Irritable bowel syndrome (IBS) is a common disorder that poses a management challenge. IBS with constipation as the dominant bowel habit is a common phenotype and features abdominal pain, infrequent bowel movements or difficult defecation, bloating and distention, as major symptoms. While laxatives increase stool frequency, they have little impact on other symptoms; prior, more specifically targeted, therapies have been withdrawn because of adverse events. Prosecretory agents represent a new drug class that act locally and have minimal systemic absorption; linaclotide is the latest to obtain regulatory approval and provides significant relief of the cardinal symptoms of IBS-constipation. Linaclotide also accelerates colonic transit and animal data suggest a visceral analgesic effect. The main side effect has been diarrhea.
{"title":"Linaclotide for irritable bowel syndrome with constipation: a review","authors":"M. Arriaga, E. Quigley","doi":"10.4155/CLI.15.25","DOIUrl":"https://doi.org/10.4155/CLI.15.25","url":null,"abstract":"Irritable bowel syndrome (IBS) is a common disorder that poses a management challenge. IBS with constipation as the dominant bowel habit is a common phenotype and features abdominal pain, infrequent bowel movements or difficult defecation, bloating and distention, as major symptoms. While laxatives increase stool frequency, they have little impact on other symptoms; prior, more specifically targeted, therapies have been withdrawn because of adverse events. Prosecretory agents represent a new drug class that act locally and have minimal systemic absorption; linaclotide is the latest to obtain regulatory approval and provides significant relief of the cardinal symptoms of IBS-constipation. Linaclotide also accelerates colonic transit and animal data suggest a visceral analgesic effect. The main side effect has been diarrhea.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"2 1","pages":"633-641"},"PeriodicalIF":0.0,"publicationDate":"2015-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89004683","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}
Background: Clinical trials are designed to test efficacy and safety of new drugs. Trials testing biologics for wound care is a fast growing field. We have analyzed clinical trials testing fibroblast cell-based agents for chronic venous stasis ulcers (VLUs). These two studies had similar objectives, study design, comparable eligibility criteria and outcomes. Objective: To assess performance and compliance in two successive clinical trials testing cell-based therapeutics for venous ulcers in order to identify trends and improvement opportunities. Methods: A systemic internal audit of two prospective, randomized wound care clinical trials was conducted at Boston Medical Center. Enrollment rates, earned values, actual and planned costs were analyzed and compared. The schedule performance index (SPI) and cost performance index (CPI) were calculated and factors affecting enrollment rates were identified. Study compliance was assessed based on study protocol deviations. Safety profile was assessed based on seve...
{"title":"Implementation of strategic management tools improves wound care clinical trial outcomes","authors":"M. Malikova, L. Shifflett, A. Farber","doi":"10.4155/CLI.15.30","DOIUrl":"https://doi.org/10.4155/CLI.15.30","url":null,"abstract":"Background: Clinical trials are designed to test efficacy and safety of new drugs. Trials testing biologics for wound care is a fast growing field. We have analyzed clinical trials testing fibroblast cell-based agents for chronic venous stasis ulcers (VLUs). These two studies had similar objectives, study design, comparable eligibility criteria and outcomes. Objective: To assess performance and compliance in two successive clinical trials testing cell-based therapeutics for venous ulcers in order to identify trends and improvement opportunities. Methods: A systemic internal audit of two prospective, randomized wound care clinical trials was conducted at Boston Medical Center. Enrollment rates, earned values, actual and planned costs were analyzed and compared. The schedule performance index (SPI) and cost performance index (CPI) were calculated and factors affecting enrollment rates were identified. Study compliance was assessed based on study protocol deviations. Safety profile was assessed based on seve...","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"28 1","pages":"653-664"},"PeriodicalIF":0.0,"publicationDate":"2015-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"85679015","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}
Tom is a founder of ePatientFinder. Before ePatientFinder, he was a partner at Chandler Morgan Consulting where he assisted numerous health IT organizations with their business development and revenue models. Before Chandler Morgan, he served as president of iMedicor, a publicly traded health information exchange company. Prior to iMedicor, he was president and CEO of NuScribe, provider of the market’s first health information exchange coupled with a professional networking platform.
