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Iatrogenic adrenal insufficiency related to corticosteroid eye drops: a case report. 与皮质类固醇滴眼液相关的医源性肾上腺功能不全1例报告。
JRSM Open Pub Date : 2025-08-14 eCollection Date: 2025-08-01 DOI: 10.1177/20542704251364598
Shruthi Rayen, Manjusha Rathi
{"title":"Iatrogenic adrenal insufficiency related to corticosteroid eye drops: a case report.","authors":"Shruthi Rayen, Manjusha Rathi","doi":"10.1177/20542704251364598","DOIUrl":"10.1177/20542704251364598","url":null,"abstract":"<p><p>Intra-ocular corticosteroids have the potential to cause adrenal insufficiency when used long term. Patients and clinicians should be counselled on the risks of long-term steroid use and consider appropriate monitoring.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 8","pages":"20542704251364598"},"PeriodicalIF":0.0,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12357029/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144873860","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}
引用次数: 0
Clinically differentiating Behcet's syndrome from Crohn's disease. 白塞氏综合征与克罗恩病的临床鉴别
JRSM Open Pub Date : 2025-08-11 eCollection Date: 2025-07-01 DOI: 10.1177/20542704251362981
Caroline J Stone, Hammad Ali, Aretha On, William N Roberts
{"title":"Clinically differentiating Behcet's syndrome from Crohn's disease.","authors":"Caroline J Stone, Hammad Ali, Aretha On, William N Roberts","doi":"10.1177/20542704251362981","DOIUrl":"10.1177/20542704251362981","url":null,"abstract":"<p><p>We aim to highlight the diagnostic challenges in differentiating Behcet's syndrome from Crohn's disease, in patients presenting with overlapping clinical features, highlighting the importance of comprehensive clinical evaluation for appropriate management and prognostication.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 7","pages":"20542704251362981"},"PeriodicalIF":0.0,"publicationDate":"2025-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12340185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144835511","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}
引用次数: 0
Corrigendum to "The association of ethnicity and migration status with agenda for change pay band in National Health Service healthcare workers: Results from the United Kingdom Research study into Ethnicity and Coronavirus Disease 2019 (COVID-19) Outcomes in Healthcare workers (UK-REACH)". “民族和移民身份与国家卫生服务保健工作者薪酬水平变化议程的关联:英国卫生保健工作者民族和2019冠状病毒病(COVID-19)结局研究结果(UK-REACH)”的勘误表。
JRSM Open Pub Date : 2025-07-23 eCollection Date: 2025-07-01 DOI: 10.1177/20542704251363054
{"title":"Corrigendum to \"The association of ethnicity and migration status with agenda for change pay band in National Health Service healthcare workers: Results from the United Kingdom Research study into Ethnicity and Coronavirus Disease 2019 (COVID-19) Outcomes in Healthcare workers (UK-REACH)\".","authors":"","doi":"10.1177/20542704251363054","DOIUrl":"10.1177/20542704251363054","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1177/20542704251330157.].</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 7","pages":"20542704251363054"},"PeriodicalIF":0.0,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12290313/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732015","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}
引用次数: 0
Cardiovascular disease reported as modes of death in the Office for National Statistics mortality data: a retrospective observational study. 在国家统计局死亡率数据中作为死亡方式报告的心血管疾病:一项回顾性观察性研究。
JRSM Open Pub Date : 2025-06-02 eCollection Date: 2025-06-01 DOI: 10.1177/20542704251330372
Joseph D Westaby, Anne-Marie Gabrawi, Mary N Sheppard
{"title":"Cardiovascular disease reported as modes of death in the Office for National Statistics mortality data: a retrospective observational study.","authors":"Joseph D Westaby, Anne-Marie Gabrawi, Mary N Sheppard","doi":"10.1177/20542704251330372","DOIUrl":"10.1177/20542704251330372","url":null,"abstract":"<p><strong>Objective: </strong>A cause of death is a specific disease or injury which directly led to the death whereas a mode of death which is a mechanism such as respiratory failure, cardiac arrest or cardiac failure but does not provide the cause of death. We sought to establish the scale of use of cardiovascular mode and other non-specific codes as causes of death.</p><p><strong>Design: </strong>We extracted the mortality statistics recorded between 2013 and 2021 and then selected cardiovascular codes.