Regulatory Open Forum

Date of approval of the application: see Q13 in the draft guidance on this subject, noting that the dates listed are out of date due to extension of the program by Congress (which you mentioned in the original post - the approval cutoff date is now the end of FY 2026). However, the guidance notes (emphasis added) "FDA may not award a voucher after September 30, 2020, unless the application is for a drug that was designated as a drug for a rare pediatric disease by September 30, 2020 and the application is approved by September 30, 2022." This is due to the fact that the voucher is issued at the time of approval (and included in the approval letter).

Note that if a given application is eligible for a rare pediatric disease priority voucher, it would also be eligible for priority review itself (also covered in the aforementioned guidance) so it would have a shorter review period which could help in meeting that approval deadline.

Additionally, while one cannot count on further extension by Congress, it is notable that the program has now been extended multiple times (first by the 21st Century Cures Act in 2016 then again in late 2020 as noted above - despite significant pushback from some organizations and researchers), so it is certainly possible it will be extended further.

Please provide the page number in the guidance document for the following details you mentioned in second paragraph (bold and underlined text) as I am unable to find them

"Note that if a given application is eligible for a rare pediatric disease priority voucher, it would also be eligible for priority review itself (also covered in the aforementioned guidance)........"

Date of approval of the application: see Q13 in the draft guidance on this subject, noting that the dates listed are out of date due to extension of the program by Congress (which you mentioned in the original post - the approval cutoff date is now the end of FY 2026). However, the guidance notes (emphasis added) "FDA may not award a voucher after September 30, 2020, unless the application is for a drug that was designated as a drug for a rare pediatric disease by September 30, 2020 and the application is approved by September 30, 2022." This is due to the fact that the voucher is issued at the time of approval (and included in the approval letter).

Note that if a given application is eligible for a rare pediatric disease priority voucher, it would also be eligible for priority review itself (also covered in the aforementioned guidance) so it would have a shorter review period which could help in meeting that approval deadline.

Additionally, while one cannot count on further extension by Congress, it is notable that the program has now been extended multiple times (first by the 21st Century Cures Act in 2016 then again in late 2020 as noted above - despite significant pushback from some organizations and researchers), so it is certainly possible it will be extended further.