The cost of gene therapy for patients with haemophilia could be as high as $2 million per person, according to US analysts.

Early estimates of the cost of gene therapy for haemophilia put the figure at $350,000 - $500,000 per patient but investment bankers Leerink Partners are predicting far higher costs by using the price of gene therapy for a rare form of blindness as a benchmark. According to, biotech analyst Joseph Schwartz believes manufacturers are exploring what costs would be acceptable to potential payers, leading him to suggest $1.5 – $2 million per patient. However, such prices assume that a single dose would cure haemophilia and there is currently doubt that this can be achieved. By the time the first products are introduced, they will have only 30 – 50 months of experience but could still attract prices around 2.5 times the cost of factor replacement therapy.

The market for gene therapy is expected to be worth about $4 billion per product per year by 2030. Two products now undergoing clinical trials are Biomarin Pharmaceutical’s valoctocogene roxaparvovec (Valrox), which is now in a Phase III trial, and Spark Therapeutics’ SPK-8011, which is in phase I and II trials. Spark’s SPK-9001, developed with Pfizer, was the gene therapy used in the recent trial in patients with haemophilia B (N Engl J Med 2017;377:2215-27).

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