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The Luxturna Debate: Why Ethics Needs a Seat at the Drug Pricing Table

By Clio Sophia Koller

Jack Hogan can now ride his bike home at dusk after an afternoon of playing with his friends. Is that childhood rite-of-passage worth $850,000?

Recently, the Health Policy and Bioethics Consortium convened by Harvard Medical School’s Center for Bioethics and the Program on Regulation, Therapeutics, and Law (PORTAL) at Brigham and Women’s Hospital met to discuss the implications of Spark Therapeutics’ new gene therapy treatment—along with its staggering price tag.

Luxturna, a novel therapy approved by the FDA last year, treats a rare form of inherited blindness known as retinitis pigmentosa. The therapeutic agent targets the RPE65 gene, associated with the disorder, and is shown to improve vision in a population with progressive vision-loss and an inability to see in dim light.

While the magnitude of improvement varied from patient to patient, the star of Massachusetts Eye and Ear’s promotional video—which was shown at the consortium—saw notable gains. In the video, Jack Hogan excitedly describes his newfound freedom: he can now get pizza with his friends and ride his bike home after dark—feats impossible before gene therapy.

While Luxturna impacted Jack’s life in other notable ways as well, it allows him to see the smart-board in school and to do his homework without assistance, Luxturna raises interesting questions of how we evaluate “health gain” and which gains are worth the significant cost.

The consortium included presentations by Dr. Jason Comander, the surgeon at Mass. Eye and Ear who operated on Jack Hogan, and Dr. Steven Pearson, the president of the Institute for Clinical and Economic Review–both of whom sang Luxturna’s praises. Each justified the steep $850,000 price tag per patient by considering the need to reward innovation, the increased vision some patients experienced (especially in lower light levels), and the subsequent improvements in quality of life.

Regarding the price, Dr. Comander asked the audience, “What kind of spending is most worth your time fighting?” suggesting the answer lay in generic drug pricing and arbitrary price hikes opposed to novel treatments.

Despite the hype, those startled by Luxturna’s cost have a right to be concerned. Luxturna does not cure blindness. It improves vision in some with a rare disorder impacting only about 1,000 to 3,000 people in the United States to begin with. If a treatment increasing sight in dim light costs $850,000, what happens if a gene therapy emerges that can cure blindness? What happens if a gene therapy emerges that can cure a disorder which usually amounts to a death sentence? As the first commercial gene therapy, what type of precedent for pricing does Luxturna set?

While we should reward scientific innovation, whatever way you cut it, the cost of Luxturna impacts the average person — those who already contribute to encouraging innovation via tax dollars used in the R&D process. The company claims that insurance covers most of the $850,000 burden, and as such, the cost is judiciously spread around entire networks. But in a country with the unfortunate mentality that healthcare is a privilege not a right, and rising drug prices that force Medicare/Medicaid eligibility tightening and cutbacks on other services, where do we draw the line of what is a “worthwhile” health gain?

If we consider the totality of the circumstances, how can we justify the ethics of some in an insurance network rationing life-sustaining medication due to cost, while rewarding such innovation through increased fees?

Luxturna isn’t the end of the conversation, it’s only the beginning, as other gene therapies are posed to enter the market, following Luxturna’s lead. Issues of healthcare economics, revenue, and scientific innovation dominate the conversation, with economists, doctors, and regulators all concerned with pricing the treatment in a cost-effective way, but where does ethics enter the picture? Who is responsible for ethical considerations of drug therapy pricing?

If Luxturna taught us anything, it’s that ethics needs a seat at the drug pricing table.

In a conversation full of lawyers dealing with regulations, scientists with innovation, and venture capitalists with revenue, the vocabulary of bioethics offers of means of orienting the varying interests at play to the reality of healthcare in the United States.

Because while $850,000 for the freedom to ride a bike might seem worth it to the 13-year-old whose life changed, and those privileged enough to be moved by his story, $850,000 for a bike ride might seem anything but for someone rationing their insulin. And if sky-high pricing isn’t a problem with insurance spreading the cost— as Dr. Comander suggested at the Consortium event — shouldn’t both perspectives have a voice as we set a precedent for a new wave of treatments?


Clio Koller is a Masters in Bioethics student at the HMS Center for Bioethics. 

Clio Sophia Koller

Clio Sophia Koller received an A.B. with honors in the history, philosophy, and social studies of science and medicine from the University of Chicago in 2018 and is currently a Master’s of Bioethics student at Harvard Medical School. Clio is interested in the intersection of law and bioethics, specifically the social, ethical, and legal implications of emerging genetic technologies. She will attend Yale Law School in the fall.

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