This rare treatment will cost you more than R10 million

Internationally, drug costs are spiraling out of control.
Internationally, drug costs are spiraling out of control.

Can you imagine a treatment so expensive that you couldn't afford it, even if you sold everything you own?

A first-of-its kind genetic treatment for blindness will cost $850,000 (±R10.4 million) per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.

The injectable treatment from Spark Therapeutics can improve the eyesight of patients with a rare genetic mutation that affects just a few thousand people in the US. Previously there has been no treatment for the condition, which eventually causes complete blindness by adulthood.

The drug, Luxturna, is intended for patients with retinal dystrophy due to a mutation of the RPE65 gene. This kind of retinal dystrophy is estimated to affect between 1 000 and 2 000 people in the USA.

According to a News24 article, a probable 150 000 South Africans suffer from retinal conditions such as macular degeneration and dystrophy, retinitis pigmentosa, and other rare conditions that cause loss of vision in over 40 million people worldwide. 

A one-time treatment

The exorbitant price of Luxturna is not an isolated problem as there is growing concern about huge price increases on older drugs, some of them generic, that have long been mainstays of treatment.

Pricing questions have swirled around the treatment due to a number of unusual factors – it is intended to be a one-time treatment, it treats a very small number of patients and represents a medical breakthrough.

Previously, Spark suggested Luxturna could be worth more than $1 million (±R12.3 million). But the company said it decided on the lower price after hearing concerns from health insurers about the affordability of the treatment.

Consternation over skyrocketing drug prices, especially in the US, has led to intense scrutiny from patients, politicians, insurers and hospitals.

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients," said CEO Jeffrey Marrazzo, in an interview with The Associated Press.

Luxturna is still significantly more expensive than nearly every other medicine on the global market, including two other gene therapies approved earlier last year in the US.

First gene therapy for an inherited disease

Pharmaceutical industry critics said the slightly lower cost is a distraction from the ongoing problem of unsustainable drug prices.

"The company very cleverly convinced everyone that they were going to charge a million dollars, so now they are being credited for being reasonable," said Dr Peter Bach, director of a policy centre at Memorial Sloan Kettering Cancer Center in New York.

Approved last month, Luxturna, is the nation's first gene therapy for an inherited disease. It requires a 45-minute operation in which a tiny needle delivers a replacement gene to the retina, tissue at the back of the eye that converts light into electric signals that produce vision. The therapy will cost $425 000 (±R5.2 million) per injection. The price does not include the cost of the operation, which Spark estimates will cost between $4 000 (±R40 000) and $5 000 (±R61 400).

The treatment is part of an emerging field of medicine that could produce dozens of new gene-targeting medications in the next few years.

Like Luxturna, these therapies are generally intended to be taken once, a fact which drug developers argue sets them apart from traditional drugs taken for months or years. Even compared to other one-time gene therapies Luxturna is still an outlier. Two customized gene therapies for blood cancer approved last year are priced at $373,000 (±R4.5 million) and $475,000 (±R5.8 million).

Drug prices not always regulated

Drug prices are not regulated in the US, as they are in many other countries, so drugmakers can price their goods like any other manufacturer. Drugmakers have historically offered little explanation for the prices they charge, other than to cite the high cost of developing a drug and the fact that so many drugs fail during trials and must be abandoned. However, some companies have begun to offer more detailed reasoning as the backlash against drug prices has grown more heated.

Fortunately medicine prices are regulated in South Africa, which means that you will be able to:

  • Know what to expect to pay when you get a prescription from your doctor.
  • Find possible generics for a branded medicine.
  • Ensure that you are not being overcharged for your medicine.

Spark Therapeutics, based in Philadelphia, has said that the cost for a lifetime of blindness – including lost earnings and caregiver wages – can easily exceed $1 million (±R12.3 million).

Even at $850,000 (±R10.4 million) a preliminary analysis by one group found that the drug would need to be priced significantly lower to be good value.

Image credit: iStock

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