Pharmaceutical Medicine最新文献

{"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}

{"title":"Disproportionality Analysis and Causal Inference in Drug Safety.","authors":"Emil Scosyrev, Sigrid Behr, Devendra Jain, Arun Ponnuru, Christiane Michel","doi":"10.1007/s40290-024-00549-4","DOIUrl":"10.1007/s40290-024-00549-4","url":null,"abstract":"<p><p>Disproportionality analysis is a method of safety signal detection based on quantitative analysis of spontaneous reports of adverse events. Disproportionality findings are often presented in medical publications as real-world evidence on drug safety. In this paper, we review theoretical properties of disproportionality analysis in the framework of causal inference theory. We show that measures of disproportionality can approximate the causal rate ratio for a specific drug-event combination when the study drug and the set of comparator drugs satisfy all of the following conditions: (1) there is no uncontrolled confounding for the drug-event association of interest, (2) under-reporting for the event of interest is either absent or has the same relative magnitude for the study drug and for the comparator drugs, and (3) reporting rates for all adverse events combined are the same for the study drug and for the comparator drug set. Because these conditions are typically not even approximately satisfied in practice, the overwhelming majority of disproportionality hits represent statistical noise rather than causal associations. Researchers choosing to report disproportionality findings in publications should explicitly acknowledge all key assumptions and the exploratory nature of this data-mining technique.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"97-107"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143542890","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 Importance of Including Underserved Populations in Research.","authors":"Kayla R Mehl, Stephanie R Morain, Emily A Largent","doi":"10.1007/s40290-025-00562-1","DOIUrl":"10.1007/s40290-025-00562-1","url":null,"abstract":"<p><p>This paper provides an overview of the ethical considerations surrounding the inclusion of underserved populations in later-phase clinical trials. Underserved populations, defined here as those with restricted access to or limited benefits from healthcare, often face systemic, logistical, and social barriers that limit their participation in research. This results in a lack of representation that undermines fairness in research and also hampers the development of effective inclusive healthcare practices. This paper argues that including underserved populations in research is crucial for promoting justice, increasing the generalizability of research findings, and building trust in medical institutions. It differentiates underserved populations from other populations of interest, including vulnerable, minority, and underrepresented groups. It then explores barriers to research participation and targeted solutions for four underserved populations: rural residents, racial and ethnic minorities, low-income individuals, and older adults. Strategies for improving participation include expanding trial sites to accessible locations, lowering financial and logistical barriers, broadening eligibility criteria, and fostering culturally tailored outreach and engagement. While some interventions may apply broadly across groups, effective solutions will often require intersectional and context-specific strategies tailored to each population's unique needs as well as coordinated efforts from multiple stakeholders. While these interventions alone cannot resolve healthcare inequities - as underrepresentation of underserved populations in research is just one contributing factor - their widespread implementation would represent meaningful steps toward advancing health equity.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"59-71"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11980435/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143763991","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":"Innovation in the Design of Clinical Trials for Infectious Diseases: Focusing on Patients Over Pathogens.","authors":"John H Powers, Robert J O'Connell","doi":"10.1007/s40290-025-00552-3","DOIUrl":"10.1007/s40290-025-00552-3","url":null,"abstract":"<p><p>Much infectious disease research focuses on the interaction of microorganisms and drugs in the laboratory, assuming biological activity of inhibiting organism growth in vitro directly translates to improving patient outcomes in the clinic. Yet in vitro testing does not consider the important role of the human immune system in causing and response to disease. Research shows that patient outcomes are still suboptimal even with disease due to organisms that maintain in vitro susceptibility to currently available drugs. Resources and discussions have focused on \"antimicrobial resistance\" yet the majority of deaths are with susceptible organisms. Studies of new interventions do not address the questions that patients and clinicians in practice ask in order to improve patient outcomes regardless of causative pathogen in patients who would receive the drugs in the real-world setting. Research in infectious diseases should shift to refocus on improving patient outcomes. This would result in changes in the research questions evaluated, the types of patients enrolled, the comparisons made, the interventions studied, the outcomes evaluated, and the types of statistical evaluations used. In turn this would provide patients and clinicians with better evidence for patient care and justify payment for new interventions.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"73-86"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11976791/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143616729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Economic Impact of Reliance on an African Medicines Regulatory Authority.","authors":"Lorraine Danks, Boitumelo Semete-Makokotlela, Regardt Gouws, Kennedy Otwombe, Stuart Walker, Sam Salek","doi":"10.1007/s40290-025-00553-2","DOIUrl":"10.1007/s40290-025-00553-2","url":null,"abstract":"<p><strong>Background and objectives: </strong>The inherited backlog of 16,000 medicines applications of the South African Health Products Regulatory Authority (SAHPRA) was cleared through facilitated review pathways that included reliance on prior work by trusted regulators. This research aimed at determining the economic impact of reliance on national regulatory authorities (NRAs) in terms of lower assessors' costs, especially to offset the financial efforts required to attain a higher World Health Organization (WHO) maturity level and understanding the way fees can sustain NRA activities.</p><p><strong>Methods: </strong>To this end, the assessor costs associated with reliance and full review applications were calculated and compared. A high-level review of African NRA fee structures was also carried out and pharmaceutical industry input was solicited regarding the feasibility of alternative tariff modalities for low- and middle-income (LMIC) NRAs.</p><p><strong>Results: </strong>The investigation showed a marked reduction in time spent in reliance assessments compared to full reviews, with an associated decrease in reviewers' costs; SAHPRA conserved US$277,413 across the 188 applications applying reliance principles. The NRA fee structure review revealed outdated fees with little differentiation between full and reliance assessment. NRAs lack the financial resources to strengthen regulatory systems; WHO Global Benchmarking Tool activities are not directly covered by levied fees. Overall, the pharmaceutical industry was supportive of advancing the maturity of African NRAs and was willing to pay increased fees for reliance reviews when authorities adhere to published timelines. More expensive fast-track services were cited, making an argument for higher fees for reliance assessment when this enables medicines to reach markets quicker.