Pharmaceutical Medicine最新文献

{"title":"The Utility of Tissue-Agnostic Drugs in Lung Cancer Treatment.","authors":"Leping Kong, Ben Grobman, Arian Mansur, Adele Lee, Connor Bondarchuk, Hannah Erdogan, Christine Y Lu","doi":"10.1007/s40290-025-00582-x","DOIUrl":"https://doi.org/10.1007/s40290-025-00582-x","url":null,"abstract":"<p><p>Tissue-agnostic therapies, which target molecular alterations across cancer types, have been approved by the US Food and Drug Administration (FDA) since 2017. These approvals were based on clinical trials enrolling patients across diverse tumor types, regardless of the tumor's site of origin. This analysis evaluated the efficacy and safety of FDA-approved tissue-agnostic therapies in patients with lung cancer, the leading cause of cancer-related deaths worldwide, harboring targetable genetic alterations. Current evidence suggests that these therapies show promise in treating NTRK gene fusions (larotrectinib, entrectinib, repotrectinib), BRAF V600E mutation (dabrafenib and trametinib), and RET fusions (selpercatinib) in lung cancer. Reported rates of grade 3 or higher treatment-related or treatment-emergent adverse events ranged from 10 to 59.7%. However, available data are limited by small sample sizes (ranging from 4 to 247 participants per trial-cohort) and the absence of control groups. Larger, controlled studies and real-world evaluations are needed to confirm these findings and inform broader clinical use.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145200547","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":"The Pharmaceutical Year That Was, 2025.","authors":"Anthony W Fox","doi":"10.1007/s40290-025-00588-5","DOIUrl":"https://doi.org/10.1007/s40290-025-00588-5","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145200550","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":"Development of a Competency Framework for Medical Affairs Professionals in Australasia.","authors":"Andrew Weekes, Matthew Britland, Cathey Saha, Simon McErlane, Niloufar Ansari, Daniel Thurley, Victoria Elegant, Orin Chisholm","doi":"10.1007/s40290-025-00585-8","DOIUrl":"https://doi.org/10.1007/s40290-025-00585-8","url":null,"abstract":"<p><strong>Background and objectives: </strong>Medical Affairs (MA) plays a critical role in bridging the gap between research, clinical practice and business strategy. With the rapid growth in this field, it is essential to have a competency framework to support individuals' professional development. A well-defined competency framework will not only empower MA professionals to excel and develop in their roles but also contribute to better patient outcomes and improved stakeholder engagement. This paper discusses the development of a competency framework for Medical Affairs professionals in Australasia.</p><p><strong>Methods: </strong>The MA competency framework was developed using an iterative method by a team of MA professionals across Australia through a series of workshops and surveys over 2 years with the cooperation of the local MA community. The core development team debated and finessed the final framework over this time via meetings and discussions to arrive at the draft framework. This was pilot tested by a local pharmaceutical organisation and feedback informed some minor changes to the final framework. This was then endorsed by the Medical Affairs of Australasia (MAPA) Executive Committee.</p><p><strong>Results: </strong>The framework consists of six domains: Scientific/Technical Knowledge, Evidence Generation, Compliance, Governance and Ethics, Leadership/Professionalism, Communication and Collaboration and Business Acumen, each with specific competencies and across four clearly defined levels from novice to expert.</p><p><strong>Conclusion: </strong>This framework has been endorsed by the Medical Affairs Professionals of Australasia Executive Committee and provides a clear framework for the professional development of medical affairs professionals across our region. It is also applicable to MA professionals more broadly.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145177434","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":"Acceptance and Utilization of Real-World Evidence among Cancer Care Physicians in the USA: A National Survey.","authors":"Thomas M Porter, Kathleen M Andersen, Wencesley Paez, Patrick Corr, Sabrina Figueiredo","doi":"10.1007/s40290-025-00586-7","DOIUrl":"https://doi.org/10.1007/s40290-025-00586-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Regulatory agencies and policy makers increasingly recognize real-world evidence (RWE) as a valuable complement to randomized controlled trials (RCTs) in oncology, yet data on how US physicians who treat cancer use and perceive RWE remain limited. The study aimed to assess attitudes toward RWE among US physicians who treat cancer, including their confidence in interpreting it and reliance across clinical decision contexts.