標題: Titlebook: Innovative Designs and Analyses for Small Population Clinical Trials; Development Strategi Jingjing Ye,Lei Nie Book 2024 The Editor(s) (if [打印本頁] 作者: 一再 時間: 2025-3-21 17:37
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作者: Ornament 時間: 2025-3-21 20:34
Master Protocol,e described in detail, separately for exploratory and confirmatory settings. The master protocol has been implemented successfully and improved the patient care in treating patients with rare cancers and pediatric cancers. One of those successful examples is discussed in the chapter.作者: Magisterial 時間: 2025-3-22 00:27
Operation and Other Considerations,sease classification, and patient monitoring. Additionally, translational relevance of preclinical models to pediatric and rare diseases and potential pharmacodynamic endpoints are included in the considerations.作者: 爭議的蘋果 時間: 2025-3-22 07:23
Case Studies,ents who receive lung transplants. The third case is Belimumab’s approval that has applied Bayesian analysis to borrow adult data into pediatric to support approval in children with system lupus erythematosus (SLE).作者: Offensive 時間: 2025-3-22 10:23
Book 2024a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patie作者: Misnomer 時間: 2025-3-22 15:43 作者: 責怪 時間: 2025-3-22 19:02 作者: 參考書目 時間: 2025-3-22 23:55 作者: 妨礙 時間: 2025-3-23 05:07
Diagnostic Criteria and Endpoint,tive multi-stake holders’ collaboration to best utilize the available information or generate the fit-for-purpose evidence. The statistical evaluation of the diagnosis and the endpoints are provided. Successful implementations in case studies are included.作者: habile 時間: 2025-3-23 06:42
Introduction,que rare diseases, affecting 300?million people worldwide, about 72% of which are genetic and about 70% of which are exclusively pediatric onset. More than 90% of rare diseases are without an FDA-approved treatment. Rare diseases are numerous, heterogenous in nature, and geographically disparate. Gi作者: grieve 時間: 2025-3-23 12:36
The Case Study of NCI-COG Pediatric MATCH Trial,various age groups presents a significant challenge for drug development program as compared to those for adults. Furthermore, modern targeted and precision cancer drug development typically are only effective in a subtype of disease, which further shrinks the targeted patient population. As a resul作者: 輕信 時間: 2025-3-23 13:52
Non-Oncology Case Study of Lonafarnib,roteins like progerin. Lonafarnib, a farnesyltransferase inhibitor, emerged as a potential treatment for HGPS. This chapter outlines lonafarnib’s development program, supported by pivotal trials comparing treated patients to a natural history cohort. Utilizing the Progeria Research Foundation Intern作者: follicle 時間: 2025-3-23 18:23
Natural History, Patient Registry, and Patient Voice,ural history of the condition and the degree of unmet medical needs. The natural history studies and the patient’s voice are critical components to rare disease drug development. In recent years, there is also strong regulatory support to incorporate natural history and patient voice into rare disea作者: fatuity 時間: 2025-3-24 00:32 作者: anticipate 時間: 2025-3-24 02:24
Design and Analysis Considerations,ive and effective drugs. We start by describing regulatory and development pathways for rare disease drug developments..We attribute the issue of chance findings in clinical trials of noneffective drugs to small sample sizes. To mitigate this, we advocate for the adoption of measures such as minimum作者: Basal-Ganglia 時間: 2025-3-24 09:40 作者: 營養(yǎng) 時間: 2025-3-24 13:54
Crossover Design,lly significant results. These designs capitalize on repeated measurements from the same subjects under different treatments, potentially enhancing the study’s efficiency and sensitivity. However, their suitability depends on specific trial characteristics, such as disease stability and reversibilit作者: 敵意 時間: 2025-3-24 18:51 作者: 閑聊 時間: 2025-3-24 19:39 作者: 持續(xù) 時間: 2025-3-25 02:50
Use of Real-World Data (RWD) and Real-World Evidence (RWE), of validated endpoints. To address these challenges, regulatory agencies like the FDA have introduced flexibilities in evidence requirements and provided guidance specifically aimed at rare disease drug development. This paper reviews the regulatory landscape and challenges in rare disease drug dev作者: 特征 時間: 2025-3-25 07:24
Modelling and Simulations, systems. This paper explores the fundamental concepts, methods, and applications of modeling and simulation in drug development for rare diseases. At its core, modeling involves constructing simplified representations of real-world phenomena, while simulation involves executing these models to obse作者: sed-rate 時間: 2025-3-25 11:20 作者: beta-cells 時間: 2025-3-25 12:37 作者: LEER 時間: 2025-3-25 16:17
Operation and Other Considerations,o enriched the possible data collection methods via digital health technologies (DHTs) and integration of siloed data generated through various sources. In the chapter, we first discuss the requirement and validations for use of the DHTs in the regulatory setting. Various emerging practical consider作者: LUMEN 時間: 2025-3-25 22:58 作者: 凹槽 時間: 2025-3-26 01:36 作者: finale 時間: 2025-3-26 04:34
