Pharmaceutical Medicine最新文献

{"title":"Impact of Regional Healthcare Policy Interventions on Biosimilar Use: A 10-year Evaluation of TNFα Inhibitors Utilization in Italy.","authors":"Giacomo Vitturi, Luca Piccoli, Andrea Spini, Giorgia Pellegrini, Luca L'Abbate, Chiara Bellitto, Ylenia Ingrasciotta, Olivia Leoni, Arianna Mazzone, Domenica Ancona, Paolo Stella, Anna Cavazzana, Angela Scapin, Sara Lopes, Valeria Belleudi, Stefano Ledda, Paolo Carta, Paola Rossi, Lucian Ejlli, Ester Sapigni, Aurora Puccini, Rita Francesca Scarpelli, Giovambattista De Sarro, Alessandra Allotta, Sebastiano Addario Pollina, Roberto Da Cas, Giampaolo Bucaneve, Antea Maria Pia Mangano, Francesco Balducci, Carla Sorrentino, Ilenia Senesi, Marco Tuccori, Rosa Gini, Ugo Trama, Francesca Futura Bernardi, Flavia Mayer, Stefania Spila Alegiani, Marco Massari, Silvia Adami, Giovanna Scroccaro, Gianluca Trifirò","doi":"10.1007/s40290-026-00629-7","DOIUrl":"10.1007/s40290-026-00629-7","url":null,"abstract":"<p><strong>Background and objectives: </strong>Tumor neurosis factor alpha (TNFα) inhibitors are widely used for the treatment of immune-mediated inflammatory diseases. Following the patent expiration of infliximab, etanercept, and adalimumab, biosimilars have become available in Europe. In Italy specifically, each region adopted heterogeneous strategies to promote biosimilar uptake. The aim of this study was to describe the impact of different regional healthcare policy interventions on the utilization of biosimilars of adalimumab, infliximab, and etanercept in the last decade.</p><p><strong>Methods: </strong>A descriptive, multiregional, retrospective cohort study was conducted in Italy in the period 2015-2023 using claims data of 14 regions from the VALORE distributed database network. First, regional policy interventions related to TNFα inhibitors biosimilars were retrieved from the regional official websites and classified into six categories: monitoring procedures, regional guidelines, regional prescribing recommendations, drug expenditure/prescription quotas, medication supply timing, and financial incentives. Secondly, the proportion of biosimilar users among incident and prevalent users of TNFα inhibitors with expired patent, stratified by active ingredient, calendar year, and region, was calculated. Then, regional policy intensity was quantified for each year using a composite score on the basis of the six categories of healthcare policy, and categorized into three levels (0, 1, ≥ 2). Short-term changes in biosimilar use were assessed as the absolute percentage change between the year of policy adoption (t) and the subsequent year (t + 1), across both policy intensity levels and policy types, and reported as medians, and interquartile ranges. Finally, the compound annual growth rate (CAGR) of the prevalence of TNFα inhibitors biologic users per 100,000 inhabitants over time was calculated to estimate the temporal trend of TNFα inhibitors use in the study period.</p><p><strong>Results: </strong>A total of 88 types of regional policy interventions in the form of official decrees or documents related to biosimilar use were issued by the involved regions. The most common healthcare policies implemented were regional prescribing recommendations and drug expenditure/prescription quotas (n = 27, 30.7%). In the years 2015-2023, a total of 128,288 prevalent users of patent-expired TNFα inhibitors, with 112,526 being incident users, were identified. Overall, prevalent biosimilar users increased from 9% in 2015 to 84% in 2022, with differences across regions. This proportion was almost always above 80% among incident users across the study period, with a more homogeneous uptake between regions. Concerning policy analyses, years in which regions implemented ≥ 2 policy interventions showed greater short-term increases in biosimilar use: 31% for adalimumab, 18% for etanercept, and 14.5% for infliximab, compared with years with no policy implementatio","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"503-518"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649869/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796372","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":"Digital Transformation in Pharmaceutical R&D in a Midsized Company: Unlocking Innovation Through Human and Organizational Factors.","authors":"Elena Businaro, Ciro Cottini, Fabio Rancati, Eva Topole, Annalisa Forlenza, Martina Binelli, Priscilla Dora Mazzoli, Tina Dell'Armi, Diego Ardigò, Riccardo Mariani","doi":"10.1007/s40290-026-00628-8","DOIUrl":"10.1007/s40290-026-00628-8","url":null,"abstract":"<p><p>Digital transformation presents a strategic imperative for the pharmaceutical industry, particularly within research and development (R&D), where it promises accelerated time to market and enhanced operational efficiency. Building on the foundation of the People Coordination, Ownable Focus Areas, Long-Term Roadmap, Common Digital Alphabet, Reporting, and Monitoring (P.O.L.A.R.) Star framework, this article delves into the critical soft elements-human and organizational factors-essential for deploying a successful digital transformation in the pharmaceutical R&D of a midsized company. While technological roadmaps provide structure and serve as a blueprint, sustainable