Pharmaceutical Medicine最新文献

{"title":"Operational Transformation in LATAM Medical Affairs: Building a Shared Regional Vision for Latin America Based on the AstraZeneca Spain Model.","authors":"Inmaculada Iglesias, Juan José López, Alberto Prado, Jorge Marinich, Noelia Sforza, Alma Neri, Diana Jaimes, Carlos Grijalba, Jaime Ortiz, Carmen Moreno, Ana Pérez Domínguez, Cecilia Marques-Toledo","doi":"10.1007/s40290-026-00639-5","DOIUrl":"https://doi.org/10.1007/s40290-026-00639-5","url":null,"abstract":"<p><p>The Medical Affairs (MA) 2030 vision positions the medical division as a strategic, patient impact-oriented function designed to translate scientific leadership, stakeholder partnerships, and evidence generation into measurable improvements in clinical practice and patient care. To align with this vision, AstraZeneca (AZ) is implementing a global transformation strategy based on common principles that can be adapted to diverse organizational and healthcare-system contexts. This transformation was first operationalized in AZ Spain through a structured MA transformation into an evolved medical operating model (OM) with data-driven, patient-centered workstreams, embedding transforming care (TC) as a working method focused on improving care pathways and health-system resilience. Scaling this approach requires maintaining a stable scientific and compliance core while allowing local configurations across diverse healthcare contexts, care pathways, and data readiness. We describe how this framework is being implemented in Latin America (LATAM) and outline the design elements intended to support phased implementation and comparability across settings. Latin American countries were organized into five clusters: Mexico, Brazil, Central America and the Caribbean (CAMCAR), the Andean region, and the Southern Cone. This structure supports phased implementation and the adaptation of priorities within a shared framework. The process comprises a four-step pathway starting with a baseline assessment, OM design and configuration, governance-led implementation, and monitoring of implementation progress, operational maturity, and longer-term impact through a focused set of indicators, an implementation roadmap, validation gates, and initial milestones. The approach combines a consistent, locally configurable transformation model and methodological assets, including CARABELA, to support implementation under different healthcare-system contexts. Key design elements include: (i) a baseline maturity assessment and cluster adaptation logic to define each setting's starting point and priority pathway gaps; (ii) a consistent set of roles, responsibilities and capability enablers to support coordinated execution; (iii) defined governance forums to align evidence planning, launch activities, and TC initiatives through standardized decision-making processes; and (iv) a monitoring approach based on leading and lagging indicators that supports implementation adjustment, assessment of maturity progression, and cross-cluster comparability despite differences in data availability. Early evidence, based on more than 25 TC active projects across clusters and all therapeutic areas reached and potentially impacted 2.5 million patients, with more than 190,000 individuals at risk of selected diseases identified through screening and case-finding initiatives. These aggregate findings indicate implementation scale and uptake, even though they cannot establish causal effectiveness. S","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149245767","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":"Galenic Modification as an Alternative Strategy for Administering Oral Anticancer Drugs: Assessment of Bioequivalence.","authors":"Pauline Claraz, Cécile Pineau, Coralie Guillemot, Etienne Chatelut, Cécile Arellano, Florent Puisset","doi":"10.1007/s40290-026-00622-0","DOIUrl":"https://doi.org/10.1007/s40290-026-00622-0","url":null,"abstract":"<p><strong>Background: </strong>Oral anticancer drugs have become widely used, improving patient autonomy and quality of life. However, patients with swallowing disorders, such as children, older adults, and patients with head and neck cancer or neurologic disease, often require galenic modifications (e.g., crushed tablets, oral suspensions) that may impact drug pharmacokinetics and clinical outcomes.</p><p><strong>Objective: </strong>The aim of this study was to assess the impact of these modifications on bioequivalence and systemic drug exposure.</p><p><strong>Methods: </strong>A systematic research of regulatory documents and scientific literature was conducted on 96 oral anticancer drugs. Data were collected on bioequivalence or relative bioavailability between standard oral solid forms and alternative manipulated or liquid formulations. Chemical and pharmaceutical properties of drugs were assessed as predictive factors for bioequivalence.</p><p><strong>Results: </strong>Data were available for 39 drugs among the 96 selected for the study. The liquid form of 26 drugs did not meet full bioequivalence criteria: maximum plasma concentration (C_max) was more frequently altered than area under the curve (AUC), suggesting that galenic modification primarily affected the absorption rate rather than overall drug exposure. Nevertheless, in 83% of cases, AUC ratios remained between 0.5 and 2. No predictive factors of bioequivalence were identified.