Tom是ePatientFinder的创始人之一。在ePatientFinder之前，他是Chandler Morgan Consulting的合伙人，在那里他协助许多医疗IT组织进行业务开发和收入模式。在Chandler Morgan之前，他曾担任iMedicor(一家公开交易的健康信息交换公司)的总裁。在加入iMedicor之前，他是NuScribe的总裁兼首席执行官，NuScribe是市场上第一个健康信息交换以及专业网络平台的提供商。
{"title":"Clinical trial recruitment in the information age","authors":"Thomas Dorsett","doi":"10.4155/cli.15.17","DOIUrl":"https://doi.org/10.4155/cli.15.17","url":null,"abstract":"Tom is a founder of ePatientFinder. Before ePatientFinder, he was a partner at Chandler Morgan Consulting where he assisted numerous health IT organizations with their business development and revenue models. Before Chandler Morgan, he served as president of iMedicor, a publicly traded health information exchange company. Prior to iMedicor, he was president and CEO of NuScribe, provider of the market’s first health information exchange coupled with a professional networking platform.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"16 1","pages":"539-542"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"75026757","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}
In solid tumors, end points such as progression-free survival are increasingly utilized as primary end points, as the use of overall survival can often be confounded by the growing use of multiple lines of therapy. In rare cancers, the choice of end points is further complicated by small and heterogeneous patient populations. In the absence of confirmed overall survival benefit, it remains unclear as to whether extending progression-free survival provides a discernible clinical benefit. Inclusion of robust patient-reported outcomes may provide valuable supporting evidence when making decisions regarding the clinical value of new costly agents. We discuss recent trials in pancreatic neuroendocrine tumors to exemplify some of the challenges faced in the trial design for rare cancers.
{"title":"Clinical trial end points relevant to patients and society for rare cancers","authors":"Shelize Khakoo, A. Georgiou, I. Chau","doi":"10.4155/CLI.15.8","DOIUrl":"https://doi.org/10.4155/CLI.15.8","url":null,"abstract":"In solid tumors, end points such as progression-free survival are increasingly utilized as primary end points, as the use of overall survival can often be confounded by the growing use of multiple lines of therapy. In rare cancers, the choice of end points is further complicated by small and heterogeneous patient populations. In the absence of confirmed overall survival benefit, it remains unclear as to whether extending progression-free survival provides a discernible clinical benefit. Inclusion of robust patient-reported outcomes may provide valuable supporting evidence when making decisions regarding the clinical value of new costly agents. We discuss recent trials in pancreatic neuroendocrine tumors to exemplify some of the challenges faced in the trial design for rare cancers.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"16 1","pages":"599-612"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"81869242","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}
Treatment of patients with systemic lupus erythematosus (SLE) is still a challenge for many physicians and often associated with a range of adverse side effects. Based on the evidence of the central role of B cells in SLE pathogenesis, several new drugs have been developed. Inhibition of BAFF/APRIL system has appeared as one of the key factors in modulating B-cell response in patients with SLE. In 2011, belimumab was approved by the European and American regulatory agencies for the treatment of SLE patients. Four years later, consistent clinical experience has been accumulated. This article aims to review the mechanisms behind this system and the evidence provided by up-to-date trials on the safety, efficacy and effectiveness of belimumab in SLE patients.