results depend on an organization's ability to evolve both in its culture and approach to change. In this context, implementing new capabilities-such as centralized R&D data governance and a common data model-not only requires a transparent approach to data, strong digital leadership, and deep expertise but also triggers an organizational rethinking to support this evolution. Based on the needs and current evolution phase of the digital transformation journey of the company, this rethinking can be represented by a \"federated\" approach based on cross-functional teams, a digital unit to centralize activities and solutions, or a hybrid model combining elements of both. In summary, this article offers a focused, actionable guide to managing digital transformation in pharmaceutical R&D. It explores the concept of a digital unit or a cross-functional team as strategic options, evaluated to guide intentional, structured operations within a digital reorganization; it also highlights the cultural shift needed for long-term sustainability.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"427-439"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649842/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761120","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":"New Therapeutic Modalities and Collingridge's Dilemma.","authors":"Torbjörn Callréus","doi":"10.1007/s40290-026-00634-w","DOIUrl":"10.1007/s40290-026-00634-w","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"423-426"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148900999","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":"Embracing Digital as a Paradigm Shift in Medical Affairs.","authors":"Amit Lalwani, Jon Zdon, Maaike Addicks, Yacin Marzouki, Densearn Seo, Orin Chisholm","doi":"10.1007/s40290-026-00623-z","DOIUrl":"10.1007/s40290-026-00623-z","url":null,"abstract":"<p><p>Digital transformation is increasingly shaping how the Medical Affairs (MA) function generates insights, engages stakeholders, and demonstrates value within the pharmaceutical industry. This article examines how MA professionals can navigate and lead the digital revolution reshaping healthcare delivery and stakeholder engagement. Digital maturity progresses from basic channel activation through tactical integration to advanced personalisation enabled by artificial intelligence (AI). Central to this transformation is the convergence of omnichannel engagement with real-world evidence generation, creating unprecedented opportunities to deliver personalised, scientifically rigorous interactions that enhance patient outcomes and healthcare professional (HCP) satisfaction. AI applications are creating new opportunities for MA to generate insights, communicate evidence, and measure impact across the healthcare ecosystem, enabling predictive analytics and intelligent content delivery at scale. Digital transformation is less likely to be effective when implemented through organisational silos. Important enablers include cross-functional collaboration, robust governance for responsible AI deployment, co-creation with external partners including HCPs and patient advocacy groups, and shared technology infrastructure that supports coordinated engagement. Best practices for digital communication strategies, fostering innovation cultures, and demonstrating measurable value are explored. As healthcare's digital evolution accelerates, MA has an important role in ensuring that digital tools are applied in ways that remain scientifically rigorous, ethically governed, and aligned with stakeholder needs. By treating digital as an enabler of high-quality scientific exchange rather than an end in itself, MA can support more relevant engagement and strengthen the translation of evidence into practice.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"469-483"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649852/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148437423","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":"From Compliance to Strategic Partner: The Transformation of Regulatory Affairs in AstraZeneca Local Affiliates.","authors":"María Cristina Fernández Montero, Hannie de Munnik, Agnieszka Sondej, Ana Pérez Domínguez","doi":"10.1007/s40290-026-00632-y","DOIUrl":"10.1007/s40290-026-00632-y","url":null,"abstract":"<p><p>The role of Regulatory Affairs (RA) within local pharmaceutical affiliates is evolving from a predominantly compliance-driven support function to a strategic partner in drug development and patient access. Traditionally, local RA focused on dossier preparation, regulatory compliance, liaison with national authorities, and maintenance of marketing authorisations, largely executing global plans downstream of clinical development. The increasing complexity of novel therapies (including advanced therapy medicinal products), evolving EU regulatory frameworks, and the broader Medical Affairs (MA) 2030 vision now require RA to be integrated earlier and to operate as a more proactive and strategic partner across functions. This article describes the transformation of local Marketing Companies Regulatory Affairs (MCRA) teams at AstraZeneca (AZ) in Europe, structured around three pillars: (1) Launch Excellence, embedding local regulatory