Report of the European Medicines Agency Conference on RNA-Based Medicines.

RNA-based medicines have potential to treat a large variety of diseases, and research in the field is very dynamic. Proactively, The European Medicines Agency (EMA) organized a virtual conference on February 2, 2023 to promote the development of RNA-based medicines. The initiative addresses the goal of the EMA Regulatory Science Strategy to 2025 to "catalyse the integration of science and technology in medicines development." The conference focused on RNA technologies (excluding RNA vaccines) and involved different stakeholders, including representatives from academia, industry, regulatory authorities, and patient organizations. The conference comprised presentations and discussion sessions conducted by panels of subject matter experts. In this meeting report, we summarize the presentations and recap the main themes of the panel discussions.

Commentary on the EMA reflection paper on the pharmaceutical development of medicines for use in the older population.

Older people are often affected by impaired organ and bodily functions resulting in multimorbidity and polypharmacy, turning them into the main user group of many medicines. Very often, medicines have not specifically been developed for older people, causing practical medication problems for them like limited availability of easy to swallow formulations, easy to open packaging and dosing instructions for enteral administration. In 2020, the European Medicines Agency (EMA) published a reflection paper 'Pharmaceutical development of medicines for use in the older population', which discusses how the emerging needs of an ageing European population can be addressed by medicines regulation. The paper intends to help industry to better consider the needs of older people during pharmaceutical/clinical medicines development by summarising data on the most relevant topics, providing early suggestions on how to move forward and prompting expert discussions and studies into knowledge gaps. Topics include patient acceptability, (dis)advantages of an administration route, formulation, dosage form, packaging, dosing device and user instruction. While the paper is directed at older people and the pharmaceutical industry, the reflections are also relevant to younger patients with similar disease-related needs and of value to other stakeholders parties, e.g., healthcare professionals, academics, patients and caregivers, as the paper makes clear what can be expected from industry and where collaborative work is needed. This commentary provides an overview of the different steps in the development of the reflection paper, discusses points considered most controversial and/or subject to (multidisciplinary) expert discussions and indicates their value for real world clinical practice.

Evaluating the impact of integrated development: are we asking the right questions? A systematic review.

Background: Emerging global transformations - including a new Sustainable Development Agenda - are revealing increasingly interrelated goals and challenges, poised to be addressed by similarly integrated, multi-faceted solutions. Research to date has focused on determining the effectiveness of these approaches, yet a key question remains: are synergistic effects produced by integrating two or more sectors?  We systematically reviewed impact evaluations on integrated development interventions to assess whether synergistic, amplified impacts are being measured and evaluated. Methods: The International Initiative for Impact Evaluation's (3ie) Impact Evaluation Repository comprised our sampling frame (n = 4,339). Following PRISMA guidelines, we employed a three-stage screening and review process. Results: We identified 601 journal articles that evaluated integrated interventions. Seventy percent used a randomized design to assess impact with regard to whether the intervention achieved its desired outcomes. Only 26 of these evaluations, however, used a full factorial design to statistically detect any synergistic effects produced by integrating sectors. Of those, seven showed synergistic effects. Conclusions: To date, evaluations of integrated development approaches have demonstrated positive impacts in numerous contexts, but gaps remain with regard to documenting whether integrated programming produces synergistic, amplified outcomes. Research on these program models needs to extend beyond impact only, and more explicitly examine and measure the synergies and efficiencies associated with linking two or more sectors. Doing so will be critical for identifying effective integrated development strategies that will help achieve the multi-sector SDG agenda.