</p><p><strong>Setting: </strong>The Office for National Statistics mortality data.</p><p><strong>Participants: </strong>Deceased individuals from England and Wales.</p><p><strong>Main outcome measures: </strong>Cause of death.</p><p><strong>Results: </strong>Of 4,852,897 deaths, 836,741 (17.2%) had cardiovascular codes. Of these, 103,160 (12.3%) were labelled as modes and 35,784 (4.3%) were non-specific causes. Modes increased from 5862 in 2013 to 14,641 in 2021. Modes included 56,291 (6.7%) as arrhythmia and 46,787 (5.6%) as heart failure. Non-specific included 12,192 (1.46%) myocardial degeneration and 6573 (0.79%) cardiomegaly. Non-specific cardiomyopathies included other cardiomyopathies (207) and cardiomyopathy, unspecified (2984).</p><p><strong>Conclusions: </strong>Modes of death are being used in a notable proportion of medical certificates and this is increasing which is worrying and does not provide the underlying cause of the death. It is important that a cause of death is given so that underlying heritable cardiac conditions, such as channelopathy or cardiomyopathy, are identified. This enables referral of blood relatives for cardiological screening and intervention. ICD-11 will help address some of the non-specific causes of death with the inclusion of codes for sudden arrhythmic death syndrome and arrhythmogenic cardiomyopathy. Autopsy is essential to establish a cause of death where only a mode of death can be given without clarification of a causative disease.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 6","pages":"20542704251330372"},"PeriodicalIF":0.0,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12130641/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144225835","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}
引用次数: 0
The association of ethnicity and migration status with agenda for change pay band in National Health Service healthcare workers: Results from the United Kingdom Research study into Ethnicity and Coronavirus Disease 2019 (COVID-19) Outcomes in Healthcare workers (UK-REACH). 民族和移民身份与国家卫生服务保健工作者薪酬水平变化议程的关系:来自英国卫生保健工作者民族与2019冠状病毒病(COVID-19)结局研究(UK-REACH)的结果。
JRSM Open Pub Date : 2025-05-19 eCollection Date: 2025-05-01 DOI: 10.1177/20542704251330157
Ji Soo Choi, Christopher A Martin, Lucy Teece, Mayuri Gogoi, Irtiza Qureshi, Daniel Pan, Joshua Nazareth, Rebecca F Baggaley, Luke Bryant, Padmasayee Papineni, Carol Woodhams, Katherine Woolf, Manish Pareek
{"title":"The association of ethnicity and migration status with agenda for change pay band in National Health Service healthcare workers: Results from the United Kingdom Research study into Ethnicity and Coronavirus Disease 2019 (COVID-19) Outcomes in Healthcare workers (UK-REACH).","authors":"Ji Soo Choi, Christopher A Martin, Lucy Teece, Mayuri Gogoi, Irtiza Qureshi, Daniel Pan, Joshua Nazareth, Rebecca F Baggaley, Luke Bryant, Padmasayee Papineni, Carol Woodhams, Katherine Woolf, Manish Pareek","doi":"10.1177/20542704251330157","DOIUrl":"10.1177/20542704251330157","url":null,"abstract":"<p><strong>Objectives: </strong>Ethnic minority and migrant healthcare workers (HCWs) constitute 24% of the UK's National Health Service. Migration status, often overlooked in Human Resources records, is associated with their placement within the Agenda for Change (AfC) pay bands. Therefore, we analysed the association between ethnicity, migration status, and AfC pay bands using data from the UK-REACH cohort study.</p><p><strong>Design: </strong>Cross-sectional study.</p><p><strong>Setting: </strong>UK-REACH cohort using baseline data collected via online questionnaires across various healthcare settings.</p><p><strong>Participants: </strong>Healthcare workers from a broad range of professional roles across the UK, recruited between December 2020 and February 2021.</p><p><strong>Main outcome measures: </strong>We used multivariable generalised ordered logistic regression models to examine the associations between ethnicity, migration status and AfC pay bands, adjusting for sex, education level, job role, and years qualified.</p><p><strong>Results: </strong>We found that Overseas-born ethnic minority HCWs were less likely to be in higher AfC pay bands compared to their White UK-born counterparts. Specifically, Asian Overseas-born and Black Overseas-born HCWs reported significantly lower odds of being in higher pay bands compared to White UK-born workers.</p><p><strong>Conclusions: </strong>Overseas-born HCWs from ethnic minorities resided in lower paid roles than White UK HCWs. Our study is the first to highlight a link between migration status and the AfC pay band and to explore interactions between ethnicity and migration within this context. Our data highlights the need for policymakers to incorporate migration status into NHS-wide electronic records to address career progression and pay inequities.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 5","pages":"20542704251330157"},"PeriodicalIF":0.0,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12089724/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111160","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}
