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Synthesis, anticancer properties, and biological profiling of synthetic glycan analogs of proscillaridin A 原黄芩苷A糖聚糖类似物的合成、抗癌特性和生物学分析
4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-10-27 DOI: 10.1007/s44395-025-00025-1
Shreya Somani, Lekhya Menta, Feng Wang-Johanning, Gary Johanning, Seoyeon Hong, Edward Njoo
{"title":"Synthesis, anticancer properties, and biological profiling of synthetic glycan analogs of proscillaridin A","authors":"Shreya Somani, Lekhya Menta, Feng Wang-Johanning, Gary Johanning, Seoyeon Hong, Edward Njoo","doi":"10.1007/s44395-025-00025-1","DOIUrl":"https://doi.org/10.1007/s44395-025-00025-1","url":null,"abstract":"Several cardiac glycosides, including digoxin, digitoxin, and proscillaridin A, have been originally identified as cardiomyocyte modulators and are currently being investigated for their anticancer properties. These cardiac glycosides are generally classified into cardenolides and bufadienolides, which bear butenolide and pyrone D-ring functionality, respectively, and have exhibited remarkable in vitro toxicity in various cancerous cell lines. As simple modifications on steroidal small molecules have demonstrated success in augmenting bioavailability or enhancing downstream biological activities, we sought to prepare synthetic analogs of proscillaridin A, a bufadienolide isolated from the genus Scilla. We synthesized five novel analogs of proscillaridin A bearing acetate esters, dimethyl ketals, or silyl ethers to investigate how strategies of ketalization, acetylation, and/or silylation of the A-ring allylic glycoside might alter its anticancer properties. The antiproliferative activity of these compounds was evaluated alongside proscillaridin A and two model cardiac glycosides—digoxin and digitoxin—across several colorectal, ovarian, and liver cancer cell lines. Through a diverse panel of cell viability and cytotoxicity experiments, reporter assays, and cell cycle and protein marker analysis by flow cytometry, we find that ketalization of the glycan of proscillaridin A provides similar, and in some cases enhanced, in vitro potency. This study establishes the foundation for current and further in vitro and in vivo evaluation of glycan analogs of proscillaridin A.","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147898990","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
A survey of regulatory recommendations for waivers of in vivo bioequivalence studies of generic products for certain dosage forms by participating regulators and organisations of the International Pharmaceutical Regulators Programme. Part 2. 参与国际药品监管机构计划的监管机构和组织对豁免某些剂型仿制药体内生物等效性研究的监管建议的调查。第2部分。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-10-20 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14721
Alfredo García-Arieta, Andrew Tam, Florencia Tiseyra, Eduardo Agostinho Freitas Fernandes, Kelen Carine Costa Soares, Raphael Sanches Pereira, Henrike Potthast, Katalina Mettke, Ya-Wen Chang, Li-Feng Hsu, Yu-Lin Su, Zulema Rodríguez Martínez, Milly Divinsky, Kevin Blake, April C Braddy, Clare Rodrigues, Tiong Toh, Erwin Guzman Aurela, Liliana Carolina Arévalo González, Ben Jones, So Hee Kim, Choongyul Ahn, Eunju Yun, Ryosuke Kuribayashi, Kohei Shimojo, Miho Kasuga, Junya Makino, Joel Raffel, Joy van Oudtshoorn, Philda Tabane, Bader Alzenaidy, Mohammed AlHarbi, Adel Alharf, Albatool BinAjlan, Chantal Walther, Matthias S Roost, Christopher Crane, Kitty Lee
{"title":"A survey of regulatory recommendations for waivers of <i>in vivo</i> bioequivalence studies of generic products for certain dosage forms by participating regulators and organisations of the International Pharmaceutical Regulators Programme. Part 2.","authors":"Alfredo García-Arieta, Andrew Tam, Florencia Tiseyra, Eduardo Agostinho Freitas Fernandes, Kelen Carine Costa Soares, Raphael Sanches Pereira, Henrike Potthast, Katalina Mettke, Ya-Wen Chang, Li-Feng Hsu, Yu-Lin Su, Zulema Rodríguez Martínez, Milly Divinsky, Kevin Blake, April C Braddy, Clare Rodrigues, Tiong Toh, Erwin Guzman Aurela, Liliana Carolina Arévalo González, Ben Jones, So Hee Kim, Choongyul Ahn, Eunju Yun, Ryosuke Kuribayashi, Kohei Shimojo, Miho Kasuga, Junya Makino, Joel Raffel, Joy van Oudtshoorn, Philda Tabane, Bader Alzenaidy, Mohammed AlHarbi, Adel Alharf, Albatool BinAjlan, Chantal Walther, Matthias S Roost, Christopher Crane, Kitty Lee","doi":"10.3389/jpps.2025.14721","DOIUrl":"10.3389/jpps.2025.14721","url":null,"abstract":"<p><p>A biowaiver generally refers to the