International – ICH targets cell and gene therapies, real-world data as topics ripe for harmonization

Officials from Health Canada and the pharmaceutical industry addressed plans to develop harmonized guidelines on cell and gene therapies and on the design of pharmacoepidemiological studies that use real-world data (RWD) to assess new medicines at the International Council for Harmonisation (ICH) regional meeting sponsored by the US Food and Drug Administration (FDA) and Health Canada.

Kathleen Francissen, global head of PT cell and gene therapies at Genentech, and Melissa Kampman, manager of the marketed health products directorate for Health Canda, provided an update on these plans as well as timetables for released harmonized documents at the meeting, which was held virtually on 22 February.

Francissen, who is the rapporteur for the ICH Cell and Gene Therapy Discussion Group (CGT DG), discussed the need for harmonized guidelines in this space. There have been “tremendous advances” in the development of cell and gene therapies over the past several years, with more clinical trials being conducted globally, she said. There are 1,400 advanced therapies in clinical trials around the world. Currently, 5,800 patients have been dosed with AAV-based gene therapies while 38,000 patients have been dosed with CAR T-cell treatments…