As mentioned in part one of this blog, while the development of cell and gene therapy (C>) products has accelerated in recent years, even the best therapeutic may not reach patients who need it if study design and execution are not done correctly. It is important to put patient safety first, start communication with regulatory agencies early, and utilize an experienced team.
Each month, Veristat's regulatory team tracks and shares a selection of new Regulatory Guidance updates from the US Food & Drug Administration (FDA) that are pertinent to the governance of clinical trials and drug, biologic, or medical device development and approval. Below are the relevant links to updates provided for both COVID-19 related and non-COVID-19 related programs in July 2021.
Cell and gene therapy (C>) products are beginning to deliver on their long-hoped-for promise. CAR-T technology has proven to be a powerful new weapon in cancer treatment, and FDA approval of Luxturna in 2017 demonstrated the potential of gene therapies to be used outside of oncology. As both cause and consequence of this success, development and clinical trial activity in these product classes is accelerating. There are over 1,200 active clinical trials in regenerative medicine and advanced cell/gene therapies in 2021, up from 700 in mid-2019.