Pharmaceutical Medicine最新文献

{"title":"Transformation of a Medical Information Department to Meet the New Healthcare Realities of the Post-Pandemic Era.","authors":"Almudena Rovirosa, Sara Jericó, Ana Muñiz, Ana Pérez","doi":"10.1007/s40290-026-00605-1","DOIUrl":"10.1007/s40290-026-00605-1","url":null,"abstract":"<p><p>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 h","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"169-178"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219166/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147639439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of a Taste-Masked, Dose-Flexible, Multiparticulate Pediatric Dosage Form: Case Study of Crizotinib, a Challenging Pediatric Formulation.","authors":"Jeremy A Bartlett, Natalie Culver, Matt Santangelo, Andrew Prpich, Erica Long, Kazuko Sagawa, Ravi Shanker, Huiping Xu, Keith Wilner","doi":"10.1007/s40290-026-00604-2","DOIUrl":"10.1007/s40290-026-00604-2","url":null,"abstract":"<p><strong>Background: </strong>Developing pediatric dosage forms remains challenging, particularly for poorly palatable drugs, which can limit tolerability and adherence. Crizotinib, an anaplastic lymphoma kinase inhibitor used to treat rare pediatric cancers, was initially available only as an oral solution or capsules, with the solution demonstrating unacceptable taste in pediatric patients.</p><p><strong>Objectives: </strong>The objectives of this work were to outline results from clinical studies supporting the crizotinib pediatric formulation development and commercialization process, summarize key clinical and formulation challenges encountered, and present lessons learned from an over-a-decade-long development process.</p><p><strong>Methods: </strong>Six clinical studies conducted in pediatric patients and healthy adult participants were collectively used to evaluate palatability, tolerability, pharmacokinetics, and bioequivalence of multiple crizotinib formulations. These studies assessed an oral solution and taste-masked microsphere formulations under fasted, fed, and proton pump inhibitor coadministration conditions, using validated palatability assessments and pharmacokinetic endpoints.</p><p><strong>Results: </strong>Early oral solution formulations showed poor palatability, contributing to reduced tolerability and discontinuation in some pediatric patients. Taste-masked microsphere formulations improved palatability but initially showed reduced drug absorption under elevated gastric pH conditions. Reformulation with a pH-independent Opadry SGR coating resulted in a microsphere formulation that achieved acceptable palatability and consistent pharmacokinetic performance and demonstrated bioequivalence to the commercial adult capsule.</p><p><strong>Conclusions: </strong>This over-a-decade-long clinical development program led to the commercialization of a palatable, dose-flexible, oral solid multiparticulate pediatric formulation of crizotinib for patients with anaplastic large cell lymphoma or inflammatory myofibroblastic tumors. The clinical outcomes and development strategy described herein provide a practical framework to support future pediatric formulation development programs.</p><p><strong>Clinicaltrials: </strong>gov trial registration number (date of registration): NCT00939770 (15 Jul 2009), NCT01606878 (28 May 2012), NCT02006277 (10 Dec 2013), NCT03137134 (02 May 2017), NCT03978143 (06 Jun 2019), NCT04856293 (23 Apr 2021, retrospectively registered).</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"193-207"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219145/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sponsor Initiated Requests to Delay Pediatric Postmarketing Studies.","authors":"Susan M Abdel-Rahman, Yutao Gong, Caleb Choi, Niquiche Guity, Gilbert J Burckart, Lynne Yao","doi":"10.1007/s40290-026-00610-4","DOIUrl":"10.1007/s40290-026-00610-4","url":null,"abstract":"<p><strong>Background and objectives: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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).</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"225-233"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147869049","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association Between Survival Benefits of Anticancer Drugs and Probability of US Food and Drug Administration Approval: A Systematic Review.","authors":"Koji Ishizuka, Shunsuke Ono","doi":"10.1007/s40290-026-00606-0","DOIUrl":"10.1007/s40290-026-00606-0","url":null,"abstract":"<p><p>BACKGROUND AND OBJECTIVES: Survival benefit constitutes the primary pillar of therapeutic efficacy in oncology. However, the survival benefits observed in registrational trials broadly range from significant to marginal, even for US Food and Drug Administration (FDA)-approved drugs. This study explores the association between survival benefits and the likelihood of FDA approval and estimates the boundary effect size that distinguishes FDA-approved from non-approved drugs.</p><p><strong>Methods: </strong>We screened 3463 phase 3 trials initiated between 1990 and 2021 on ClinicalTrials.gov. Eligibility was restricted to randomized phase 3 trials for novel anticancer agents with overall survival (OS) as a primary or co-primary endpoint. Included trials required published results, including the OS hazard ratio (HR) and 95% confidence interval (CI). A total of 189 eligible trials were identified, encompassing 208 arm-pairs and 158,250 participants. Clinical data were extracted from published reports, while regulatory outcomes were adjudicated at the trial-and-indication level using US Prescribing Information on Drugs@FDA database. The association between OS benefit and approval status was modeled using logistic regression, with generalized estimating equations (GEE) to account for trial-level clustering. Publication bias was assessed via funnel plots and the trim-and-fill method.