A Futurism article this week put into very human terms the ongoing question we face when it comes to rare disease treatment and Pharma:
What is the cost of life?
Nearly a year ago, the FDA approved the first-ever treatment for spinal muscular atrophy (SMA), a rare genetic disorder that causes mild to severe muscle weakness and degeneration. The drug is called Spinraza, which is manufactured by Biogen – and it costs $750,000 for the initial year of treatment.
$750,000.
After the first year, the total annual cost drops to $375,000. And the drug must be taken for life.
This topic – soaring costs of certain drugs and treatments – is nothing new, but it’s worth debating and re-debating because it just doesn’t seem right that the only drug available to treat a debilitating condition which affects a lot of children would cost nearly one million dollars.
The article profiles Jana Gundy and Amanda Chaffin, whom live within two hours of each other in Oklahoma and each has a child with SMA. Gundy’s son Kyle was on private insurance, so he began getting treatment immediately, whereas Chaffin’s son Kayden did not, because he was a Medicaid enrollee and Oklahoma regulators debated whether to offer it to children like him who required ventilators to breathe.
And this dilemma plays out all across the country.
Private insurers and thinly-spread state Medicaid programs wrestle with what, if any, limits to place on patients’ access to high-cost drugs, weighing the needs of the sick against budget realities.
“It looks like a drug that works for a tragic condition that afflicts children and cripples and kills them. That’s the good news,” said Jerry Avorn, a professor at Harvard Medical School. But “how in the world did the price of $750,000 a year get chosen?”
Biogen, the makers of the drug, defended the steep price, saying they compared industry norms for other drugs in rare disease. So in other words, they are saying “Hey – every other drug company does it, so why can’t we?”
And to be completely honest, Biogen does have programs that help covers the cost of the drug for patients who are denied by their insurers. According to Biogen, 20% of Spinraza doses were provided free in the U.S.
But that is only slapping a bandage on the bigger problem of exorbitant drug costs.
Consider this: Spinraza was granted orphan drug status by the FDA, which provides tax credits for research. So in all likelihood, Biogen’s research and development costs were not unusually high.
And it’s not like drug companies publicly report their manufacturing and research costs that justify these ridiculously high price tags.
So we just need to take them at their word...
So in Kayden’s case, while Oklahoma’s Medicaid program approved rules barring coverage of Spinraza for patients dependent on ventilators, he was ultimately denied. But luckily he was accepted into Biogen’s patient-assistance program, which covers the cost of the drug.
And Medicaid will cover the 2k per injection to cover doctor and hospital costs.
But not everyone gets these opportunities. Some government programs — Medicare and Medicaid — restrict efforts to eliminate copayments for drugs. And the time and effort required to fill out paperwork and apply to programs and appeal decisions – this can be taxing on working families.
We can’t forget the faces and lives behind these issues – no one chooses to be sick, least of all children. So it isn’t asking much for these drug companies to keep that in mind so they can hopefully find a more ethical approach to determining drug prices.
