Editor’s Memo

‘That needs to stop’


Peter Wehrwein

When Kaiser Health News published its investigation of orphan drugs in January, it shed light on a troubling corner of the pharmaceutical market.


The Orphan Drug Act is now 34 years old, and it has accomplished what it set out to do—and then some.


The economics of developing treatments for rare diseases were unworkable. Now they are veritable gold mines for pharmaceutical companies.


EndPoint News published its “10 most expensive drugs on the planet” list in April, and every one of them was, no surprise, an orphan drug. Topping the tally is Horizon Pharma’s Ravicti with a wholesale acquisition cost of $793,632. The company disputes that figure. BioMarin’s Brineura was second on the list, coming in at $702,000, although according to Bloomberg, BioMarin also says that price is unrealistic and that after mandatory government discounts (most of the patients are on Medicaid) the price will be $486,000.


(Yep, we definitely need some price transparency.)


High drug prices for truly rare diseases do present a dilemma. Private companies need a return on investment. The markets and the patients—infants and children—are often tiny. But high prices are one thing. These astronomical ones, another.


Besides, as Kaiser and Contributing Editor Joseph Burns reports in this issue, the intent of the orphan drug law is being undermined. Drugs approved as orphans now rack up blockbuster sales as they accrue multiple indications and are used off label. Common diseases are “salami sliced” by biomarker into rare ones so the drugs to treat them can gain orphan status and the attendant benefits (subsidies, market exclusivity, etc.). “That needs to stop,” David Mitchell told Burns. Mitchell is the founder of a new—and much-needed—group, Patients for Affordable Drugs.


Mitchell’s right. It is time to tighten up the Orphan Drug Approval so its original good intentions are realized, not taken advantage of.

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