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Clinical trials glossary & taxonomy
Evolving Terminology for Emerging Technologies
Comments? Questions? Revisions? 
Mary Chitty MSLS
Last revised January 09, 2020

SCOPE NOTE  Clinical trials are clinical research studies involving human participants assigned to an intervention in which the study is designed to evaluate the effect(s) of the intervention on the participant and the effect being evaluated is a health-related biomedical or behavioral outcome.  NIH Clinical Trial Definition FAQ

Clinical trials include Phase 1 Phase II Phase III, patient recruitment, auditing, monitoring, budgeting & resource allocation, Clinical Research Organizations outsourcing to, clinical trial software

Clinical trials is a sub-category of  Drug discovery & development     Molecular Medicine
Related glossaries include
Drug safety & pharmacovigilance    Ethics      Molecular Diagnostics  
   Pharmacogenomics     Regulatory Affairs
Algorithms     Clinical informatics     Research  

Summit for Clinical Ops Executives (SCOPE)
Summit for Clinical Ops Executives (SCOPE)
 February 18-21, 2020 • Orlando, FL

SCOPE Europe, 17-18 October, 2019 Barcelona, Spain

adaptive clinical trials: The pharma industry is gradually coming to realize that the classically structured clinical trial does not offer enough flexibility to make use of continuously emerging knowledge that is generated as the trial progresses. This report is a comprehensive assessment of the benefits, challenges, and accumulated industry experience with regard to adaptive clinical trials. Herman Mucke, Adaptive Clinical Trials: Innovations in clinical trial design, management and analysis, Insight Pharma Reports, 2007  

Adaptive Clinical trials webcast Jerald Schindler, VP Biostatistics and Research Decision Sciences Late Stage Clinical Statistics, Merck Research Laboratories  See related pivotal clinical trials

adaptive licensing: Under adaptive licensing, the clinical-development program is restructured to allow for early approval of a new compound for a limited, typically high-risk population based on valid clinical measures from smaller human studies. Approval would be revisited at several points along the clinical-development pathway as candidate populations are broadened, longer-term outcomes are evaluated, and risks of treatment are better understood. … also called “staggered approval” and “progressive licensing”. Dan Ollendorf, If Everyone Hates the FDA Approval Process, Let’s Fix It, HBR Blog Network, Harvard Business Review, Oct 18, 2013  

Analytics-Driven Feasibility, Site Selection and Study Activation October 16-17, 2018 Barcelona | Site Selection and Study Activation Improving Protocol Development, Global Site Selection, Feasibility and Site Management 17-19 OCTOBER 2019 Barcelona SPAIN  Analytics and data-driven global site selection, an optimized protocol development and feasibility assessment process, and effective site management are critical to improving clinical trial timelines and outcomes. Too often companies fail to learn from past mistakes and take the same approach to trial planning and execution. In order to overcome challenges in clinical trial planning, operations and site management leaders should learn from the best practices of their peers, utilize multiple data sources and technology to support decision making, and improve communication and relationships between Sites, CROs, and Sponsors.

Artificial Intelligence in Clinical Research  February 20-21, 2020  Orlando, FL | Artificial intelligence (AI) and machine learning (ML) have propelled many industries toward a new, highly functional and powerful state. Now they are starting to make their way into the clinical research realm. Many pharmaceutical companies and larger CROs are starting projects involving some elements of AI, ML and robotic process automation in clinical trials.

attrition: Unacceptable levels of attrition in the clinical stage of development are driving profound changes in the architecture, design, and analysis of clinical trials. The majority of respondents to our survey said that reduction in patient numbers, less exposure to study drug, and drops in overall trial duration were key points in favor of adaptive designs; however, a majority also had specific concerns with adaptive trials―concerns that involved methodological, logistical, and regulatory uncertainties: Herman Mucke, Adaptive Clinical Trials: Innovations in clinical trial design, management and analysis, Insight Pharma Reports, 2007  

basket trials: (or studies) test the effect of one drug on a single mutation in a variety of tumor types, at the same time. These studies also have the potential to greatly increase the number of patients who are eligible to receive certain drugs relative to other trials designs.   Related term: umbrella trials

Clinical biomarkers strategy and innovation:  February 19-20, 2020  Orlando, FL  |  The concept of personalized or precision medicine has brought to life several types of clinical trials that involve biomarkers and require biospecimen collection and management. Effective management of these trials can be complicated and requires specific operational approaches.