</p><p><strong>Conclusion: </strong>Liquid formulation and galenic modification of anticancer drugs primarily increases drug exposure, especially C_max. A priori dose adjustments could not be recommended due to the low and unpredictable pharmacokinetic impact of galenic modifications. Consequently, therapeutic drug monitoring is required when possible, and manipulation should be performed under supervised and standardized conditions.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149063589","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":"Manufacturer-Perceived Development and Market-Entry Risks Associated with Discontinuation and Non-launch of Generic Medicines in Japan: A Cross-Sectional Survey of Marketing Authorization Holders.","authors":"Hiroaki Mamiya, Harumi Tagoku, Ken-Ichi Izutsu","doi":"10.1007/s40290-026-00636-8","DOIUrl":"https://doi.org/10.1007/s40290-026-00636-8","url":null,"abstract":"<p><strong>Background: </strong>Generic medicines are essential for pharmaceutical affordability and access, yet some products are never launched or their development is discontinued even after patent expiry. Limited empirical evidence is available on how manufacturers perceive the development and market-entry risks associated with these outcomes.</p><p><strong>Objective: </strong>The aim of this study was to identify and structure manufacturer-perceived development and market-entry risk factors associated with generic medicine development discontinuation and non-launch in Japan.</p><p><strong>Methods: </strong>We conducted a cross-sectional survey of companies involved in generic medicine development and commercialization in Japan. The primary survey outcome was company-reported experience of development discontinuation or post-approval non-launch during fiscal years 2020-2024. Respondent characteristics were compared between companies with and without such experience. Among companies with such experience, development and market-entry risk factors were assessed in terms of both experience and perceived importance. All responding companies were asked to rate policy and regulatory options on five-point importance scales.</p><p><strong>Results: </strong>Among 122 companies with valid outcome data, 52 (42.6%) reported experience of development discontinuation or non-launch. Such experience was more frequently reported by companies with larger generic product portfolios. The market size and profitability domain was rated highest among the risk domains (top-2 box importance, 94.2%), but regulatory and clinical-study requirements (75.0%), formulation technology and manufacturing complexity (72.5%), and competition and institutional uncertainty (48.0%) were also rated as important. Among individual risk factors, active pharmaceutical ingredient (API) supply, patient-based bioequivalence or clinical-study requirements, dedicated equipment investment, and special formulation requirements were, in an exploratory descriptive map, among the factors with relatively high reported experience and perceived importance.</p><p><strong>Conclusions: </strong>Generic medicine development discontinuation and non-launch in Japan were perceived by manufacturers as involving multiple development and market-entry risks extending beyond pricing to include API supply, manufacturing complexity, regulatory uncertainty, and competitive dynamics. Respondents prioritized reimbursement, regulatory, and manufacturing-related options for further consideration; the study did not evaluate the effectiveness, budget impact, or net social benefit of these options.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148970514","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":"Managing Inhaler Environmental Sustainability and Effective Respiratory Care: Current Evidence, Challenges, and Opportunities.","authors":"Amy Booth, Jasmin Abbott, Samantha Workman, Fionnuala Plumart, Bethan Davies, Michael Okorie","doi":"10.1007/s40290-026-00633-x","DOIUrl":"https://doi.org/10.1007/s40290-026-00633-x","url":null,"abstract":"<p><p>Growing global concern about climate change, alongside the widespread use of inhaler therapies, has intensified interest in the environmental sustainability of inhalers. The respiratory care community is increasingly focused on how to balance effective disease management with the need to avoid exacerbating environmental determinants of respiratory illness. Since Pharmaceutical Medicine last examined this topic in its article Sustainability in Inhaled Drug Delivery, the field has evolved rapidly, with notable developments. This Leading Article reviews the current evidence on the carbon footprints of inhalers and discusses strategies-along with their implications for patient care-aimed at reducing the environmental impact of inhaler therapies. We focus on strategies at the (a) health system level, including optimisation of respiratory care and inhaler choice, adherence, and technique, and (b) pharmaceutical industry level, including innovation and action related to next-generation lower-carbon propellants such as HFA-152a and inhaler circularity. While sustainability strategies exist, we acknowledge that progress is slow, and highlight key implementation, financial, and regulatory challenges that constrain their uptake. The article concludes by outlining recommendations to help support sustainable and effective respiratory care.