Therapeutic innovation & regulatory science最新文献_第6页

Provision of Drug Information Using Database Surveys-Enhancing Clinical Information for Patients with Specific Backgrounds. 利用数据库调查提供药物信息--为具有特殊背景的患者提供更多临床信息。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-24 DOI: 10.1007/s43441-024-00715-5

Kiyohito Nakai, Mie Ikeda, Yumiko Nomura

引用次数: 0

Turning the Page on Hardcopy Risk Management Plan Educational Materials: Digitalization Made Possible. 翻开硬拷贝风险管理计划教材的新一页：数字化成为可能。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-11-25 DOI: 10.1007/s43441-024-00717-3

Anabel Ng, Ayn Nova Celo, Beliz Fernandes

{"title":"Turning the Page on Hardcopy Risk Management Plan Educational Materials: Digitalization Made Possible.","authors":"Anabel Ng, Ayn Nova Celo, Beliz Fernandes","doi":"10.1007/s43441-024-00717-3","DOIUrl":"10.1007/s43441-024-00717-3","url":null,"abstract":"'Risk Management Plan Educational Materials' (RMP EMs) are additional risk minimization measures (aRMMs) intended to prevent or reduce the occurrence of adverse reactions associated with the exposure to a medicine, or to reduce their severity or impact on the patient. While the healthcare sector is embracing various digital tools and platforms for educational and/or awareness building purposes, paper-based materials have remained the mainstay approach for implementation of aRMMs by pharmaceutical companies. Novartis in Singapore conducted a pilot on the feasibility of distributing electronic copies of RMP EMs (e-RMP). Post-pilot, e-RMP was officially implemented in Novartis Singapore. A year following the launch of e-RMP, a survey was performed with healthcare professionals (HCPs) to understand end-users' experience. The survey responses revealed a general preference by both HCPs and patients towards e-RMP. Digital methods of delivering educational aRMMs offer great benefits over traditional paper-based programs. e-RMP significantly reduces the time needed for updated RMP EMs to reach HCPs and their patients/caregivers. This is important to ensure that HCPs and patients/caregivers are made aware of any updates in key safety messages of the products in a timely manner to ultimately ensure patient safety. The successful transition to digital solutions requires purposeful collaborations between key stakeholders of the healthcare ecosystem including regulatory authorities, pharmaceutical companies, HCPs, patients and caregivers. This article aims to provide insights on the digitalization journey of e-RMP, a case study in Singapore, outlining the value but also some of the challenges faced during this transformation.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"164-172"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706838/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142717154","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Experimental Study of the Promotional Implications of Proprietary Prescription Drug Names. 处方药名称的促销意义实验研究》。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-09-28 DOI: 10.1007/s43441-024-00704-8

Susana Peinado, Amie C O'Donoghue, Kevin R Betts, Ryan S Paquin, Kristen Giombi, Jennifer E Arnold, Bridget J Kelly, Christine Davis

{"title":"Experimental Study of the Promotional Implications of Proprietary Prescription Drug Names.","authors":"Susana Peinado, Amie C O'Donoghue, Kevin R Betts, Ryan S Paquin, Kristen Giombi, Jennifer E Arnold, Bridget J Kelly, Christine Davis","doi":"10.1007/s43441-024-00704-8","DOIUrl":"10.1007/s43441-024-00704-8","url":null,"abstract":"Background: The meaning and characteristics embedded in proprietary drug names have the potential to affect name recall, perceptions of drug benefits and risks, and attitudes toward a drug. In this study, we examined: (1) whether names that reference the drug's medical indication affect consumers' and primary care physicians' (PCPs') perceptions of the drug and (2) whether names that overstate the drug's efficacy affect consumers' and PCPs' perceptions of the drug.Methods: We conducted an online experiment with 455 PCPs and 450 consumers to test the effects of fictitious proprietary prescription drug names. Participants were randomized to view one neutral drug name, one name that overstated the drug's efficacy, and five names that referenced the drug's medical indication.Results: Names that referenced the drug's medical indication and names that overstated the drug's benefit both influenced perceptions of efficacy and risk compared to neutral names. For several outcomes, names evoking medical indications had similar effects to those designed to overstate the drug's efficacy. The patterns of effects were similar for PCPs and consumers.Conclusion: Findings suggest drug names alone can be sufficient to produce attitudes and risk and benefit perceptions about drugs, even in the absence of any information beyond the drug's medical indication.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"80-88"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354377","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Better Medicines for Children: Lessons Learnt and Share Learnings at the EFGCP Annual Paediatric Conferences. 为儿童提供更好的药物：在 EFGCP 年度儿科会议上吸取经验教训并分享心得。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-11-13 DOI: 10.1007/s43441-024-00710-w

