STAT+: Why a British hospital, and not a drugmaker, is trying to get a rare disease therapy approved
LONDON — Claire Booth, a gene therapy researcher in London, had hoped that a biotech company would take her team’s work on an experimental medication for an ultra-rare children’s disease and get it to market. It didn’t happen.
Now, in an unusual step, the hospital where she works is trying to get the medicine approved on its own.
Booth, who leads the stem cell gene therapy program at London’s Great Ormond Street Hospital for Children, is trying to overcome the fact that many biopharma companies have abandoned promising genetic therapies for ultra-rare diseases or halted their research programs in these conditions. The snag isn’t one of science. Rather, it’s that drug companies often don’t see a possibility of making a return on these medicines, which are immensely expensive to design, treat diseases that only a small number of patients have, and are running into reimbursement obstacles.
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