Drug development for children presents unique challenges and is highly regulated. Novel approaches, such as the use of extrapolation to address for example the need to avoid unethical studies, whilst supporting robust evidence generation have been developed in support of benefit/risk considerations by regulatory authorities. This is only one step in the decision-making process towards access, which in Europe also includes health technology assessment (HTA) bodies. Discussions related to evidentiary requirements in small populations through the use of evidence transfer has been identified as a priority action by EMA/ EUnetHTA 21. We describe the outcome of this work; reflect on the discussions taken place how to leverage prior knowledge through identifying and addressing uncertainties during life cycle management to support regulatory and HTA decision making. Through examples, we discussed the spectrum of use to support evidence generation, and developed regulatory and HTA reflections on general design considerations important for robust evidence generation; reflective of the joint ambition. Early interactions with all respective stakeholders, particularly between regulators and HTA bodies are key to optimise data generation and utility in children. In Europe, the HTA regulation will offer opportunities for collaborations, which are important for all development efforts. We collaboratively explored the unique specific challenges relating to paediatric drug development, ethically and in its ability to leverage prior knowledge, as exemplified using extrapolation. Learnings from these offers opportunities to further develop methodology how to leverage uncertainties across a product’s life cycle for small populations generally.

Purpose: There is increasing recognition of the importance of transparency and reproducibility in scientific research. This study aimed to quantify the extent to which programming code is publicly shared in pharmacoepidemiology, and to develop a set of recommendations on this topic. Methods: We conducted a literature review identifying all studies published in “Pharmacoepidemiology and Drug Safety” (PDS) between 2017 and 2022, Data was extracted on the frequency and types of programming code shared, and other key open science practices (clinical codelist sharing, data sharing, study pre-registration, and use of reporting guidelines). We developed six recommendations for investigators who choose to share to programming code and gathered feedback from members of the International Society of Pharmacoepidemiology (ISPE). Results: Programming code sharing by articles published in PDS ranged from 2.4% in 2017 to 13.4% in 2022. It was more prevalent among articles with a methodological focus, simulation studies, and papers which also shared record-level data. We recommend that reporting of open science practices, including code sharing, is standardised to enable continued monitoring. When sharing programming code, we recommend the use of permanent digital identifiers, appropriate licenses, and, where possible, adherence to good software practices around the provision of metadata and documentation, computational reproducibility, and data privacy. Conclusion: Programming code sharing is rare but increasing in pharmacoepidemiology studies published in Pharmacoepidemiology and Drug Safety. We recommend improved and consistent reporting of code sharing, and adherence to good programming practices in order to maximize the utility of code when this is shared.