The eyes have it

What is “it,” you ask? Spark Therapeutics’ new gene therapy Luxturna which can cure blindness in a single treatment. The condition causing the blindness only affects a few thousand people, so the FDA has designated it as an orphan drug. The single dose and orphan drug aspects of this gene therapy combine for a rather expensive R&D bill, and translate into a bit of sticker shock at the selling price: $425,000 per eye. Spark is trying out some interesting commercialization practices to get the drug to patients, like giving rebates to patients whose eyes don’t see better over time. They’re also considering selling the therapy directly to insurance companies so that health care providers don’t have to pay and store the treatment without a guarantee it’ll ever be used.

2. Hope Santa brought cash for Christmas

Happen to have a spare $750,000 USD in your bank account? If so, you could afford the initial year of treatment on Biogen’s new spinal muscular atrophy drug, Spinraza. After the first year, you’d need another $375,000 per year for subsequent treatment. Our collective wallets shuddered just thinking about that. Keep in mind—this is a drug for an ultra-orphan disease so companies typically set high price points given the small patient populations. Nonetheless, the price tag is turning heads in the media. Patients and their families will be waiting with bated breath to gauge payers’ reactions. So far into 2017, it looks like there is no end in sight for drug pricing controversies.

3. Grants aim to make rare diseases even rarer

What’s a good way to put a dent in rare diseases? Well, you can always throw $23 million at it. At least that’s what the FDA did this past week. Through the Orphan Products Clinical Trials Grants Program, 21 new clinical trial research grants were awarded to studies developing drugs and devices for 21 different diseases. And they’re not just hoping that something good will come out of it, they have proof that it works. Five studies that received funding from this grants program supported product approvals in 2015 alone. If they keep this up, one day rare diseases will be extinct. And with names like lymphangioleiomyomatosis (try saying that three times fast), who needs ‘em? That’s just being pretentious.

3. It probably works. Maybe.

The first drug therapy for Duchenne muscular dystrophy (DMD), eteplirsen, is now on the accelerated approval pathway by the FDA. It’s a gene therapy that allows 13% of DMD patients to partially produce an otherwise absent protein, which slows the progression of DMD. At least, that’s what the supposed clinical benefit is. Trials run by Sarepta Therapeutics, the drug’s manufacturer, do show an increase in the otherwise missing protein, but haven’t effectively linked those increased levels to a clinical benefit yet. The trials performed so far weren’t randomized, which our readers may recognize as… not the best way to collect unbiased data. But the increased protein levels seem promising enough for the FDA. The accelerated approval also comes with priority review and an orphan drug designation.

1. For once, some good news about drug pricing

Since the Orphan Drug Act was passed in 1983, the number of FDA approvals for orphan drugs has increased to records levels – 41 in 2015 alone. No shocker, orphan drugs have been criticized for commanding hefty prices. But a new study published in the journal Health Affairs found that orphan drug spending is only about 8.9 percent of total pharmaceutical spending. While that’s up from 4.8 percent before the ODA, the study projects spending will level off. That’s a big deal because *some people* blame rising drug spending on these rare diseases. The ODA has resulted in the approval of more than 552 orphan drugs overall, in many cases for diseases that had little or no previous treatments. Forward to your friend who thinks “big pharma is evil, bro.”