News Feature | July 14, 2014

FDA Looks To Further Develop Pediatric Safety, Efficacy

By Nick Otto


It’s been a busy few weeks for the FDA, and the agency recently released another forward-thinking plan on ways it plans to enhance the safety and study of products used in treating pediatric populations.

As part of the Food and Drug Administration Safety and Innovation Act (FDASIA), the agency is required to take two actions furthering the development of new therapies in children: Hold stakeholder meetings to consider new ways to inspire better ways of developing pediatric therapies to treat rare diseases; and issue a report that includes a strategic plan for achieving this goal.

PMLiVE points to several key findings from a recent FDA stakeholder meeting, including the need for “more comprehensive information about the natural history of rare diseases, greater involvement of patients and caregivers in the therapeutic development process and – potentially – willingness for very sick children and their families to accept a greater level of risk.”

In an FDA Voice blog post, the agency says it plans to focus on four primary objectives through their Strategic Plan for Accelerating the Development of Therapies for Pediatric Rare Diseases:

  • Enhance foundational and translational science for pediatric rare diseases
  • Strengthen communication, collaboration, and partnering for pediatric rare diseases within and outside FDA
  • Advance the use of regulatory science to aid clinical trial design and performance for pediatric rare diseases
  • Enhance the FDA’s review process for pediatric rare disease products

The Regulatory Affairs Professionals Society adds that the FDA, in a fifth objective, vows to reconsider current safety and efficacy standards in favor of a more patient-centric approach.

“We also plan to … establish a patient engagement panel as part of the medical device advisory committee process,” Jill Hartzler Warner, associate commissioner for special medical programs at the FDA, wrote in an FDA Voice blog post.

The FDA also recently released its five-year plan with an overall focus on safety, as well as a globalized regulatory approach.