insight into early-asset, launch, and brand planning to support evidence generation, risk anticipation and implementation-ready launches; (2) External Engagement and Advocacy, strengthening strategic dialogue with national Health Authorities and contributing local perspectives to policy discussions on key EU initiatives such as the Health Technology Assessment (HTA) Regulation, New Pharmaceutical Legislation, Clinical Trials Regulation, Biotech Act, Critical Medicines Act, digital labelling, accelerated pathways and adaptive licensing; and (3) Digitalisation and Process Optimisation, leveraging standardised tools, RA platforms and emerging artificial intelligence (AI) solutions to simplify ways of working, improve alignment, and support more efficient submission and lifecycle management. Central to this transformation is \"One-Team\" Local-Global RA culture of AZ, operationalised through the MCRA Operating Model. Local MCRA professionals are fully embedded in affiliate cross-functional teams while maintaining bi-directional collaboration with Global RA, enabling local insights to inform global strategy and helping ensure that global decisions are implementable at the country level. To monitor progress, a contribution-based measurement framework is proposed, combining quantitative and qualitative indicators aligned with the three transformation pillars and the \"One-Team\" model. The framework focuses on predictability, launch readiness, environment-shaping activities and organisational effectiveness, recognising that regulatory outcomes result from cross-functional decision making across the product lifecycle. Overall, early and strategic RA involvement, structured around the three pillars and underpinned by a \"One-Team\" culture, helps accelerate patient access to innovative medicines across Europe.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"457-468"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649880/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148970458","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":"Evaluating AI-Enabled Voice to Text for Medical Affairs Documentation and Insight Capture: The RAPID Randomized Crossover Trial.","authors":"Riaz Abbas, Jeongin Son, Marloes Oosterhof, Yosuke Amagai, Jackie Tang, Cheng Zhang, Bin Li, Caidan Luo, Seigo Susa, Ka Weng Mah, Helen Lin","doi":"10.1007/s40290-026-00630-0","DOIUrl":"10.1007/s40290-026-00630-0","url":null,"abstract":"<p><strong>Background and objectives: </strong>Artificial intelligence (AI) transcription systems using large language models (LLMs) and natural language processing (NLP) may reduce documentation burden. Medical science liaisons (MSLs) face challenges documenting scientific interactions with health care professionals (HCPs). This Amgen study evaluated whether AI-enabled transcription improved MSL documentation experience and insight documentation compared with existing practice (EP) of manual customer relationship management (CRM) entry.</p><p><strong>Methods: </strong>This randomized crossover study enrolled 27 MSLs from six Amgen Asia-Pacific affiliates; 78% used non-English languages during V2T documentation. Participants were randomized 1:1 to voice-to-text (V2T) using Microsoft Copilot then EP, or EP then V2T (Copilot), with four-week study periods. The primary outcome was composite MSL Satisfaction Questionnaire (MSLSQ) score across efficiency, speed to CRM entry, cognitive load, ease of use, and insight structuring. Secondary assessment compared blinded therapeutic area (TA) expert ratings of insight quality. Exploratory analyses assessed language effects, interaction counts, and qualitative feedback.</p><p><strong>Results: </strong>All participants completed both periods. Composite MSLSQ scores were higher with V2T (Copilot) than EP (mean difference + 0.763; p = 0.0114, two-way repeated-measures analysis of variance (ANOVA)). Domain-level primary comparisons favored V2T (Copilot) across all five MSLSQ domains, while Wilcoxon sensitivity analyses most consistently supported improvements in speed to CRM entry, cognitive load, and insight structuring. TA expert-rated insight quality and documented interaction counts were similar. Satisfaction was lower among Japanese and Korean-speaking participants owing to lower transcription accuracy.</p><p><strong>Conclusions: </strong>V2T (Copilot) improved documentation experience without improving insight quality or field activity. Limitations related to scientific terminology recognition, multilingual transcription, highlight opportunities to optimize AI-supported documentation in Medical Affairs. V2T (Copilot) improved the composite MSL-reported documentation satisfaction score, with the greatest improvements observed in cognitive load, speed to CRM entry, and insight structuring. Performance strengths included reduced documentation burden and improved structuring, while practical limitations included recognition of scientific terminology, lower transcription accuracy in Japanese and Korean, and lack of native CRM integration.