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Insight Brief
Practical Insights for Cell and Gene Therapy Long-term Follow-up Studies
Helping regulators develop new tools, standards, and approaches for assessing the safety, efficacy, quality, and performance of regulated medical products.
Dec 15, 2023
To inform upcoming guidance on long-term follow up (LTFU) in cell and gene therapy (CAGT), FDA's Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) conducted a public listening meeting titled, "Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products."1 In addition to the live meeting, FDA invited written public comments, (via Docket No. FDA-2023-N-0398), with the intent to use the information received in preparing new guidance for sponsors in this area.
IQVIA responded to the opportunity by coordinating an exchange with the FDA for the purpose of communicating our perspective surrounding in four topic areas:
1) Alternative study designs, including decentralized studies;
2) Development and establishment of product-based and/or disease-based registries;
3) Real-world data collected in clinical settings, through digital health technologies, electronic health records (EHR), insurance claims databases, and other administrative databases, and population-based data sources; and,
4) Determination of specific safety or efficacy outcomes (e.g., development of malignancies, effects on fertility, or confirmation of benefits) for which collection of post-approval safety or efficacy data may be necessary for cell or gene therapies.
IQVIA is committed to helping regulators to develop new tools, standards, and approaches for assessing the safety, efficacy, quality, and performance of regulated medical products. In responding to OTP's solicitation, we shared our clinical, technical and operational insights with conducting long-term follow-up studies for CAGTs to help FDA develop future guidance in this area for study sponsors.
More than a dozen IQVIA staff members who work with sponsors on CAGT studies volunteered to participate in a working group, organized though our Real-World Regulatory Science and Strategy team. We collected their ideas in response to the FDA's prompts and synthesized them into four presentations that were to be delivered by three senior subject matter experts at the live meeting.
Read our takeaway messages, submitted to FDA, based on IQVIA's presentations and the questions posed by panelists during the meeting.
IQVIA responded to the opportunity by coordinating an exchange with the FDA for the purpose of communicating our perspective surrounding in four topic areas:
1) Alternative study designs, including decentralized studies;
2) Development and establishment of product-based and/or disease-based registries;
3) Real-world data collected in clinical settings, through digital health technologies, electronic health records (EHR), insurance claims databases, and other administrative databases, and population-based data sources; and,
4) Determination of specific safety or efficacy outcomes (e.g., development of malignancies, effects on fertility, or confirmation of benefits) for which collection of post-approval safety or efficacy data may be necessary for cell or gene therapies.
IQVIA is committed to helping regulators to develop new tools, standards, and approaches for assessing the safety, efficacy, quality, and performance of regulated medical products. In responding to OTP's solicitation, we shared our clinical, technical and operational insights with conducting long-term follow-up studies for CAGTs to help FDA develop future guidance in this area for study sponsors.
More than a dozen IQVIA staff members who work with sponsors on CAGT studies volunteered to participate in a working group, organized though our Real-World Regulatory Science and Strategy team. We collected their ideas in response to the FDA's prompts and synthesized them into four presentations that were to be delivered by three senior subject matter experts at the live meeting.
Read our takeaway messages, submitted to FDA, based on IQVIA's presentations and the questions posed by panelists during the meeting.