ROME — The total estimated costs from inherited retinal diseases in the United States amounts to $31.8 billion. And yet, research barriers slow down the progress toward innovative treatment pathways, such as gene and cell therapies.
“Rare disease is costly and is difficult to get into. But there is an immeasurable benefit to cure ... that we must continue to strive for,” Suber S. Huang, MD, MBA, said at the FLORetina meeting.
Clinical trials for rare diseases are challenging to implement. The treatments are expensive, with limited return on investments and a large financial