Cell and gene therapies offer groundbreaking opportunities to treat previously intractable diseases. Rather than only managing symptoms, gene therapy addresses a disease’s root cause by modifying the expression of a patient's genes or by repairing or replacing abnormal genes. The common goal of cell and gene therapy researchers, clinicians, manufacturers, and regulators is translating these vital therapies into safe and effective options to improve patients’ lives. Proper oversight from regulatory authorities is critical to ensure that this is accomplished in a practical, ethical, and clinical evidence-based manner.
Due to their specialized nature, cell and gene therapies come with their own unique impediments to approval, including evolving regulatory expectations, rare study design considerations, and a lack of animal models. In terms of regulatory roadblocks, it isn’t possible to make general policy that can be applied across the spectrum of these therapeutics. Download the full article to learn more about why it is crucial for sponsors to leverage broad collaboration and engagement with both patients and regulatory agencies to increase the potential of cell and gene therapies and improve accessibility.