Gene therapy clinical trials are logistically complex studies that require highly qualified sites and engaged patients for success. The ability to select the right sites and patients can be the difference between product failure and market approval. In this final...
To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients, pairing them with qualified sites, providing informed consent, and confirming...
The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. To bring these treatments to market, sponsors are tasked with conducting clinical trials...
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of...