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148901031","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":"From Invisible to Enrolled: How Artificial Intelligence Is Reshaping Clinical Trial Recruitment.","authors":"Sangzin Ahn","doi":"10.1007/s40290-026-00635-9","DOIUrl":"https://doi.org/10.1007/s40290-026-00635-9","url":null,"abstract":"<p><p>Clinical trial recruitment continues to fail at scale, with eligible patients often invisible to routine screening despite being present in electronic health records. Disease stage, treatment response, biomarker results, and clinical reasoning are frequently documented in narrative form rather than structured fields, placing eligibility-relevant information beyond the reach of conventional recruitment workflows. This narrative review examines applications of artificial intelligence (AI), and particularly large language models, across the recruitment pipeline. The evidence is organized around two ceilings. The discovery ceiling reflects the limits of identifying eligible candidates in clinical data, and current evidence suggests AI can raise it, improving discovery and screening efficiency in defined workflows. The enrollment ceiling reflects the human, logistical, ethical, and institutional barriers that persist after a patient has been identified. AI can make invisible patients visible, but visibility is not enrollment, and AI alone cannot remove these barriers. Upstream applications in trial design, predictive enrichment, site selection, and patient-facing engagement are also reviewed, together with governance requirements for equity, privacy, regulation, and workflow integration. Current evidence remains concentrated in retrospective evaluations, with limited prospective, multicenter validation and few demonstrated gains in enrollment, representativeness, or cost effectiveness. Recruitment AI is therefore best treated as a governed, human-supervised support tool that is locally validated and evaluated against downstream outcomes such as enrollment, diversity, burden, and cost.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148901054","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":"Empowering Medical Affairs Professionals in Their Critical Role in Integrated Evidence Generation Planning.","authors":"Judith Nelissen, Angela Williams, Cerise James, Gary Male","doi":"10.1007/s40290-026-00625-x","DOIUrl":"10.1007/s40290-026-00625-x","url":null,"abstract":"<p><p>An integrated evidence generation plan (IEGP) can transform the effectiveness of how pharmaceutical companies demonstrate the value of new therapies. Medical Affairs professionals are uniquely positioned to lead and shape an IEGP, leveraging their scientific and clinical expertise, while maintaining a focus on patient access needs among competing cross-functional priorities. In this article, we explore key subfunctions within Medical Affairs, describing their contributions to the development of an IEGP and discussing the impact of the evolving healthcare environment on their responsibilities. Finally, we consider how to measure the impact of an IEGP for pharmaceutical companies and describe what success looks like for Medical Affairs in the development of an IEGP. Our companion article in this issue of Pharmaceutical Medicine provides more details of the process of integrated evidence generation planning and considers the impact of evolving regulatory and health technology assessment landscapes.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"357-364"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13545031/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796420","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":"Medication Safety in India and Southeast Asia: A SWOT Analysis of Policies and Practices.","authors":"Sangeeta Sharma, Parminder Gautam, Deepika Sharma, Jitendra Nath Srivastava, Atul Kotwal","doi":"10.1007/s40290-026-00626-w","DOIUrl":"10.1007/s40290-026-00626-w","url":null,"abstract":"<p><p>Medication safety is a critical component of patient care, yet fragmented regulations, under-reporting of adverse events, and resource constraints continue to pose risks in India and Southeast Asia. Addressing these challenges requires system-level interventions supported by robust policies and technology-driven solutions. This review analyzes medication safety frameworks in India and World Health Organization South-East Asia Region countries using a Strengths, Weaknesses, Opportunities, and Threats (SWOT) approach, identifying internal strengths and weaknesses alongside external opportunities and threats. The goal is to propose strategic actions that enhance regulatory oversight, capacity building, health-system resilience, and hospital-level practices. A comparative narrative review of national regulations, World Health Organization and Association of Southeast Asian Nations frameworks, policy documents, and published literature was conducted. Recurring themes were consolidated into four domains such as regulatory systems, health-system strengthening, workforce capacity building, and hospital-level strategies and synthesized using a SWOT-based analytical approach to identify shared