Lagging pediatric safety and effectiveness data increase the risks to children associated with off-label drug use. The objective of this study was to delineate the frequency of, and reasons behind, delays in the completion of mandated pediatric postmarketing requirement (PMR) studies. Publicly accessible and internal US Food and Drug Administration (FDA) data were aggregated to characterize pediatric PMRs issued from 2012 to 2024, including relevant dates, durations, and deferral extension (DE) requests. Sponsor size, and clinical trial enrollment status were also examined. There were 1160 pediatric PMRs identified, 459 of which were associated with 1176 DE requests. Despite a significant decline in the annual number of PMRs issued (slope [95% confidence interval]: -3.64 [-6.50, -0.79] year-1, p = 0.02), an increase in the annual number of DE requests (5.24 [2.16, 8.31] year-1, p < 0.01) was observed. Declines in negotiated timelines for PMR completion and durations of time granted per request were also seen. Issues related to clinical trial conduct predominated DE rationale in every year and accounted for 48% of all requests. One in 10 delayed trials failed to initiate enrollment before the PMR due date and 1 in 5 cited problems with clinical trial conduct before enrollment began. Smaller companies (<500 employees) exhibited higher DE request rates, lower PMR success rates, and accounted for an increasing proportion of PMRs over the review period (from 17% in 2012 to 72% in 2024). Year-on-year increases in DE requests reinforce ongoing challenges to timely and successful pediatric trial completion. Difficulties with clinical trial conduct appear to be the primary driver for these delays although the underlying root causes have yet to be elucidated. Regulators, sponsors, and stakeholder partners should work cooperatively to remediate delayed pediatric drug development timelines and reduce the duration of off-label drug use in children.

The role of Medical Information in the pharmaceutical industry is undergoing a profound transformation, driven by evolving stakeholder expectations, digital innovation, and the increasing complexity of therapeutic landscapes. Traditionally perceived as a reactive function focused on providing accurate and timely responses to healthcare professionals (HCPs), AstraZeneca's Medical Information department has now evolved into a proactive, strategic partner within Medical Affairs (MA). This shift has been accelerated by the COVID-19 pandemic, which amplified the demand for rapid, accessible, and reliable medical information among HCPs, regulatory agencies, patients, and caregivers. This article describes the transformation of AstraZeneca's Medical Information department, highlighting the integration of advanced digital platforms, artificial intelligence (AI)-enabled tools, and self-service channels that enhance the efficiency, consistency, and accessibility of responses. Initiatives, such as the medical information leaders in Europe centralized hub and the Medical Information website, exemplify how technology enables the real-time, multi-channel delivery of evidence-based content. In parallel, Medical Information processes have been standardized to ensure regulatory compliance, uphold ethical standards, and maintain the quality and personalization of interactions. Beyond information provision, Medical Information now functions as an analytical hub, systematically capturing, synthesizing, and interpreting insights from medical inquiries and other MA touchpoints. These insights inform the refinement of core medical messaging, support evidence generation priorities, and identify knowledge gaps or misconceptions within the healthcare community. The department's cross-functional collaboration with Regulatory Affairs, Pharmacovigilance, Market Access, and Commercial ensures that global scientific content is adapted to local healthcare realities, strengthening both internal alignment and external engagement. Key performance indicators (KPIs) have been implemented to measure Medical Information's impact, including enquiry volume trends (related to the number of requests), digital adoption rates (such as the volume of interactions per digital channel), stakeholder satisfaction (measured through qualitative feedback collected in surveys), and insight utilization (i.e., number and impact of generated insights). Such metrics enable continuous performance monitoring and facilitate data-driven decision making. Looking ahead, the Medical Information function is poised to further integrate real-world evidence, expand multi-channel communication capabilities, and deepen its role as a "listening post" for unmet needs and patient-centric innovation. By combining operational agility, technological innovation, and scientific expertise, Medical Information can continue to enhance informed decision making, improve patient outcomes, and deliver sustained value to the healthcare ecosystem.

Abstract Artificial intelligence (AI) is being applied across the pharmaceutical and health tech sectors, The chapter provides insight into the application of AI and the emerging regulatory framework and available guidance at the EMA and the MHRA. The five key principles for regulatory use of AI are covered alongside descriptions of the guidance documents of SPIRIT AI and CONSORT AI

Abstract T-tests are useful for comparing the means of two samples. The paired and unpaired approaches are discussed.

Abstract A description of the Scottish Intercollegiate Guidelines Network (SIGN) evidence-based clinical practice guidelines for the NHS in Scotland are provided, including the guideline development process.

Abstract Confidence intervals show the range within which the true treatment effect is likely to lie. Information on how to calculate and interpret a confidence interval are included.

Abstract Clinical data management (CDM) is the process of collecting, cleaning, and management of subject data in compliance with regulatory standards. Regulatory guidance is covered with a focus on the data management plan and data validation plan.

Abstract The requirements and potential incentives on offer for development of medicines for the paediatric population are described for Europe (paediatric investigation plan, PIP; USA, Best Pharmaceuticals for Children Act).

Abstract Tools and practical tips to deliver effective presentations are described including understand your audience, having a logical structure, use of simple language and visuals and practice.

Abstract Health economics is the formal analysis of direct and indirect costs and benefits that are a consequence of a healthcare intervention, programme, or strategy. The key elements of economic valuation in healthcare are described: cost-minimization analysis, cost-benefit analysis, cost-effectiveness analysis, and cost-utility analysis.