The International Council for Harmonization has identified the key scientific issues pediatric drug developers must address early on to improve the prospects for a drug’s approval later in the development process, in a draft that updates a 16-year-old efficacy guideline.

In the past decade-and-a-half, both the U.S. and the European Union have separately mandated that drugmakers perform studies in relevant pediatric populations when pursuing a new adult indication, and these development programs are becoming increasingly global in the pursuit of larger sample sizes.

To streamline development, the ICH is recommending that sponsors begin working early to reach consensus with international regulatory agencies, and address the following issues: identifying the key pediatric populations and subgroups; the methodological approaches and specific design elements that can be considered; whether juvenile animal studies will be needed; and what gaps exist in knowledge of developmental physiology, disease pathology, and data in adult populations or from related compounds.