Pharmaceutical Medicine最新文献

{"title":"Correction: The Value and Importance of a Professional Ethical Code for Medicines Development: IFAPP International Ethics Framework.","authors":"Sandor Kerpel-Fronius, Alexander L Becker","doi":"10.1007/s40290-025-00563-0","DOIUrl":"10.1007/s40290-025-00563-0","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"259"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12246024/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143720564","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":"Embracing Medicines Development as a Profession.","authors":"Pravin Chopra","doi":"10.1007/s40290-025-00556-z","DOIUrl":"10.1007/s40290-025-00556-z","url":null,"abstract":"<p><p>Medicines development has dramatically transformed in the preceding decades. It has evolved from a task undertaken by a small team to a complex series of activities, involving several functions and qualified professionals, across multiple, interrelated, scientific disciplines, worldwide. Conceptualized as a medical specialty, concerned with the research, development, and monitoring of medicines, and spearheaded largely by pharmaceutical physicians, the discipline has extended to embrace non-medically qualified scientists progressively taking on traditional roles within the medicines development ambit. There is expanded engagement of professional backgrounds. Each individual function contributes unique expertise and skills, but they all share a sense of identity, a collective commitment, and a common goal of improving human health and well-being, through innovative treatments. At the same time, milestone advancements in the research and development environment, in healthcare delivery, the regulatory ethos, data sharing with greater transparency, the exponential digitalization, and the emphasis on patient outcomes have imposed greater accountability in systems and processes, and across all global stakeholders. Increasingly, this demands a joint purpose, fostering continuous learning, and engendering a professional identity within the medicines development community, a coinage gaining incremental affirmation as equivalent to pharmaceutical medicine. The aligned set of related competencies and capabilities across the multi-professional teams involved, the sense of identifying concurrently across different, albeit complementary, communities of practice, the obligation of ongoing specialized knowledge training, the mandated enterprise-wide codes of ethics and moral conduct, and the intertwined career paths within the larger business organizational construct of academic institutions, regulatory agencies, and the biopharmaceutical industry-all profess to meet the criteria, and merit medicines development/pharmaceutical medicine be acknowledged, regarded, and recognized as a distinct profession of stature, in its own standing. The concept of pharmaceutical medicine has been floated since 1957, and has received considerable attention in scientific literature over the years. Yet, the discipline struggles to be universally understood and appreciated. This perspective scrutinizes related engendering influences and hindering elements, while advocating the furtherance of medicines development from a traditional occupation, emerging as a vocation, and transforming into a mature profession, with the vision and purpose of advancing better medicines for improved health outcomes for people, globally.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"215-224"},"PeriodicalIF":3.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12246006/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616727","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":"Safety Warnings on the Same Harmful Effects of Medicines: A Comparison of Four National Regulators.","authors":"Lucy T Perry, Alice Bhasale, Ashleigh Hooimeyer, Eliza J McEwin, Annim Mohammad, Barbara Mintzes","doi":"10.1007/s40290-025-00573-y","DOIUrl":"https://doi.org/10.1007/s40290-025-00573-y","url":null,"abstract":"<p><strong>Background: </strong>Safety advisories provide critical information to clinicians and patients on the harms of medicines. Previous research has shown that national regulators vary in their decisions to issue safety warnings. However, it is not known whether clinicians receive similar information when regulators communicate about the same medicines' harms.</p><p><strong>Aim: </strong>Our aim was to assess whether content provided to clinicians in safety advisories on risk, fatal outcomes, evidence and clinician advice was comparable.</p><p><strong>Methods: </strong>This retrospective content analysis examines safety advisories issued by the Australian Therapeutic Goods Administration, Health Canada, the United Kingdom Medicines and Healthcare products Regulatory Agency, and the US Food and Drug Administration between 2007 and 2016. Content was extracted from advisories issued on the same medicine and harm (n = 40), including evidence, risk quantification, fatal outcomes and clinician advice. A case study on pioglitazone and bladder cancer illustrates differences in regulatory communications.</p><p><strong>Results: </strong>Variation was seen in the detail and presentation of information on evidence, deaths, risk quantification and advice to clinicians. Specific advice to clinicians was provided in 70% (96/155) of advisories with no significant differences between regulators (p = 0.19). Evidence of harm was presented in 81% (130/160) of advisories and risk quantification in 61% (98/160). The type of evidence presented and directness of information differed however. In the pioglitazone case study, for example, regulators differed in how bladder cancer risks were characterised and advice provided.</p><p><strong>Conclusions: </strong>Our analysis of safety advisories on the same harms of medicines indicates that while regulators provide similar content elements in safety advisories, risk messages to clinicians vary. This may lead to differences in knowledge and awareness between countries and potentially impact public health outcomes. Further transparency around regulatory decisions on safety advisories is needed.