Multidrug-resistant pathogens cause severe infections with substantial morbidity and mortality in neonates, infants and children, as they do in adults. However, the approval of therapies for multidrug-resistant infections in paediatric patients often lags years behind approval for adults, which creates an untenable situation for people who care for affected children. Many issues contribute to the current unacceptably long period between adult and paediatric new drug approval, including the requirement for sequential age group enrolment in clinical trials, the types of regulatory studies required and a lack of alignment between regulatory authorities. To streamline paediatric drug development, more efficient approaches to investigational and approval processes that meet acceptable safety and efficacy metrics are required. For products that address an urgent unmet need in children, approval should be based primarily on matching drug exposure to levels in adults and on extrapolating safety and efficacy data from adults, where appropriate, rather than on requiring clinical trials in all paediatric age groups for each approved indication in adults. In addition, real-world and post-approval data could be used to support early, limited drug approval for paediatric patients. However, there are economic challenges in developing anti-infective drugs for paediatric populations and increased funding is needed to ensure that neonates, infants and children globally have timely access to safe, effective and life-saving therapies.

Traditional epidemiological surveillance methods are often limited by delays in reporting and fragmented data systems. Saudi Arabia faces additional public health challenges from mass gatherings during Hajj and Umrah, an increasing burden of noncommunicable diseases and rapid urbanization, highlighting the need for investing in digital epidemiology.

Madagascar has a high number of unvaccinated (zero-dose) children. In 2023, an estimated 74% received any dose of the diphtheria-tetanus-pertussis (DTP) vaccine. In southern Madagascar, service gaps, poverty, drought and limited access leave many children unreached by routine immunization.

Fragmented primary health care in China fails to tackle the growing burden of noncommunicable diseases. Despite substantial investment, fewer than half of patients with diabetes achieve glycaemic control.

The World Health Organization recommends a benchmark for uncorrected visual acuity of ≥ 6/12 in 80% of patients at 4 to 6 weeks following cataract surgery. In low-resource countries, reaching this benchmark is challenging and assessing progress is difficult.

Umbrella protocols have recently come to be widely used in clinical trial designs. However, the value of this approach in public health is less well recognized. The coronavirus disease 2019 (COVID-19) pandemic highlighted the need for rapid, reliable and scalable evaluation of diagnostic technologies. In the United Kingdom of Great Britain and Northern Ireland, this need prompted the development of an umbrella research protocol enabling multiple clinical evaluation studies of similar designs to be undertaken under a single overarching preapproved ethics and governance framework. We describe the development, implementation and evolution of this protocol, which was designed to support timely assessment of the performance of in vitro diagnostic devices and associated testing approaches in various settings. The umbrella protocol allowed studies to be started quickly during the pandemic, reduced administrative burdens, supported regulatory submissions and enabled prospective collection of samples for surveillance. While the system described reflects British governance structures, the principles underpinning this approach, including proportionality (ensuring oversight requirements are appropriate to the risk level), standardization and preapproved flexibility, are applicable to many settings. The protocol now forms part of the United Kingdom's wider pandemic preparedness structure and illustrates how preapproved, adaptable research frameworks can accelerate evidence generation during outbreaks. The world is now assessing lessons from the COVID-19 pandemic and it is timely to consider how research systems can support innovative designs such as umbrella protocols. We therefore summarize lessons learnt and practical considerations to support other countries seeking to adopt similar approaches within their own ethical and regulatory systems.