Haemophilia: what is the potential breakthrough?
Around 14,000 people in Europe have haemophilia B, of which 60% live with a severe form of the disease, putting them at risk of bleeds. Gene therapy Phase I/II trials have seen results sufficient to transform the disease into a mild form, while further trials could see a cure. In time, gene therapy approaches could also tackle other diseases like cancer and infections.
“Gene therapies have the potential to relieve or even cure rare diseases, such as haemophilia B, where the patient’s quality of life and prognosis are poor.”
The cost of current treatment is approximately €6 million over a single patient’s lifetime. Gene therapy could decrease emergency hospitalisations by reducing the number of patients suffering serious bleeds. It could also lessen the burden on infusion clinics and cut drug expenditure.
Not all aspects of innovations fit the traditional pharmaceutical delivery pathway, and therefore, it is necessary to upgrade or develop new infrastructure to bring innovative therapies to patients. For example, upfront reimbursement for gene therapy is a challenge because there is currently limited evidence of lasting clinical or economic benefit, leading to uncertainty around its value. In the future, new finance schemes and integrated budgets may help to fund this type of innovation.