Pharmaceutical Medicine最新文献

{"title":"Potential of Artificial Intelligence to Accelerate Drug Development for Rare Diseases.","authors":"Giulio Napolitano, Canan Has, Anne Schwerk, Jui-Hung Yuan, Carsten Ullrich","doi":"10.1007/s40290-023-00504-9","DOIUrl":"10.1007/s40290-023-00504-9","url":null,"abstract":"<p><p>The growth in breadth and depth of artificial intelligence (AI) applications has been fast, running hand in hand with the increasing amount of digital data available. Here, we comment on the application of AI in the field of drug development, with a strong focus on the specific achievements and challenges posed by rare diseases. Data paucity and high costs make drug development for rare diseases especially hard. AI can enable otherwise inaccessible approaches based on the large-scale integration of heterogeneous datasets and knowledge bases, guided by expert biological understanding. Obstacles still exist for the routine use of AI in the usually conservative pharmaceutical domain, which can easily become disillusioned. It is crucial to acknowledge that AI is a powerful, supportive tool that can assist but not replace human expertise in the various phases and aspects of drug discovery and development.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"79-86"},"PeriodicalIF":2.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139692586","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":"Physician Awareness of the Safe Use of Cyproterone Acetate in Europe: A Survey on the Effectiveness of Additional Risk Minimization Measures.","authors":"Carolyn Sweeney, Alicia Gilsenan, Brian Calingaert, Carsten Moeller, Gesa Schomakers, Alen Sok, Ruth Holzmann, Federica Pisa","doi":"10.1007/s40290-023-00510-x","DOIUrl":"10.1007/s40290-023-00510-x","url":null,"abstract":"<p><strong>Background: </strong>Cyproterone acetate (CPA) is a synthetic progesterone derivative introduced in the 1970s and prescribed as antiandrogenic therapy for inoperable prostate cancer, sexual deviations in men, and signs of androgenization in women. In 2020, the CPA summary of product characteristics (SmPC) was revised to include an updated special warning and precaution about (1) the risk of meningioma with increasing cumulative dose and (2) contraindication in patients with meningioma or history of meningioma. A Direct Healthcare Professional Communication (DHPC) was distributed. The European Medicine Agency's Pharmacovigilance Risk Assessment Committee requested that marketing authorization holders in Europe conduct a survey to assess physicians' knowledge of the updated key safety information. The primary objective of this study was to measure physicians' awareness (i.e., did they receive and review the revised SmPC and DHPC) and level of knowledge and understanding of the key safety information pertaining to the restricted use of CPA monotherapy because of the risk of meningioma.</p><p><strong>Methods: </strong>This cross-sectional web-based survey was administered to dermatologists, endocrinologists, gynecologists, urologists, oncologists, psychiatrists, and general practitioners in France, Germany, Poland, Spain, and the Netherlands who had prescribed CPA monotherapy in the previous 12 months to assess awareness of the risk of meningioma associated with CPA monotherapy.</p><p><strong>Results: </strong>Of the 613 physicians who participated, 85% correctly indicated that CPA monotherapy should be prescribed with the lowest effective dose, 75% correctly indicated that the risk of meningioma increases with increasing cumulative CPA monotherapy doses, and 73% correctly indicated that treatment with CPA-containing products must be stopped permanently if a patient is diagnosed with meningioma. Overall, 40% of physicians reported having received the DHPC, and 42% reported having received the revised SmPC.</p><p><strong>Conclusions: </strong>Despite low recall of receipt of the updated SmPC and DHPC, most physicians surveyed are aware of the meningioma risk and actions to mitigate the risk.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"145-156"},"PeriodicalIF":2.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10948458/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139651372","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":"Female Problems: Women's Health Mustn't be Ghettoized in the Uterus.","authors":"Peter J Pitts","doi":"10.1007/s40290-023-00514-7","DOIUrl":"10.1007/s40290-023-00514-7","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"75-77"},"PeriodicalIF":2.5,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139542818","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":"Rare Immune-Related Adverse Events (irAEs): Approach to Diagnosis and Management.","authors":"Anadil Javaid, Catherine Bennett, Aparna Rao, Lavinia Spain","doi":"10.1007/s40290-023-00508-5","DOIUrl":"10.1007/s40290-023-00508-5","url":null,"abstract":"<p><p>Immune checkpoint inhibitors (ICIs) have revolutionised the treatment landscape across many solid organ malignancies and form part of routine clinical practice in many tumours. As indications for monotherapy, doublet therapy and combination approaches with chemotherapy and targeted agents expand, clinicians must be aware of the wide range of possible immune-related adverse events (irAEs). Common toxicities, including rash, colitis, hepatitis and pneumonitis are well described in the literature, and have established diagnostic and