Clinical Data Strategy and Analytics  February 19-20, 2020  Orlando, FL   E-clinical technologies have changed the landscape of the clinical research industry and healthcare IT in general. Digitalization of healthcare data, mobile data capture technologies, and cloud storage of data are a few of the main technological advances that influence clinical data management and analytics. These technological advances have been coupled with novel data visualization solutions, and this powerful duo is helping to develop a new paradigm of data-driven clinical trials.

clinical forecasting: It is clear that late-stage clinical failures account for a large proportion of the expenses. This can be as a result of both the large out-of-pocket investments in Phase III clinical trials and because unsuccessful trials tie up capital resources during their conduct, and potentially also for the time spent during any attempted recovery following regulatory rejection. So, there is an interest in strategies that could halt, as early as possible, the development of drugs that eventually fail. Clinical forecasting in drug development, Asher D. Schachter and Marco F. RamoniNature Reviews Drug Discovery 6, 107-108 (February 2007) | doi:10.1038/nrd2246 

Narrower term: Bayesian clinical forecasting, Bayesian clinical trials

clinical research: Clinical trials are clinical research studies. Clinical research includes all research involving human participants. It does not include secondary studies using existing biological specimens or data collected without identifiers or data that are publicly available.

Clinical Research & Translational Informatics Transforming Biological Data to Clinical Development 2020 April 21-23 Boston MA Advancing clinical trials and translational research requires transforming biological insights and raw research data into clean, actionable data using innovative techniques for its integration, visualization and analysis. The Clinical Research & Translational Informatics track explores new approaches to the integration, visualization, analysis, and application of biological and clinical trial data, including machine learning, artificial intelligence, big data analytics, and additional technologies with case studies from across pharma and academia.

Clinical Supply Management  February 19-20, 2020  Orlando, FL  Successful, patient-centric clinical trials depend upon streamlined clinical trial supply processes that ensure that the study drug is properly handled and delivered to the right patient whether at the trial site, pharmacy or in their home.

Clinical Technology and Innovation  February 20-21, 2020  Orlando, FL  |  Digital technology, mobile solutions, novel data collection modalities and integrative systems are becoming game-changing features of modern clinical trials. However, the adoption of novel technology solutions to improve overall outcomes and garner operational efficiencies has been slower than expected. Will include  blockchain technology, machine learning, digital trends, and their adoption and implementation in clinical research.

Clinical Trial Design Task Force: The Clinical Trial Design Task Force (CTD-TF) of the National Cancer Institute (NCI) Investigational Drug Steering Committee (IDSC) has published a series of discussion papers on phase II trial design in Clinical Cancer Research. The IDSC has developed formal recommendations about aspects of phase II trial design that are the subject of frequent debate, such as endpoints (response versus progression-free survival), randomization (single-arm designs versus randomization), inclusion of biomarkers, biomarker-based patient enrichment strategies, and statistical design (e.g., two-stage designs versus multiple-group adaptive designs). Although these recommendations in general encourage the use of progression-free survival as the primary endpoint, randomization, inclusion of biomarkers, and incorporation of newer designs, we acknowledge that objective response as an endpoint and single-arm designs remain relevant in certain situations. The design of any clinical trial should always be carefully evaluated and justified based on characteristic specific to the situation. The Design of Phase II Clinical Trials Testing Cancer Therapeutics: Consensus Recommendations from the Clinica l Trial Design Task Force of the National Cancer Institute Investigational Drug Steering Committee. Seymour L, et. al,   Clin Cancer Res. 2010 Mar 9. [Epub ahead of print]

Clinical Trial Innovation Summit April 6-8, 2020 • Cambridge, MA  | Through case studies, workshops, panel & roundtable breakout discussions and an active exhibit hall, the summit delivers the real-world experiences and best practices needed to optimize clinical trial innovation, planning and management.
clinical trial simulation:
A relatively new effort to devise in silico simulations of human physiology and genetic variation to help identify which compounds will eventually fail in the drug development process.   