</p><p><strong>Methods: </strong>A cross-sectional national survey was administered in November 2024 to licensed US physicians who treat cancer, recruited from the American Society of Clinical Oncology (ASCO) member directory, using a random sample stratified by state population. Inclusion criteria were active US medical licensure and current involvement in oncology patient care. The survey instrument included sections on demographics and practice characteristics; RWE familiarity and usage frequency; comparative reliance on RWE versus RCTs in treatment selection, dosing, and outcome prediction (scales ranging from 0 to 10: 0 = complete reliance on RCT data, 10 = complete reliance on RWE); perceived barriers to adoption (4-point scale); and potential facilitators (4-point scale). Categorical data were summarized as counts and percentages, and continuous variables were summarized as means and standard deviations (SD). Chi-squared tests were used to compare categorical variables across groups, paired t tests were used to assess differences in mean reliance scores, and Spearman's rho was used to evaluate correlations. Statistical significance was set at p < 0.05.</p><p><strong>Results: </strong>In total, 128 completed surveys were received. Overall, 94% of respondents (n = 120) were at least \"somewhat familiar\" with RWE, 14% (n = 18) used it \"often,\" and 3% (n = 4) reported daily use. 49% (n = 63) felt confident interpreting RWE studies, with late-career physicians (> 20 years of experience) less confident than their early and mid-career peers. Reliance on RWE was lower for treatment selection (mean 3.0, SD 1.7) than for dosing (mean 3.7, SD 2.0) or outcome prediction (mean 3.8, SD 2.0) (p < 0.001). Top barriers included reconciling conflicting RWE versus RCT data, data completeness, and bias. Key facilitators included improved analytical standards, guideline integration, and additional training.</p><p><strong>Conclusions: </strong>While awareness of RWE is high among US physicians who treat cancer, they apply it selectively on the basis of clinical context, showing notably lower reliance for treatment selection. Addressing concerns about methodological rigor, data quality, and interpretive skills may strengthen RWE's integration into oncology care.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113474","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":"The Utilization, Reimbursement, and Cost of Targeted Therapies for Duchenne Muscular Dystrophy (DMD) in US Medicaid Programs: A Descriptive Trend Analysis from 2017 to 2022.","authors":"Yazeed Ghawaa, Alex C Lin, Jeff Jianfei Guo","doi":"10.1007/s40290-025-00587-6","DOIUrl":"https://doi.org/10.1007/s40290-025-00587-6","url":null,"abstract":"<p><strong>Background: </strong>Duchenne muscular dystrophy (DMD) is a rare hereditary neuromuscular disorder caused by mutations in the dystrophin gene, leading to progressive muscle deterioration, loss of ambulation, and reduced life expectancy. The US Food and Drug Administration (FDA) has approved several novel drugs for DMD; however, their utilization, reimbursement, and cost patterns within US Medicaid programs remain unexamined.</p><p><strong>Objectives: </strong>The purpose of this study was to evaluate the utilization, reimbursement, and cost trends of targeted therapies for DMD in the US Medicaid population from 2017 to 2022.</p><p><strong>Methods: </strong>A retrospective descriptive drug utilization study was conducted using the national Medicaid drug utilization files that were extracted from the Centers for Medicare and Medicaid Services (CMS) from 2017 to 2022. The study drugs comprised the following four FDA-approved drugs: eteplirsen (Exondys 51^®), golodirsen (Vyondys 53^®), viltolarsen (Viltepso^®), and casimersen (Amondys 45^®). The annual number of prescriptions represented drug utilization and the annual amount of reimbursement represented the total spending, which were calculated for time-series secular trend analyses. The average cost per prescription was also computed as an estimate of drug cost trends.</p><p><strong>Results: </strong>Over the 6-year period, the total count of DMD prescriptions increased significantly by 2988%, from 643 prescriptions in 2017, when only eteplirsen (Exondys 51^®) was available, to a peak of 19,855 prescriptions in 2022, including all four novel DMD drugs. Additionally, the overall reimbursement grew by nearly 2809%, increasing from US$22,027,999 in 2017 to US$640,890,515 in 2022. However, the average cost per prescription decreased by about 6%, from US$34,258 in 2017 to US$32,279 in 2022.</p><p><strong>Conclusion: </strong>The considerable rise in the utilization and spending of novel DMD drugs has imposed a significant burden on the Medicaid budget, underlining the need for policy measures to manage rising costs and maintain equal access to treatment.