Download Clinical Trial

Regulatory
Clinical Trials
Clinical Trials
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Definition: research studies to find ways to
improve health
Each trial tries to answer scientific questions
Types of trials
– Treatment trials: test new treatments (new
drug)
– Prevention trials: test new approaches to
prevent illness (vaccines)
– Screening trials: test new methods (new test
method)
– Quality of Life trials: (supportive care) test for
ways to improve comfort and quality of life
Pre Clinical Trials
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Performed on animals
– Try to use animal model which most
closely relates to human subject
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i.e. pigs may be used to study effects on skin
Performed to predict potential toxic
effects on humans
– Minimize the risk of harm during clinical
trial
Clinical Investigation
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Definition: any experiment that
involves a test article and one or more
human subjects and that is either
subject to requirements for submission
to FDA or is not subject for submission
but results are intended to be
submitted later.
Clinical Trial: why do it?
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To understand if the drug or device
does what it claims to do (efficacy)
To ensure safety to public
To ensure safety to participants
Clinical Trial: Who
oversees?
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FDA establishes guidelines for clinical
trials
– 21CFR50: Title 21 Section 50 of Code of
Federal Regulations
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Regulatory Department
– Within any organization this group
oversees the actual clinical trial
Why did the FDA need to
establish guidelines?
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To ensure patients knew they were
participating
– Informed consent: was not always
obtained
– HIPAA came on board to protect the patient’s
privacy
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To ensure guidelines were in place for
controlling the drug which was being
investigated
– Accountability; prevent abuses of drugs and
devices
Informed Consent
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Written document
Outlines the following:
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Purpose and method of the study
Alternative methods of treatment
Benefits and Risks of participation
Right to refuse or withdraw from study
Does not include the right to see the results
of the study
International Guidelines
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ICH: International Conference on
Harmonization….
– Regulatory authorities in Europe, Japan,
and US got together to establish
guidelines for clinical trials and product
registration
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Declaration of Helsinki
– World Medical Association
– 1st adopted in 1964
Phases of Clinical Trials
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Phase 1: small number of patients, (10-20)
– Can take 9-18 months to complete
– May continue with animal studies for long term data
Phase 2: continues to test safety and evaluates how
well the new drug is working. More patients enrolled
(100-300)
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– May take 1-2 years to complete
Phase 3: most extensive study,1-3 years
– Fully assess safety and effectiveness of drug
– 100-1000 patients involved, diverse group, multiple
sites
– May compare old and new drug treatments
– Evaluating side effects
Phase 4: long term side effects and effectiveness
Participants in clinical
trials
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Investigator
Sponsor
Sponsor Investigator
Human Subject
Institution
Institution Review Board
Investigator
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Individual who actually conducts the
clinical investigation or is the
leader of the team conducting the trial
Main person responsible for the
safety of the human subject
participating in the study
– however, all healthcare workers are
responsible for protecting the human
subjects
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Nurse, investigator, IRB, etc.
Sponsor and Sponsor
Investigator
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Sponsor: person who initiates a
clinical investigation, but does not
actually conduct the investigation
Sponsor Investigator: person who both
initiates AND actually conducts the
clinical investigation
Human Subject and
Institution
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Human participating in the clinical
investigation
Institution: any public or private entity
or facility. Typically the facility where
the clinical investigation is being
conducted
Institutional Review
Board
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Definition: any board, committee or
group formally designated by an
institution to review biomedical
research involving human subjects
IRB approval MUST be
obtained before an
investigator can begin a study
Institutional Review
Board
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Usually comprised of medical and lay
persons (at least 5 members, no conflict of interest)
Established to protect the human
subjects and ensure fair enrollment
Review and Approve Informed
Consent
Conduct periodic reviews to ensure
integrity of the study
When is IRB not
required?
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Studies for cosmetics
Studies for taste of foods
Studies for veterinary use
New devices (drugs, reagents or
hardware such as pacemakers,
artificial joints) require IRB
Clinical Trial Data
Analysis
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Data analyzed by Statistics or
Biostatistics group
Use well defined, accepted and predetermined data analysis formulas and
methods.
– Looking for levels of significance 99% still
means 1% error possible of 1/100 (0.01).
Is that good enough?
 How do you balance the data with the
delay in releasing a product?
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Outlier: data point outside of the
average range and is usually larger or
smaller than the rest of the points
Clinical Trial Data
Analysis
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INCLUSION/EXCLUSION CRITERIA:
The medical or social standards determining
whether a person may or may not be
allowed to enter a clinical trial. These
criteria are based on such factors as age,
gender, the type and stage of a disease,
previous treatment history, and other
medical conditions. It is important to note
that inclusion and exclusion criteria are not
used to reject people personally, but rather
to identify appropriate participants and keep
them safe.
Single and Double Blinded
Study
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Single-Blind study: clinical trial
study in which the human subjects
do not know if they are receiving
the active product or a placebo.
Double-Blind study: study in which
neither the subject nor the
investigator knows the treatment
assigned. (usually subjects, investigators,
monitors and data analysts are unaware)