{"title":"Updating the clinical evidence on belimumab role modulating B-cell response and treatment of systemic lupus erythematosus","authors":"I. Rodríguez-Pintó, G. Espinosa, R. Cervera","doi":"10.4155/CLI.15.16","DOIUrl":"https://doi.org/10.4155/CLI.15.16","url":null,"abstract":"Treatment of patients with systemic lupus erythematosus (SLE) is still a challenge for many physicians and often associated with a range of adverse side effects. Based on the evidence of the central role of B cells in SLE pathogenesis, several new drugs have been developed. Inhibition of BAFF/APRIL system has appeared as one of the key factors in modulating B-cell response in patients with SLE. In 2011, belimumab was approved by the European and American regulatory agencies for the treatment of SLE patients. Four years later, consistent clinical experience has been accumulated. This article aims to review the mechanisms behind this system and the evidence provided by up-to-date trials on the safety, efficacy and effectiveness of belimumab in SLE patients.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"93 1","pages":"561-571"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76063755","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}
S. Morris, Karla Childers, J. Berlin, J. Waldstreicher
The Johnson & Johnson vision for data sharing We believe in responsible sharing of clinical trial data that advances science and respects individual participants, who give their time and even take risks to contribute to medicine. In keeping with that belief, our company, Johnson & Johnson (NJ, USA), has established a data sharing agreement with the Yale Open Data Access Project, an independent academic institution, to review and decide, based on scientific merit, whether requests for access to our clinical trial data, for both pharmaceutical and medical device products, will be fulfilled [1]. Sharing clinical trial data has the potential to improve public health by providing a better understanding of physiology and pathophysiology, as well as both the benefits and risks of all types of treatments, including medicines and devices. Importantly, it also enables a powerful approach to address important questions that could not be addressed within individual studies by using either a combined analysis or metaanalysis, enabling a far deeper understanding of diseases, subgroups of patients who might respond differently and strengthening the evidence base for future studies, treatment guidelines, r egulatory, payer and medical decisions [2]. Recently, the Institute of Medicine released a comprehensive report, which offers recommendations for responsible data sharing and a vision of an ecosystem in which clinical trial data from all sources are more broadly accessible to the research community [3]. We support the Institute of Medicine’s recommendations and agree on the need to move forward, through multistakeholder groups, some of which are already working on various aspects of data transparency and approaches to harmonization, including the multiregional clinical trial forum [4] and TransCelerate [5]. We agree that the greatest value from data sharing efforts will only be realized if data sharing is agreed and embraced broadly. This will require all stakeholders who conduct clinical trials of all types and of all interventions to participate in data sharing, as well as an effort towards more common data collection tools and standards. Of course, all efforts have to be aimed at maintaining the highest possible scientific principles.
我们相信，负责任的临床试验数据共享能够促进科学发展，并尊重每位参与者，他们为医学付出了时间，甚至承担了风险。本着这一信念，强生公司(NJ, USA)与独立学术机构耶鲁大学开放数据获取项目(Yale Open data Access Project)签订了数据共享协议，根据科学价值审查并决定是否满足获取我们的制药和医疗器械产品临床试验数据的请求[1]。共享临床试验数据有可能通过更好地了解生理学和病理生理学以及所有类型治疗(包括药物和设备)的益处和风险来改善公众健康。重要的是，它还提供了一种强大的方法来解决无法通过使用组合分析或荟萃分析在单个研究中解决的重要问题，从而能够更深入地了解疾病，可能有不同反应的患者亚组，并为未来的研究、治疗指南、监管、付款人和医疗决策加强证据基础[2]。最近，医学研究所发布了一份综合报告，提出了负责任的数据共享建议，并提出了一个生态系统的愿景，在这个生态系统中，来自所有来源的临床试验数据都可以更广泛地为研究界所获取[3]。我们支持医学研究所的建议，并同意有必要通过多方利益相关者团体向前推进，其中一些团体已经在研究数据透明度和协调方法的各个方面，包括多区域临床试验论坛[4]和transelerate[5]。我们一致认为，只有在数据共享得到广泛同意和接受的情况下，才能实现数据共享的最大价值。这将要求开展所有类型临床试验和所有干预措施的所有利益攸关方参与数据共享，并努力建立更通用的数据收集工具和标准。当然，所有的努力都必须以维护尽可能高的科学原则为目标。
