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Some scientists tout gene therapies—drugs that modify a patient’s genes to treat or cure a disease—as the future of medicine, with the potential to treat the most pressing diseases, like cancer, diabetes, and heart disease.
Gene therapies also have massive cost-savings potential, particularly when it comes to treating rare diseases. Most rare diseases are caused by a single gene mutation, which means a gene therapy treatment could fix the root cause of the condition.
The economic burden of rare diseases is staggering. It cost the US almost $1 trillion in 2019 from direct medical costs, non-medical and uncovered healthcare costs, and productivity loss, according to Rachel Salzman, global head of corporate strategy at precision therapeutics company Armatus Bio.
But these gene therapies come with a hefty price tag.
A gene therapy called Hemgenix, developed by CSL Behring and uniQure, costs $3.5 million per dose. And that’s not far out of the ordinary for gene therapies. The Institute for Clinical and Economic Review estimates the average cost for gene therapy is between $1 million and $2 million per dose.
The cost of the therapies means many patients lack access, Salzman said, speaking at the American Society of Gene and Cell Therapy’s annual meeting on May 16. Fewer than 10,000 patients received treatment with three gene therapy drugs in 2021, she said.
The three drugs—two from Novartis called Zolgensma and Kymriah, and one from Kite Pharma called Yescarta—brought in a cumulative $3.5 billion in revenue in 2021. If 10,000 patients were treated with the drugs in 2021, that translates to $350,000 in revenue per patient.
Financial toxicity, or obstacles patients face due to the cost of their medical treatment, is a leading cause of healthcare disparities, Salzman said.
With the high costs of gene therapies, she posed the question: “Are gene therapies a tool for accelerating existing disparities?”
Correction, May 22, 2023: An earlier version of this article misstated the cost per-dose of Hemgenix gene therapy.