As innovation within gene therapy technologies continues to advance, drugmakers are looking to create commercial gene therapy medicines for specialized diseases, such as hemophilia. Although the medicines are extremely expensive, drugmakers claim that gene therapy will save health insurance providers money in the long run, since it provides a “one-off” cure for rare diseases, as opposed to conventional medications that patients usually need to take for many years.

Over the past 5 years, 2 gene therapies have been approved in Europe, though only 3 patients have been given commercial medications. Complicated reimbursement processes, inconsistent clinical trial records and a lack of presence are thought to be contributing factors in why gene therapies have not sold well, according to Spark chief executive Jeffrey Marrazzo. However, the commercialization of gene therapies is still a complex question, as health care systems usually pay for drugs on a monthly basis as opposed to paying an enormous bill upfront. A “one-off” cure is thought to ultimately decrease the need for expensive treatment and care, but gene therapy companies are looking to create a new business model in order to be rewarded for the time and R&D costs spent in developing the gene therapy medication. One option is a “pay-for-performance” model, in which insurers or the government would pay companies and stop payments if the medication became ineffective.

Currently, hemophilia is forecast as being the first significant commercial opportunity for gene therapy companies.

Source: Reuters