Public Pressure Works: Gilead Asks FDA To Rescind Orphan Drugs Designation For Possible COVID-19 Treatment

from the good-to-see dept

Earlier this week, we wrote about the sham orphan drug designation that the FDA gave to Gilead Sciences for remdesivir. As we explained, remdesivir was developed with mostly public funds, and Gilead Sciences already held three patents on it, with a fourth one pending. Orphan drugs designations are supposed to be extra incentives for drug makers to target rare diseases. The issue here was that part of the definition of a rare disease under the Orphan Drugs Act is that it has to affect fewer than 200,000 people in the United States. Ridiculously, the law does not take into account the rate at which the disease spreads -- just how many people have it at the time a drug maker requests the designation. Even worse, the law explicitly says that the FDA cannot remove the designation if the affected group later grows to over 200,000.

Over the last few days, anger continued to grow at Gilead and the FDA -- including with the news that the FDA won't even say when Gilead applied for Orphan Drug status, because that's apparently "a commercial secret." Either way, this morning a ton of public interest groups, organized by Public Citizen, sent a letter to Gilead asking it to renounce its claim for orphan drug status:

We were shocked to learn that your company sought a lucrative orphan drug designation from the Food & Drug Administration for remdesivir, one of relatively few medicines being explored as a possible treatment for COVID-19 this year.

This is an unconscionable abuse of a program designed to incentivize research and development for treatments for rare diseases. COVID-19 is anything but a rare disease. Some estimates suggest that half or more of all Americans may ultimately contract the disease.

We are writing to demand you reverse course and renounce your claim to orphan drug designation privileges for remdesivir.

As you know, Gilead was able receive an orphan drug designation only by rushing to file its application while there were fewer than 200,000 COVID-19 U.S. cases.

The United States most likely will surpass 200,000 COVID-19 reported cases in a matter of days. The real number of people suffering with the new coronavirus likely already has passed this mark. Calling COVID-19 a rare disease mocks people’s suffering and exploits a loophole in the law to profiteer off a deadly pandemic.

And here's the crazy thing. It worked!

Almost immediately after that letter was sent, Gilead announced that it has asked the FDA to rescind the designation:

Gilead has submitted a request to the U.S. Food and Drug Administration to rescind the orphan drug designation it was granted for the investigational antiviral remdesivir for the treatment of COVID-19 and is waiving all benefits that accompany the designation. Gilead is confident that it can maintain an expedited timeline in seeking regulatory review of remdesivir, without the orphan drug designation. Recent engagement with regulatory agencies has demonstrated that submissions and review relating to remdesivir for the treatment of COVID-19 are being expedited.

In early March, Gilead sought and was subsequently granted an orphan drug designation for the remdesivir as a potential treatment for COVID-19. Orphan drug designation is granted by the FDA in situations where the disease affects fewer than 200,000 patients in the United States.

The notice tries to give some justification for the orphan designation, suggesting that it would have made approval easier:

Among the benefits of orphan drug designation, this status results in a waiver of the requirement to provide a pediatric study plan prior to the submission of a New Drug Application – a process that can to take up to 210 days to review.

Gilead recognizes the urgent public health needs posed by the COVID-19 pandemic. The company is working to advance the development of remdesivir as quickly as possible, and will provide updates as they become available

So, in the end, public pressure worked, and the right thing was done.

As a side note, I've seen some people point to this article to suggest that because the FDA had only designated remdesivir as an orphan drug for COVID-19, and not yet approved it, that this story was blown out of proportion. This is wrong. The designation almost certainly eventually leads to the approval, and the mere designation creates a massive chilling effect on others working in the space, as it effectively tells them not to go down that path at all. The designation was a big deal, and a terrible scam pulled off by the FDA and Gilead, and it's good to see that Gilead was realizing that it was being seen as a pandemic profiteer and has backed off.

Filed Under: covid-19, exclusivity, fda, greed, orphan drugs, pandemic, patents, public pressure, remdesivir
Companies: gilead sciences

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  1. icon
    Atkray (profile), 26 Mar 2020 @ 12:45pm

    Close the loophole

    It would be nice if congress would just add a line stating that any drug grated orphan status that subsequently climbs above 200,000 cases in the US automatically becomes public domain.

    That would probably fit better with the original intent of getting companies to research drugs for rare diseases instead of the newest disease.

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