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Clinical trials glossary & taxonomy
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
https://grants.nih.gov/grants/policy/faq_clinical_trial_definition.htm#5219
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
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
Clinical biomarkers strategy and innovation:
February
19-20, 2020
Orlando, FL
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. Ramoni, Nature
Reviews Drug Discovery 6, 107-108 (February 2007) | doi:10.1038/nrd2246 http://www.nature.com/nrd/journal/v6/n2/full/nrd2246.html
Clinical Supply Management
February 19-20, 2020
Orlando,
FL
|
Clinical Technology and
Innovation
February 20-21, 2020
Orlando, FL
|
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.https://grants.nih.gov/policy/clinical-trials/definition.htm
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
http://rethinkingclinicaltrials.org/resources/ehr-phenotyping/#intro-definitions
CONSORT
Consolidated Standards of Reporting Trials: http://www.consort-statement.org/
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
https://en.wikipedia.org/wiki/Consolidated_Standards_of_Reporting_Trials
Clinical trials
are increasingly being run by outsourcing to these groups.
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.
https://www.clinicaltrialsummit.com/data-technology
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.
https://www.ncbi.nlm.nih.gov/books/NBK44024/
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
https://www.scopesummit.com/Patient-Recruitment/
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
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. https://www.scopesummit.com/Forecasting-Budgeting/
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
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 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.
http://rethinkingclinicaltrials.org/about-nih-collaboratory/
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. https://www.scopesummit.com/Outsourced-Trials/
patient:
People
with a specific disease or condition, particularly those being treated
by a health professional.
Patient Engagement, Enrollment and Retention through Communities and
Technology
February 20-21, 2020
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 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
http://rethinkingclinicaltrials.org/cores-and-working-groups/patient-reported-outcomes-2/
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
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.
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
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3148611/
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.
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.
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
http://rethinkingclinicaltrials.org/
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
Protocol Development, Global Site Selection, Feasibility and Site
Management
February 19-20, 2019
Real World Data Leveraging for Clinical and
Observational Research
February 20-21, 2020
Orlando, FL
Resource Management and Capacity Planning for Clinical Trials
February
20-21, 2020
Orlando, FL
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.
https://www.scopesummiteurope.com/risk-based-monitoring/
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
http://en.wikipedia.org/wiki/Site_management_organization
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.
https://www.clinicaltrialsummit.com/site-selection
Site-Study Activation and Performance Improving
February
20-21, 2020
Orlando, FL
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.
Clinical trials resources
Clinical
trials Conferences
http://www.healthtech.com/conferences/upcoming.aspx?s=CTL
Evolving Terminology for Emerging Technologies
Comments? Questions? Revisions?
Mary Chitty MSLS
mchitty@healthtech.com
Last revised
January 09, 2020
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
Informatics
Algorithms
Clinical
informatics
Research
Summit for Clinical Ops Executives
(SCOPE)
February
18-21, 2020 • Orlando, FL
https://www.scopesummit.com/
SCOPE Europe,
17-18 October, 2019 Barcelona, Spain
https://www.scopesummiteurope.com/#
Artificial Intelligence in Clinical Research
February 20-21, 2020
Orlando, FL |
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.
FAQ NIH Clinical trial definition
https://grants.nih.gov/grants/policy/faq_clinical_trial_definition.htm
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
https://www.cancer.gov/about-cancer/treatment/clinical-trials/search
developmental
site agreements:
Companies
work with hospitals to develop instrumentation and clinical
research
applications.
Outsourcing Strategy Mastering
February
19-20, 2020
Orlando, FL
|
https://www.scopesummit.com/Outsourcing-Strategy/
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.
https://c-path.org/programs/pro/
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
Related terms: disease resistant individuals, lure of initial value,
placebo responders
Terminology related to the Revised Common Rule
https://www.hhs.gov/ohrp/regulations-and-policy/regulations/terminology/index.html
https://www.cancer.gov/publications/dictionaries/cancer-terms/def/randomized-clinical-trial
https://www.scopesummit.com/Resource-Management-Capacity-Planning/
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.
https://www.scopesummit.com/QbD-Risk/
Part
2 February
20-21 2020 Orlando FL
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
https://www.cancer.gov/publications/dictionaries/cancer-terms/def/risk-ratio
Bandolier,
Glossary,
http://www.bandolier.org.uk/glossary.html
CDISC, Glossary
https://www.cdisc.org/standards/glossary
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
http://www.cochrane.org/glossary/5
FDA,
CDER Glossary,
Drugs@FDA, https://www.fda.gov/animal-veterinary/guidance-industry/chemistry-manufacturing-and-controls-cmc-guidances-industry-gfis
FDA Clinical trials guidance documents
https://www.fda.gov/RegulatoryInformation/Guidances/ucm122046.htm
FDA
Review.org Glossary, Independent Institute, 2003, about 60 terms
http://www.fdareview.org/glossary.shtml
NIH Collaboratory of
Pragmatic Clinical Trials: Rethinking Clinical trials
http://rethinkingclinicaltrials.org/
NIH Grants Important Clinical Trial
related Terms,
https://grants.nih.gov/policy/clinical-trials/glossary-ct.htm
PharmaSchool JargonBuster,
http://pharmaschool.co/jargon02.asp?name=
Clinical trial terminology, about 400 terms.
WHO glossary,
http://www.who.int/ictrp/glossary/en/index.htm
BioIT World Expo
https://www.bio-itworldexpo.com/
Clinical trial innovation
summit
https://www.clinicaltrialsummit.com/
SCOPE Summit for Clinical Operations Executives
https://www.scopesummit.com/
SCOPE Europe,
Sept 17-18, 2019 Barcelona Spain
https://www.scopesummiteurope.com/#
Clinical Trials Barnett Publications
https://www.barnettinternational.com/EducationalServices/Publications.aspx
Clinical Trials Barnett Core Curriculum
https://www.barnettinternational.com/EducationalServices/Seminars.aspx
Clinical Trials, Barnett Training courses
https://www.barnettinternational.com/training-courses/
Clinical Trials Barnett Web Seminars
https://www.barnettinternational.com/EducationalServices/Webinars.aspx
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to look for other unfamiliar terms
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