gaps, contextual differences, and strategic priorities across India and South-East Asia Region countries. The SWOT analysis across four domains revealed shared patterns between India and World Health Organization South-East Asia Region countries. Regulatory frameworks show strong institutional structures in India and Association of Southeast Asian Nations Good Manufacturing Practices harmonization, but both lack centralized drug registries and face under-reporting of adverse events. Capacity building benefits from professional networks, yet rural workforce shortages and inconsistent training persist region wide. Health systems leverage multi-tiered structures and national programs, but fragmentation and weak digital infrastructure hinder medication error reporting. At the hospital level, limited resources, staff resistance, and complex policies restrict adoption of electronic health records and safety technologies. Opportunities include harmonizing Good Manufacturing Practice standards, scaling accreditation, and deploying digital tools, while threats such as counterfeit medicines, antimicrobial resistance, and punitive reporting cultures remain significant. Strategic priorities include establishing centralized drug registries, enforcing prescription-only rules, implementing QR codes for high-risk medicines, and promoting blame-free reporting. Medium-term actions involve expanding digital health solutions, integrating medication safety into curricula, and scaling accreditation. Long-term goals focus on regional harmonization, collaborative research, and cultural transformation toward safety. Leveraging India's regulatory strengths and lessons learned can accelerate medication safety improvements across Southeast.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"373-398"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796345","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":"Medical Affairs in the Age of Complexity: From Scientific Support to Strategic Enterprise Integrator.","authors":"Mussa Arvani","doi":"10.1007/s40290-026-00621-1","DOIUrl":"10.1007/s40290-026-00621-1","url":null,"abstract":"<p><p>Regulatory acceleration, expanded reliance on real-world evidence (RWE), and increasing integration of artificial intelligence (AI) into healthcare systems have substantially reshaped expectations placed on Medical Affairs. Accelerated and conditional approval pathways are progressively shifting confirmatory evidence generation into the post-marketing phase, while regulatory agencies have formalized frameworks for incorporating RWE into benefit-risk evaluation and lifecycle decision making. In parallel, healthcare systems are increasingly conceptualized as learning health systems in which routine-care data inform iterative improvement. This Leading Article proposes a conceptual but operationally applicable framework that positions Medical Affairs as a Strategic Enterprise Integrator, responsible for coordinating lifecycle evidence architecture, scientific exchange, insight translation, and governance across development and real-world settings. The framework is structured around four interdependent capability domains: lifecycle evidence architecture; scientific translation and insight management; cross-functional governance; and AI/data governance, including oversight of large language model (LLM)-supported workflows in medical information and analytics. The proposed model explicitly delineates interfaces with Clinical Development, Pharmacovigilance, Market Access/Health Economics and Outcomes Research, and Commercial functions to reduce redundancy while preserving functional accountability. Methodological considerations for RWE are anchored in principles of fit-for-purpose data, study design discipline, and transparent interpretation, while AI-enabled processes are addressed through a scientific governance lens emphasizing validation, auditability, and context-of-use. Illustrative operational examples and a staged maturity model with measurable indicators are introduced to support practical implementation. The framework positions Medical Affairs as a scientifically accountable integrator of lifecycle evidence within evolving regulatory science. As a conceptual model this framework requires further empirical validation to assess its applicability and generalizability across organizational contexts.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"365-371"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148430864","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":"Animal Models for Drug Discovery in Schizophrenia: A Critical View from a Clinical Perspective.","authors":"Inga Dammann-Bawadkji, F Markus Leweke, Cathrin Rohleder","doi":"10.1007/s40290-026-00617-x","DOIUrl":"10.1007/s40290-026-00617-x","url":null,"abstract":"<p><p>Schizophrenia is a complex neuropsychiatric disorder manifesting with diverse positive and negative symptoms as well as cognitive impairments. Current antipsychotics primarily address positive symptoms and frequently cause substantial side effects, highlighting the need for novel therapeutics targeting alternative pathomechanisms. Animal models are widely used to investigate schizophrenia's neurobiology and guide drug discovery, yet their clinical relevance for proof-of-concept (PoC) studies remains controversial. This