{"title":"The need for even further progress with clinical trial data sharing efforts: patients are waiting","authors":"S. Morris, Karla Childers, J. Berlin, J. Waldstreicher","doi":"10.4155/CLI.15.22","DOIUrl":"https://doi.org/10.4155/CLI.15.22","url":null,"abstract":"The Johnson & Johnson vision for data sharing We believe in responsible sharing of clinical trial data that advances science and respects individual participants, who give their time and even take risks to contribute to medicine. In keeping with that belief, our company, Johnson & Johnson (NJ, USA), has established a data sharing agreement with the Yale Open Data Access Project, an independent academic institution, to review and decide, based on scientific merit, whether requests for access to our clinical trial data, for both pharmaceutical and medical device products, will be fulfilled [1]. Sharing clinical trial data has the potential to improve public health by providing a better understanding of physiology and pathophysiology, as well as both the benefits and risks of all types of treatments, including medicines and devices. Importantly, it also enables a powerful approach to address important questions that could not be addressed within individual studies by using either a combined analysis or metaanalysis, enabling a far deeper understanding of diseases, subgroups of patients who might respond differently and strengthening the evidence base for future studies, treatment guidelines, r egulatory, payer and medical decisions [2]. Recently, the Institute of Medicine released a comprehensive report, which offers recommendations for responsible data sharing and a vision of an ecosystem in which clinical trial data from all sources are more broadly accessible to the research community [3]. We support the Institute of Medicine’s recommendations and agree on the need to move forward, through multistakeholder groups, some of which are already working on various aspects of data transparency and approaches to harmonization, including the multiregional clinical trial forum [4] and TransCelerate [5]. We agree that the greatest value from data sharing efforts will only be realized if data sharing is agreed and embraced broadly. This will require all stakeholders who conduct clinical trials of all types and of all interventions to participate in data sharing, as well as an effort towards more common data collection tools and standards. Of course, all efforts have to be aimed at maintaining the highest possible scientific principles.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"10 1","pages":"531-533"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"85444948","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}
The use of social media is tremendously increasing since years and will continue its growth. Recent examples show that social media can also be used to attract patients to clinical trials and improve recruitment rates. In addition, retention of the patient is increased. Social media help in designing clinical trials by obtaining early feedback from possible patients, informing patients about clinical trials and facilitating their participation by providing tools to guide them through the trial. To achieve this, social media use three different channels, listen – inform – engage. For listening and feedback, blogs can best be used, for information purposes social networking sites. To engage patients, apps are very useful.
{"title":"Is social media suitable for patient recruitment","authors":"W. Eglmeier","doi":"10.4155/CLI.15.21","DOIUrl":"https://doi.org/10.4155/CLI.15.21","url":null,"abstract":"The use of social media is tremendously increasing since years and will continue its growth. Recent examples show that social media can also be used to attract patients to clinical trials and improve recruitment rates. In addition, retention of the patient is increased. Social media help in designing clinical trials by obtaining early feedback from possible patients, informing patients about clinical trials and facilitating their participation by providing tools to guide them through the trial. To achieve this, social media use three different channels, listen – inform – engage. For listening and feedback, blogs can best be used, for information purposes social networking sites. To engage patients, apps are very useful.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"78 1","pages":"573-583"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"79689540","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}
M. Thomason, M. Spyer, Nicola Joffe, J. Boles, A. Burke, H. Wilkes, M. Rauchenberger
The Medical Research Council Clinical Trials Unit has coordinated HIV clinical trials in Africa for almost 15 years. Approaches to monitoring trial data have been developed using a combination of on-site and central (database) monitoring. Tools and templates have been designed to supplement trial protocols and help standardize trial processes. Local monitors supplement infrequent visits from the sponsor, enabling monitoring at the required intensity and allowing for capacity building. Database strategies have evolved to complement on-site visits, allowing more effective monitoring of data quality, and providing functionality in a cost-effective manner. Ongoing training and support of monitors and site staff is given via teleconferences, emails and meetings. Mentoring of site staff by monitors is encouraged, including cross-site visits where resources allow.
