In 2023, the cell and gene therapy sector experienced significant growth, with seven FDA approvals in the U.S. and one in the European Union, as reported by the Alliance for Regenerative Medicine. Looking ahead to 2024, the organization anticipates up to 17 approvals in the U.S. and EU combined. The projected approvals include five gene therapies for rare genetic diseases, the first-ever approval of adoptive cell therapy for solid tumors, and the initial U.S. approval of an allogeneic T-cell therapy. Notable milestones expected in 2024 involve therapies for hemophilia A and B, as well as dystrophic epidermolysis bullosa. The Alliance for Regenerative Medicine is optimistic about the progress in the field and believes many of these advancements will reach successful completion. However, challenges such as manufacturing issues, clinical development timelines, and global regulatory requirements pose critical considerations for the future of gene therapy. The high cost of gene therapies, exemplified by recent approvals with multimillion-dollar price tags, raises concerns about the sustainability of these treatments within the U.S. healthcare system. Despite the high upfront costs, proponents argue that gene therapies can be economically viable by offsetting expenses associated with traditional treatments for rare diseases. The Alliance for Regenerative Medicine cites an Institute for Clinical and Economic Review (ICER) report, indicating substantial cost offsets in gene therapies for hemophilia A and B due to the elimination of expensive prophylactic treatments. This perspective aims to demonstrate the potential long-term affordability and cost-effectiveness of gene therapies.
By Greg Slabodkin, News Editor at BioSpace