management algorithms. Rarer toxicities, often with an incidence of less than 1%, are less defined. These syndromes can be poorly recognised, may take on a fulminant course and do not have established or evidence-based diagnostic and management strategies. As such, patients may experience increased morbidity, mortality and poorer outcomes, related both to these irAEs as well as how the treatment of these may affect the management of their underlying malignancy. In this review, we aim to explore the incidence, potential biomarkers, pathogenesis, diagnostic work-up and clinical sequelae of a selection of uncommon irAEs, with a focus on myocarditis, neurological and haematologic syndromes. Further prospective research is required to accurately define the incidence and pathogenesis of these conditions, with the aim of increasing clinician awareness of rare irAEs and to assist with a more personalised and mechanism-based approach to these syndromes.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"25-38"},"PeriodicalIF":3.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10824871/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139403934","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":"Medicinal Products and Environmental Pollution.","authors":"Noel Snell","doi":"10.1007/s40290-023-00502-x","DOIUrl":"10.1007/s40290-023-00502-x","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"5-7"},"PeriodicalIF":2.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71413407","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 Assessing Drug Exposure and Medication Adherence in Evaluating Investigational Medications: Ensuring Validity and Reliability of Clinical Trial Results.","authors":"Bernard Vrijens, Antoine Pironet, Eric Tousset","doi":"10.1007/s40290-023-00503-w","DOIUrl":"10.1007/s40290-023-00503-w","url":null,"abstract":"<p><p>The objective of this current opinion paper is to draw global attention to medication adherence, emphasizing its crucial role in drug trials. Frequently, trialists lean on traditional approaches to assess medication adherence, which, while comfortable, may only reveal what trialists desire rather than offering the essential insights needed for informed decision making in drug development. Understanding drug exposure and medication adherence is paramount when evaluating the effectiveness and safety of investigational medications. Without a comprehensive understanding of how patients adhere to their prescribed treatment regimens, the integrity and dependability of clinical trial results can be compromised. This paper emphasizes the need for measures that accurately and reliably assess medication intake behaviors, enabling the differentiation between minor dosing errors and significant deviations that may impact the drug's efficacy and safety. Accurate knowledge of drug exposure empowers researchers to make informed decisions, identify potential confounding factors, and appropriately interpret study outcomes, ultimately ensuring the validity and reliability of the research findings. By prioritizing drug exposure assessment and medication adherence measurement, clinical trials can enhance their scientific rigor, contribute to more accurate evaluations of investigational medications, and ultimately speed up the development process.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"9-18"},"PeriodicalIF":3.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10824809/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138885618","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":"Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease.","authors":"Kerrie-Anne Ho, Anna Pierce, Meredin Stoltenberg, Thais Tarancon, Carol Mansfield","doi":"10.1007/s40290-023-00509-4","DOIUrl":"10.1007/s40290-023-00509-4","url":null,"abstract":"<p><strong>Introduction: </strong>Qualitative and quantitative methods provide different and complementary insights into patients' preferences for treatment.</p><p><strong>Objective: </strong>The aim of this study was to use a novel, mixed-methods approach employing qualitative and quantitative approaches to generate preliminary insights into patient preferences for the treatment of a rare disease-generalized myasthenia gravis (gMG).</p><p><strong>Methods: </strong>We conducted a mixed-methods study to collect exploratory qualitative and quantitative patient preference information and generate informative results within a condensed timeline (about 4 months). Recruitment was facilitated by an international health research firm. Study participants first reviewed a brief document describing six treatment attributes (to facilitate more efficient review of the material during the focus groups) and were then provided a link to complete an online quantitative survey with a single risk threshold task. They then participated in online focus groups, during which they discussed qualitative questions about their experience with gMG treatment and completed up to three quantitative threshold tasks, the first of which repeated the threshold task from the online survey.</p><p><strong>Results: </strong>The study elicited both quantitative data on 18 participants' risk tolerance and qualitative data on their treatment experience, additional treatment attributes of importance, the reasoning behind their preferences, and the trade-offs they were willing to make. Most participants (n = 15) chose the same hypothetical treatment in the first threshold task in the online survey and the focus groups. Focus group discussions provided insights into participants' choices in the threshold tasks, confirmed that all the attributes were relevant, and helped clarify what was important about the attributes.