{"title":"Monitoring randomized clinical trials in Africa; pragmatic approaches and experiences from HIV trials","authors":"M. Thomason, M. Spyer, Nicola Joffe, J. Boles, A. Burke, H. Wilkes, M. Rauchenberger","doi":"10.4155/CLI.15.18","DOIUrl":"https://doi.org/10.4155/CLI.15.18","url":null,"abstract":"The Medical Research Council Clinical Trials Unit has coordinated HIV clinical trials in Africa for almost 15 years. Approaches to monitoring trial data have been developed using a combination of on-site and central (database) monitoring. Tools and templates have been designed to supplement trial protocols and help standardize trial processes. Local monitors supplement infrequent visits from the sponsor, enabling monitoring at the required intensity and allowing for capacity building. Database strategies have evolved to complement on-site visits, allowing more effective monitoring of data quality, and providing functionality in a cost-effective manner. Ongoing training and support of monitors and site staff is given via teleconferences, emails and meetings. Mentoring of site staff by monitors is encouraged, including cross-site visits where resources allow.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"96 1","pages":"585-597"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"77426830","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}
Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme acid β-glucosidase. Enzyme replacement therapy is the standard of care for the treatment of GD type I. Currently, three preparations, including imiglucerase (Cerezyme®, Genzyme Corporation, MA, USA), taliglucerase alfa (Elelyso®, Pfizer Inc., NY, USA) and velaglucerase alfa (VPRIV®, Shire Human Genetic Therapies Inc., Dublin, Ireland), are commercially available. Here, we will review the recent literature addressing the safety and efficacy of velaglucerase, particularly as compared with the other enzyme replacement therapy products, as well as the treatment of GD type 1 with velaglucerase alfa.
{"title":"Velaglucerase alfa in the treatment of Gaucher disease type 1: an update","authors":"K. Weaver, G. Grabowski, T. Burrow","doi":"10.4155/CLI.15.19","DOIUrl":"https://doi.org/10.4155/CLI.15.19","url":null,"abstract":"Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme acid β-glucosidase. Enzyme replacement therapy is the standard of care for the treatment of GD type I. Currently, three preparations, including imiglucerase (Cerezyme®, Genzyme Corporation, MA, USA), taliglucerase alfa (Elelyso®, Pfizer Inc., NY, USA) and velaglucerase alfa (VPRIV®, Shire Human Genetic Therapies Inc., Dublin, Ireland), are commercially available. Here, we will review the recent literature addressing the safety and efficacy of velaglucerase, particularly as compared with the other enzyme replacement therapy products, as well as the treatment of GD type 1 with velaglucerase alfa.","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"47 1","pages":"543-549"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"86794631","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}
Clinical research involving human subjects is driven mainly by the availability of novel products, devices or interventions that require further investigations to determine their safety and efficacy. The designs of clinical trials are critical to ensuring scientific validity and reproducibility of the results and it is essential that ethical approval is obtained by well-informed ethical committees in all participating countries. The WHO has developed a roadmap for 17 poverty related diseases, collectively called neglected tropical diseases (NTDs) [1]. However, the WHO focus NTDs do not include malaria, HIV/AIDs and tuberculosis which account for nearly two-thirds of all R&D funding associated with poverty related or neglected diseases. According to the 2014 G-FINDER survey report [2], in 2013, US$3.2 billion were invested in R&D for 132 poverty related diseases referred to as ‘neglected diseases’ [2]. The investment targeted 138 products that included drugs, vaccines, diagnostics, microbicides and vector control products. The big three diseases, HIV/AIDS, malaria and TB received 69% of the global neglected disease R&D funding and less than 10% was invested in the WHO focus NTDs. Of the US$2.1 billion contributed by the public sector, over US$2 billion were provided by high income countries (HIC) that influenced the diseases to focus on. This may partly explain the huge disparity between funding for the big three diseases and the 17 WHO focus NTDs. Nevertheless, in 2012 and 2014, 22 partners from the public and private sectors, including WHO, Bill & Melinda Gates Foundation, pharmaceutical companies and the US and UK governments committed through the London Declaration on NTDs ‘to