As we know, many drug products are approved initially only for adult patients. The Pediatric Research Equity Act (PREA) of 2003 “gives FDA the authority to require pediatric studies in certain drugs and biological products. Studies must use appropriate formulations for each age group. The goal of the studies is to obtain pediatric labeling for the product.” This is especially important for drugs where the extrapolation from adult to pediatric patient may not be straightforward. The Best Pharmaceuticals for Children Act (BPCA) “provides an incentive of additional marketing exclusivity to sponsors who voluntarily complete pediatric clinical studies outlined in a Written Request issued by FDA.” The exclusivity period awarded is 180-day added to any existing patent or exclusivity period for the sponsor at the time the pediatric exclusivity is awarded.
Lately there does not seem to be a lot of action with regards to the written requests or the pediatric exclusivity awards. This may be due to the more rigorous enforcement of PREA where pediatric studies are required when the original application is submitted. Anyway, an updated list of pediatric exclusivity determinations was just published by FDA a few days ago and I took a look to see what was new in this particular regulatory minefield. It turns out that there was only one addition to the list (here) for Blinatumomab, a drug to treat Acute Lymphocytic Leukemia (ALL) and the determination for the award of pediatric exclusivity was made on June 13, 2023. The list now contains a copy of the written requests (WR) and any amendments to the WR for all WRs made after September 27, 2007. Older WR can be viewed in the FDA archives, but the amount of information may not be as extensive as those on the current listing.
The FDA correspondence with the sponsors may provide valuable information regarding how the BPCA program works in practice. But the interesting issue from my perspective is that there have only been 16 determinations made in all of 2022 and through today in 2023. When BPCA came into play, it seemed as if there were pediatric exclusivity awards on almost every product and generic firms were always scrabbling for information about whether they were going to get approved on a specific date of patent or exclusivity expiration or if a pediatric exclusivity determination would put their launch off for another 180 days.
Well, this is today and BPCA is still in play, but somehow it does not seem to be as great of a problem for generics firms because of regulatory changes in the timing required to obtain written requests prior to patent or exclusivity expiration firms, except, of course, if it impacts one of your applications!