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092305","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":"Exosomes as Emerging Therapeutic Platforms in Neurological and Psychiatric Disorders.","authors":"Ram Mohan Ram Kumar, Logesh Rajan, Saravana Babu Chidambaram","doi":"10.1007/s40290-025-00584-9","DOIUrl":"https://doi.org/10.1007/s40290-025-00584-9","url":null,"abstract":"<p><p>Exosomes, small extracellular vesicles (sEVs) that range in Size from 30 to 150 nm in diameter, have emerged as crucial mediators of intercellular communication within the central nervous system (CNS). They play significant roles in the pathogenesis and progression of various neurological and psychiatric disorders, including Alzheimer's disease, Parkinson's disease, ischemic stroke, depression, bipolar disorder, and autism spectrum disorder. Exosomes carry a diverse cargo of proteins, nucleic acids, lipids, and other bioactive molecules that can influence neuronal function and synaptic plasticity. In disease states, exosomes derived from stressed neurons or glial cells can propagate neuroinflammation, synaptic dysfunction, and cognitive decline. They may also mediate the spread of abnormal proteins or microRNAs, disrupting neuronal connectivity and neurotransmitter signaling and contributing to the development of proteinopathies and neurotoxicity. Owing to their presence in bodily fluids such as blood plasma, cerebrospinal fluid, and saliva, exosomes hold promise as biomarkers for these disorders. Moreover, their regulatory roles present new opportunities for developing novel diagnostic biomarkers and therapeutic interventions. This review provides an overview of the multifaceted roles of exosomes in neurological and psychiatric disorders. We delve into their contributions to disease pathogenesis, their potential as diagnostic biomarkers, and the innovative therapeutic strategies leveraging exosome-based delivery systems. By exploring the current state of research, we aim to highlight the translational potential of exosomes in revolutionizing the diagnosis and treatment of these disorders.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145086173","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":"Survey on the Role of Comparative Efficacy Studies Required for Biosimilar Monoclonal Antibody Approval in Japan to Justify the Quality Attribute Differences Between Biosimilars and Their Reference Products Based on the Pharmaceuticals and Medical Devices Agency (PMDA) Assessments.","authors":"Kanoko Goto, Aya Hariu, Ryosuke Kuribayashi","doi":"10.1007/s40290-025-00583-w","DOIUrl":"https://doi.org/10.1007/s40290-025-00583-w","url":null,"abstract":"<p><strong>Background and objective: </strong>Since the approval of the first biosimilar monoclonal antibody (mAb) in 2014, 18 biosimilar mAbs have been approved in Japan as of December 2023. These biosimilar mAbs are typically developed using data that exhibit comparability with their reference products (RPs) in terms of quality, pharmacokinetics, safety, and efficacy. Although comparative efficacy studies are not necessarily required under Japanese biosimilar Guidelines, such studies have been conducted for all 18 approved biosimilar mAbs. Meanwhile, there is growing global interest in reevaluating the absolute necessity of these studies. The objective of this original research article was to analyze the data packages of biosimilar mAbs using publicly available review reports from 2014 to 2023 to assess the role of comparative efficacy studies.</p><p><strong>Methods: </strong>First, we identified quality attributes (QAs) that differed between each biosimilar mAb and its RP on the basis of publicly available review reports from the Pharmaceuticals and Medical Devices Agency (PMDA) website. Subsequently, we examined the evidence used to justify these differences.</p><p><strong>Results: </strong>The results showed that 64% of QA differences were justified using data from other QAs. Conversely, only 6% of QA differences were justified through findings from comparative efficacy studies.</p><p><strong>Conclusions: </strong>These findings indicate that comparative efficacy studies play a limited role in establishing comparability between biosimilars and their RPs. Therefore, sponsors may consider whether a comparative efficacy study is necessary to determine if differences in comparative quality studies are clinically meaningful, rather than automatically conducting such studies.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145086109","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":"Pioneering the Future of Medical Affairs: A Strategic Transformation to Meet the Spanish Healthcare Ecosystem's Evolving Trends.","authors":"Ana Pérez Domínguez, Jorge Marinich, Gema Monteagudo, Carmen Moreno, Lucía Regadera, Inmaculada Iglesias","doi":"10.1007/s40290-025-00574-x","DOIUrl":"10.1007/s40290-025-00574-x","url":null,"abstract":"<p><p>The role of medical affairs (MA) in the pharmaceutical industry is undergoing a profound transformation, driven by the increasing complexity of healthcare ecosystems, the rise of digital technologies, and the need for enhanced stakeholder engagement. In alignment with the MA 2030 vision, AstraZeneca Spain has implemented a forward-thinking transformation strategy, positioning MA as a strategic healthcare partner through a structured, data-driven, and patient-centered approach. Our transformation journey has been anchored in five strategic priorities: boosting MA leadership, integrating end-to-end data and analytics, refocusing resources using data-backed strategies, aligning evidence generation with stakeholder needs, and orchestrating omnichannel scientific engagement. To achieve this, we developed and implemented innovative methodologies such as regional archetyping, healthcare account characterization, and stakeholder mapping to systematically analyze and tailor engagement strategies