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497618","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":"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":"https://doi.org/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":""},"PeriodicalIF":3.1,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144182443","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":"Cost and Duration of Clinical Trials in Drug Development by Japanese Pharmaceutical Companies.","authors":"Norihiro Okada, Yosuke Takahashi","doi":"10.1007/s40290-025-00558-x","DOIUrl":"10.1007/s40290-025-00558-x","url":null,"abstract":"<p><strong>Background: </strong>Understanding parameters such as cost and duration, which reflect the scale of clinical trials, is essential for government agencies to formulate pharmaceutical policies and for pharmaceutical companies to strategically allocate resources. As most studies on clinical trial costs and durations have primarily focused on pharmaceutical companies developing drugs in the USA, understanding actual trial costs and durations for Japanese pharmaceutical companies engaged in global development or development within their own countries remains challenging.</p><p><strong>Objectives: </strong>This study aimed to clarify the actual phase-specific costs and durations of clinical trials conducted by leading Japanese pharmaceutical companies and to identify the factors influencing these parameters.</p><p><strong>Methods: </strong>We conducted a questionnaire-based survey among 22 pharmaceutical companies headquartered in Japan. Respondents from each company retrospectively identified clinical trials for new drug applications or for expanding approved disease indications, conducted between 2012 and 2022, providing cost and duration data of each clinical trial. We calculated summary statistics for costs and durations by phase and conducted an exploratory analysis of the variables influencing these parameters.</p><p><strong>Results: </strong>In total, 631 projects with clinical trials conducted by these companies were analyzed. The median costs in the analysis population were 859 million Japanese yen (JPY) for Phase 1, 1,739 million JPY for Phase 2, and 4,183 million JPY for Phase 3. For reference, the median annual average exchange rates during the study period were 109 JPY per US dollar (USD) and 130 JPY per euro (EUR). The median durations were 20 months for Phase 1, 29 months for Phase 2, and 31 months for Phase 3. For projects targeting approval only in Japan, the median Phase 3 cost was 3,050 million JPY, while for projects targeting approval involving the USA or Europe, it was 8,083 million JPY.</p><p><strong>Conclusions: </strong>This study quantifies the costs and durations incurred by Japanese pharmaceutical companies in conducting clinical trials, revealing the high costs associated with projects targeting approval involving the USA or Europe. The findings highlight the substantial investments required by the Japanese pharmaceutical industry to bring new drugs to the global market.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"199-207"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144064344","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":"Why the Pharmaceutical Industry Needs Implementation Science for Sustainable Innovation.","authors":"Anthony J Messina","doi":"10.1007/s40290-025-00566-x","DOIUrl":"10.1007/s40290-025-00566-x","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"147-150"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126355/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031165","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":"Association Between Health-Related Quality of Life Measures and Survival Endpoints in Oncology Clinical Trials and in Clinical Decision Making: A Narrative Review.","authors":"Anna Amela Valsecchi, Massimo Di Maio","doi":"10.1007/s40290-025-00568-9","DOIUrl":"10.1007/s40290-025-00568-9","url":null,"abstract":"<p><p>For decades, oncology research has primarily relied on survival-based endpoints, such as progression-free survival and overall survival, to evaluate treatment efficacy. However, recent studies and international guidelines underscore the importance of incorporating patient-reported outcomes through patient-reported outcomes measures (PROMs). PROMs provide a more comprehensive view of treatment effectiveness, integrating the concepts of 'living longer' and 'living better.' Health-related quality of life (HRQoL) improvements have an intrinsic value for the patient, with importance in the overall definition of treatment value. These findings have sparked discussions regarding the relationship between HRQoL and traditional survival endpoints, influencing both oncology clinical trials and their interpretation for decision-making processes in practice. To effectively integrate PROMs into research, the choice of study design, appropriate PROMs questionnaires, and timing of administration are critical. The clinician's ability to interpret HRQoL data with awareness is equally important to ensure good clinical decision making. A pivotal concept in this context is the minimum clinically important difference (MCID), which is essential to inform the interpretation of treatment effect size in terms of clinically relevant HRQoL changes. Incorporating PROMs fosters a patient-centered approach to cancer care, aligning treatment goals with individual preferences and values. By balancing survival outcomes with quality of life, and through empathetic communication, healthcare providers can deliver treatments that are not only effective but also resonate with patients' experiences and priorities.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"171-182"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126343/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144079229","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 Model for an Academia-Industry Collaboration for Pharmacovigilance and Pharmacoepidemiology.","authors":"Alfred I Neugut, Vinu George, Judith S Jacobson, Michael D Parkinson, Leslie E Segall, Michelle Lebo, Charles C Branas, Daniel E Freedberg, Mirza I Rahman","doi":"10.1007/s40290-025-00567-w","DOIUrl":"10.1007/s40290-025-00567-w","url":null,"abstract":"<p><p>Collaborations between academia and the pharmaceutical industry are common for drug development and clinical trials, but rare for pharmacovigilance. Here we describe a novel model for collaboration between academia and the pharmaceutical industry, focused on post-marketing pharmacovigilance, that others may wish to emulate. For the past 5 years, Otsuka Pharmaceutical, a global Japan-based pharmaceutical company, has collaborated with