Written by Neil Morrison

Neil Morrison is Director, Strategic Development at Alpharmaxim Healthcare Communications

Gene therapy, the process whereby genetic material is inserted into cells to compensate for abnormal genes or to make a beneficial protein, has left the realms of science fiction and is now a reality. Diseases that in the past could only be controlled, at best, by regular medication now have the potential to be cured in a single treatment. But this brings a problem for pharmaceutical companies and healthcare payers alike. How do you pay for a treatment that only requires a single dose to potentially last a lifetime?

Much of the early work in gene therapy focused on rare diseases. Glybera, designed to treat lipoprotein lipase deficiency, was priced at $1,000,000 per treatment when it was approved by the European authorities. The price proved too much, and only a single patient was commercially treated – a German woman who had suffered such debilitating illness that she had been hospitalised in intensive care more than 40 times.

“How do you pay for a treatment that only requires a single dose to potentially last a lifetime?”

Despite the successful outcome of that treatment and the clinical trial programme before it, within two years Glybera’s European marketing licence was left to expire. The three remaining manufactured doses were all but given away. At a cost of €1 each, three final patients were treated – all responded well to the treatment.

As gene therapy research evolves into treating more-common diseases, the issue of pricing and reimbursement will become ever more important. Gene therapy for haemophilia is currently a target for several companies and, although no pricing has been announced, the question of funding the six- or seven-figure cost per patient is one that is taxing governments and insurance companies alike.

Payers are already beginning to examine ways to spread the potential costs of gene therapy. The ‘pay-up-front’ model used for most treatments is not ideal, particularly as real-world data on the long-term efficacy of these products are not yet available. Incremental payments have been suggested as an alternative – where an individual patient gains and maintains benefit, payments would continue. Conversely, payments could be revoked if the patient falls out of remission. This model is already in use. Novartis, which markets tisagenlecleucel, a chimeric antigen receptor T-cell therapy for cancer treatment, only charges its price of $475,000 per patient if they enter remission within the first month.

Of course, current treatment for haemophilia and the complications it can bring is already extremely expensive – a recent study suggested almost a €200,000 annual cost per patient1 – so a ‘one-off’ charge for a gene therapy product that ‘cures’ haemophilia could be cost-effective whatever its price.

As gene therapies begin to enter the market, payers, insurance companies, governments and pharmaceutical companies will need to work together, whatever the arguments for cost-effectiveness. Designing a payment system that rewards the pharmaceutical company for the time and cost of their research and development, while also maintaining the accessibility of these exciting new treatments for the patients that need them, will become a high priority.


1. O’Hara J, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet Journal of Rare Diseases 2017;12(1):106

If you’d like to know discuss more about this topic, please contact Will Hind at will.hind@alpharmaxim.com or on +44 (0)161 929 0400