Pharmaceutical Medicine最新文献

{"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":"Correction: Shifting Paradigms in Medicines Development as a Profession: To Be or Not To Be.","authors":"Soneil Guptha, Domenico Criscuolo","doi":"10.1007/s40290-025-00570-1","DOIUrl":"https://doi.org/10.1007/s40290-025-00570-1","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111556","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":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144064344","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":"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":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031165","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 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":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144079229","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":"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":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022848","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":"Challenges to Establishing Pharmaceutical Medicine as a Profession: Needs for Professional Identity and a Career Pathway: Role of Education and Training.","authors":"Honorio Silva, Varvara Baroutsou, Domenico Criscuolo, Soneil Guptha, Anna Jurczynska, Sandor Kerpel-Fronius, Heinrich Klech, Ingrid Klingmann","doi":"10.1007/s40290-025-00559-w","DOIUrl":"https://doi.org/10.1007/s40290-025-00559-w","url":null,"abstract":"<p><p>Pharmaceutical Medicine (PM) was defined by its founding pharmaceutical physicians as a \"medical/scientific discipline concerned with the discovery, development, evaluation, registration, monitoring, and medical aspects of the commercialization of medicines, for the benefit and the health of the community.\" Conceived as a medical specialty, the discipline encompassed several health-related professions and occupations, such as medicine, pharmacy, clinical pharmacology, drug safety and pharmacovigilance, pharmaceutical sciences, biology, health economics and others. Historically, non-medically qualified medicines development scientists from health-related disciplines have gradually grown into the medical roles within pharmaceutical organizations, regulatory agencies, and contract research organizations and today physicians and non-physicians lead drug development groups. Medicines Development Sciences (MDS) has been accepted as an alternative name. The practice of the disciplines involved in PM/MDS has evolved from an occupation to be accepted as a distinct profession, by meeting well established criteria. Despite thousands of health professionals participating today in medicines development, their awareness of the existence of this discipline, and its evolution from occupation to profession is low. The lack of professional identity and a clear career path have been identified as key factors limiting the advancement of this profession. This position paper aims to consolidate PM/MDS as an advanced profession in health sciences and propose a career path that is envisioned and championed by educational standards setting organizations like the International Federation of Association of Pharmaceutical Physicians and Pharmaceutical Medicine (IFAPP), PharmaTrain, and Global Medicines Development Professionals (GMDP) Academy with education and training as pillars for its achievement.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034472","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":"Shifting Paradigms in Medicines Development as a Profession: To Be or Not To Be.","authors":"Soneil Guptha, Domenico Criscuolo","doi":"10.1007/s40290-025-00565-y","DOIUrl":"10.1007/s40290-025-00565-y","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015961","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}