Question: Assuming the results from the Rett trial are good enough for AVXL to get FDA approval, wouldn't that automatically qualify them to receive the Priority Review Voucher from the FDA?
And once they receive the voucher, couldn't AVXL use it to get expedited approval for PDD?
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The US Congress created the priority review voucher program in 2007 based on a 2006 Health Affairs paper (Ridley et al. 2006). The voucher entitles the bearer to regulatory review in about six months rather than the standard ten months. The Food and Drug Administration (FDA) awards a voucher following approval of a treatment for a neglected disease, rare pediatric disease, or medical countermeasure. Two drugs receive priority review for each voucher: the drug winning a voucher for a neglected or rare pediatric disease, and the drug using a voucher for another indication.
The voucher may be sold. For example, a small company might win a voucher for developing a drug for a neglected disease, and sell the voucher to a large company for use on a commercial disease.
The voucher program is intended to reduce two types of inefficiency. First, the voucher program motivates more treatments for neglected diseases, rare pediatric diseases, and medical countermeasures. Second, the voucher program speeds approval of potential blockbuster therapies in the US, getting US patients access to these treatments more quickly.
By moving a drug to faster review, there is the potential to slow other drugs. To provide FDA with more resources and mitigate this cost, the voucher holder must pay the FDA an additional user fee ($2,116,167 in fiscal year 2020). The FDA can include in its budget request the expectation of redeemed vouchers. For example, if the FDA consistently has 4 vouchers redeemed each year, it can consistently request an extra $10 million.
The voucher has three effects on commercial value: the competitive effect, the time-value of money effect, and the exclusivity effect (Ridley and Régnier 2016).
