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Clinical trials glossary & taxonomy
Evolving Terminology for Emerging Technologies
Comments? Questions? Revisions?  Mary Chitty 
mchitty@healthtech.com
Last revised September 10, 2014
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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 http://blogs.hbr.org/2013/10/if-everyone-hates-the-fda-approval-process/  

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  

Bayesian clinical trials: Clinical  Informatics

clinical genomics -  clinical trials impact: Clinical genomics is the application of large-scale, high-throughput genomics technologies in clinical settings, such as clinical trials or primary care of patients. Clinical genomics promises to allow a molecular understanding of disease and drug response, with benefits in all areas of medicine. Contributing to the growth of genomics, in 2005 the FDA issued guidelines for applications of genomics in drug development, with the stated hope that genomics will improve the safety and effectiveness of medicines. Given this mandate, clinical genomics applications appear to have crossed a threshold with the recent approval of several clinical genomics products.  Insight Pharma Reports, Impact of Genomics on Clinical Trials, 2006 http://www.insightpharmareports.com/reports/2006/61_Clinical_Genomics/overview.asp    

clinical trials: The current clinical evaluation process is fraught with inefficiencies, resulting in numerous compound failures and exploding development costs. Until recently, the industry has reacted to the clinical evaluation problem essentially by "streamlining" the existing processes and by introducing information technology in a cautious and evolutionary fashion. While the FDA’s Critical Path Initiative of 2004 showed that the agency is willing to take the lead in working with representatives from industry and academia towards a remedy, this report suggests that a more radical solution is needed. Insight Pharma Reports, Clinical Trial in 2015: A new paradigm for clinical development, 2006 http://www.insightpharmareports.com/reports/2005/59_Clinical_Trials/overview.asp   

The NIH defines a clinical trial as a prospective biomedical or behavioral research study of human subjects that is designed to answer specific questions about biomedical or behavioral interventions (drugs, treatments, devices, or new ways of using known drugs, treatments, or devices). Clinical trials are used to determine whether new biomedical or behavioral interventions are safe, efficacious, and effective. Behavioral human subjects research involving an intervention to modify behavior (diet, physical activity, cognitive therapy, etc.) fits this definition of a clinical trial. Human subjects research to develop or evaluate clinical laboratory tests (e.g. imaging or molecular diagnostic tests) might be considered to be a clinical trial if the test will be used for medical decision making for the subject or the test itself imposes more than minimal risk for subjects. Biomedical clinical trials of experimental drug, treatment, device or behavioral intervention may proceed through four phases: See also Phase I, Phase II, Phase III, Phase IV , Phase Zero

CenterWatch  http://www.centerwatch.com/  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
, cancer.gov, National Cancer Institute, NIH
http://www.nci.nih.gov/clinical_trials/
IBM Life Sciences, Pharmaceutical Clinical Development, 2002 http://www-935.ibm.com/services/uk/index.wss/summary/igs/a1008011?cntxt=a1006867   
Related terms: CRO Clinical Research Organization, clinical informatics, comparative data mining, FDA drug approvals, Phase I, Phase II, Phase III, Phase IV/ postmarketing surveillance, preclinical, predictive data mining  Narrower terms: adaptive clinical trials, computer trial simulations, explanatory trials, management trials, randomized clinical trials  
clinical trials - Europe:
A vast new opportunity for clinical trials has emerged in Europe as a result of the collapse of the Soviet Union and events during the last decade of the 20th century. The result is the emergence of dozens of sovereign countries and gone is the political dividing line between East and West Europe. Now these countries join the countries of Western Europe as well as the CIS to offer a spectrum of clinical trial options. Among these are: Treatment-naïve populations, Ease of patient recruiting, Superb trial administration, Lower costs, Applicable EMEA standards or equivalents. Insight Pharma Reports, Conducting Clinical Trials in Europe: An insider's analysis, 2008

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. An overview of drug development, Barnett/Parexel, 2000 http://www.barnettinternational.com/EducationalServices_Publication.aspx?p=6464&id=97141 

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

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

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 http://www.bmj.com/cgi/content/full/316/7127/285  Compare pragmatic trials   

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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.  

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

patient recruitment: Patient recruitment and retention are critical to drug development programs. Patient recruitment, if not adequately planned for, can extend your development timeline. 

Patient Reported Outcomes Consortium: The Critical Path Institute (C-Path), in cooperation with the U.S. Food and Drug Administration (FDA) and the medical products industry, has formed the Patient-Reported Outcomes Consortium for the purpose of developing, evaluating, and qualifying PRO instruments with the FDA for use in clinical trials designed to evaluate the safety and effectiveness of medical products. http://www.c-path.org/PRO.cfm   

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 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 http://books.google.com/books?id=HXMUEjZ4vyAC&pg=PA14&lpg=PA14&dq=Phase+IIIb+clinical+trials+FDA+DOSING+OR+COMPASSIOANTE&source=bl&ots=Y2P9rfVklb&sig=Idowe-Y

phase II risk reduction: See Chemistry glossary  backup compounds

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 http://en.wikipedia.org/wiki/Clinical_trial#Phase_III

Phase IV/ postmarketing surveillance: At this stage, after a drug has been launched, pharmaceutical companies may conduct further studies of its performance, often examining long- term safety.  See also Regulatory Affairs Phase Zero, also known as microdosing

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 

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 http://www.bmj.com/cgi/content/full/316/7127/285   Compare explanatory trials