Solange Corriol-Rohou, Sabine Ingeborg Fürst-Recktenwald, Elin-Haf Davies, Martine Dehlinger-Kremer, Mark A Turner

{"title":"Better Medicines for Children: Lessons Learnt and Share Learnings at the EFGCP Annual Paediatric Conferences.","authors":"Solange Corriol-Rohou, Sabine Ingeborg Fürst-Recktenwald, Elin-Haf Davies, Martine Dehlinger-Kremer, Mark A Turner","doi":"10.1007/s43441-024-00710-w","DOIUrl":"10.1007/s43441-024-00710-w","url":null,"abstract":"For many years, the European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party has organised a Paediatric conference annually. In the past, this event was organised jointly with the European Medicines Agency who was used to host it, along with the Drug Information Association (DIA). This conference is the opportunity for all involved in paediatric drug development, i.e., regulators, HTA bodies, patients' representatives, academia and industry, to share learnings and raise awareness about new regulatory requirements of interest to optimise paediatric drug development. The theme of the 2021 conference was \"Challenges and Solutions - the path forward\" while in 2022 it focused on \"Progress made and Continuing Challenges\". Because of the COVID-19 pandemic these two conferences were organised virtually. However, this has not impacted the attendance and value of the conference, since because of a broad and attractive agenda there was a wide stakeholder participation, which provided a compendious overview of the leading issues to improve children's access to innovative medicines.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"184-189"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A Clustering Ensemble Method for Drug Safety Signal Detection in Post-Marketing Surveillance. 上市后监测中药物安全信号检测的聚类组合方法。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-20 DOI: 10.1007/s43441-024-00705-7

Shubhadeep Chakraborty, Ram Tiwari

{"title":"A Clustering Ensemble Method for Drug Safety Signal Detection in Post-Marketing Surveillance.","authors":"Shubhadeep Chakraborty, Ram Tiwari","doi":"10.1007/s43441-024-00705-7","DOIUrl":"10.1007/s43441-024-00705-7","url":null,"abstract":"Post-marketing surveillance refers to the process of monitoring the safety of drugs once they reach the market, after the successful completion of clinical trials. In this work, we investigate a computational approach using data mining tools to detect safety signals from post-market safety databases, or in other words, to identify adverse events (AEs) with disproportionately high reporting rates compared to other AEs, associated with a particular drug or a drug class. Essentially, we view this as a problem of cluster analysis-based anomaly detection on post-market safety data, where the goal is to 'unsupervisedly' detect the anomalous or the signal AEs. Our findings demonstrate the potential of using a clustering ensemble method to detect drug safety signals. It employs multiple clustering or anomaly detection algorithms, followed by a performance comparison of the candidate algorithms based on a collection of appropriate measures of goodness of clustering results. The method is general enough to include any number of clustering or anomaly detection algorithms and goodness measures, and performs better than any of the candidate algorithms in identifying the signal AEs. Extensive simulation studies illustrate that the ensemble method detects the AE signals from synthetic post-market safety datasets pretty accurately across the different scenarios explored. Based on the cases reported to the FDA Adverse Event Reporting System (FAERS) between 2013 and 2022, we further demonstrate that the ensemble method successfully identifies and confirms most of the adverse events that are known to occur most frequently in reaction to antiepileptic drugs and <math><mi>β</mi></math> -lactam antibiotics.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"89-101"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475472","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Expanding Pharmaceutical Access Via Over the Counter Drugs. 通过非处方药扩大药品供应。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-20 DOI: 10.1007/s43441-024-00709-3

Terra Marie M Jouaneh, Vrushab Gowda, Brian J Miller

引用次数: 0

Pediatric-Specific Drug Loss Issue in Japan: Comparison of Pediatric Development Status Between Japan and the United States. 日本的儿科专用药物流失问题：日本与美国儿科发展状况的比较。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-23 DOI: 10.1007/s43441-024-00714-6