</p><p><strong>Results: </strong>Of the 208 arm-pairs, 79 (38%) supported FDA approval, and 129 (62%) did not. The dataset spanned 27 cancer types, with a mean sample size of 761 participants. The pooled OS HR was 0.70 (95% CI 0.68-0.73) for approved drugs and 0.95 (95% CI 0.93-0.97) for non-approved drugs. Logistic regression revealed a sharp sensitivity of approval probability to the HR for OS. A boundary was observed ranging from 0.74 to 0.86 in the HR for OS, with a 50% probability of FDA approval at HR 0.80. GEE analysis confirmed the robustness of these estimates against trial-level clustering. While funnel plot asymmetry suggested potential publication bias in the non-approved group, trim-and-fill analysis confirmed that the relative disparity in OS HR between approved and non-approved drugs remained consistent.</p><p><strong>Conclusion: </strong>FDA approval for anticancer drugs is characterized by distinct OS HR patterns. While these findings provide a clear efficacy benchmark for OS-driven trials, they should be interpreted cautiously given the evolving therapeutic landscape and potential publication bias in negative trials. Our results underscore the central role of survival benefit in regulatory decisions and provide a quantitative metric to support oncology drug development.</p><p><strong>Funding: </strong>This study was funded by the Ministry of Education, Culture, Sports, Science and Technology (MEXT): Shunsuke Ono KAKEN-HI: 25K10043.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"179-191"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219144/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147581485","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Paediatric Phase I/II Clinical Trial Assessments Under the New EU Clinical Trial Regulation: A Descriptive Analysis.","authors":"Laura Fankhauser, Franca Ligas, Giovanni Lesa, Anette Solli Karlsen, Marianne Lunzer, Monique Al, Sabine Scherer, Sylvie Benchetrit, Claudia Riedel, Annette Kopp-Schneider, Ruth Witt, Olaf Witt, Dominik Karres, Cornelis M van Tilburg","doi":"10.1007/s40290-026-00607-z","DOIUrl":"10.1007/s40290-026-00607-z","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Children remain underrepresented in clinical research, despite regulatory frameworks like the EU Paediatric Regulation and Paediatric Investigation Plans (PIPs). The 2024 Declaration of Helsinki revision now recognises excluding vulnerable populations from research as a potential ethical concern. The Clinical Trial Regulation (EU) No 536/2014 aims to harmonise clinical trial application assessment procedures through a coordinated process involving a Reporting Member State (RMS) and Member States Concerned (MSCs).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Aims: &lt;/strong&gt;To determine if harmonisation is occurring, we conducted the first systematic analysis of Phase I/II paediatric trial application assessments across Member States (MS) in the European Union (EU) focusing on assessment variability, evidence requirements, PIP integration, and adolescent inclusion practices in adult trials.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;In the context of a fellowship at the European Medicines Agency, non-public data of 160 paediatric Clinical Trial Applications (CTAs) submitted through the Clinical Trial Information System (January 2022-July 2024) were screened, with inclusion of 55 Phase I/II trials in the main analysis (selected for their focus on innovative treatments and complex risk-benefit assessments). For each CTA, requests for information (consisting of considerations stated by the RMS and MSCs), assessment reports, sponsor responses, and PIP documentation were reviewed. The number of considerations in the requests for information per MS was analysed with median values calculated. Also, considerations were systemically categorised and assessment patterns between RMS and MSC roles were compared. It was examined how PIPs were integrated into assessments and approaches to adolescent inclusion in adult trials was analysed through systematic review of MS considerations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Of the 160 paediatric CTAs screened, 145 received authorisation, 10 were not authorised, and 5 were withdrawn. Among the 145 authorised CTAs, 61 were Phase I/II trials, 55 of which were included in the main analysis. Analysis of these applications revealed significant heterogeneity in both the number and type of considerations raised across MS, with the median number of considerations per CTA ranging from 5.5 to 26 across MSs (p = 0.025), with particularly marked variation when MSs acted as MSCs compared to RMSs-where additional considerations ranged from zero to 25 per CTA on top of those raised by the RMS. In 36% (20/55), MSs raised concerns about insufficient (pre-)clinical evidence pointing to divergent interpretations of evidence requirements. The degree to which PIPs were integrated into CTA assessments varied considerably-ranging from strict adherence to PIP elements to minimal consideration. In 92% (11/12), MSs showed reluctance to include adolescents in adult Phase I/II trials.