FDA's Priority Review Voucher Program is Broken | Pharmaguy's Insights Into Drug Industry News | Scoop.it

There isn’t much profit in drugs or vaccines targeting rare diseases, especially when those rare diseases afflict people who don’t have a lot of money.

Since 2007, the US government has attempted to draw companies into this sparsely occupied space by creating an incentive program. Manufacturers who license products for specified “neglected” diseases are granted the right to an expedited Food and Drug Administration review when they want to bring another product to market.

It seems like a good idea — and may still be. But a number of groups that supported the program aren’t happy with how it is playing out so far.

The program is called the Priority Review Voucher Program for Neglected Diseases. When it was created, it covered specific diseases — among them, leprosy, malaria, and schistosomiasis and onchocerciasis, known as river blindness.

The vouchers don’t have to be used by the companies that earn them; they can be sold. Originally they could only be sold once. Last year that rule was changed and now a company that buys a voucher can resell it at a later date.

David Ridley and some colleagues at Duke University’s Fuqua Business School dreamed up the priority vouchers scheme — they published an articleproposing it in Health Affairs in 2006. Ridley always hoped the vouchers would earn big money for companies that receive them. And they have.

The first sold for $67.5 million. The next went for almost double that, $125 million. Then $245 million. In August, United Therapeutics sold a priority review voucher to Abbvie for $350 million. The others remain unused and if they have been sold, it hasn’t yet been disclosed.

But more vouchers mean there are more drugs for neglected diseases, right?

Not really. And that’s the problem.

Some of the vouchers have been awarded to drugs that have been on the market in other parts of the world for years, but have just been put through the licensure process in the United States. In fact, the first voucher was awarded to Novartis in 2009 for a combination malaria drug that had been licensed outside the US since 2001 and was already widely in use.

“This was not in any way an actual new treatment,” said Rachel Cohen, regional executive director for the North American division of the Drugs For Neglected Diseases Initiative. She noted the Rare Pediatric Diseases program does require that a treatment is novel in order to earn a voucher.

Another problem: Drug makers that earn priority review vouchers don’t have to guarantee that the drugs will actually be available, or sold at an affordable price. Janssen was awarded a voucher for Sirturo, used to treat multidrug resistant tuberculosis. Cohen said the drug is prohibitively priced in many parts of the world.