</p><p><strong>Conclusions: </strong>Reliance is a tool to safeguard NRA resources and support regulatory and information systems strengthening. The study illustrates the return on investment of reliance for NRAs and, if optimally implemented, the benefits for patients.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"109-123"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11976329/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143537726","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":"Compliance with Cyproterone Contraindications and Meningioma Risk: Resource-Efficient Use of Aggregated Statistics from Swedish National Health Registers.","authors":"Karl Mikael Kälkner, Anders Sundström, Rickard Ljung","doi":"10.1007/s40290-025-00560-3","DOIUrl":"10.1007/s40290-025-00560-3","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"143-145"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634319","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":"Suggested Improvements to the Current East African Community Medicines Regulatory Harmonization Joint Review Process and a Proposed New Review Model for this Initiative.","authors":"Nancy Ngum, Chimwemwe Chamdimba, Dedith Mbonyingingo, Fred Siyoi, Emile Bienvenu, Mawien Atem, Adam Fimbo, David Nahamya, Burhani Simai, Stuart Walker, Sam Salek","doi":"10.1007/s40290-025-00554-1","DOIUrl":"10.1007/s40290-025-00554-1","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;In 2012, the East African Community Medicines Regulatory Harmonization (EAC-MRH) initiative was established to improve access to safe, effective, and high-quality medical products to patients in the East African region. The East African Community (EAC) Partner States, the Republic of Burundi, Democratic Republic of Congo, Republic of Rwanda, United Republic of Tanzania, Republic of Kenya, Republic of South Sudan, and the Republic of Uganda, have a population of 290 million inhabitants. The timely access to medical products for this population was to be achieved through harmonisation of regulatory requirements, joint assessments, joint inspections of manufacturing sites, and the strengthening of regulatory systems. The aims of this study were (1) to investigate ways in which the regional initiative could be a well-coordinated and functioning regional assessment and inspection process on which national registration decisions can rely; (2) to investigate whether a sustainable semi-autonomous regional agency could provide regulatory guidance and coordination for the entire region; and (3) to propose a new and improved model for the EAC-MRH.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Three established questionnaires were used to collect and analyse data on the EAC national regulatory authorities (NRAs) and EAC-MRH initiative 2020-2023: (1) The Optimising Efficiencies in Regulatory Agencies (OpERA) questionnaire was completed by senior officials in the seven authorities that were leading the medicine registration departments about their own respective NRA. The heads of authorities of these NRAs further validated the completed questionnaire, which documented the general organisation of the authorities in terms of their structure, organisation, resources, review process, and timelines. (2) The Process Effectiveness and Efficiency Rating (PEER) questionnaire was completed by the seven authorities to obtain the views of the individual medicines regulatory authorities of the EAC-MRH initiative to identify the strengths and challenges regarding the performance of the joint assessment of the EAC-MRH initiative. (3) The PEER questionnaire, modified for the pharmaceutical industry, was completed by the heads of regulatory units in the pharmaceutical companies that had used the EAC-MRH process for the review and approval of their applications.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The number of applications received for joint reviews increased from nine applications in 2015 to 44 applications in 2023, and the median review time reduced from 553 calendar days in 2015 to 259 calendar days in 2023. A key benefit for pharmaceutical companies using the work-sharing initiative to apply for marketing authorisation was the reduced burden associated with the need to prepare only one application for submission and eventual access to several markets simultaneously.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;The EAC-MRH initiative can only be effective and efficient","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"125-141"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11976760/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143557628","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":"Visionary Health Architects and Leaders: The Strategic Role of Medical Advisors in Modern Pharma.","authors":"Selene García-García, Joan Rius-Tarruella","doi":"10.1007/s40290-025-00557-y","DOIUrl":"10.1007/s40290-025-00557-y","url":null,"abstract":"<p><p>This paper reviews the pivotal role of the Medical Advisor (MA) within the medical department of the pharmaceutical industry, highlighting their essential contribution to the development, commercialization, and appropriate use of medicines. In an environment characterised by stringent regulations and increasing demands for transparency, the MA role has become an indispensable position for ensuring optimal integration between scientific innovation and commercial strategy. The work of MAs facilitates cross-functional collaboration between a board spectrum of internal teams and external stakeholders, ensuring that scientific and clinical knowledge is effectively integrated at every stage of the product lifecycle. This includes leveraging emerging new technologies such as artificial intelligence and digital health solutions, as well as using machine learning to enhance predictive analytics. Such integration is critical to addressing unmet medical needs while aligning these initiatives with broader business objectives to drive innovation, market competitiveness, and patient outcomes. We explore the key responsibilities of today's MAs, which include contributing to the generation of data through clinical trials and post-authorization studies, providing continuing medical education, and communicating accurate information to diverse audiences, including healthcare providers, regulators, and patients. Furthermore, MAs promote innovation and therapeutic progress, acting as guardians of medical ethics. This review aims to provide a comprehensive understanding of the strategic role of MAs in bridging the gap between scientific research and clinical practice. Their contributions are critical to addressing current industry challenges, ensuring that companies remain competitive and making a significant contribution to patient wellbeing and medical progress.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"87-95"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634326","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":"Acknowledgement to Referees.","authors":"","doi":"10.1007/s40290-024-00548-5","DOIUrl":"https://doi.org/10.1007/s40290-024-00548-5","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040668","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}