Replies to post #219082 on Anavex Life Sciences Corp (AVXL)

[makemydaze]

Thanks for that info Roy - I was wondering why the FDA would throw that voucher bone out there:

"The voucher program is intended to reduce two types of inefficiency. First, the voucher program motivates more treatments for neglected and rare diseases. 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)."

https://www.priorityreviewvoucher.org/

[Stockdoggy]

Very interesting, thanks for that link. The priority voucher really is rare-chart shows only 32 have been given since the program started in 2007.

[polarbear77]

Thanks for that chart Roy showing the 32 PRV issuances by FDA since 2007 passage.

Here’s updated FDA guidance from
7/29/19:


https://www.fda.gov/news-events/fda-brief/fda-brief-fda-updates-draft-guidance-rare-pediatric-disease-priority-review-voucher-program

““Despite continued progress, too many rare diseases still have no approved therapies. One of the FDA’s top priorities is to provide incentives and clear advice to medical product developers so that they can advance innovations that can help address the significant unmet medical needs for patients with rare diseases. This holds especially true in rare diseases that impact children,” said Janet Maynard, M.D., director of the FDA’s Office of Orphan Products Development, which along with the Office of Pediatric Therapeutics, oversees the Rare Pediatric Disease Designation program. “One program in place intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases is the Rare Pediatric Disease Priority Review Voucher Program. Under this program, a drug company that receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. To provide clarity and advice to drug developers, today we are issuing an updated draft guidance about how this program works. This updated draft guidance reflects input from rare disease and pediatric experts from across the agency, highlighting our commitment to addressing the needs of patients with rare diseases. When finalized, this draft guidance will provide the agency’s current thinking regarding the updated definition of rare pediatric disease and explain the statutory requirements to earn a rare pediatric disease priority review voucher.””