We are experiencing an exciting and unprecedented period in the history of children’s medicines globally. Milestone developments unfolding in recent years include the formation of the International Alliance for Better Medicines for Children in 2006 and landmark initiatives by the EU, the World Health Assembly (WHA), and the WHO in 2007. However, the challenges of optimizing the development, wider availability, and routine use of effective, safe, and affordable medicines addressing important child health needs are considerable. Each aspect of this continuum has so far received differential attention. Major initiatives in the US and EU have focused on stimulating research into children’s medicines, largely driven by drug regulatory reforms, but with important gaps remaining. Many countries are lacking similar reforms, so the benefits of these initiatives are currently not well reflected in the rest of the world. A systematic approach to knowledge translation to improve use of best evidence and deliver quality use of medicines (QUM) to children routinely has also been largely a ‘missing link’ so far. The WHO’s Essential Medicines List for Children and related initiatives are addressing improving children’s access to needed medicines. Priority research gaps, especially in the developing world, are also being pursued by the WHO. However, in many countries, including developed nations such as Australia, the policy response to the WHA resolution on ‘Better Medicines for Children’ has been inconsistent or fragmented. A better integrated overall approach, linking global medicines research efforts to child health needs and actual medicines use is needed. International networking to support the conduct, synthesis, and rapid dissemination of pediatric medicines research will help close knowledge gaps at a global level. Harmonization of pediatric drug regulation will support this goal and facilitate improving access to needed medicines. Increasing research into and dissemination of effective strategies to promote QUM is an essential component to maximize return (in health benefits) on increased investment in medicines research. A greater focus on QUM should also help create demand for better evidence from clinicians. Delivering on the promise of better medicines for children, wherever in the world they may be, depends on achieving successful integration between the science, the policy, and the practice of pediatric medicines.