request to waive an <i>in vivo</i> bioequivalence study. A biowaiver may be granted not only based on the Biopharmaceutics Classifications System (BCS) but also for many immediate-release dosage forms based on pre-defined criteria. The current paper summarises the results from a survey of the biowaiver requirements for cutaneous/topical products (topical solutions, gels, suspensions, ointments, and creams), ear/otic and ophthalmic solutions and suspensions, enemas in solution and suspension, and vaginal solid dosage forms and suppositories defined by the participants of the Bioequivalence Working Group for Generics (BEWGG) of the International Pharmaceutical Regulators Programme (IPRP). A review of the results from the survey indicates that there is a trend towards convergence when the dosage forms are less complex; however, the most common approach used by each of the participants was a case-by-case approach given that most participants do not have well-defined guidelines to support all possible scenarios. Notwithstanding the differences, disseminating information is the first step towards regulatory convergence regarding biowaivers for certain dosage forms and will be useful for pharmaceutical companies currently developing generic medicinal products for countries represented by IPRP participants.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14721"},"PeriodicalIF":5.5,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12580124/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145446520","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
The need to redefine diabetic cardiomyopathy as a unique clinical entity that requires pharmacotherapy. 需要重新定义糖尿病心肌病作为一个独特的临床实体,需要药物治疗。
IF 4.3 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-09-23 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.15503
Alexa N King, John R Ussher
{"title":"The need to redefine diabetic cardiomyopathy as a unique clinical entity that requires pharmacotherapy.","authors":"Alexa N King, John R Ussher","doi":"10.3389/jpps.2025.15503","DOIUrl":"10.3389/jpps.2025.15503","url":null,"abstract":"","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"15503"},"PeriodicalIF":4.3,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12500475/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145253425","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
Personalizing immunosuppressive therapy: predictors of drug switches in Malaysian kidney transplant recipients. 个体化免疫抑制治疗:马来西亚肾移植受者药物转换的预测因素。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-09-08 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14632
Chiau Ling Choong, Farida Islahudin, Mohd Makmor-Bakry, Nor Asyikin Mohd Tahir, Hin-Seng Wong, Rosnawati Yahya
{"title":"Personalizing immunosuppressive therapy: predictors of drug switches in Malaysian kidney transplant recipients.","authors":"Chiau Ling Choong, Farida Islahudin, Mohd Makmor-Bakry, Nor Asyikin Mohd Tahir, Hin-Seng Wong, Rosnawati Yahya","doi":"10.3389/jpps.2025.14632","DOIUrl":"10.3389/jpps.2025.14632","url":null,"abstract":"<p><strong>Objective: </strong>Tacrolimus-mycophenolic acid (MPA)-prednisolone immunosuppression remains the first-line management of kidney transplantation. Despite this, a switch to low-dose tacrolimus in combination with an mTOR inhibitor may be inevitable in some patients due to various factors. This study aims to identify the reasons and factors influencing the switch of tacrolimus-MPA to other combination immunosuppressive agents among kidney transplant recipients (KTRs).</p><p><strong>Methods: </strong>This retrospective observational cohort study included adult KTRs between year 2011-2019 at the two main kidney transplant centers in Malaysia. Demographic data, clinical, laboratory and medication information were collected. Multiple logistic regression was used to determine factors associated with the initial switch of tacrolimus-MPA immunosuppressive therapy.</p><p><strong>Results: </strong>From the 257 KTRs studied, 81 KTRs had their immunosuppressive agents switched from tacrolimus-MPA-prednisolone immunosuppressive regimen, with majority (96.3%, n = 78) switching to everolimus, an mTOR inhibitor in combination with low-dose tacrolimus. The average time switch was 125.8 ± 100.9 days. The main reasons for the initial switch include unresolved transaminitis (n = 15, 18.5%), cytomegalovirus (CMV) infection (n = 13, 16.0%) and BK virus (BKV) infection (n = 10, 12.3%). In the multiple logistic analysis, Malay ethnicity (P < 0.001), KTRs without post-transplant hypertension (P = 0.004) and KTRs with BKV infection (P < 0.001) were predictors for the initial switch of tacrolimus-MPA-prednisolone immunosuppressive therapy.