引用次数: 0
Incidental finding of lung adenocarcinoma following routine bullectomy and pleurodesis on a young patient: a case report. 一个年轻病人在常规大球切除和胸膜切除术后偶然发现肺腺癌:一个病例报告。
JRSM Open Pub Date : 2025-05-08 eCollection Date: 2025-05-01 DOI: 10.1177/20542704241304218
Michelle Lee, Al-Rehan Dhanji, Gowthanan Santhirakumaran, Periklis Perikleous, David Waller
{"title":"Incidental finding of lung adenocarcinoma following routine bullectomy and pleurodesis on a young patient: a case report.","authors":"Michelle Lee, Al-Rehan Dhanji, Gowthanan Santhirakumaran, Periklis Perikleous, David Waller","doi":"10.1177/20542704241304218","DOIUrl":"https://doi.org/10.1177/20542704241304218","url":null,"abstract":"<p><p>Spontaneous pneumothorax is a rare complication of lung cancer. In this report, we present a case of a patient with recurrent pneumothorax undergoing routine bullectomy and pleurodesis and lung adenocarcinoma is diagnosed incidentally. The prognosis for patients with untreated lung cancer has always been unfavourable with a median survival time of only 10 to 14 months, even for early-stage disease. Once the diagnosis is established, an effective treatment should be instituted without delay. Spontaneous pneumothorax is a rare manifestation of lung cancer and the relative risk for developing lung cancer should be considered with the patients with recurrent spontaneous pneumothorax.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 5","pages":"20542704241304218"},"PeriodicalIF":0.0,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12062624/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144030978","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}
引用次数: 0
The importance of multiculturalism in medical education: a global comparison of perspectives from medical and health professions students at 21 universities. 多元文化在医学教育中的重要性:21所大学医学和卫生专业学生观点的全球比较。
JRSM Open Pub Date : 2025-04-10 eCollection Date: 2025-04-01 DOI: 10.1177/20542704251322244
Anette Wu, Radhika Patel, Jason Luong, Sean McWatt, Rahul Goel, Cecilia Brassett, Jane Dutton, Mandeep Gill Sagoo, Carol Kunzel, Alexander Green, Geoffroy Noel
{"title":"The importance of multiculturalism in medical education: a global comparison of perspectives from medical and health professions students at 21 universities.","authors":"Anette Wu, Radhika Patel, Jason Luong, Sean McWatt, Rahul Goel, Cecilia Brassett, Jane Dutton, Mandeep Gill Sagoo, Carol Kunzel, Alexander Green, Geoffroy Noel","doi":"10.1177/20542704251322244","DOIUrl":"https://doi.org/10.1177/20542704251322244","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to quantitatively assess the baseline level of self-perceived cultural competency preparedness and skillfulness among medical and health professions students from 21 universities around the world utilizing a previously validated and standardized testing tool.</p><p><strong>Design: </strong>Cross-sectional study.</p><p><strong>Setting: </strong>The International Collaboration and Exchange Program (ICEP), a global exchange initiative for junior medical and health professions students spanning 21 universities across four continents.</p><p><strong>Participants: </strong>A total of 753 students from the 2021 and 2022 ICEP cohorts.</p><p><strong>Main outcome measures: </strong>Students self-evaluated their cultural competency skills on a 5-point Likert-type scale encompassing different areas of competency. Multiple linear regression was performed to identify contributors to cultural competency levels.</p><p><strong>Results: </strong>Upon rating how skillful they are at interacting with culturally diverse patients, North American students reported the highest scores with a mean of 3.22, while Australian students showed the lowest score of 2.82. When analyzing students' stages of study, those in clinical years of medical schools scored the highest at 3.29. Significant variations were observed in the cultural competency self-rating scores among students based on their respective regions (<i>p </i>< .005) and program types/stages (<i>p</i> < .05). Notably, students in their clinical years of school consistently rated themselves higher compared to their preclinical counterparts (<i>p</i> < .05). Furthermore, students from Europe displayed elevated self-ratings compared to the other regions (<i>p</i> < .005).