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. It is the patient's choice to be in a randomized trial. National Cancer Institute  Dictionary of Cancer Terms http://www.cancer.gov/Templates/db_alpha.aspx?CdrID=45858 

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  http://www.ncbi.nlm.nih.gov/pubmed/19604080

risk ratio: The ratio of risk in the treated group (EER) to the risk in the control group (CER). This is used in randomised trials and cohort studies and is calculated as EER/CER.  [Glossary of EBM Evidence Based Medicine] Terms, Centre for Evidence Based Medicine, Mt. Sinai Hospital, 2000 http://www.cebm.utoronto.ca/glossary/  

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Single Controlled Trial SCT: Since 1998, the FDA has allowed drug developers to use what is known as the single controlled trial (SCT) to support their drug approval applications. The SCT provision allows applicants to prove the effectiveness of new drugs by submitting data from only one controlled clinical study instead of multiple studies.  

site management organization: Wikipedia http://en.wikipedia.org/wiki/Site_management_organization  

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. 

streamlined clinical trials :  A Parkinson's research and treatment center and the genetic testing company 23andMe, both in California's Silicon Valley, are experimenting with an unusual new approach to clinical trials: have participants assess themselves from their home computers potentially using everything from videos of tremors to a mouse that senses motor abilities. If it works—still a big if—the strategy could greatly reduce the need for doctors' visits and make trial participation vastly cheaper and possible from anywhere in the world. Researchers have long considered how the Web might enhance clinical trials, in particular its ability to aggregate data from the thousands of people needed for studies tracking genetic and environmental factors linked to common diseases. But the new alliance comes with many uncertainties. Will there be a bias in who completes the assessment? Can the approach capture subtle changes in disease symptoms? Will participants understand what they're signing up for, and how will the data they supply be used? Streamlined Clinical Trials from a Home Computer Science 30 May 2008: Vol. 320 no. 5880 p. 1143
DOI: 10.1126/science.320.5880.1143
http://www.sciencemag.org/content/320/5880/1143.full 

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

women and minorities and research:  Inclusion of Women and Minorities As Participants In Research Involving Human Subjects - Policy Implementation Page, NIH, Office of External Research, 2007  http://grants1.nih.gov/grants/funding/women_min/women_min.htm

Bibliography
Bandolier, Glossary of Diagnostic Terms,  2004, about 150 terms http://www.jr2.ox.ac.uk/bandolier/glossary.html  [URL change?]
CDISC, Glossary Terms Version 7.0 http://www.cdisc.org/search/index.php?findtext=glossary  [download]
CDISC, Acronym, Abbreviations and Initials, 2010 http://www.cdisc.org/stuff/contentmgr/files/0/08a36984bc61034baed3b019f3a87139/misc/act1210_049_058_acronyms.pdf 
CenterWatch Glossary, 100+ definitions
http://www.centerwatch.com/patient/glossary.html
ClinicalTrials.gov Glossary of Clinical Trials Terms, National Library of Medicine, 2007. http://www.clinicaltrials.gov/ct/info/glossary 
Cochran Collaboration, Cochran Glossary 30+ definitions http://www2.cochrane.org/resources/handbook/glossary.pdf 

FDA, CDER Glossary, Drugs@FDA,  2004, 30+ definitions. http://www.fda.gov/cder/drugsatfda/glossary.htm
FDA, Innovation or stagnation: Challenge and opportunity on the critical path to new medical products, 2004 http://www.fda.gov/oc/initiatives/criticalpath/whitepaper.html
FDA Review.org Glossary, Independent Institute, 2003, about 60 terms http://www.fdareview.org/glossary.shtml 
IRB Institutional Review Board Glossary http://www.hhs.gov/ ohrp/archive/irb/irb_glossary. htm Mt. Sinai Hospital, Glossary of EBM [Evidence Based Medicine] Terms, Centre for Evidence Based Medicine,  2000
http://www.cebm.utoronto.ca/glossary/
Novartis Clinical Trials Glossary, Novartis E Trials  http://www.novartisclinicaltrials.com/etrials/clinicalTrialGlossary.do About 80 terms.
PharmaSchool JargonBuster, http://pharmaschool.co/jargon02.asp?name=  Clinical trial terminology, about 400  terms.
Roche, Key Clinical Trial Terms: Glossary of Terms and Abbreviations, 2009
http://www.rochetrials.com/glossary.action 
Tufts Center for the Study of Drug Development, Glossary of Terms, 20+ terms.
http://csdd.tufts.edu/InfoServices/Glossary.asp  
WHO glossary, 2007, 28 definitions
http://www.who.int/ictrp/glossary/en/index.htm 

Clinical trials Conferences http://www.chicorporate.com/Conferences/Search.aspx?k=&r=&s=CTL
BioIT World Expo http://www.bio-itworldexpo.com/
SCOPE Summit for Clinical Operations Executives http://www.scopesummit.com/

Clinical trials CDs, DVDs  http://www.chicorporate.com/Conferences/CompactDiscs.aspx?s=CTL
Clinical Trials Short courses http://www.healthtech.com/Conferences_Upcoming_ShortCourses.aspx?s=CTL

Insight Pharma Reports Clinical trials series http://www.insightpharmareports.com/Clinical-Trials-Reports/


Clinical Trials Barnett books   http://www.barnettinternational.com/EducationalServices/Publications.aspx  
Clinical Trials Barnett Live Seminars http://www.barnettinternational.com/EducationalServices/Seminars.aspx
Clinical Trials Barnett Web Seminars http://www.barnettinternational.com/EducationalServices/Webinars.aspx

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