Move to value-driven health system to reap rewards of gene therapies, claims PhRMA

US trade group the Pharmaceutical Research and Manufacturers of America (PhRMA) has pointed out the savings that gene therapies can deliver for patients that otherwise require lifelong treatment.

PhRMA has cited an analysis by Health Advances looking at two particularly burdensome hematological diseases — beta thalassemia and hemophilia A — showing that gene therapies in the pipeline could reduce per patient costs by as much as 41% and 90%, respectively, over five years.

"Gene therapies such as these harness the latest scientific advances and have the potential to transform the trajectory and even curing some of the most complex and devastating diseases facing patients today"Depending on disease severity, the Health Advances analysis indicates that direct medical costs and indirect costs associated with hemophilia A can range between $441,000 and $1.94 million per patient over a five-year period.