clinical trials: NIH Definition of a Clinical Trial  A research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.
FAQ NIH Clinical trial definition    Why changes to clinical trial policies?
CenterWatch  A listing of more than 41,000 industry- and government- sponsored clinical trials as well as new drug therapies recently approved by the FDA. 
Clinical trials
,, National Cancer Institute, NIH 

Related terms: CRO Clinical Research Organization, clinical informatics, comparative data mining, FDA drug approvals,  postmarketing surveillance, preclinical, predictive data mining  Narrower terms: adaptive clinical trials, computer trial simulations, explanatory trials, management trials, randomized clinical trials  Phase I, Phase II, Phase III, Phase IV , Phase Zero

computable phenotypes in the context of electronic health records (EHRs), a computable phenotype or simply phenotype refers to a clinical condition or characteristic that can be ascertained via a computerized query to an EHR system or clinical data repository using a defined set of data elements and logical expressions. These queries can identify patients with a condition, such as diabetes mellitus, obesity, or heart failure, and can be used to support a variety of purposes and data needs for observational and interventional research.  NIH Collaboratory of Pragmatic Clinical Trials

CONSORT  Consolidated Standards of Reporting Trials: encompasses various initiatives developed by the CONSORT Group to alleviate the problems arising from inadequate reporting of randomized controlled trials. .. The main product of the CONSORT Group is the CONSORT Statement,[1] which is an evidence-based, minimum set of recommendations for reporting randomized trials. It offers a standard way for authors to prepare reports of trial findings, facilitating their complete and transparent reporting, reducing the influence of bias on their results, and aiding their critical appraisal and interpretation. Wikipedia accessed 2018 Sept 2

CRO Clinical Research Organization : An organization that conducts all or some of the clinical research involved in the drug or product development process on behalf of pharmaceutical research companies. Barnett/Parexel,  

Clinical trials are increasingly being run by outsourcing to these groups. Disambiguation: CRO may also refer to Contract Research Organization which focuses on drug discovery and development. 

Data & Technology April 7-8, 2020 Cambridge, MA | Technology and data are at the forefront driving clinical trial decision-making. With further advancements in new technologies (such as mobile devices and wearables) and the rise of online communities, the pharma and biotech industries are poised to capitalize on these advancements to innovate existing clinical trial processes and systems.

developmental site agreements: Companies work with hospitals to develop instrumentation and clinical research applications.  

efficacy trials: (explanatory trials) determine whether an intervention produces the expected result under ideal circumstances. Effectiveness trials (pragmatic trials) measure the degree of beneficial effect under “real world” clinical settings. 2 Hence, hypotheses and study designs of an effectiveness trial are formulated based on conditions of routine clinical practice and on outcomes essential for clinical decisions.

Efficacy and effectiveness exist on a continuum. Generalizability depends largely on the viewpoint of the observer and the condition under investigation. Baseline patient characteristics (e.g., sex, age, severity of the disease, racial groups) are primary factors in generalizability; thus, depending on the population of interest, generalizability of the same study can range from low to high. Geographic settings (urban versus rural) and health care systems can also be significant, factors, 1 although geography may have less influence on generalizability of drug trials than trials of other interventions (e.g., screening programs, behavioral therapy). Criteria for Distinguishing Effectiveness From Efficacy Trials in Systematic Reviews Technical Reviews, No. 12. Gartlehner G, Hansen RA, Nissman D, et al. Rockville (MD): Agency for Healthcare Research and Quality (US); 2006 Apr.

Efficacy and effectiveness: Archie Cochrane, British famous clinical epidemiologist, defined two concepts related to assessing healthcare interventions. Efficacy is the extent to which an intervention does more good than harm under ideal circumstances. Effectiveness assesses whether an intervention does more good than harm when provided under usual circumstances of healthcare practice.1)  Research on efficacy and effectiveness has also been described as explanatory and pragmatic trials. Kim SY. Efficacy versus Effectiveness. Korean Journal of Family Medicine. 2013;34(4):227. doi:10.4082/kjfm.2013.34.4.227.

enrichment strategies: For the purposes of this guidance, the term enrichment is defined as the prospective use of any patient characteristic to select a study population in which detection of a drug effect (if one is in fact present) is more likely than it would be in an unselected population…. Enrichment strategies fall into three broad categories:   1. Strategies to decrease heterogeneity  2. Prognostic enrichment strategies 3. Predictive enrichment strategies  Guidance for Industry Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products, Dec. 2012   DRAFT GUIDANCE  Enrichment Strategies for Clinical Trials to Support Approval of - FDA .

