FDA conference highlights: Agency focuses on pediatric therapies to treat rare diseases

In a monthly FDA-focused blog published exclusively by The Pharma Letter, Dr Nicola Davies looks the continuing impact of the FDA 2014 conference.

On January 6, 2014, the US Food and Drug Administration held a workshop aiming to develop procedures and protocols that fast track promising and risky treatments for rare diseases. The workshop fulfills a requirement that Congress placed on the agency, when it passed the Food and Drug Administration Safety and Innovation Act in 2012. That Act requires that the FDA host public meetings for the purpose of accelerating the development of therapies designed to address pediatric rare diseases (PDRs), and issue a report encouraging such therapies. The FDA released this report in July of 2014.

The January 2014 workshop was spread over two days and the FDA invited internationally recognized medical leaders, scientists, researchers and practitioners to educate colleagues, patients, caregivers, practitioners and other stakeholders. Richard Moscicki, a pediatric expert and the deputy center director for Science Operations at the FDA, forcefully espoused a pediatric focus. He also presented a report noting that the USA continues to lead the way in experimental protocols. In 2013, 72% of novel drugs approved by US regulatory agencies went on to receive approval from internationally recognized medical oversight bodies, as well as the local FDA equivalent, in most industrialized nations around the world.