faculty at Columbia University, a major university, for epidemiology support. The primary aim of this collaboration is to provide expert research guidance for Otsuka's pharmacovigilance group on questions involving pharmacoepidemiology. University epidemiologists are also consulted by other industry divisions, such as the clinical trials group. University epidemiologists help to determine the incidence, prevalence, and outcomes of diseases; draft the epidemiology components of risk management plans for drugs; and plan retrospective database analyses. A second major aim of this collaboration is to provide educational services to the company by conducting workshops on basic epidemiology and biostatistics; leading a monthly lecture/journal club series; hosting seminars on medical topics; and providing a writing workshop to assist in preparing abstracts and papers for presentation and publication. University epidemiologists provide oversight/evaluation through quarterly presentations and updates to the industry partner's external advisory committee as well as to university leadership. This type of epidemiologic collaboration has key advantages for industry over the alternatives of building an in-house epidemiology department or hiring outside consulting firms: lower cost; rapid access to university experts for potentially esoteric medical topics; and, for education, deep university experience in terms of assembling didactic programming and recruiting speakers. We offer this model for collaboration for others performing mandatory regulatory post-marketing pharmacovigilance activities.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"151-156"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144132635","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":"Importance of Health Economics and Outcomes Research in the Product Lifecycle.","authors":"Amit Dang","doi":"10.1007/s40290-025-00564-z","DOIUrl":"10.1007/s40290-025-00564-z","url":null,"abstract":"<p><p>Health economics and outcomes research (HEOR) has become an integral part of healthcare systems, through its ability to authentically demonstrate the value of the product. HEOR provides healthcare stakeholders with important insights to make informed decisions regarding healthcare delivery. This review aims to highlight the pivotal role of HEOR across the product lifecycle and the value of integrating HEOR activities during the various phases of drug development. Pharmaceutical companies are increasingly realizing that the integration of HEOR activities from early phases of product development through product launch, also during the postmarketing phase, to generate real-world evidence (RWE) can be crucial for their product's continued commercial success. HEOR helps validate the value of a pharmaceutical product, enabling its success in distinct regulatory and health technology assessment (HTA) landscapes across varied geographies. Regardless of several challenges in data collection and analysis, technological advancements facilitate opportunities to improve the value of HEOR. With rising demands for robust clinical evidence by global regulators and economic evidence by HTA agencies and payers, HEOR will become even more crucial in establishing long-lasting value of a pharmaceutical product for all stakeholders, including regulators, patients, prescribers, and payers.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"157-170"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144035965","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":"Artificial Intelligence: Applications in Pharmacovigilance Signal Management.","authors":"Jeffrey Warner, Anaclara Prada Jardim, Claudia Albera","doi":"10.1007/s40290-025-00561-2","DOIUrl":"10.1007/s40290-025-00561-2","url":null,"abstract":"<p><p>Pharmacovigilance is the science of collection, detection, and assessment of adverse events associated with pharmaceutical products for the ongoing monitoring and understanding of those products' safety profiles. Part of this process, signal management, encompasses the activities of signal detection, signal validation/confirmation, signal evaluation, and ultimately, final assessment as to whether a safety signal constitutes a new causal adverse drug reaction. Artificial intelligence is a group of technologies including machine learning and natural language processing that are revolutionizing multiple industries through intelligent automation. Here, we present a critical evaluation of studies leveraging artificial intelligence in signal management to characterize the benefits and limitations of the technology, the level of transparency, and our perspective on best practices for the future. To this end, PubMed and Embase were searched cumulatively for terms pertaining to signal management and artificial intelligence, machine learning, or natural language processing. Information pertaining to the artificial intelligence model used, hyperparameter settings, training/testing data, performance, feature analysis, and more was extracted from included articles. Common signal detection methods included k-means, random forest, and gradient boosting machine. Machine learning algorithms generally outperformed traditional frequentist or Bayesian measures of disproportionality per various metrics, showing the potential utility of advanced machine learning technologies in signal detection. In signal validation and evaluation, natural language processing was typically applied. Overall, methodological transparency was mixed and only some studies leveraged \"gold standard\" publicly available positive and negative control datasets. Overall, innovation in pharmacovigilance signal management is being driven by machine learning and natural language processing models, particularly in signal detection, in part because of high-performing bagging methods such as random forest and gradient boosting machine. These technologies may be well poised to accelerate progress in this field when used transparently and ethically. Future research is needed to assess the applicability of these techniques across various therapeutic areas and drug classes in the broader pharmaceutical industry.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"183-198"},"PeriodicalIF":3.1,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12126317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022848","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}