The Shocking Cost of One Girl’s Spinal Muscular Atrophy Treatment

One of the most impressive moments of childhood is when a young child takes his or her first steps. Natalie Essex, age four, loves to walk. For Natalie, spinal muscular atrophy makes walking a real challenge. A new drug is helping Natalie regain some muscle, but there’s a cost. Keep reading to learn more about Natalie’s story, or click here to read more at the original source.

The drug helping Natalie is called Spinraza. It sounds sort of like a miracle, but the drug therapy enables Natalie to get back some of the muscle use she’s lost as a result of spinal muscular atrophy. The biggest problem, however, is that Spinraza costs roughly $100,000. That’s per dose.

Spinal muscular atrophy patients like Natalie require one dose every four months. That’s an expenditure of nearly $300,000 per year just for one drug.

Natalie is fortunate enough to be part of a clinical trial. Her costs will be covered as a result. The drug isn’t funded in British Columbia though, so even if it does get approved, not all patients will have access to it.

Even more troubling, this kind of treatment isn’t uncommon. New drugs often come to market with price tags that could stretch to the moon. Especially when treatments involve rare disease, patients could be expected to pay anywhere from tens to hundreds of thousands of dollars per dose.

Researchers studying the pharmaceutical industry are quick to point out the injustice of this system. In their terms, drug company pricing is essentially ridiculous, without grounding in real life.

Steve Morgan, a health economist and professor with UBC’s School of Population and Public Health, has a strong stance on this issue. He explains that those responsible for the creation of drugs will often point to the costs of research and development as an explanation for high prices. “But that actually doesn’t necessarily have anything to do with the actual prices they’re paying, in part because they never fully disclose what they did spend on research and development.”

“At the end of the day, the cost of research and development is not necessarily the determinant of the price of the drug.”

Even at the urging of government, corporations often do not lower prices. The government of British Columbia has frequently asked pharmaceutical companies to provide justification for their prices. Little to nothing has changed.

Health minister Adrian Dix does not seemed surprised. “They’ve now found kind of a profit pipeline for themselves,” he says of pharmaceutical companies. Still, he wants them to understand. He believes that what people need and what companies want can find a compromise that is healthy for both. Dix describes the solution as a “way going forward so that people aren’t caught in this position at the end.”

Fortunately, the Essex family won’t have to worry much over this issue. Natalie will continue to receive life changing treatment for the duration of the trial. It’s an amazing opportunity. The question of how to extend it to others, however, may remain for a long time to come.

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