Legislators and regulators on both sides of the Atlantic have spent the past decade trying to improve access to safe and effective pediatric formulations and drug products—but it’s unclear whether pharma companies are taking notice.
The US Congress urged pediatric studies with the 1997 FDA Modernization Act, followed by the 2002 Best Pharmaceuticals for Children Act and the 2003 Pediatric Research Equity Act. In 2007, the FDA Amendments Act extended and amended these regulations so that every new drug application, unless granted a waiver or deferral, must include pediatric testing and evaluation. Europe followed suit by requiring, as of July 2008, that all new products contain the results of studies based on a pediatric investigation plan.
One of the new US stipulations involves alerting the public about available pediatric formulations that have not reached the market. If a drug sponsor has not introduced a pediatric formulation that was found to be safe and effective for children to the market within one year of receiving pediatric exclusivity, FDA has the authority to publish information about the drug product formulation. Unfortunately, according to the FDA pediatric drug development web page, it looks like this is starting to happen.
In February 2009, Wyeth published that it had found a pediatric formulation to be safe and effective for Protonix (pantoprazole sodium) for delayed release oral suspension. A year later, that formulation is still not on the market.
I hope this is not the start of a trend in which companies simply complete the required testing for pediatric populations and then leave their discoveries on the lab floor. Children in need of certain medication would be denied access because a company has chosen, perhaps, to focus on the adult formulation (that is, the main revenue source). It would be no surprise if this were to become common practice—the bottom line is the priority in any for-profit business—but it’s sad when sick children bear the consequences.
See related article, “Pediatric Formulations: Technical and Regulatory Considerations”