy products in the JAPAC region, please click here.

References:

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