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"519-531"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649876/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148901083","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":"Application of Actor Network Theory to Evaluate Supply and Demand Strategies Used to Encourage International Biosimilar Uptake.","authors":"Chad Rieger, Judith A Dean, Gregory Merlo, Lisa Hall","doi":"10.1007/s40290-026-00627-9","DOIUrl":"10.1007/s40290-026-00627-9","url":null,"abstract":"<p><p>Biosimilar medicines have the potential to reduce costs and expand patient access, yet uptake rate remains uneven world-wide. This policy review will (1) evaluate and compare biosimilar medicine uptake and policy frameworks across eight countries in the Organisation for Economic Co-operation and Development (OECD) including Australia and (2) apply Actor Network Theory (ANT) translation to interpret the mechanisms underlying the observed differences in adoption. Targeted searches of academic databases and grey literature were conducted for the period 2017 to 2025. Policies from Australia, Belgium, Denmark, France, Germany, Italy, Canada, and UK were reviewed and classified as either supply-side or demand-side interventions. Maximum market penetration rates of six selected biosimilar products were assessed as the principal indicators of successful adoption. Actor network theory was applied to interpreting how policy makers align other actors in the network to various interventions and to explain differences in effectiveness. Correlation exists between countries achieving high levels of uptake and implementation of two distinct policy uptake methodologies. The first combined multiple supply- and demand-side measures, including incentives, substitution policies, and educational initiatives. The second relied primarily on coordinated national tendering systems operating across hospital and retail markets. Moderate-performing countries employed similar measures but with less consistent implementation, while lower-uptake countries tended to rely predominantly on price controls with limited demand-side engagement. Overall, policy approaches that either integrate multiple complementary interventions or implement robust tendering frameworks appear to be correlated with highest rates of biosimilar uptake. Applying ANT highlights how policy design interacts with healthcare networks, offering insights that may inform more effective biosimilar policy development. Actor network theory illustrates why the policy combinations may be associated with higher levels of uptake: they translate diverse actors into more stable configurations that support adoption.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"441-455"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13649829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761478","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":"Global Mapping of National Health Economic Evaluation Guidelines: Evidence from 40 Countries.","authors":"Thu Anh Do, Tung Hoang","doi":"10.1007/s40290-026-00618-w","DOIUrl":"10.1007/s40290-026-00618-w","url":null,"abstract":"<p><p>Health economic evaluation (HEE) guidelines play an important role in health technology assessment (HTA) by supporting the consistent and transparent use of economic evidence in decision making. As countries continue updating their guidelines, substantial methodological variation persists. This review aimed to provide an updated global comparison of national HEE guidelines. National HEE guidelines were identified from the ISPOR and GEAR repositories and supplemented by hand-searching and searches of governmental and institutional sources. Two reviewers independently extracted data across 23 methodological domains, including analytical perspective, comparators, cost categories, outcome measures, modeling, discounting, evidence synthesis, and uncertainty analysis. Forty national guidelines were identified (25 high-income countries, 9 upper-middle-income countries, and 6 lower-middle-income countries). Most guidelines had been updated between 2015 and 2025. Forty-eight percent were recommendatory, 43% mandatory, and 10% voluntary. Cost-utility analysis was preferred in 48% of guidelines, and quality-adjusted life-years (QALYs) were recommended in 53%. Systematic review and the use of real-world evidence was commonly emphasized. Discount rates ranged from 1.5 to 5%, largely reflecting national economic conditions. Nearly all guidelines required clear reporting of assumptions, parameter values, and sensitivity analyses. Variation remained in preferred perspectives, comparator selection, inclusion of indirect costs, and utility measurement methods. This review provides an updated global overview of national HEE guidelines, highlighting areas of convergence in analytical methods, evidence requirements, and approaches to uncertainty. Persistent differences across countries and income settings underscore the need for context-specific but methodologically robust guidance to support HTA and healthcare decision making.