Current Opinion critically examines the key limitations of animal models in schizophrenia research from a clinical perspective. The disorder's multifaceted nature, involving genetic, environmental, and neurodevelopmental factors, makes accurate replication in animals challenging. Additionally, core human-specific symptoms, like hallucinations and thought disorders, cannot directly be modeled, although some underlying cross-species constructs can be operationalized. We argue that animal models are most informative when used to test specific, well-defined mechanistic hypotheses and when readouts are anchored to human-relevant biomarkers and neurophysiology, rather than interpreted as proxies for diagnostic categories. Accordingly, translational utility is not uniform: predictive performance depends on the induction paradigm, the construct validity of behavioral and neurophysiological readouts, and the clinical endpoint being modeled. We advocate for close collaboration between preclinical and clinical researchers to refine existing models, establish new and translationally relevant paradigms, and clearly define their interpretive scope. Integrating complementary advanced in vitro and in silico models may enhance mechanistic understanding and help prioritize hypotheses and candidates. Still, these approaches should be viewed as adjuncts, not superior replacements, because they cannot yet capture the circuit- and systems-level dynamics relevant to schizophrenia.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"335-349"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13545137/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148437383","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":"Placebo Response in Randomised Controlled Trials of Systemic Therapies for Cutaneous Lupus Erythematosus: A Systematic Review and Meta-Analysis.","authors":"Stéphanie Légaré, Sherry Lin, Sarah Vahey, Juan Gabriel Ovalles-Bonilla, Pina D'Angelo, Jasmina Jankicevic, Robert Bissonnette","doi":"10.1007/s40290-026-00631-z","DOIUrl":"10.1007/s40290-026-00631-z","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Lupus erythematosus is a heterogeneous autoimmune disease with manifestations ranging from skin-limited forms to severe systemic disease. Despite the high prevalence of skin symptoms among lupus participants, no therapies are currently approved specifically for cutaneous lupus erythematosus (CLE). An increasing number of clinical trials have evaluated systemic therapies in lupus participants with cutaneous manifestations; however, placebo response in CLE has never been systematically examined. Characterising the magnitude and predictors of placebo response is essential to optimising trial design and improving the interpretability of efficacy outcomes in this population with significant unmet medical needs.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To characterise placebo response in randomised controlled trials (RCTs) of systemic therapies for CLE and explore clinical and design factors associated with increased placebo responses.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A systematic literature review and meta-analysis were conducted on double-blind, randomised, placebo‑controlled trials published between 2005 and June 2024 that evaluated systemic therapies in CLE and reported outcomes using the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI‑A). Searches were performed in PubMed, Cochrane Library, and Scopus. The review was registered on the International Prospective Register of Systematic Reviews (PROSPERO). Risk of bias was evaluated using the Cochrane RoB2 tool. Random-effects meta-analyses were performed to estimate pooled placebo response rates (95% confidence intervals [CIs]), with further exploration of clinical and design factors associated with increased placebo responses through subgroup analyses, meta‑regression, and Pearson correlation analyses.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Overall, 33 RCTs, described in 43 publications and including 2382 placebo-treated participants, were included in this analysis. At the primary endpoint timepoint, the pooled proportion of placebo-treated participants (n = 424 in 14 trials) achieving ≥ 50% reduction in CLASI-A (CLASI‑A50) was 42% (95% CI, 35-50). The CLASI‑A50 placebo responses were 21% at Week 8, 33% at Week 24, and reached 46% at Week 52 for up to 8 trials (n = 150 to 278). Univariate analyses revealed higher placebo responses in studies initiated after 2016, those with follow‑up durations &gt; 24 weeks, and those requiring stable background therapy. Study start year and primary endpoint timepoint were identified as the most influential predictors of placebo response in a meta-regression analysis. A positive correlation was also observed between the proportion of White participants and CLASI-A50 placebo responses.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;These findings suggest that limiting background therapy, shortening follow-up durations, and ensuring balanced racial representation should be further explored to reduce placebo responses in future CLE trials. Such ","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"399-412"},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796410","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}