Ryohei Osako, Naoki Matsumaru, Katsura Tsukamoto

{"title":"Pediatric-Specific Drug Loss Issue in Japan: Comparison of Pediatric Development Status Between Japan and the United States.","authors":"Ryohei Osako, Naoki Matsumaru, Katsura Tsukamoto","doi":"10.1007/s43441-024-00714-6","DOIUrl":"10.1007/s43441-024-00714-6","url":null,"abstract":"Background: The lack of label information for the pediatric population has been a global issue, leading to the introduction of several countermeasures by major health authorities. Despite various efforts by Japanese health authorities, some drugs are approved only for adults in Japan, while the United States (US) label includes information on pediatric usage for the same drugs. This suggests a potential for pediatric-specific drug loss in Japan, where overall drug loss has recently become a major concern.Methods: In this study, we compared the pediatric usage status between Japan and the US, focusing on the indications approved in both countries.Results: Of the 404 indications, 70 (17.3%) and 102 (25.2%) included pediatric usage in Japan and the US, respectively. The proportion of indications, including pediatric usage, was significantly higher in the US than in Japan (χ2 test, p < 0.001). Multivariate analysis of indications for pediatric usage in the US demonstrated that simultaneous development with adults (odds ratio (OR), 24.9; 95% confidence interval (CI), 6.79-91.1) and Japan-first development (OR, 31.5; 95% CI, 2.59-384) were significantly affecting the inclusion of pediatric usage in Japan.Conclusions: Our results suggest that there was pediatric-specific drug loss in Japan compared to that in the US. The multivariate analysis demonstrated that US-first development and non-simultaneous development had a negative impact on the inclusion of pediatric usage in Japan; however, pediatric assessment request was not a significant factor. Further frameworks to promote pediatric drug development should be introduced in Japan to address pediatric-specific drug loss issues.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"142-149"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Pharmaceutical Company's Choices of Indication for the First Clinical Projects in Oncological Drug Development in the United States. 制药公司对美国肿瘤药物开发首个临床项目适应症的选择。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-31 DOI: 10.1007/s43441-024-00718-2

Can Wu, Shunsuke Ono

{"title":"Pharmaceutical Company's Choices of Indication for the First Clinical Projects in Oncological Drug Development in the United States.","authors":"Can Wu, Shunsuke Ono","doi":"10.1007/s43441-024-00718-2","DOIUrl":"10.1007/s43441-024-00718-2","url":null,"abstract":"We analyzed factors shaping the choice of the lead indication (i.e., cancer type) in the first clinical development projects of new oncological drugs in the United States (US), and how the type of pharmaceutical company is related to this choice. We selected 576 new clinical development projects in the US since 2000 for analysis. These projects were characterized according to three potential perspectives detected by multiple correspondence analysis: the morbidity of the cancer type which corresponds to market size of each cancer type, the company's previous experience with the cancer type, and the company's attitude to development risks. Mega firms tend to choose cancer types with higher morbidity (and large-market), previously experienced cancer types, while diverse small firms choose both major and rare cancers and both high- and low-risk projects, indicating that different sizes of firms utilize different development entry patterns. Common tendencies concerning the choice of lead indication were found across all companies. Cancer types the company had developed and launched in the past were more likely to be chosen; cancer types with high five-year survival rates and those with high competition were less likely to be chosen. The study showed that pharmaceutical companies seem to enter clinical development from cancer types where they can demonstrate their strengths and advantages through experience, depending on each cancer type's different market sizes and development difficulties. The results could provide clues for considering what support measures and incentives are appropriate to balance the efficiency of industrial development and the fulfillment of society's unmet medical needs.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"9-19"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142558862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Abstract Versus Concrete Risk Identification in Clinical Research in Japan: Randomized and Prospective Pilot Research on the Effect of Risk Reduction Activities in a Risk-Based Approach. 日本临床研究中的抽象与具体风险识别：基于风险的方法中降低风险活动效果的随机和前瞻性试点研究。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-28 DOI: 10.1007/s43441-024-00702-w