advance R&D through partnerships and provision of funding to find nextgeneration treatments and interventions for neglected diseases’ [3]. Many of the WHO focus NTDs are considered tools ready, and targeted for eradication or elimination by 2020 [1]. These include Guinea worm disease, lymphatic filariasis, leprosy, human African trypanosomiasis and blinding trachoma. Other focus NTDs including schistosomiasis, soil-transmitted helminthiasis (STH), Chagas disease, visceral leishmaniasis and river blindness (onchocerciasis) are targeted for control as a public health problem by 2020. Alternative intervention strategies based on new drugs, vaccines and novel devices have been proposed as additional tools that could fast-track the fight against NTDs [4–7]. However, many challenges exist for the conduct of clinical trials that will determine the safety and efficacy of the proposed new products and interventions. NTDs are diseases of neglected people living in lowand middle-income countries (LMIC) with little influence over the allocation of the substantial R&D funding required for the development of new products for “Making an investment case for the R&D for vaccines of tool ready diseases have been challenging but researchers advocating for new products, including vaccines,
{"title":"The challenges of conducting clinical trials for neglected tropical diseases","authors":"M. Rebollo, M. Bockarie","doi":"10.4155/CLI.15.24","DOIUrl":"https://doi.org/10.4155/CLI.15.24","url":null,"abstract":"Clinical research involving human subjects is driven mainly by the availability of novel products, devices or interventions that require further investigations to determine their safety and efficacy. The designs of clinical trials are critical to ensuring scientific validity and reproducibility of the results and it is essential that ethical approval is obtained by well-informed ethical committees in all participating countries. The WHO has developed a roadmap for 17 poverty related diseases, collectively called neglected tropical diseases (NTDs) [1]. However, the WHO focus NTDs do not include malaria, HIV/AIDs and tuberculosis which account for nearly two-thirds of all R&D funding associated with poverty related or neglected diseases. According to the 2014 G-FINDER survey report [2], in 2013, US$3.2 billion were invested in R&D for 132 poverty related diseases referred to as ‘neglected diseases’ [2]. The investment targeted 138 products that included drugs, vaccines, diagnostics, microbicides and vector control products. The big three diseases, HIV/AIDS, malaria and TB received 69% of the global neglected disease R&D funding and less than 10% was invested in the WHO focus NTDs. Of the US$2.1 billion contributed by the public sector, over US$2 billion were provided by high income countries (HIC) that influenced the diseases to focus on. This may partly explain the huge disparity between funding for the big three diseases and the 17 WHO focus NTDs. Nevertheless, in 2012 and 2014, 22 partners from the public and private sectors, including WHO, Bill & Melinda Gates Foundation, pharmaceutical companies and the US and UK governments committed through the London Declaration on NTDs ‘to advance R&D through partnerships and provision of funding to find nextgeneration treatments and interventions for neglected diseases’ [3]. Many of the WHO focus NTDs are considered tools ready, and targeted for eradication or elimination by 2020 [1]. These include Guinea worm disease, lymphatic filariasis, leprosy, human African trypanosomiasis and blinding trachoma. Other focus NTDs including schistosomiasis, soil-transmitted helminthiasis (STH), Chagas disease, visceral leishmaniasis and river blindness (onchocerciasis) are targeted for control as a public health problem by 2020. Alternative intervention strategies based on new drugs, vaccines and novel devices have been proposed as additional tools that could fast-track the fight against NTDs [4–7]. However, many challenges exist for the conduct of clinical trials that will determine the safety and efficacy of the proposed new products and interventions. NTDs are diseases of neglected people living in lowand middle-income countries (LMIC) with little influence over the allocation of the substantial R&D funding required for the development of new products for “Making an investment case for the R&D for vaccines of tool ready diseases have been challenging but researchers advocating for new products, including vaccines, ","PeriodicalId":10369,"journal":{"name":"Clinical investigation","volume":"44 1","pages":"535-537"},"PeriodicalIF":0.0,"publicationDate":"2015-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"81021466","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}
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