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"485-501"},"PeriodicalIF":4.7,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148796339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Galenic Modification as an Alternative Strategy for Administering Oral Anticancer Drugs: Assessment of Bioequivalence.","authors":"Pauline Claraz, Cécile Pineau, Coralie Guillemot, Etienne Chatelut, Cécile Arellano, Florent Puisset","doi":"10.1007/s40290-026-00622-0","DOIUrl":"https://doi.org/10.1007/s40290-026-00622-0","url":null,"abstract":"<p><strong>Background: </strong>Oral anticancer drugs have become widely used, improving patient autonomy and quality of life. However, patients with swallowing disorders, such as children, older adults, and patients with head and neck cancer or neurologic disease, often require galenic modifications (e.g., crushed tablets, oral suspensions) that may impact drug pharmacokinetics and clinical outcomes.</p><p><strong>Objective: </strong>The aim of this study was to assess the impact of these modifications on bioequivalence and systemic drug exposure.</p><p><strong>Methods: </strong>A systematic research of regulatory documents and scientific literature was conducted on 96 oral anticancer drugs. Data were collected on bioequivalence or relative bioavailability between standard oral solid forms and alternative manipulated or liquid formulations. Chemical and pharmaceutical properties of drugs were assessed as predictive factors for bioequivalence.</p><p><strong>Results: </strong>Data were available for 39 drugs among the 96 selected for the study. The liquid form of 26 drugs did not meet full bioequivalence criteria: maximum plasma concentration (C_max) was more frequently altered than area under the curve (AUC), suggesting that galenic modification primarily affected the absorption rate rather than overall drug exposure. Nevertheless, in 83% of cases, AUC ratios remained between 0.5 and 2. No predictive factors of bioequivalence were identified.</p><p><strong>Conclusion: </strong>Liquid formulation and galenic modification of anticancer drugs primarily increases drug exposure, especially C_max. A priori dose adjustments could not be recommended due to the low and unpredictable pharmacokinetic impact of galenic modifications. Consequently, therapeutic drug monitoring is required when possible, and manipulation should be performed under supervised and standardized conditions.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149063589","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Manufacturer-Perceived Development and Market-Entry Risks Associated with Discontinuation and Non-launch of Generic Medicines in Japan: A Cross-Sectional Survey of Marketing Authorization Holders.","authors":"Hiroaki Mamiya, Harumi Tagoku, Ken-Ichi Izutsu","doi":"10.1007/s40290-026-00636-8","DOIUrl":"https://doi.org/10.1007/s40290-026-00636-8","url":null,"abstract":"<p><strong>Background: </strong>Generic medicines are essential for pharmaceutical affordability and access, yet some products are never launched or their development is discontinued even after patent expiry. Limited empirical evidence is available on how manufacturers perceive the development and market-entry risks associated with these outcomes.</p><p><strong>Objective: </strong>The aim of this study was to identify and structure manufacturer-perceived development and market-entry risk factors associated with generic medicine development discontinuation and non-launch in Japan.</p><p><strong>Methods: </strong>We conducted a cross-sectional survey of companies involved in generic medicine development and commercialization in Japan. The primary survey outcome was company-reported experience of development discontinuation or post-approval non-launch during fiscal years 2020-2024. Respondent characteristics were compared between companies with and without such experience. Among companies with such experience, development and market-entry risk factors were assessed in terms of both experience and perceived importance. All responding companies were asked to rate policy and regulatory options on five-point importance scales.</p><p><strong>Results: </strong>Among 122 companies with valid outcome data, 52 (42.6%) reported experience of development discontinuation or non-launch. Such experience was more frequently reported by companies with larger generic product portfolios. The market size and profitability domain was rated highest among the risk domains (top-2 box importance, 94.2%), but regulatory and clinical-study requirements (75.0%), formulation technology and manufacturing complexity (72.5%), and competition and institutional uncertainty (48.0%) were also rated as important. Among individual risk factors, active pharmaceutical ingredient (API) supply, patient-based bioequivalence or clinical-study requirements, dedicated equipment investment, and special formulation requirements were, in an exploratory descriptive map, among the factors with relatively high reported experience and perceived importance.</p><p><strong>Conclusions: </strong>Generic medicine development discontinuation and non-launch in Japan were perceived by manufacturers as involving multiple development and market-entry risks extending beyond pricing to include API supply, manufacturing complexity, regulatory uncertainty, and competitive dynamics. Respondents prioritized reimbursement, regulatory, and manufacturing-related options for further consideration; the study did not evaluate the effectiveness, budget impact, or net social benefit of these options.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148970514","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}