Enrollment Planning and Patient Recruitment  February 19-20, 2020  Orlando, FL   |  Patient recruitment and up-front enrollment planning are critical to drug development programs. Patient recruitment if not adequately planned for can extend your development timeline by a number of years. Retention of patients throughout the life of a clinical trial is essential in order have complete data sets for your analysis and subsequent filings. In order to optimize both you have to have a plan and effectively leverage analytics and technology without losing site of the participant’s user experience

expansion cohort trials FIH FIrst in Human Guidance:

explanatory trials:  Explanatory trials generally measure efficacy—the benefit a treatment produces under ideal conditions, often using carefully defined subjects in a research clinic. Martin Roland, David J Torgerson, Understanding controlled trials: What are pragmatic trials? BMJ 316: 285 24 January, 1998  Compare pragmatic trial

Forecasting, Budgeting and Contracting Clinical Trials   February 19-20, 2020  Orlando, FL As clinical trials become more complex and take on innovative designs, it is more critical than ever to develop proper strategies for forecasting, budgeting, negotiating, and contracting both internally and externally with sites, CROs, and other partners. Finance and operations teams must continue to evolve and adapt, especially in light of new and changing regulations and laws.

HIPAA Health Insurance Portability and Accountability Act: Research

intervention:  As related to the definition of a clinical trial, a manipulation of the subject or subject's environment for the purpose of modifying one or more health-related biomedical or behavioral processes and/or endpoints. Examples include: drugs/small molecules/compounds; biologics; devices; procedures (e.g., surgical techniques); delivery systems (e.g., telemedicine, face-to-face interviews); strategies to change health-related behavior (e.g., diet, cognitive therapy, exercise, development of new habits); treatment strategies; prevention strategies; and, diagnostic strategies. NIH, Important Clinical Trial terms  

large simple trials: A large simple trial (LST) is a type of randomized clinical trial (RCT) ideally suited to answer many important clinical questions and because it typically answers only one or 2 questions in a broader patient population, is generally more efficient and less expensive than other large RCTs. LSTs have a large sample size and statistical power to detect clinically relevant treatment effects, providing unambiguous results and minimizing the effects of random errors. Yet, LSTs are not often employed; Clinical trials transformation initiative

lure of initial value: What we observe [in clinical trials] is not so much a placebo response, but a so- called lure of initial value, where extreme symptoms tend to get spontaneously better. To get a handle on this problem, it would be useful to identify genes that are predictive of rapid disease remission, because many of our problems in terms of testing drugs are driven by the placebo group.  ... One needs very large groups to detect that small difference. Obtaining genes predictive of response might also lead to defining the group of people who, because they are likely to get better spontaneously, are going to get minimal benefit from the drug.  

measurements: are used to collect data, while interventions are used to modify health-related endpoints. A manipulation or modification in one’s behavior or environment for the purpose of measurement alone is not considered a clinical trial. NIH Clinical Trial Definition

NIH Collaboratory: Supported by the Common Fund at the National Institutes of Health (NIH), the NIH Health Care Systems Research Collaboratory aims to improve the way clinical trials are conducted by creating a new infrastructure for collaborative research with healthcare systems. The ultimate goal is to ensure that healthcare providers and patients can make decisions based on the best available clinical evidence.

Outsourced Clinical Trials Managing  February 20-21, 2020  Orlando, FL   |  As more clinical trial activities are outsourced to contract research organizations (CROs) and other third-party vendors, sponsors and their partners must form effective and quality partnerships. Features lessons learned from sponsors and CROs on vendor quality and performance in light of the new ICHE6 R2 changes, as well as how to build beneficial partnerships that effectively mitigate and manage risks and resources.

