Gilead has asked the Food and Drug Administration to rescind a special regulatory status for its experimental coronavirus drug, but not before stirring up a controversy that had critics accusing the company of manipulating a law designed to spur research into therapies for rare diseases.
So-called Orphan Drug status, which the FDA granted Monday, would have limited competitors from filing applications for generic versions of Gilead's drug for seven years. In addition, Gilead would have qualified for tax credits to defray the costs of developing the drug, called remdesivir and currently in testing as a treatment for COVID-19.
Gilead's quick reversal — the biotech requested the FDA designate remdesivir an orphan drug earlier this month — came after some industry watchers noted the number of patients likely to be infected by the new coronavirus could easily exceed the 200,000-patient threshold the FDA uses to define orphan diseases. The FDA's designation also raised questions about how much Gilead might charge for remdesivir.
“Here's an opportunity for Gilead to come out smelling like roses, and instead they've just created a stink,” said Tahir Amin, co-founder of I-Mak, an advocacy group seeking patent reforms, in an interview.
Gilead has maintained that it does not currently view remdesivir as a commercial opportunity for the company. "We are committed to making the medicine both accessible and affordable to governments and patients around the world," Gilead said in a statement.
At a press conference earlier this month, Gilead CEO Daniel O'Day said the company hasn't yet been discussing pricing with governments.
In its statement, the company also said it is "waiving all benefits that accompany the designation," and believes the trial and review process will be expedited without the additional benefit of an Orphan Drug designation.
Remdesivir was originally intended as a treatment for the Ebola virus. Gilead, with consent from government health agencies, rushed it into trials in patients with severe cases of coronavirus disease, called COVID-19, citing preclinical findings that it's active against the SARS and MERS viruses, which share genetic similarities.
Five mid- to late-stage studies in the U.S., China and elsewhere in Asia are currently ongoing, with the first results expected next month. In anticipation of potentially obtaining positive results, Gilead has begun manufacturing remdesivir to prepare for huge demand, at a cost of tens of millions of dollars to the company, O'Day said at the press conference this month.
Orphan Drug status protects treatments from generic competition, gives a 50% tax credit for research & development costs and offers a streamlined FDA approval process that allows for small datasets. It's not clear whether the designation would have protected remdesivir for longer than its patents would.
The designation was created through a 1983 law. But because many drugs with massive sales, ranging from Novartis' cancer drug Gleevec to AbbVie's Humira, the best-selling pharmaceutical in the world, have secured Orphan Drug designations, many advocates have called for reform of the law.
In the case of a fast-spreading pandemic, the need to speed research and development and ensure broad access may call for a different set of regulations that would need to be enabled with new laws. I-Mak's Amin, along with Christopher Tang, a professor at the University of California, Los Angeles, suggest a "public good" model that would allow governments to license experimental drugs from companies and fast-track research, approval and distribution.
"This is a wake up call," Tang, who specializes in global supply chain management, said of the pandemic. "I fear these pharmaceutical companies would restrict the availability and jack up the price."