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Despite the CTR's harmonisation goals, ","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"209-223"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219154/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147819284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of the Quality Decision-Making Practices Utilized by the Zambia Medicines Regulatory Authority Technical Committees.","authors":"Constance Sakala Chisha, Makomani Siyanga, Stephanie Leigh, Juan Lara, Stuart Walker","doi":"10.1007/s40290-026-00608-y","DOIUrl":"10.1007/s40290-026-00608-y","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;A consistent and transparent approach is essential for quality decision-making in the review and approval of medicines. This is achieved through the use of a standard and well-defined framework, as the decisions made should not be influenced by any biases. While most National Medicines Regulatory Authorities (NMRAs) have designed frameworks to assess the quality of their decisions, many have not been implemented. A well-structured framework improves consistency and predictability in the decision-making process. The Quality of Decision-Making Orientation Scheme (QoDoS) has been widely adopted as a leading framework for this purpose. This study aimed to assess the decision-making processes of two technical committees operating within the Zambia Medicines Regulatory Authority (ZAMRA) using the QoDoS framework developed by the Centre for Innovation in Regulatory Science (CIRS); determine areas of improvement for routine assessment of quality of decision-making and its acceptability with respect to ZAMRA; and suggest ways of improving the lowest-scoring Quality Decision-Making Practices (QDMPs) and how these may be implemented into the decision-making framework to ensure consistency.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The framework was used to assess the quality of the decision-making process and subsequent implementation of the ten QDMPs. The study included five members from the Technical Committee for Human Medicines (TCHM) and seven members from the Technical Committee for Veterinary Medicines (TCVM) at ZAMRA, all responsible for recommending the approval or rejection of applications following scientific review. The validated QoDoS questionnaire was electronically distributed to the members, and data were analysed using descriptive statistics.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Analysis of the QDMPs across the 12 committee members indicated that both human and veterinary medicines technical committees generally perceived their individual and organizational decision-making practices as favourable. Favourable QDMPs included QDMP 2 (assigning clear roles and responsibilities of the stakeholders involved in the review and approval of medicines), QDMP 4 (evaluate both internal and external influences or biases), QDMP 6 (considering uncertainty), QDMP 7 (re-evaluate new information as it becomes available), QDMP 9, (ensure transparency and keep a record trail) and QDMP 10 (effective communication of the basis of the decision). However, areas for improvement were identified in QDMP 1 (systematic structured approach), QDMP 3 (decision criteria), QDMP 5 (alternatives) and QDMP 8 (impact analysis) within the TCVM, as well as in QDMP 3 and QDMP 8 within the TCHM.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;This study assessed committee members' perceptions of the implementation of QDMPs in the review and approval of medicines. It was demonstrated that most of the best QDMPs were implemented, except for four QDMPs that needed improvement, name","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"235-248"},"PeriodicalIF":4.7,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13219053/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147874846","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Best Practices for Medical Information Global Response Documents from phactMI and MILE.","authors":"Susan Wnorowski, Faith Niemoller, Anja Glaetzer, Robert Tamburri, Elissa Vine, Franziska Flick, Meera Patel, Evelyn R Hermes-DeSantis","doi":"10.1007/s40290-025-00598-3","DOIUrl":"10.1007/s40290-025-00598-3","url":null,"abstract":"<p><p>Medical information departments within pharmaceutical companies play a critical role in addressing unsolicited healthcare professionals' inquires with accurate, evidence-based, and balanced medical information through scientific response documents. Global response documents (GRDs) aim to harmonize information between global and local teams and the end product, the scientific response document. To address the challenges associated with the development and maintenance of global response documents and this harmonization, phactMI and Medical Information Leaders Europe (MILE) convened an international expert panel representing 11 major pharmaceutical companies. Over months of discussion and deliberation, the panel developed consensus-based best practices for the creation and management of global response documents. The experts contributed nuanced insights regarding optimal document structure and format that consider various stakeholders while maintaining medical accuracy. Consensus was achieved via a structured survey with a 75% approval threshold for each best practice. Overall, GRDs are essential tools for pharmaceutical companies, offering accurate evidence-based responses to healthcare professionals. While companies may respond to patient