Outsourcing Strategy Mastering  February 19-20, 2020  Orlando, FL   |  Understanding outsourcing needs and optimizing the selection process of vendors lays the foundation for an efficient, cost-effective clinical trial.

patient:  People with a specific disease or condition, particularly those being treated by a health professional. Compare subject

Patient-Centric Enrollment Planning and Engagement Sept 17-18, 2019 Barcelona   |  Patient recruitment and up-front enrollment planning are critical to drug development programs and if not adequately planned for or properly executed can extend your development timeline by a number of years. Clinical researchers and study teams are working hard to better identify, understand and engage patients. Developing both a patient-centric culture and systems are key to enrolling and retaining patients throughout the life of a clinical trial. There are strategies, new technologies and techniques to empower patients, improve outreach and better match trials to the patients who need them.

Patient Engagement & Enrollment April 7-8, 2020 Cambridge  MA  Recruitment, retention and continued engagement of clinical trial participants is essential to successfully completing a clinical trial on time. With the rise of social media platforms and digital technology, the pharma industry stands to leverage these to improve patient experience, engagement, and adherence.

Patient Engagement, Enrollment and Retention through Communities and Technology  February 20-21, 2020 Orlando, FL   |  Enrollment planning, patient recruitment and a more patient-centric approach to study planning and execution are critical to drug development programs and garner a lot of attention by study teams. However, once the hard work of identifying and recruiting a trial subject has been accomplished they must be retained and remain in compliance. Retention of patients throughout the life of a clinical trial is essential to have complete data sets for your analysis and subsequent filings. There are strategies, tools and data-driven techniques such as social media platforms and mobile technology, empowered patient communities, and a more informed patient population that need to be understood and engaged.

Patient Recruitment & Site Selection April 7-8 , 2019  Cambridge, MA | Delays in patient recruitment can set back drug development by years and millions of dollars. Effective patient recruitment plans that leverage the latest analytics and technologies, as well as involve patient insight, are critical to successful clinical trials. 

Patient Reported Outcomes Consortium: The Patient-Reported Outcome (PRO) Consortium was formed in late 2008 by the Critical Path Institute (C-Path) in cooperation with the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research and the pharmaceutical industry, and formally launched in March 2009. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

Patient-reported outcome (PRO) data are defined by the FDA as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” These data are increasingly used to inform and guide patient-centered care, clinical decision-making, and health policy decisions  NIH Collaboratory, Patient Reported Outcomes Working Group

Phase I: The main aim of this phase is to determine drug safety. At this stage, drugs are tested in a small group of healthy volunteers to determine the drug’s activity. 

Phase II: These trials are aimed at identifying the optimal dose to be used in Phase III trials and, ideally, they identify drugs that will not make it through the next phase of testing. Typically, Phase II trials are double-blinded and have placebo controls.   

phase II clinical trials design: The optimal design of phase II studies continues to be the subject of vigorous debate, especially studies of newer molecularly targeted agents. The observations that many new therapeutics "fail" in definitive phase III studies, coupled with the numbers of new agents to be tested as well as the increasing costs and complexity of clinical trials, further emphasize the critical importance of robust and efficient phase II design. The Design of Phase II Clinical Trials Testing Cancer Therapeutics: Consensus Recommendations from the Clinica l Trial Design Task Force of the National Cancer Institute Investigational Drug Steering Committee. Seymour L, et. al,   Clin Cancer Res. 2010 Mar 9. [Epub ahead of print]

Phase IIa and b: Some pharmaceutical companies further differentiate this phase into phases IIA and IIB. Clinical studies designed to evaluate dosing are referred to as Phase IIA and studies designed to determine the effectiveness of the drug are called phase  IIB. Design and Analysis of Clinical Trials Shein-Chung Chow, Jen-pei Liu Wiley 2004

Phase III: These studies, which take several years, can involve thousands of patients at multiple trial centers. They are aimed at definitively determining the drug’s effectiveness and its side- effect profiles. These studies are also typically double- blinded and placebo- controlled.   