Jingjing Ye,Lei Niets, it gives clear guidance based on the fundamental principles of moral philosophy, explaining them in simple language and illustrating them with abundant clinical examples and case studies. The strength of this text is in its emphasis on normative ethics and critical thinking, and that there is tr作者: FICE 時間: 2025-3-26 11:12 作者: BURSA 時間: 2025-3-26 12:58 作者: Bombast 時間: 2025-3-26 19:11
Jingjing Ye,Lei Nieto set up those committees is linked to an analysis of ethical aspects in medical research projects as published in an American medical journal of high reputation. The results prompted the National Instituts of Health (NIH) to require an assessment of research projects by an Institutional Review Boa作者: 與野獸博斗者 時間: 2025-3-26 23:56 作者: chuckle 時間: 2025-3-27 04:57
Jingjing Ye,Lei Nien relation with mental health care, an introduction of the concepts of necessary care and solidarity, as well as need and outcome based approaches. While the needs of mental health patients are widely recognised, needs are generally seen as subjective and difficult to distinguish from preferences or作者: cajole 時間: 2025-3-27 09:04
Jingjing Ye,Lei Nieed on the information gleaned from the data. Such data-intensive processes lead to unique ethical issues relating to autonomy of the data generator, privacy, confidentiality and justice. In this chapter, we attempt to describe the ethical issues in data handling in epidemiology using two examples, p作者: GENRE 時間: 2025-3-27 09:53
Jingjing Ye,Lei Nie. Very few public health education programs in India have an explicit module on public health ethics, and even where imparted, it is skewed towards biomedical and/or research ethics. This chapter takes the readers through a journey on designing and transacting a course on Ethics in Public Health Pra作者: 關(guān)節(jié)炎 時間: 2025-3-27 17:13
Jingjing Ye,Lei Nie. Very few public health education programs in India have an explicit module on public health ethics, and even where imparted, it is skewed towards biomedical and/or research ethics. This chapter takes the readers through a journey on designing and transacting a course on Ethics in Public Health Pra作者: Presbyopia 時間: 2025-3-27 18:48 作者: CLAM 時間: 2025-3-28 01:23
Jingjing Ye,Lei Niees several ethical issues that are implicated in this burgeoning industry, the foremost being the stigmatization of childlessness and essentialization of motherhood that push women particularly?to access ARTs?(Assisted Reproductive Technologies). Secondly, even when regulation was initiated, many co作者: 無禮回復(fù) 時間: 2025-3-28 02:42
Jingjing Ye,Lei Nieg health inequities, among others, have raised serious concerns about public health goals and practices and necessitate an ethical lens to the “l(fā)essons learned” from such challenges. The last two decades or so have witnessed an increasingly explicit engagement with public health ethics in academic a作者: PLIC 時間: 2025-3-28 06:45 作者: 密碼 時間: 2025-3-28 13:38
Jingjing Ye,Lei Nielysis. The primary goal of the framework is to provide students with a way to systematically consider ethical concerns when evaluating health policy options. A secondary goal of the framework is to give the students and myself a common language to move forward in the context of the material presente作者: 他姓手中拿著 時間: 2025-3-28 16:20 作者: 加強防衛(wèi) 時間: 2025-3-28 21:52
Introduction,e discussed. The rest of the book will dive deep into providing a systematic framework that require a collaborative infrastructure including natural histories, diagnosis criteria, identification of clinical endpoints and engagement with the stakeholders. Each of the book chapters will discuss the cl作者: COMMA 時間: 2025-3-28 23:44
The Case Study of NCI-COG Pediatric MATCH Trial,al, is discussed in detail. The NCI-COG Pediatric MATCH trial overcomes many challenges and has created a collaborative framework for efficient collection, processing, and sequencing of refractory pediatric cancers. The study successfully facilitates evaluation of molecular-targeted agents in biomar作者: VAN 時間: 2025-3-29 06:56 作者: FATAL 時間: 2025-3-29 08:24 作者: 敲竹杠 時間: 2025-3-29 13:22 作者: 騷擾 時間: 2025-3-29 15:40 作者: 步履蹣跚 時間: 2025-3-29 23:21
Use of Real-World Data (RWD) and Real-World Evidence (RWE), and natural history studies have emerged as valuable resources, aiding in clinical trial planning and endpoint modeling. However, challenges such as data bias and variability require careful consideration. Case examples, such as the approval of Lutathera for neuroendocrine tumors, demonstrate the u作者: Fallibility 時間: 2025-3-30 01:24 作者: Accord 時間: 2025-3-30 07:33 作者: beta-carotene 時間: 2025-3-30 10:57 作者: antidepressant 時間: 2025-3-30 15:08
2199-0980 s, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.978-3-031-60845-2978-3-031-60843-8Series ISSN 2199-0980 Series E-ISSN 2199-0999 作者: neoplasm 時間: 2025-3-30 17:31 作者: ASSAY 時間: 2025-3-30 22:42
Jingjing Ye,Lei Nie stake that create duties for the pharmacist. This is the balancing act of normative ethics, and of deciding which duties should prevail in a given clinical situation. This work presents a clear-cut pathway for resolving ethical dilemmas encountered by pharmacists, based on foundational principles a