</p><p><strong>Conclusion: </strong>Early identification of factors associated with the switch may prepare healthcare professionals for KTRs risk stratification, allowing ample time for appropriate optimization of tacrolimus-MPA-prednisolone immunosuppressive therapy based on individual patient's needs. This can possibly be a cost-effective alternative to switching to mTOR inhibitors for improved transplant outcomes.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14632"},"PeriodicalIF":5.5,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12450749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145132521","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
Corrigendum: Metabolic flux in macrophages in obesity and type-2 diabetes. 更正:肥胖和2型糖尿病中巨噬细胞的代谢通量。
IF 4.3 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-09-03 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.15426
Angela Wong, Qiuyu Sun, Ismail Ibrahim Latif, Qutuba G Karwi
{"title":"Corrigendum: Metabolic flux in macrophages in obesity and type-2 diabetes.","authors":"Angela Wong, Qiuyu Sun, Ismail Ibrahim Latif, Qutuba G Karwi","doi":"10.3389/jpps.2025.15426","DOIUrl":"10.3389/jpps.2025.15426","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.3389/jpps.2024.13210.].</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"15426"},"PeriodicalIF":4.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12440828/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145088044","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
A systematic review of real-world evidence on the clinical relevance, characterization, and utility of CYP2D6 biomarker testing. 对CYP2D6生物标志物检测的临床相关性、表征和效用的真实世界证据进行系统回顾。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-08-29 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14708
Patrick Rodriguez, Emma Kikerkov, Nora Emmott, Christine Y Lu, Rachele M Hendricks-Sturrup
{"title":"A systematic review of real-world evidence on the clinical relevance, characterization, and utility of <i>CYP2D6</i> biomarker testing.","authors":"Patrick Rodriguez, Emma Kikerkov, Nora Emmott, Christine Y Lu, Rachele M Hendricks-Sturrup","doi":"10.3389/jpps.2025.14708","DOIUrl":"10.3389/jpps.2025.14708","url":null,"abstract":"<p><p>Pharmacogenomic (PGx) research investigates how an individual's genetic make-up impacts their drug metabolism. PGx testing can therefore inform therapeutic decision-making, especially as compelling evidence develops over time to substantiate its clinical and personal utility across a range of therapeutic areas. PGx biomarker <i>CYP2D6</i>, in particular, is widely implicated in drug metabolism and across several therapeutic areas. Real-world evidence (RWE) derived intentionally using electronic health record (EHR) and insurance claims data presents an opportunity to explore clinical-behavioral outcomes and implementation barriers and facilitators for PGx testing in real-world clinical settings. In this systematic review, we explored these areas with a focus on PGx biomarker <i>CYP2D6</i>, investigating drug-gene pairs with strong evidence (Level A, Final classification by the Clinical Pharmacogenetics Implementation Consortium [CPIC]). Across 25 studies that met our study inclusion criteria, nine (9) drug-gene pairs that met the CPIC Level A, Final, strong evidence category for <i>CYP2D6</i> were described. Overarching qualitative themes across studies were 1) variation in CYP2D6 biomarker testing and interpretation, and 2) PGx test implementation and data considerations. CYP2D6-drug pairs were reported across four therapeutic areas (analgesia [n = 21], psychiatry [n = 17], oncology [n = 7], gastroenterology [n = 6]) with the two most researched drugs being codeine (n = 21) and tramadol (n = 18). Six (6) of 25 articles reported PGx clinical outcomes, considered to be a \"measurable change in symptoms, overall health, ability to function, quality of life, or survival outcomes\" in relation to PGx testing. Special EHR and claims data considerations for future work include but are not limited to addressing inconsistent phenotype categorizations (i.e., natural genotype versus phenoconversion); lack of reliable racial, ethnic, and genetic ancestry data within EHR and claims data sources; and data inoperability issues between PGx test results and EHRs.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14708"},"PeriodicalIF":5.5,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12425829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145066321","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