Phase III success rates for the pharmaceutical industry are low, but recent advances in simulation & modeling, clinical trial design, proof-of-concept, and pre-clinical decision making are set to reduce the attrition rate.
Phase IIIb: It is common practice that certain Phase III trials will continue while the regulatory submission is pending at the appropriate regulatory agency. This allows patients to continue to receive possibly lifesaving drugs until the drug can be obtained by purchase. Other reasons for performing trials at this stage include attempts by the sponsor at "label expansion" (to show the drug works for additional types of patients/diseases beyond the original use for which the drug was approved for marketing), to obtain additional safety data, or to support marketing claims for the drug. Studies in this phase are by some companies categorised as "Phase IIIB studies."[25][26]  Wikipedia accessed Feb 15 2011

Phase IV Planned post-marketing studies of diagnostic, therapeutic, or prophylactic drugs, devices, or techniques that have been approved for general sale. These studies are often conducted to obtain additional data about the safety and efficacy of a product. This concept includes phase IV studies conducted in both the U.S. and in other countries. MeSH  Clinical trials, Phase IV as topic 2008 (1993) 

Not all Phase IV studies are post-marketing surveillance (PMS) studies but every PMS study is a phase IV study. Phase IV is also an important phase of drug development. In particular, the real world effectiveness of a drug as evaluated in an observational, non-interventional trial in a naturalistic setting which complements the efficacy data that emanates from a pre-marketing randomized controlled trial (RCT). No matter how many patients are studied pre-marketing in a controlled environment, the true safety profile of a drug is characterized only by continuing safety surveillance through a spontaneous adverse event monitoring system and a post-marketing surveillance/non-interventional study.  Phase IV of Drug Development Dr Viraj Suvarna Perspect Clin Res. 2010 Apr-Jun; 1(2): 57–60. PMCID  PMC3148611  See also Post-Marketing Surveillence

phase zero: Phase 0 studies are exploratory studies that often use only a few small doses of a new drug in a few patients. They might test whether the drug reaches the tumor, how the drug acts in the human body, and how cancer cells in the human body respond to the drug. The patients in these studies might need extra tests such as biopsies, scans, and blood samples as part of the study process.The biggest difference between phase 0 and the later phases of clinical trials is that there’s almost no chance the volunteer will benefit by taking part in a phase 0 trial – the benefit will be for other people in the future. Because drug doses are low, there’s also less risk to the patient in phase 0 studies compared to phase I studies. Phase 0 studies help researchers find out whether the drugs do what they’re expected to do. If there are problems with the way the drug is absorbed or acts in the body, this should become clear very quickly in a phase 0 clinical trial. This process may help avoid the delay and expense of finding out years later in phase II or even phase III clinical trials that the drug doesn’t act as expected to based on lab studies. Phase 0 studies aren’t used widely, and there are some drugs for which they wouldn’t be helpful. Phase 0 studies are very small, often with fewer than 15 people, and the drug is given only for a short time. They’re not a required part of testing a new drug. American Cancer Society, Phase Zero Clinical trials  Related term: Drug discovery & Development microdosing

pivotal clinical trials: The intermediate-sized clinical trials supported through this RFA are a pivotal decision point in the NCI chemoprevention drug development program. The consensus view of a Working Group from the NCI and the FDA acknowledges that "the interim analysis of a validated surrogate endpoint of cancer incidence may facilitate the timely and cost-effective marketing of efficacious drugs (Kelloff et al., Cancer Epidemiol. Biomark. Prev. 4: 1-10, 1995)." Thus, the efficacy and safety data from these studies potentially supports FDA marketing approval (NDA applications) for chemoprevention indications, and certainly facilitates decisions regarding the most appropriate recommendations for subsequent large, community-based efficacy studies. Pivotal clinical trials for chemoprevention clinical development  National Cancer Institute, NIH RFA: CA-98-001 1997  Related term: adaptive clinical trials  

placebo non- responders: Non- responders on placebo] define a group that would never improve their condition unless given the drug. They may be a group that, if we could identify them, could be used to reduce clinical trial size. Using this group in a proof- of -concept, it may be possible to test a drug even without a comparative placebo and determine whether it is likely to be active.   
Related terms: disease resistant individuals, lure of initial value, placebo responders

placebo responders: Most people think of the placebo response as a true response. But much of it is actually regression to the mean.  Clinical trial subjects with more extreme symptoms are often selected because it is desirable to see a dramatic effect upon treatment with the drug.   Related terms: disease resistant individuals, lure of initial value, placebo non- responders 

