Under the recently enacted Pediatric Research Equity Act (PREA), sponsors must study their developing drugs in children in particular cases.
The FDA can grant extensions for deferred pediatric studies at a sponsor’s request if there is good cause for a delay in completing the studies, the agency says. For example, if the sponsor is having difficulty recruiting enough pediatric patients to complete the study, FDA can grant a deferral extension.
However, if a sponsor fails to seek or obtain a deferral extension, fails to submit deferred pediatric studies by the final due date, or fails to request approval for a required pediatric formulation, the FDA can send a non-compliance letter to the sponsor and publish the letter and the company’s response online.
CRO Uptick?
This latest move by the FDA may end up further encouraging companies to speed up their pediatric trials, which may in turn lead them to seek out a CRO that can expedite the process.
“There’s definitely increased interest in setting up these clinical trials,” Fabio Thiers, MD, PhD, CEO at ViS Research Institute, told Outsourcing-Pharma.com.
In collaboration with the industry group BIO, ViS is using its online analytics platform to work with sponsors and CROs to evaluate global pediatric clinical research infrastructure, identify pediatric patient populations and empower more collaborations.
“We’re basically using our technology to capture the infrastructure on running worldwide pediatric trials,” Thiers said, noting the tech can help sponsors adjust to find where pediatric trials are growing and “where there’s more or less saturation,” so that sponsors and CROs can meet these deadlines.
Example of Genzyme
The most recently posted letter to the FDA’s website was sent to Genzyme in April after the company failed to submit a pediatric assessment for its chronic kidney disease drug Renvela (sevelamer carbonate), though the deadline was set for 2009.
Genzyme requested a deferral extension on December 5, 2012, but the FDA said that its request does not qualify for an extension.
In its response, Genzyme said that a final protocol for the trial was agreed upon after an amendment was submitted in November 2011. Genzyme also said it remains committed to conducting the trials and in May 2012, the first patient consented for the study. Currently there have been 40 patients randomized in the US and clinical sites in Europe are preparing for start-up.