Gene therapy is a hot topic across all fields of medicine. Starting with the US Food and Drug Administration (FDA) approval of tisagenlecleucel (Kymriah; Lucentis) for pediatric B-cell acute lymphoblastic leukemia in August 2017, followed quickly in December 2017 by voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) for confirmed biallelic RPE65 mutation–associated retinal dystrophies, there are currently 27 approved therapies listed on the FDA website. This number is going to balloon, with more than 1,000 gene therapy studies currently registered on ClinicalTrials.gov . The FDA expects to approve 10 to 20 new gene therapies per year starting in 2025.

This is obviously a very exciting time, but at what cost to the health-care system? Many early therapies were for indications without a treatment, and the costs were not really discussed. What cold-hearted medical director would say no to a parent of a child with spinal muscle atrophy to receive treatment, despite the million-dollar price tag?

By 2024, the cost of gene therapies is expected to be more than $16 billion in the United States alone. Although used rarely, the average cost of Luxturna is around $850,000. This is a bargain compared to the $3 million for Bluebird Bio’s Skysona for cerebral adrenoleukodystrophy or $2.8 million for its Zynteglo for beta thalassemia. The excuse for these ridiculous prices is that it is justified because it delivers a lifelong cure that saves the health-care system money in the long term. The problem is that we don’t know if this is even true. Will additional treatments be required? In Bluebird’s case, they are offering a “refund” of 80% of Zynteglo’s price for 2 years if patients still need blood transfusions. Why is it only for 2 years?

In this issue, we explore the latest in gene therapy for retinal disease. Some diseases being targeted in ophthalmology are not orphan indications with only a few patients. They are diseases with huge patient numbers, such as age-related macular degeneration and diabetic retinopathy. What will be the cost of these medications? We obviously do not know. It is doubtful that insurance companies will be as willing to stomach high prices for gene therapy in these common maladies. For example, why would a commercial payer who covers diabetic patients be willing to foot the bill for a diabetic retinopathy gene therapy that will benefit competing insurance companies when the patient changes their plan?

The current gene therapy pricing situation is unsustainable. It is estimated that a large proportion of our health-care dollars is being spent on these specialty drugs for a very small fraction of our population. It is vital that we tackle the problem of adequately reimbursing ophthalmic gene therapy. We need development to continue at the pace it is going, but we also cannot bankrupt our system.

I asked ChatGPT about pricing in the future, and received this unhelpful response: “the price may change based on various factors, such as supply and demand, market conditions, and other economic factors. Additionally, the price may also vary based on the location and the health-care system.” RP