inquiries as well in different formats (verbal vs written), GRDs are exclusively intended for healthcare professionals currently. Best practices for GRD development include: being focused on a single topic, developed in a label-agnostic manner, utilize the most robust scientific information available, developed up to 12-15 months prior to launch, have regular feedback in a structured approach from local teams, follow internal guidelines and work instructions, and be peer reviewed internally whenever new data are added. In addition, local response documents or scientific response documents should be based on a GRD that is localized with appropriate documentation. Overall, GRD development is a collaborative and innovative approach (including interactive formats), supported by defined metrics (including response time) and ongoing updates to ensure global relevance and compliance.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"87-94"},"PeriodicalIF":4.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12988992/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146011427","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Shaping the Future of Evidence Generation: Real-World Data to Drive Healthcare Transformation and Patient-Centered Decisions.","authors":"Jesús Medina, Joaquín Sánchez-Covisa, Javier Nuevo, Marta Galvez-Fernandez, Alberto García, Belén Pimentel, Eunice Fitas, Eduardo Fernández, Ryan N Walton, Meri Petrovska, Ana Pérez Domínguez","doi":"10.1007/s40290-025-00599-2","DOIUrl":"10.1007/s40290-025-00599-2","url":null,"abstract":"&lt;p&gt;&lt;p&gt;The increasing complexity of healthcare decision making and the demand for real-world evidence call for a transformation in how Medical Affairs departments generate and communicate evidence. In response, AstraZeneca Spain implemented a structured stakeholder-driven framework aligned with the Clinical Evidence 2030 and Medical Affairs 2030 visions, aiming to deliver high-impact, timely, and tailored insights across each therapeutic area. An ambitious evidence generation vision was developed around two core components: (1) enhanced therapeutic area knowledge leading to improvements in guideline-directed medical therapy for patients and (2) strategic use of evidence for the company, leading to improved efficiency in delivering healthcare innovation for patients. The first component encompasses a broad spectrum of study modalities, such as the generation of real-world data on the use of medicines, health economics and outcomes research, predictive analytics, clinical management, and patient experience studies using diverse data sources, including real-world databases, registries, and electronic health records. Studies address different research questions and data gaps, including disease burden, treatment patterns, economic modeling, patient experience through ethnographic research, and knowledge derived from artificial intelligence-driven analytics. The second component seeks to ensure that the evidence generated is used strategically. First, it should be the basis for the development of a powerful Value Story that conveys the clinical, economic, and humanistic value of a pharmaceutical product and creates confidence in regulators, payers, and prescribers, thereby strengthening its therapeutic positioning. Second, the evidence generated needs to be presented to stakeholders in a dynamic manner, enhancing decision-making processes and fostering real-time engagement with healthcare providers and payers. Here, we present a data visualization tool that was developed to this end, termed ATLAS. Third, results from real-world evidence studies can be used to inform outcome-based agreements with payers. This type of innovative value strategy that goes beyond traditional financial agreements represents a method to ensure that the clinical and economic value delivered by a product is reflected in healthcare pricing and reimbursement models, facilitating broader access and timely patient care. AstraZeneca Spain's evidence generation approach represents a significant contribution to the evolution of Medical Affairs into a strategic driver of healthcare innovation and, ultimately, patient benefit. It implies an effort in the consolidation of unique features such as the increase in robust methodological capabilities and versatility in the design of studies, the leverage of predictive analytics, the development of advanced data visualization tools, the emphasis on patient-centered research, and the collaboration with partners expert in emerging technologies. T","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"95-106"},"PeriodicalIF":4.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12988914/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146065844","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Publicly Available Clinical Trial Safety Data: Review and a Call for Standardization and Improved Reporting Practices.","authors":"Barbara A Hendrickson, Cynthia McShea, Tarek A Hammad, Jaishri Meer, Wei Michelle Zhang, Edward Whalen, Susan Talbot, Ranjeeta Sinvhal, Maria Beatrice Panico, Li-An Lin, Dimitri Bennett","doi":"10.1007/s40290-025-00596-5","DOIUrl":"10.1007/s40290-025-00596-5","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objectives: &lt;/strong&gt;Comprehensible reporting of clinical trial safety data is essential for multiple stakeholders, including ongoing safety reporting by sponsors to the health authorities. However, the consistency and completeness of information about safety events of interest (EOIs) in public sources is not well characterized. This study examined the availability and transparency of adverse event (AE) information from public clinical trial data sources, with a focus on their utility in similar patient populations for signal detection and contextualizing rates of anticipated EOI reported as serious or severe (grade ≥ 3).