-
Americas
-
Asia & Oceania
-
A-I
J-Z
EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more -
Middle East & Africa
EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
Regions
-
Americas
-
Asia & Oceania
-
Europe
-
Middle East & Africa
-
Americas
-
Asia & Oceania
-
Europe
Europe
- Adriatic
- Belgium
- Bulgaria
- Czech Republic
- Deutschland
- España
- France
- Greece
- Hungary
- Ireland
- Israel
- Italia
EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more -
Middle East & Africa
EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
Learn more
SOLUTIONS
-
Life Sciences Solutions
-
Healthcare Solutions
-
Medical Device and Diagnostic
-
U.S. Government
Life Sciences Solutions
Bringing together unparalleled healthcare data, advanced analytics, innovative technologies, and healthcare expertise to create intelligent connections that speed the development and commercialization of innovative medicines to improve patient lives.
Learn MoreLife Sciences Solutions
Healthcare Solutions
Accelerate digital innovation to enable smarter decisions that reduce cost, modernize patient and consumer engagement, and improve health outcomes.
Learn MoreHealthcare Solutions
Medical Device and Diagnostic Solutions
Your world is unique – and quite different from pharma. In the U.S., decision-making has shifted from individual physicians to integrated networks--GPOs, IDNs and payers. These groups have heightened the focus on proving your solution’s value, demanding outcomes analyses and putting pressure on pricing.
Learn MoreMedical Device and Diagnostic Solutions
U.S. Government Solutions
For government agencies and organizations at every level—from federal or national to regional and local—Big Data can have a huge impact on public health.
Learn MoreU.S. Government Solutions
U.S. PROGRESS POINT
A curation of IQVIA's best thinking on topics and trends driving change, disruption, and progress in the United States healthcare market.
LEARN MOREU.S. BLOGS
Get the latest insights on our life sciences, healthcare, and medical technology solutions in the United States. Follow our blog today!
LEARN MORE
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.