</p><p><strong>Conclusions: </strong>Though these participants represent a highly motivated subgroup of students, potentially limiting result generalizability, the findings emphasize that regional differences exist. Given the multifaceted nature of cultural competency, the results suggest that factors such as educational stage, age, and region may influence students' perceived competency levels.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 4","pages":"20542704251322244"},"PeriodicalIF":0.0,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12032487/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017378","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}
引用次数: 0
Structured medication reviews for patients with polypharmacy in primary care: a cross-sectional study in North West London, UK. 针对基层医疗机构多药合用患者的结构化药物审查:英国伦敦西北部的横断面研究。
JRSM Open Pub Date : 2025-04-01 DOI: 10.1177/20542704251325056
Linwei Li, Geva Greenfield, Benedict W J Hayhoe, Derryn Lovett, Vesselin Novov, Azeem Majeed, Paul Aylin, Hadar Zaman, Thomas Woodcock
{"title":"Structured medication reviews for patients with polypharmacy in primary care: a cross-sectional study in North West London, UK.","authors":"Linwei Li, Geva Greenfield, Benedict W J Hayhoe, Derryn Lovett, Vesselin Novov, Azeem Majeed, Paul Aylin, Hadar Zaman, Thomas Woodcock","doi":"10.1177/20542704251325056","DOIUrl":"10.1177/20542704251325056","url":null,"abstract":"<p><strong>Objectives: </strong>To identify the number and characteristics of patients with polypharmacy receiving structured medication reviews (SMRs) and medication reviews in primary care in 2022, and to evaluate whether the provision of these services is equitable across different demographic and socio-economic groups.</p><p><strong>Design: </strong>Cross-sectional study.</p><p><strong>Setting: </strong>Primary care networks in North West London, UK.</p><p><strong>Participants: </strong>Adults registered with a general practitioner (GP) and regularly prescribed at least five medicines or more.</p><p><strong>Main outcome measures: </strong>Receipt of at least one SMR and any kind of medication review during the study period (2022).</p><p><strong>Results: </strong>Among 515,042 adults regularly prescribed with medication, 167,482 were regularly prescribed at least five medicines, defined as polypharmacy. 53.3% (89,220) of these patients received at least one kind of medication review and 17.2% (11,954) of them received SMRs. Patients who were males, black, more affluent, and frailer, were more likely to receive medication reviews, while those who were males, less affluent, and frailer, were more likely to receive SMRs.</p><p><strong>Conclusions: </strong>Although polypharmacy was common in North West London, only about half of eligible patients received medication reviews, and only 17.2% received SMRs. Different distributions of medication reviews and SMRs by demographic and socio-economic characteristics may indicate inequities in the provision of these services. Policy makers should consider effective ways to incentivise the equitable provision of SMRs.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 4","pages":"20542704251325056"},"PeriodicalIF":0.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963712/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143780233","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}
引用次数: 0
Therapeutic value of oncology products with a conditional approval from Health Canada: a cross-sectional study. 加拿大卫生部有条件批准的肿瘤产品的治疗价值:横断面研究。
JRSM Open Pub Date : 2025-03-19 eCollection Date: 2025-03-01 DOI: 10.1177/20542704251325314
Joel Lexchin