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A structured review of 44 EOI for ten medicinal products approved for different indications in the past 7 years in the United States (US) and European Union was conducted. The selected EOIs were events likely to be reported as serious or severe in clinical trials and additionally were either anticipated AEs or of customary high interest for the patient population. Safety data from journal publications, ClinicalTrials.gov, US Food and Drug Administration (FDA) review documents, European Public Assessment Reports (EPARs), and product labeling (US Prescribing Information, Summary of Product Characteristics) were evaluated. Parameters assessed for availability included demographic data, disease severity measures, serious AE (SAE) frequency, and exposure-adjusted rates. Clarity in approach for EOI identification also was evaluated, specifically whether based on expert adjudication or delineated pre-specified or ad hoc groupings of Medical Dictionary for Regulatory Activities (MedDRA) Preferred Terms (PTs) or alternatively not specified.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Overall, clinical summaries available at Drugs@FDA and EPARs provided the most information about enrolled trial participant characteristics. Greater than 95% of journal publications sampled provided information regarding enrolled participant demographics and disease severity; geography of enrolled participants was available in 43% of instances. The percentage of participants experiencing an SAE was typically available in all sources except product labeling. Information about selected serious/severe EOIs was most often found at Drugs@FDA and in the EPARs, followed by journal publications. Gaps in ClinicalTrials.gov included event adjudication details, if applicable, and lack of exposure-adjusted rates, including for medical conditions that require MedDRA PT grouping for assessment. Significant issues across all sources were frequent omission of patient years of treatment exposure and clear definitions of how EOIs were identified. Published standardized queries (e.g., Standardized MedDRA Queries) were uncommonly used across all sources.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Public clinical trial data sources often lack consistency and sufficient level of detail, hindering their value in contextualizing serious/severe","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"145-161"},"PeriodicalIF":4.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12988996/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145763598","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Combat Against Antimicrobial Resistance: An Overview of EU Recent Health Policies and Drug Regulations Promoting the Development of New Antimicrobials.","authors":"Lena Daher, Ingrid Vilimelis-Piulats","doi":"10.1007/s40290-026-00601-5","DOIUrl":"10.1007/s40290-026-00601-5","url":null,"abstract":"<p><p>Antimicrobial resistance, including antibiotic resistance, constitutes a major global public health threat. In Europe, antimicrobial resistance is recognized among the top three health threats, in line with the World Health Organization's prioritization. This narrative review examines recent key health and pharmaceutical policies and drug regulations in the European Union, aimed at combating antimicrobial resistance and promoting the development of novel antibiotics and alternative therapeutic approaches. It also highlights potential limitations within these initiatives. Between 2015 and 2024, the authors have identified only 14 newly authorized antibiotics by the European Medicines Agency (excluding withdrawn applications, generics, and new formulations of existing medicines). This underscores the stagnation in the development of antibiotics, potentially attributable to limited investments because of fears of low profitability, and a loss of technical expertise within the pharmaceutical industry. At the European Union level, the One Health Action Plan (2017) established a strategic framework to implement anti-antimicrobial resistance measures and stimulate research on antimicrobials. Furthermore, the forthcoming reform of the European Union general pharmaceutical legislation introduces new incentives to support development of antimicrobials. In parallel, the European Medicines Agency has reinforced its efforts to support development of antimicrobials through updated, or planned updates, of its regulatory guidelines and through its innovation-support regulatory tools (e.g., \"Priority Medicine\" scheme). Concurrently, interest in bacteriophage-based therapies as antibiotic alternatives is increasing, as reflected in recent updates to the European Pharmacopoeia and the work initiated on the European Medicines Agency guidelines for bacteriophage therapy. While all these initiatives represent meaningful progress, significant challenges remain, necessitating sustained collaborative efforts across stakeholders.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"107-128"},"PeriodicalIF":4.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147290574","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}