Members of the Senate's health committee voted Wednesday in favor of a bill that would add Zika to the list of 21 neglected, rare tropical diseases eligible for a voucher to fast-track approval by the U.S. Food and Drug Administration.
Sen. Susan Collins (R-Maine), however, said the voucher program needed fixing to make sure it wasn't exploited by drugmakers.
The FDA's Priority Review Voucher program was launched in 2007 to incentivize companies to develop treatments that might not result in high profits. The group of diseases included in the program mostly affect poor people. The voucher reduces a drug's review time from the usual 10 months to six. In 2012, a second review voucher program was created for rare pediatric diseases.
But companies can obtain a voucher even if they are not actually developing a drug. There is also nothing that prohibits a company from seeking approval for a drug that has already been marketed and used outside of the U.S.
Such was the case recently when pharmaceutical firm KaloBios, under the direction of former CEO Martin Shkreli, planned to purchase the rights to sell the generic drug benznidazole, a medicine used to treat Chagas disease. Though the drug had been used for years throughout South America at a cost of about $100 for a course of treatment, the company announced it would seek FDA approval to sell the same drug in the U.S., and raise the price to $100,000.
The company's true incentive became clear once it announced it would ask the FDA to award them a priority review voucher, since Chagas is one of the eligible diseases.
“There's a problem in the way the present system works that people like Martin Shrkeli are exploiting,” Sen. Collins said. “We really need to fix this problem before this bill goes to the floor.”
Since 2009, a total of nine vouchers have been awarded, three of which were for tropical diseases and six under the pediatric program. The majority of those vouchers have been sold to other drug firms for hundreds of millions of dollars.
The first sale of a voucher occurred in 2014, when drug makers Sanofi and Regeneron purchased a voucher held by BioMarin for $67 million that the companies redeemed to expedite review of its new cholesterol-lowering drug Praluent, which was approved last July. Since then the price has gone up, with the most recent sale occurring last August in a deal valued at $350 million.
Also in 2014 Knight Therapeutics was awarded a review voucher for its drug miltefosine to treat the parasitic disease Leishmaniasis. It later sold the drug to Gilead Sciences for $125 million. The drug had been in use in other countries for years and Knight had simply been the one to register it for use in the U.S.
The FDA earlier this month suggested the voucher program, at least for pediatric diseases, should end because was not useful in sparking innovation. The Government Accountability Office stated it was “too early” to determine the effectiveness of the pediatric voucher program. The report made no mention of the tropical disease voucher program.
In fact, experts have pushed the FDA to add requirements that would call on companies to report on the affordability of the drugs developed through a voucher.
“Our observation of the mechanism is that it has important shortcomings in its design,” said Judit Rius Sanjuan,U.S. legal policy adviser for Doctors Without Borders.
In November, the group was one of seven medical organizations that signed a letter asking the FDA to reject vouchers for products that were not new and already used in other countries.
She said making changes to the program would be a way of separating "the good apples from the bad apples."