$ 850,000 against blindness: a gene therapy for the rich and those who go into debt

Luxturna® is the first gene therapy approved in the US for a hereditary disorder affecting the functioning of the retina, the layer of sensory tissue lining the back of the eye. The total cost of treatment should be close to one million dollars!

After approval by the Food and Drug Administration, Luxturna® is expected to be offered in the US for a modest $ 850,000. According to its maker, Spark Therapeutics, a single dose of $ 425,000 is needed to treat one eye. As the genetic mutation affects both eyes and affects the functioning of the retina at the same rate, most patients should require a treatment for both eyes, $ 850,000 will be needed ... This drug against blindness is happening to become the most expensive treatment in the world.

A transplant of a new functional gene

It is actually a gene therapy: it aims to repair the gene behind this rare type of blindness. The drug is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene. This rare genetic abnormality causes severe visual impairment early in childhood and can eventually lead to blindness.
Luxturna® is administered by an injection made by a specialist directly into the eye with a micro-needle. The drug aims to provide a third RPE65 gene normal to the cells of the retina. This third version of the gene does not eliminate or replace the two mutated genes that cause the disease. However, the normal gene delivered via the injection of a vector into the eye is able to function in some retinal cells and correct vision problems.

Rare, innovative and refundable

Even though the drug is innovative, the laboratory knew that a shower of criticism was waiting for it when it revealed the exorbitant price of the treatment. To facilitate "access to all", Spark Therapeutics has provided some financial arguments, beyond the mere clinical efficacy.
First of all, there are very few patients carrying the genetic anomaly and therefore concerned by this treatment: in the United States, 1,000 people are currently eligible for this drug and only 10 to 20 cases per year will be added . This is called a "niche" treatment. No risk therefore according to them to destabilize the health accounts.
Secondly, this gene therapy may be available as part of a "results-based repayment agreement". Clearly, they promise a refund in case of failure of treatment. A drug close to a million dollars: yes, but only if it works!
Finally, third development, Spark Therapeutics also announced a proposal for installment payments for different paying agencies in the US. Credit to rescue the caisses of the Secu!
It is not sure that this proposal is satisfactory while swelling in all countries, including the United States, a controversy on the high cost of new innovative drugs.

An approach that will become common

However, this is an approach that is "generalized": in hematological cancers, CAR-T cell that rescue patients suffering from refractory forms of leukemia or lymphoma, will be charged also nearly a million of dollars (for the entire treatment, including resuscitation) and the Novartis lab, which markets the first CAR-T cell promised to reimburse its drug in case of failure.

It should be known that CAR-T cells are potentially usable in many cancers and in AIDS, and there, we are on populations of much larger patients.

Video: re:publica 2019. tl;dr. Stage 1 - Day 2 - ENGLISH (November 2019).