</p><p><strong>Conclusions: </strong>Patient preference information can be collected using a variety of approaches, both qualitative and quantitative, tailored to fit the research needs of a study. The novel mixed-methods approach employed in this study efficiently captured patient preference data that were informative for exploratory research, internal decision making, and future research.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"55-62"},"PeriodicalIF":2.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10824859/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138830892","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":"A Review of the Current FDA-Approved Antibody-Drug Conjugates: Landmark Clinical Trials and Indications.","authors":"Meghana Kesireddy, Srikanth Reddy Kothapalli, Sai Giridhar Gundepalli, Samia Asif","doi":"10.1007/s40290-023-00505-8","DOIUrl":"10.1007/s40290-023-00505-8","url":null,"abstract":"<p><p>Despite considerable treatment progress, cancer remains among the leading causes of death worldwide. Antibody-drug conjugates (ADCs), a rapidly growing class of systemic therapy, show promise by combining the properties of conventional chemotherapy and targeted therapy. Antibody-drug conjugates have been shown to be more efficacious than traditional chemotherapy. To date, there are 13 ADCs approved by the United States Food and Drug Administration (FDA) for treating various hematological and solid organ cancers. There are several new promising ADCs that are being developed and are in clinical trials. This review provides an overview of the current FDA-approved ADCs, the landmark clinical trials that led to their approval, the common toxicities seen in the landmark trials, the challenges associated with ADCs, and the potential future directions.</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"39-54"},"PeriodicalIF":2.5,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138452079","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":"Evaluation of Physician Knowledge of Safety and Safe Use Information for Intravitreal Aflibercept Injection in Europe: A Second Survey of Physicians Following Dissemination of Updated Risk-Minimization Materials.","authors":"Laurie J Zografos, Elizabeth Andrews, Dan L Wolin, Brian Calingaert, Eric K Davenport, Alexander Michel, Margarete Latocha, Ursula Maria Schmidt-Ott, Nejra Lovic, Lynne R Brunck, Kristian T Johnson, Kiliana Suzart-Woischnik","doi":"10.1007/s40290-023-00506-7","DOIUrl":"10.1007/s40290-023-00506-7","url":null,"abstract":"<p><strong>Background: </strong>Materials have been distributed in the European Union to inform physicians on the safe use of intravitreal aflibercept (IVT-AFL) as part of the risk-minimization plan for IVT-AFL.</p><p><strong>Objective: </strong>We aimed to measure physician knowledge and understanding of key safety information for IVT-AFL.</p><p><strong>Methods: </strong>The current study was a follow-up cross-sectional survey ('wave 2') to an earlier survey ('wave 1') examining the effectiveness of the IVT-AFL educational materials by assessing physician knowledge of the key safety information. Based on wave 1 results, the educational materials were revised to focus more on items of key concern (e.g., use in women of childbearing potential, procedural information); physicians in France, Germany, Italy, Spain, and the UK completed a questionnaire to evaluate their knowledge of key safety information in the revised educational materials.</p><p><strong>Results: </strong>Among 454 physician respondents (of 4715 invited; response rate 9.6%), most reported having received the IVT-AFL Summary of Product Characteristics (SmPC; 89%) and Prescriber Guide (82%). More than half reported receiving the Injection Procedure Video (54%) and Patient Booklet (65%). The highest percentage of correct answers was observed for questions concerning procedural steps, the most important risks, and safe use as emphasized by the educational materials and the SmPC.</p><p><strong>Conclusion: </strong>Physician knowledge and understanding of safe use of IVT-AFL, including for questions that prompted revisions to the educational materials, suggests the need to reconsider methods for developing educational materials to follow best practices (e.g., focusing on only key messages and pretesting with end users).</p>","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":" ","pages":"63-73"},"PeriodicalIF":3.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10824811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138482786","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":"“Regulatory Sandboxes” Could Solve the Regulatory Problems Encountered in Europe and Arising from Innovation in Biological Medicinal Products","authors":"Mathieu Guerriaud","doi":"10.1007/s40290-023-00507-6","DOIUrl":"https://doi.org/10.1007/s40290-023-00507-6","url":null,"abstract":"","PeriodicalId":19778,"journal":{"name":"Pharmaceutical Medicine","volume":"8 6","pages":""},"PeriodicalIF":2.5,"publicationDate":"2023-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138585426","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}