post-marketing surveillence: Drug safety

pragmatic clinical trials:  are performed in real-world clinical settings with highly generalizable populations to generate actionable clinical evidence at a fraction of the typical cost and time needed to conduct a traditional clinical trial. They present an opportunity to efficiently address critical knowledge gaps and generate high-quality evidence to inform medical decision-making. However, these trials pose different challenges than are typically encountered with traditional clinical trials. NIH Collaboratory of Pragmatic Clinical Trials

pragmatic trials: Pragmatic trials measure effectiveness—the benefit the treatment produces in routine clinical practice  Martin Roland, David J Torgerson, Understanding controlled trials: What are pragmatic trials? BMJ 316: 285 24 January, 1998   Compare explanatory trials

protection of human subjects: Revised common rule: The U.S. Department of Health and Human Services and fifteen other Federal Departments and Agencies have issued final revisions to the Federal Policy for the Protection of Human Subjects (the Common Rule). A final rule was published in the Federal Register (FR) on January 19, 2017, and was amended to delay the effective and compliance dates on January 22, 2018 and June 19, 2018. 
Terminology related to the Revised Common Rule

Protocol Development, Global Site Selection, Feasibility and Site Management  February 19-20, 2019 Orlando, FL  Data-driven global site selection, an optimized protocol development and feasibility assessment process, and effective site management are critical to improving clinical trial timelines and outcomes. Too often companies fail to learn from past mistakes and take the same approach to trial planning and execution. In order to overcome challenges in clinical trial planning, operations and site management leaders should learn from the best practices of their peers, utilize data and analytics to support decision making, and improve communication and relationships between Sites, CROs, and Sponsors

randomized clinical trials RCTs:  A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. National Cancer Institute  Dictionary of Cancer Terms  

randomized controlled clinical trials: Are the gold standard for determining the efficacy of therapeutic interventions. However, medical practice has not evolved around the concept of randomized trials, but around the idea of careful observations, (anecdotal) case studies and the evaluation of retrospective data. Interventions discovered by these means and taken forward into clinical practice became standard practice as they continued to be superior when compared with prior or alternative types of treatment. Back to the future: why randomized controlled trials cannot be the answer to pharmacogenomics and personalized medicine, Frueh FW. Pharmacogenomics. 2009 Jul;10(7):1077-81

Real World Data Leveraging for Clinical and Observational Research  February 20-21, 2020  Orlando, FL The abundance of data generated during routine health care is growing in significance and should be used for clinical and observational research. Patient electronic records, registries, data from pharmacy and social media, and wearable sensors have been increasingly used as eSources. This process requires strategizing, utilizing novel data technologies, as well as close collaboration between pharmaceutical companies and organizations that possess the data. See related pragmatic clinical trials.

Resource Management and Capacity Planning for Clinical Trials  February 20-21, 2020  Orlando, FL Resource management and capacity planning is as complex as the clinical trial protocol itself – and the need to properly manage staff, workload, and outsourced partners is more important than ever to execute trials on time and within budget. To properly understand resource needs, the scope of the pipeline, and the types of complex protocols to be expected, resource managers need input and information from the project to the portfolio level, from finance and operations teams. All teams must ultimately find the best balance between cost savings and high performance.

Risk-Based Monitoring Europe  Sept 17-18, 2019 Barcelona  | With the passing of ICH E6 R2, the biopharma industry now places greater emphasis on clinical trial quality and oversight than ever before. Ensuring clinical trial quality from the onset of clinical trial planning lays the foundation for successful trials and risk-based monitoring (RBM) implementation. As industry adoption of risk-based monitoring increases, it is clear that although RBM takes many forms – remote, centralized, and risk-based monitoring – successful risk-based monitoring implementation from pilot studies to full-scale rollout requires proper change management, analytics and processes. Once established RBM and its resulting data can be leveraged to drive future clinical trial decision making.

Risk-Based Monitoring Implementing (Part 1)  February 19-20, 2020  Orlando, FL  Poor quality and risk management of clinical trials significantly impacts the success, timeliness and cost-effectiveness of clinical trials.  lessons learned, case studies, and ample discussion on building and maintaining proper clinical quality management systems with emphasis on the latest quality standards and guidelines, including the recent ICH-E6 R2 addendum changes, thereby ensuring higher quality clinical trials and laying the foundation for successful risk-based monitoring. The program will also focus on successful RBM implementation tactics employed by small and mid-sized organizations.

