Looking for a simple explanation of how gene therapy safety is evaluated? In this video, part of our GT-FAQ video series, Karen, a veteran pediatric nurse and member of our Duchenne Nurse Educators team, explains the testing, monitoring and review that happen before – and even after – a therapy is approved by the U.S. FDA. Watch for a clear, simple explanation of the basics.
Read the video transcript below.
Video Transcript
How is gene therapy safety evaluated?
Before a gene therapy is approved by regulators like the FDA, it goes through years of testing to demonstrate that it’s safe and effective.
During a gene therapy clinical trial, participants are closely monitored during and after treatment. Researchers monitor and record safety data. This information can help researchers respond and potentially adjust treatment plans to protect the health of participants.
Regulators review this information when deciding if a therapy should be approved. This safety data may also be included in the product’s prescribing information.
How long is safety monitored?
People who participate in gene therapy clinical trials are monitored long-term – often for years after treatment. Gathering long-term safety data provides important information on the long-term effects of gene therapy. This data may be shared at a medical meeting or in a medical journal – and can help doctors weigh treatment benefits against risks and discuss with their patients.
Let’s review!
- Approved medicines are evaluated for years.
- Trial participants are monitored long-term.
- Regulators review safety before approving a medicine.
- Long-term safety data may be shared in medical meetings/journals…
- …to help doctors weigh benefits & risks.
If you have questions about gene therapy, talk to your healthcare provider or email our Patient Affairs team at [email protected].