across different healthcare settings. A core component of this strategy is the CARABELA initiative, a structured approach aimed at fostering the optimization clinical pathways, promoting public-private collaborations, and driving practice-changing interventions to improve healthcare efficiency. In addition, we have transitioned from descriptive to predictive analytics through advanced real-world evidence models, ensuring that MA-led initiatives remain proactive and impact-driven. This transformation serves as a scalable framework for MA evolution globally, reinforcing its role as a catalyst for healthcare innovation. By integrating real-world data, digital engagement, and strategic collaboration, MA departments should position themselves to navigate the evolving healthcare landscape while delivering tangible benefits for patients, healthcare professionals, and systems worldwide.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"281-292"},"PeriodicalIF":4.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12443896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144619729","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":"A Framework for Considering the Role of the Public Sector in R&D of Health Technology.","authors":"Juan Carlos Rejon-Parrilla, David Epstein, Jorge Mestre-Ferrandiz, Jaime Espin","doi":"10.1007/s40290-025-00576-9","DOIUrl":"10.1007/s40290-025-00576-9","url":null,"abstract":"<p><p>The development of health technologies involves complex, costly, and risky investments, with significant contributions from both public and private sectors. Recent EU pharmaceutical directives propose transparency on public funding to aid pricing negotiations and affordability. However, questions remain regarding how public investments should be measured and their influence on pricing and reimbursement (P&R) decisions. In this paper, we characterise public sector institutions as \"payers,\" \"R&D investors,\" and \"regulators\". Through a myriad of agencies and decisions, these institutions directly, indirectly or sometime unexpectedly influence risk and return on private research and development (R&D) through P&R, direct investments, and regulatory policy. P&R decisions by payers for innovative therapies influence risk and expected return of future R&D. Value-based pricing offers a more reliable signal of payers' priorities than cost-plus or (international) reference pricing. For greatest impact, public R&D investment should be directed to areas where markets are deficient, such as basic science, translational research, real-world studies, and towards emerging fields like AI and gene editing that will play an increasing role in healthcare and drug development. Applied R&D should be conducted on a financially sustainable basis. Licensing arrangements can be used to recover those investments, while promoting spillover benefits to wider society. Market access regulators are aware of the need for scrupulous transparency and neutrality, but other public sector actors (payers and R&D investors) also must recognise their policies affect the level playing field.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"325-340"},"PeriodicalIF":4.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12443882/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144799857","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":"Transforming Care in China by Using Public-Private Partnerships to Unlock the Power of Patient-Centered Real-World Data.","authors":"Maigeng Zhou, Feng Sun, Fuzhong Xue, Lena Sjögren, Shirley Xiao, Shaosen Zhang, Pingyan Chen, Siyan Zhan, Alexander Bedenkov","doi":"10.1007/s40290-025-00571-0","DOIUrl":"10.1007/s40290-025-00571-0","url":null,"abstract":"<p><p>The transformation of China's healthcare system is increasingly driven by the development of real-world data (RWD) and real-world evidence (RWE). In the context of the \"Healthy China 2030\" initiative, which emphasizes addressing the growing burden of non-communicable diseases (NCDs), this article assesses the current state and potential of RWD/RWE development. Key areas of focus include the policy environment, guideline systems, data foundations and tools, and talent development. The article outlines a comprehensive roadmap for advancing high-quality RWD/RWE in China, emphasizing the construction of nationwide RWD sources and the harmonization of patient-centered RWD public-private partnerships. As a call to action, the article proposes specific recommendations for various healthcare stakeholders (government, regulatory authorities, industry, academia, clinical researchers and institutions, and data service providers) to collaboratively establish a robust and sustainable RWE ecosystem in China. By leveraging these efforts, the ultimate goal is to significantly improve healthcare outcomes, enhance the efficiency of healthcare delivery, and support evidence-based policymaking, thereby contributing to the overall health and wellbeing of the population.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"261-270"},"PeriodicalIF":4.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12443901/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144182443","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}