Adverse drug events associated with tiotropium: a real-world pharmacovigilance study of FDA adverse event reporting system database. 与噻托溴铵相关的药物不良事件:FDA不良事件报告系统数据库的现实世界药物警戒研究。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-08-29 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14917
Yang Rui, Tianyuan Xin, Yu Chen, Beiyi Xiang, Changwen Chen, Lin Zhang, Zhe Chen
{"title":"Adverse drug events associated with tiotropium: a real-world pharmacovigilance study of FDA adverse event reporting system database.","authors":"Yang Rui, Tianyuan Xin, Yu Chen, Beiyi Xiang, Changwen Chen, Lin Zhang, Zhe Chen","doi":"10.3389/jpps.2025.14917","DOIUrl":"10.3389/jpps.2025.14917","url":null,"abstract":"<p><strong>Introduction: </strong>Tiotropium, a long-acting muscarinic antagonist, is commonly employed for the maintenance treatment of chronic obstructive pulmonary disease (COPD) and asthma. While its efficacy has been validated through numerous randomized controlled trials, safety concerns in real-world post-marketing settings necessitate further evaluation.</p><p><strong>Aim: </strong>This study aimed to analyze the adverse events (AEs) associated with tiotropium reported in the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS) database to identify potential safety signals.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on adverse reaction reports related to tiotropium in the FAERS database from the first quarter of 2004 to the fourth quarter of 2024. The AE names in the FAERS database were systematically classified using the Preferred Terms (PTs) and System Organ Classes (SOCs) provided by the latest version of the Medical Dictionary for Regulatory Activities (MedDRA 27.1). After deduplication, a combination of methods, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS), was employed for disproportionality analysis.</p><p><strong>Results: </strong>A total of 129,763 AE reports related to tiotropium were included in the analysis, affecting 65,045 patients. These reports encompassed 27 different SOC categories, identifying 264 AEs associated with tiotropium. After excluding certain AEs deemed clinically insignificant, the most common AEs reported were dyspnea (n = 8,600), cough (n = 2,440), and pneumonia (n = 2080). The AEs exhibiting the highest signal strength included aggravated dyspnea (ROR: 162.04), hoarseness (ROR: 43.42), and aggravated chronic obstructive airway disease (ROR: 43.17). Additionally, we identified potential risks not mentioned in the instructions (United States Prescribing Information and the Canadian Product Monograph), such as epiglottic cancer, halo vision, and malignant lung tumors.</p><p><strong>Conclusion: </strong>This study offers a more comprehensive understanding of tiotropium by uncovering previously unreported adverse reactions. Physicians should take these newly identified adverse reactions into account when prescribing this medication.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14917"},"PeriodicalIF":5.5,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12425831/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145066270","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
Optimized Ginkgo biloba extract EGb 761®: boosted therapeutic benefits with minimized CYP enzyme interference. 优化银杏提取物EGb 761®:提高治疗效益,最大限度地减少CYP酶的干扰。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-08-26 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14614
Sunbeom Kwon, Suji Jeong, Seulah Lee
{"title":"Optimized <i>Ginkgo biloba</i> extract EGb 761<sup>®</sup>: boosted therapeutic benefits with minimized CYP enzyme interference.","authors":"Sunbeom Kwon, Suji Jeong, Seulah Lee","doi":"10.3389/jpps.2025.14614","DOIUrl":"10.3389/jpps.2025.14614","url":null,"abstract":"<p><strong>Object: </strong>The development of cognitive-enhancing drugs from <i>Ginkgo biloba</i> extract is actively pursued worldwide. This study compares the chemical compositions of different <i>G. biloba</i> extracts and their formulated drugs, highlighting the distinguishing characteristics and potential benefits of optimized <i>G. biloba</i> extract, EGb 761<sup>®</sup>.