{"id":3238,"date":"2024-06-10T11:22:36","date_gmt":"2024-06-10T15:22:36","guid":{"rendered":"https:\/\/cytel.agencyukdev.com\/perspectives\/new-fda-guidelines-on-pediatric-studies-and-potential-effects-on-opportunities-for-market-exclusivity\/"},"modified":"2024-08-22T05:56:39","modified_gmt":"2024-08-22T09:56:39","slug":"new-fda-guidelines-on-pediatric-studies-and-potential-effects-on-opportunities-for-market-exclusivity","status":"publish","type":"perspectives","link":"https:\/\/cytel.agencyukdev.com\/fr\/perspectives\/new-fda-guidelines-on-pediatric-studies-and-potential-effects-on-opportunities-for-market-exclusivity\/","title":{"rendered":"New FDA Guidelines on Pediatric Studies and Potential Effects on Opportunities for Market Exclusivity"},"content":{"rendered":"

Legislation on pediatric studies has existed for more than 20 years in the US, yet additional guidance from the FDA has been relatively scarce. However, two new draft guidelines have now been published, including policy changes that may affect current opportunities for pediatric exclusivity and when data may be extrapolated from adults. How might this affect pediatric drug development?<\/p>\n

What has the regulatory framework looked like so far?<\/strong><\/p>\n

To improve the safety and efficacy of drug use and dosage for children, two acts were passed in recent decades to encourage drug companies to document their products for use in children. The first \u2014 the Best Pharmaceuticals for Children Act (BPCA) of 2002 \u2014 incentivized the inclusion of children in clinical studies by offering market exclusivity, becoming a \u201ccarrot\u201d for drug developers. However, it didn\u2019t have the intended effect quickly enough, so Congress later developed a \u201cstick\u201d: the Pediatric Research Equity Act (PREA) of 2003, which gives the FDA<\/a> the authority to require pediatric studies in certain cases.<\/p>\n

PREA affects the majority of pharmaceutical projects, though some products are automatically exempted, and companies can apply for waivers or deferrals for certain product types and indication areas. In all other cases, pediatric studies must be carried out, though studies in children are typically started with some lag compared to studies in adults.<\/p>\n

However, for sponsors that voluntarily conduct pediatric studies, the BPCA offers six months extra pediatric market exclusivity. Keep in mind, the six months can be added to any existing market exclusivity and patent periods.<\/p>\n

Some requirements must be met to enable pediatric exclusivity. The FDA<\/a> must have issued a Written Request (WR), something the company can request. The WR describes which studies are to be carried out and within which time frames. The company must then conduct the pediatric studies and submit study reports to the FDA in accordance with the agreed time frames. However, positive results in the studies are not required for exclusivity.<\/p>\n

 <\/p>\n

What\u2019s new? Announced changes that may affect market exclusivity<\/strong><\/p>\n

Intended to replace the previous \u201cHow to Comply with the Pediatric Research Equity Act\u201d of 2005, the new draft guidelines published May 2023 include:<\/p>\n