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EMEA Thought Leadership
Developing IQVIA’s positions on key trends in the pharma and life sciences industries, with a focus on EMEA.
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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
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Americas
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Asia & Oceania
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Europe
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Middle East & Africa
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Americas
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Asia & Oceania
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Europe
Europe
- Adriatic
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- Bulgaria
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- Deutschland
- España
- France
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- Hungary
- Ireland
- Israel
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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
WHO WE HELP
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Pharmaceutical Manufacturers
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Pharmaceutical Manufacturers
Harness unparalleled healthcare data, advanced analytics, and cutting-edge technologies with our expertise to accelerate the development and commercialization of innovative medicines, enhancing patient lives.
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Emerging Biopharma (Global page)
Boost your innovation with IQVIA's expert team. Our comprehensive solutions integrate top-tier insights, technology, and expertise to maximize your asset's success in a competitive landscape.
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Navigating MedTech's unique landscape: U.S. decision-making now rests with GPOs, IDNs, and payers, who demand solid value proof through rigorous outcomes analyses and pricing scrutiny.
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Invest in healthcare with confidence. Stay ahead of pharma and medical device trends using real-time, high-quality data. Access market insights, historical sales, prescription trends, and emerging product information for informed, risk-reduced decisions.
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Health Plans
Accelerate digital innovation with our data-driven expertise in data science and healthcare, optimizing business processes for smarter decisions.
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Leverage digital innovation to cut costs, boost efficiency, and elevate patient engagement, all while enhancing quality and health outcomes.
Streamline DecisionsHospital and Health Systems
Insurers and Risk
Enhance your ability to evaluate and forecast population risk swiftly and accurately by integrating unmatched data, expertise, and technology.
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Medical Specialty and Patient Advocacy
Transform into a data-driven organization that enhances your specialty and showcases value to your members through digital transformation.
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A curation of IQVIA's best thinking on topics and trends driving change, disruption, and progress in the United States healthcare market.
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Explore our library of insights, thought leadership, and the latest topics & trends in healthcare.
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German White Paper
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.