“However small the chance might be of striking lucky, the chance was there.”
― Roald Dahl, Charlie and the Chocolate Factory
Big money needed for a small market
How does a rare disease company chasing a treatment for disease with just a handful of patients come up with a compelling commercialization plan that would convince investors, grant reviewers etc, that there is a pot of gold at the end? That is the challenge for many small companies (such as my own).
Everyone is well aware that drug development is incredibly costly. It can be in the billions of dollars. For drugs that treat major diseases like diabetes or hypertension, the investment is certainly worth it. But what about ultra rare diseases that only have a handful of patients?
These are not blockbuster diseases, but examples of precision medicine, treatments almost tailored to the individual. Treatments that could also be transformational to the patients and their families. Money has always been a good incentive to get people to do something. But instead of providing cash, the government provides a piece of paper, a priority review voucher which enables the winner to use it for another of their drugs to be reviewed under FDA's priority review system. This therefore acts as a way to speed up drug reviews also. This is essentially a ‘golden ticket’ because if applied to one of those blockbuster drugs, it could give a 3-month head start to the market which would add hundreds of millions of dollars in revenue.
The golden tickets
These vouchers were initially developed to work on treatments for selected tropical diseases in 2007. Then in 2012 the Rare Pediatric Disease Priority Review Voucher was created under the Food and Drug Administration Safety and Innovation Act (FDASIA)
. A "rare pediatric disease" in this case is defined specifically as one which "primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children and adolescents," and is a rare disease according to federal statute (200,000 persons in the US or fewer).
These vouchers can be used by the winner or sold to others for their use or resale. Which is exactly what has happened in most cases. The first rare pediatric disease voucher was awarded to BioMarin in 2014 and they sold it to Sanofi and Regeneron for $67 million (Figure 1). The second voucher was awarded to United Therapeutics in 2015 and is so far unused. In 2014, Knight Therapeutics sold their tropical disease priority voucher for $125 million. The third pediatric voucher was awarded in 2015 to Asklepion Pharmaceuticals, but was passed on to Retrophin when they bought the company. Their voucher was then sold to Sanofi for $245 million. It would appear that the price of these vouchers has skyrocketed and this could be due to their scarcity.
These golden tickets may not last forever
contained a clause which limited the FDA to awarding as few as 3 of the pediatric disease vouchers. The FDASIA
legal wording writes "[FDA] may not award any priority review vouchers … after the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section". In other words, this means the Rare Pediatric Disease Priority Review Voucher program will formally end on March 17, 2016—1 year after Retrophin received the priority review voucher—unless Congress takes additional action.
21st Century Cures Act extends the voucher program
So what needs to happen for the voucher program to continue? There is legislation now being considered in the US House of Representatives (the 21st Century Cures Act
), which would extend the Rare Pediatric Disease Priority Review Voucher system for 3 years.
The extension would only apply for rare pediatric diseases which are serious or life-threatening. Further, the new legislation would not allow companies to double dip (obtain both a tropical disease voucher and
a pediatric voucher for the same drug/ disease).
Of course the irony of all of this is that here I am as a biotech, using the hope of eventually winning a golden ticket as the bait to get funding from venture capitalists and the NIH to develop a treatment for a pediatric rare disease that dramatically shortens the lives of those affected. I believe this is exactly what the Rare Pediatric Disease Priority Review Voucher was set out to do. And yet the very uncertainty of whether it will continue undermines the very effort itself and the dreams of all of those in a similar position trying to develop treatments for the few.
The Golden ticket is the last chance to strike it lucky for those who are determined to find treatments for rare diseases but do not have the millions or billions of funding of a biotech or pharma, respectively.