Hidenobu Kondo, Shih-Wei Chiu, Yukikazu Hayashi, Naoto Takahashi, Takuhiro Yamaguchi

{"title":"Abstract Versus Concrete Risk Identification in Clinical Research in Japan: Randomized and Prospective Pilot Research on the Effect of Risk Reduction Activities in a Risk-Based Approach.","authors":"Hidenobu Kondo, Shih-Wei Chiu, Yukikazu Hayashi, Naoto Takahashi, Takuhiro Yamaguchi","doi":"10.1007/s43441-024-00702-w","DOIUrl":"10.1007/s43441-024-00702-w","url":null,"abstract":"Background: The risk-based approach (RBA) of clinical trial was first introduced in 2011-2012. RBA necessitates implementing risk reduction activities that are proportionate to risk in order to reduce avoidable quality issues. However, there is no consistent methodology or research for identifying and evaluating risks and planning risk reduction activities. We aimed to evaluate risk reduction activities and their effects by using two risk identification and evaluation methods.Methods: Among the risk identification and evaluation methods, we selected one method with the lowest number of categories for identifying risks [risk assessment form (RAF)] and one with the highest number [risk assessment tool (RAT)]. Each method was used to identify and evaluate risks in and plan risk reduction activities for the research on ponatinib blood concentration and treatment outcome in patients with chronic phase chronic myelogenous leukemia. RAF and RAT can identify risk using abstract questions and a list of concrete risks, respectively. The sites were randomized into two groups to implement planned risk reduction activities using RAF and RAT and to compare the mean of errors and protocol deviation per subject visit between the two groups.Results: The mean of errors per subject visit and the mean of protocol deviation per subject visit were lower in the RAF group than in the RAT group.Conclusions: Our study indicates that risk reductions can be successfully implemented by using a method to identify and evaluate risks in a small number of abstract categories that are critical to quality of clinical research.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"71-79"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142523190","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Expert Consensus Recommendations on a Biosimilars Value Framework for the Gulf Cooperation Council Countries. 关于海湾合作委员会国家生物仿制药价值框架的专家共识建议。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-01-01 Epub Date: 2024-10-30 DOI: 10.1007/s43441-024-00716-4

Khalid A Alnaqbi, Ahmed Al-Jedai, Mohamed Farghaly, Mohammed A Omair, Anas Hamad, Fatemah M A Abutiban, Ali Al Shirawi, Hanan Al Rayes, Sarah Aldallal, Sahar Fahmy, Steven Simoens

{"title":"Expert Consensus Recommendations on a Biosimilars Value Framework for the Gulf Cooperation Council Countries.","authors":"Khalid A Alnaqbi, Ahmed Al-Jedai, Mohamed Farghaly, Mohammed A Omair, Anas Hamad, Fatemah M A Abutiban, Ali Al Shirawi, Hanan Al Rayes, Sarah Aldallal, Sahar Fahmy, Steven Simoens","doi":"10.1007/s43441-024-00716-4","DOIUrl":"10.1007/s43441-024-00716-4","url":null,"abstract":"Objective: This paper aims to develop a biosimilar value framework with local stakeholders in Gulf Cooperation Council (GCC) countries.Methods: A convenience sample of ten key opinion leaders from the United Arab Emirates, Kingdom of Saudi Arabia, Kuwait, Oman and Qatar participated in an expert panel meeting in November 2022 that examined factors positively influencing biosimilar adoption in these countries. The discussion was structured around a conceptual biosimilar value framework and an overview of biosimilar policies as derived from a targeted review of the peer-reviewed and grey literature.Results: The expert panel agreed on a biosimilar value framework for the GCC countries that is founded on trust, cost savings and contextual considerations. They emphasized the importance of launching educational initiatives that build trust in and expand knowledge of all stakeholders about biosimilars. This also includes making stakeholders aware of the various value propositions of biosimilars as an instrument to produce, for example, cost savings. Finally, they stressed that biosimilar adoption is influenced by contextual factors such as incentives and implementation efforts.Conclusion: Our proposed biosimilars value framekwork is the first set of recommendations in the Arab countries designed to help policymakers and decision-makers promote biosimilar adoption, both in high-income GCC countries and in low- and middle-income countries.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"153-163"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706834/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142547634","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0