Part 2 February  20-21 2020 Orlando FL 
Risk-based monitoring (RBM) approaches promise to improve clinical trial efficiency while ensuring data quality. As industry adoption of RBM increases, it is critical to reflect on lessons learned to refine the process as well as focus on leveraging RBM data for clinical operations.

Risk Based Monitoring Mastering April 7-8, 2020  Cambridge, MA  Ensuring quality from the outset of a clinical trial leads to higher quality, lower risk clinical trials. The ensuing risk assessment and mitigation from the design and planning of clinical trials with the establishment of clinical quality management systems lays the foundation for successful risk-based monitoring (RBM)

risk ratio: 
A measure of the risk of a certain event happening in one group compared to the risk of the same event happening in another group. In cancer research, risk ratios are used in prospective (forward looking) studies, such as cohort studies and clinical trials. A risk ratio of one means there is no difference between two groups in terms of their risk of cancer, based on whether or not they were exposed to a certain substance or factor, or how they responded to two treatments being compared. A risk ratio of greater than one or of less than one usually means that being exposed to a certain substance or factor either increases (risk ratio greater than one) or decreases (risk ratio less than one) the risk of cancer, or that the treatments being compared do not have the same effects. Also called relative risk. NCI Dictionary

Sensors, Wearables and Digital Biomarkers in Clinical Trials: Digital Endpoints and Connectivity FEBRUARY 19-20 2020, Orlando FL Clinical research industry is moving toward end-to-end digital clinical trials. The data collection should stay in line with this inevitable change and wearables and point-of-care sensors address this need. Furthermore, digital biomarkers translate new data sources into clinically actionable insights.

site management organization: Wikipedia  

Site Selection & Feasibility Strategies for Selecting High Performing Sites April 7-8, 2020 Cambridge MA The ability to identify and select high performing sites is key to effectively launching a clinical trial. CHI’s “Site Selection & Feasibility” conference features best practices and case studies on successful site selection techniques using novel, patient-centric and data-driven approaches.

Site-Study Activation and Performance Improving  February 20-21, 2020  Orlando, FL  Clinical trial site activation and efficient study start-up are critical to drug development programs, in terms of time, cost and quality of data. To improve start-up times and outcomes, one needs an experienced clinical research investigator, motivated and capable team members and efficient communication by all. Everyone (Sponsor, CRO, Site) must communicate and execute effectively in order to improve: the study feasibility process, contract and budget negotiations, standardization of source documents and other study-related materials, development of patient and staff educational materials, and development of patient recruitment and retention programs.

stratification- clinical trials: The FDA has been cautious in forwarding any policy on genotyping and clinical trial stratification, while at the same time trying to engage the industry in discussions on the subject. 

subject:  People being studied as part of clinical trials or other investigation, including those serving as controls.  Compare patient.    

umbrella trials (or studies): have many different treatment arms within one trial. People are assigned to a treatment arm of the trial based on their type of cancer and the specific molecular makeup of their cancer. ASCO American Society Clinical Oncology, Clinical trial Design and Methodology  Related term: basket trials

Clinical trials resources
Bandolier, Glossary,
CDISC, Glossary
CDISC, Acronym, Abbreviations and Initials, 2010 
CenterWatch Glossary, 100+ definitions Glossary of Clinical Trials Terms, National Library of Medicine, 2007. 
Cochran Collaboration, Cochran Glossary  
FDA, CDER Glossary, Drugs@FDA
FDA Clinical trials guidance documents
FDA Glossary, Independent Institute, 2003, about 60 terms 
NIH Collaboratory of Pragmatic Clinical Trials: Rethinking Clinical trials
NIH Grants Important Clinical Trial related Terms,

PharmaSchool JargonBuster,  Clinical trial terminology, about 400  terms.
WHO glossary, 

Clinical trials Conferences
BioIT World Expo
Clinical trial innovation summit
SCOPE Summit for Clinical Operations Executives

SCOPE Europe,  Sept 17-18, 2019 Barcelona Spain

Clinical Trials Barnett  Publications
Clinical Trials Barnett Core Curriculum
Clinical Trials, Barnett Training courses
Clinical Trials Barnett Web Seminars

How to look for other unfamiliar  terms

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