{"title":"Therapeutic value of oncology products with a conditional approval from Health Canada: a cross-sectional study.","authors":"Joel Lexchin","doi":"10.1177/20542704251325314","DOIUrl":"10.1177/20542704251325314","url":null,"abstract":"<p><strong>Objectives: </strong>Investigate the additional therapeutic value compared to existing medicines of new oncology drugs given a conditional approval (Notice of Compliance with conditions, NOC/c) by Health Canada using therapeutic ratings from four independent organisations.</p><p><strong>Design: </strong>A list of all new oncology drugs with an NOC/c from the start of the programme in 1998 to the end of 2023 was constructed. First-in-class and orphan drug status was determined for all drugs. Therapeutic ratings were obtained from the Canadian Patented Medicine Prices Review Board, the French drug bulletin Prescrire International, the French agency Haute Autorité de Santé and the German Institute for Quality and Efficiency in Health Care. If more than one organisation rated the drug, the highest rating was used.</p><p><strong>Setting: </strong>Canada.</p><p><strong>Participants: </strong>Oncology drugs with a conditional approval.</p><p><strong>Main outcome measures: </strong>Additional therapeutic gain compared to existing products.</p><p><strong>Results: </strong>Fifty-four oncology drugs were approved. Conditions were fulfilled for 29, fulfilment was still pending for 22 and 3 drugs were either discontinued by the manufacturer or placed on restricted access. Eighteen drugs had both orphan drug and first-in-class status. Therapeutic evaluations were available for 50 drugs, and the distribution of additional therapeutic value was examined for the entire group of 50 drugs, for 29 drugs that had fulfilled their conditions and for 18 drugs with both orphan drug and first-in-class status. In the three groups, 8.0%, 10.3% and 11.7%, respectively, offered major therapeutic improvement.</p><p><strong>Conclusions: </strong>Few new oncology drugs approved through the NOC/c pathway offer major therapeutic improvements over existing drugs.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 3","pages":"20542704251325314"},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11924082/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143692635","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}
引用次数: 0
The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data. 英国早期获得药物计划10年:使用公开数据的评估。
JRSM Open Pub Date : 2025-02-24 eCollection Date: 2025-02-01 DOI: 10.1177/20542704251317916
Pandora Pound, Rebecca Ram, Kathy Archibald
{"title":"The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data.","authors":"Pandora Pound, Rebecca Ram, Kathy Archibald","doi":"10.1177/20542704251317916","DOIUrl":"10.1177/20542704251317916","url":null,"abstract":"<p><strong>Objectives: </strong>To investigate the drugs and indications that have passed through the UK's Early Access to Medicines Scheme (EAMS) to date, the type of evidence the regulator considers when accepting a drug into the EAMS, and potential risks to patients.</p><p><strong>Design: </strong>Analysis of publicly available data: MHRA Public Assessment Reports; Electronic Medicines Compendium database; interactive Drug Analysis Profiles database; Eudravigilance database.</p><p><strong>Setting: </strong>United Kingdom.</p><p><strong>Participants: </strong>The 51 'scientific opinions' available on the MHRA website in June 2024.</p><p><strong>Main outcome measures: </strong>Public Assessment Reports, pharmacovigilance data.</p><p><strong>Results: </strong>After exclusions, there were 48 EAMS submissions, consisting of 48 indications and 32 drugs. 60% of indications were for cancer. Only 7% of EAMS submissions were based on double-blind, placebo-controlled randomised trials. The average sample size of studies conducted for the EAMS was 654. Most studies used surrogate (76%) and/or survival (57%) outcomes. Only 17% used subjective outcomes. For 17% of the indications, no ongoing studies were being conducted. Animal studies were conducted preclinically for all drugs and 35% also conducted in vitro studies. 47% of the drugs had elevated rates of suspected adverse reaction reports according to pharmacovigilance data.</p><p><strong>Conclusions: </strong>We recommend that the EAMS drugs with elevated reporting rates are reviewed, that future studies of EAMS drugs use patient-centred outcomes, that preclinical studies make greater use of human biology-based approaches, that post-approval trials are conducted, and that future reviews of the EAMS centre the experience of patients.</p>","PeriodicalId":17674,"journal":{"name":"JRSM Open","volume":"16 2","pages":"20542704251317916"},"PeriodicalIF":0.0,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11848878/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501936","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}
引用次数: 0
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