Gene therapy is coming to us in a big way, a number of gene therapy clinical trials are underway, and Glybera, a gene therapy agent for lipoprotein esterase deficiency, has been approved for marketing in Europe, but the price is as high as 1.11 million euros, setting the highest record for drug pricing. Even in Europe and the United States, where payments are based on government health insurance and insurance companies, such prices are considered “sky-high” and unacceptable.
But gene therapy does have its own special features. In the case of Glybera, the 1.11 million euro treatment is basically a cure, that is to say, a one-shot deal, unlike most current drugs, which require long-term maintenance.
As an example, with type 1 diabetes, current insulin replacement therapy requires a lifetime of use, and although it costs only $10,000 to $20,000 per year, the medication lasts for decades. With gene therapy, on the other hand, patients may gain the ability to secrete their own insulin with a single treatment, and are also spared the pain of daily insulin injections and the risk of infection, so shouldn’t the price be higher than the total price of lifelong insulin replacement therapy? On the other hand, diabetes is a common disease with hundreds of millions of patients worldwide, and a huge audience is naturally able to dilute the cost. The number of patients with rare diseases is much smaller, and gene therapy, as an emerging treatment, has high initial research and development costs, and the upfront investment in phase I, II, and III clinical trials cannot be less, so the cost of individual patient treatment is bound to rise with the water.
But in reality, such high prices are still daunting. Now that gene therapy is halfway into the clinical field, how to reasonably price it is a hot topic of discussion. The special case of gene therapy pricing. Nat Biotechnol. 2014 Sep;32(9):874-6. doi : 10.1038/nbt.3003. Morrison C. $1-million price tag set for Glybera gene therapy. Nat Biotechnol. 2015 Mar 6;33(3):217-8. doi: 10.1038/nbt0315-217. ), discuss the pricing mechanisms of gene therapy. There are currently three models; first, a direct one-time fee, but at an exorbitant cost. Second, installment payment of the treatment cost, similar to the consumer loan model. The third, and currently considered more reasonable, is called “pay for performance”. Based on the once-and-for-all nature of gene therapy, the treatment is paid for every year for as long as it works. This spreads the cost of the treatment and makes the patient and family willing to pay for the treatment in the face of real results. In the Western world, where credit is what people are built on, patients who receive treatment and their insurance companies are able to do so by paying for the initial treatment every year. In developing countries, however, there is a lack of discipline if patients receive treatment and then stop paying for it. Therefore, there is a question mark as to whether it is appropriate for the Chinese context.
Experience has taught us that the emergence of something new inevitably brings a series of changes and we must shift with it. The technological revolution is changing every aspect of our lives, and the way we see and treat patients. Are you ready for it?