- When a company gains approval for a rare pediatric or tropic disease drug, it receives a priority review voucher from the FDA, which allows a company to expedite the review process from 10 months to six months.
- The goal of priority review vouchers is to encourage companies to focus on rare pediatric or tropical diseases, but not all voucher holders are doing that.
- There is concern among some members of the FDA, including the New Drugs Director, Jon Jenkins, that some companies are using the vouchers for primary care treatments, which involve numerous large-scale trials and are time-consuming to review.
When the program was started in 2007, the FDA may not have realized how popular it would be. Apparently saving four months is worth hundreds of millions of dollars given the hot market for these vouchers.
Last year, BioMarin sold a voucher to Sanofi and Regneron for $67.5 million—which ultimately helped the company win approval for Praluent (alirocumab) before its competitor Amgen introduced its PCSK9 inhibitor to the market. Then, Gilead bought a voucher for $125 million, which it wants to use to expedite review for one of the drugs in its HIV pipeline. Sanofi spent $245 million for a voucher which it bought from Knight Therapeutics; and now, AbbVie has allocated $350 million to buy a voucher from United Therapeutics.
Vouchers are a hot business and the FDA has not said that they can't be used to expedite review of drugs that don't fall in the category of rare pediatric or tropical diseases. The FDA has been considering whether the current voucher program, as it exists, might compromise the agency's core mission and make it hard for them to do their jobs effectively.
For now, the priority review program will remain as it is, but the prices of the vouchers are most likely going to become more expensive over time.