Rare disease designation for coronavirus drug is just a tax break [Updated] (original) (raw)

How is a pandemic “rare”?!

When we say "rare disease," it's generally accepted that this means a condition that affects fewer than 200,000 individuals in the United States. Obviously the predictions for COVID-19 are for many times that number—it's a pandemic, after all. However, the Orphan Drug Act does have an exception for more common diseases with unmet needs. So the FDA is allowed to consider granting orphan drug status to a treatment for "[a] disease affecting over 200,000 persons in the US, but for which there is no reasonable expectation that the cost of developing and making available a drug for such disease will be recovered from sales in the US." It's this provision that Gilead used with its remdesivir orphan drug application.

The FDA has not approved remdesivir as an orphan drug

At this point, it's important to make something clear: the FDA has not approved remdesivir as an orphan drug. It has designated it as an orphan drug (as of yesterday), which means that Gilead can start tracking all of its expenses for studying remdesivir as a treatment for COVID-19 to satisfy the IRS. But getting something designated as an orphan drug by the FDA is pretty easy—the agency tries to respond to applications within 90 days and doesn't even charge user fees.

Getting FDA approval as an orphan drug is another matter—that requires actual clinical data, and approval typically happens four or five years after designation. (In remdesivir's case, I could see approval happening much quicker given the public health emergency, but again it would still require clinical trial data to be collected, analyzed, and then reviewed.) This approval is by no means guaranteed; Gilead applied for orphan drug status for remdesivir in September 2014 as a treatment for Ebola. But as of March 2020, that approval has not been granted.

Is it time to grab my torch and pitchfork?

In its article, The Intercept notes that Gilead is no longer allowing new patients access to remdesivir through a "compassionate use" exemption that allows a drug to be given to a patient for a disease for which that drug was not FDA approved. But we should also note that, until the clinical trials are complete, it's yet to be proven that remdesivir will actually be effective with SARS-CoV-2 infections. A Phase 3 clinical trial is underway in the US, but it only began in February. (Another trial is also underway in China.) And the seven years of orphan drug exclusivity—and those potentially outrageous prices—can only happen after any FDA approval.