</p><p><strong>Methods: </strong>We analyzed three <i>G. biloba</i> extracts and fifteen formulated drugs using HPLC, principal component analysis, and LC-MS/MS to identify key compositional differences. Molecular docking analysis was conducted to evaluate the binding affinity of the key component with a target protein involved in cognitive enhancement. CYP inhibition assays were performed on selected extracts and their derived products to examine drug-drug interactions.</p><p><strong>Results: </strong>EGb 761<sup>®</sup> and its formulated drugs displayed a unique composition, characterized by a significantly higher level of protocatechuic acid (PCA). PCA demonstrated strong interactions with the M<sub>1</sub> receptor, acetylcholinesterase, glycogen synthase kinase-3, which are the key targets for cognitive enhancement. CYP inhibition assays indicated that EGb 761<sup>®</sup> and the drugs derived from EGb 761<sup>®</sup> had lower inhibitory activity compared to other samples.</p><p><strong>Conclusion: </strong>The high PCA content in EGb 761<sup>®</sup> may contribute to cognitive benefits. With low CYP inhibition, it suggests minimal interference with drug metabolism, highlighting its potential as a safer cognitive enhancer. Ultimately, this study indicates that the composition of EGb 761<sup>®</sup> can be effectively leveraged for its pharmacological benefits.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14614"},"PeriodicalIF":5.5,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12417213/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145042107","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
A review to determine regulatorily and reimbursement successes of studies conducted using data from Canadian patient support programs based on the real-world evidence guidelines published by Canadian drug agency and health Canada. 根据加拿大药品管理局和加拿大卫生部公布的真实世界证据指南,审查使用加拿大患者支持方案数据进行的研究在监管和报销方面的成功情况。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-08-08 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14587
Catherine Y Lau
{"title":"A review to determine regulatorily and reimbursement successes of studies conducted using data from Canadian patient support programs based on the real-world evidence guidelines published by Canadian drug agency and health Canada.","authors":"Catherine Y Lau","doi":"10.3389/jpps.2025.14587","DOIUrl":"10.3389/jpps.2025.14587","url":null,"abstract":"<p><strong>Introduction: </strong>Patient Support Programs (PSPs) are growing globally to support early reimbursement, disease and medication dosing management. In Canada, the lack of public health support has promoted the rapid expansion of company-supported disease-specific or drug-product-specific PSPs. Data collected from these programs generate unique Canadian data serving as a valuable source of real-world data (RWD), generally adopted in EU and US as a source of evidence generation. This review evaluates the suitability of PSP data for regulatory or reimbursement submissions, based on recently published Real World Evidence guidelines by the Canadian Drug Agency (CDA-AMC).</p><p><strong>Methods: </strong>Peer-reviewed publications evaluating patients with chronic diseases enrolled in a PSP from 1 January 2020, to 31 March 2025, were selected for review. The checklist in the CDA-AMC RWE Guideline was used to measure the quality and suitability of the PSP data.</p><p><strong>Results: </strong>Nine studies were reviewed against the checklist. Based on the criteria required to inform decision-making, most studies failed to meet key criteria for regulatory submissions. One recently published study, \"Therapeutic Drug Monitoring of Infliximab\" met most regulatory and reimbursement submission requirements.</p><p><strong>Conclusion: </strong>Data quality validation, data source transparency, validated methodology to manage study bias, measured or unmeasured confounders, and robust outcome analysis, including sensitivity and quantitative bias analysis, are essential to ensure PSP data analysis results in successful decision-making.</p>","PeriodicalId":50090,"journal":{"name":"Journal of Pharmacy and Pharmaceutical Sciences","volume":"28 ","pages":"14587"},"PeriodicalIF":5.5,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12371237/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144977114","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
Utilizing transformative learning theory to enhance professional identity formation. 运用变革学习理论促进职业认同的形成。
IF 5.5 4区 医学
Journal of Pharmacy and Pharmaceutical Sciences Pub Date : 2025-06-05 eCollection Date: 2025-01-01 DOI: 10.3389/jpps.2025.14605
Christine Pan, Jennie B Jarrett, Kathryn Sawyer
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