This document summarizes a seminar on clinical trials and their design presented by Mhaske Sujit Ramesh. It discusses the different types of clinical trial designs including observational designs like cohort studies, cross-sectional studies, and case-control studies as well as experimental designs like randomized controlled trials. It provides details on each study design including their characteristics, strengths, and limitations. The document also discusses sources of bias in clinical trials and how they can be reduced or avoided in trial design, conduct, and analysis.
Clinical Research Regulation:
Various types of Clinical Study, Phases of Clinical Trial, Clinical Trial Protocol..
Drug discovery and development together are the complete process of identifying a new drug and bringing it to market.
Discovery may involve
Screening of chemical libraries
Identification of the active ingredient from a natural remedy.
Design resulting from an understanding of the target.
Development includes 0
Studies on microorganisms and animals.
Analytical method development and validation
Clinical trials and ultimately regulatory approval.
Drug Development/Approval Process..
Types of Clinical Study: Descriptive and Analytical..
Difference between Case Control and Cohort Study
Phases of Clinical Study
Phase 0: Phase 0 includes exploratory, first-in-human (FIH) trials that are carried out in accordance with FDA regulations.
The single subtherapeutic doses are administered to 10 to 15 volunteers in phase 0 trials, which are also known as human micro dose studies. These trials provide pharmacokinetic data or aid in the imaging of certain targets without causing pharmacological effects.
Phase 1: The initial evaluation of a medicine is done in phase I studies, which use fewer healthy human participants.
Phase 1 typically involves 20 to 80 healthy volunteers who have the illness or condition. Patients are often only utilized when a drug's mechanism of action indicates that healthy individuals will not tolerate it.
Nonetheless, researchers carry out Phase 1 studies in patients with that particular form of diabetes if a new medication is recommended for use in diabetic patients.
Phase 2: The purpose of phase II studies, which involve larger patient populations (a few hundreds), is to confirm the results of phase I safety evaluations and examine the drug's efficacy.
These tests are insufficient to determine whether the medicine will be effective in treating patients.
Phase 3: Researchers plan Phase 3 studies to prove whether a product deals an action benefit to a specific people or not.
Sometimes known as pivotal studies/ pilot, these studies comprise 300 to 3,000 volunteers.
Phase 3 studies deliver most of the safety data.
Phase 4: Phase 4 trials are conducted when the drug or device has been approved by FDA.
These trials are also recognized as post-marketing surveillance involving pharmacovigilance and continuing technical support after approval.
There are numerous observational strategies and assessment patterns used in Phase 4 trials to evaluate the efficacy, cost-effectiveness, and safety of an involvement in real-world settings.
RANDOMIZED CONTROL trials
an assessment method
questions validity and applicability of many preventive and therapeutic procedures
reference Park's Preventive and social medicine
CRISPR-Cas9, a revolutionary gene-editing tool, holds immense potential to reshape medicine, agriculture, and our understanding of life. But like any powerful tool, it comes with ethical considerations.
Unveiling CRISPR: This naturally occurring bacterial defense system (crRNA & Cas9 protein) fights viruses. Scientists repurposed it for precise gene editing (correction, deletion, insertion) by targeting specific DNA sequences.
The Promise: CRISPR offers exciting possibilities:
Gene Therapy: Correcting genetic diseases like cystic fibrosis.
Agriculture: Engineering crops resistant to pests and harsh environments.
Research: Studying gene function to unlock new knowledge.
The Peril: Ethical concerns demand attention:
Off-target Effects: Unintended DNA edits can have unforeseen consequences.
Eugenics: Misusing CRISPR for designer babies raises social and ethical questions.
Equity: High costs could limit access to this potentially life-saving technology.
The Path Forward: Responsible development is crucial:
International Collaboration: Clear guidelines are needed for research and human trials.
Public Education: Open discussions ensure informed decisions about CRISPR.
Prioritize Safety and Ethics: Safety and ethical principles must be paramount.
CRISPR offers a powerful tool for a better future, but responsible development and addressing ethical concerns are essential. By prioritizing safety, fostering open dialogue, and ensuring equitable access, we can harness CRISPR's power for the benefit of all. (2998 characters)
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Clinical Research Regulation:
Various types of Clinical Study, Phases of Clinical Trial, Clinical Trial Protocol..
Drug discovery and development together are the complete process of identifying a new drug and bringing it to market.
Discovery may involve
Screening of chemical libraries
Identification of the active ingredient from a natural remedy.
Design resulting from an understanding of the target.
Development includes 0
Studies on microorganisms and animals.
Analytical method development and validation
Clinical trials and ultimately regulatory approval.
Drug Development/Approval Process..
Types of Clinical Study: Descriptive and Analytical..
Difference between Case Control and Cohort Study
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Phase 0: Phase 0 includes exploratory, first-in-human (FIH) trials that are carried out in accordance with FDA regulations.
The single subtherapeutic doses are administered to 10 to 15 volunteers in phase 0 trials, which are also known as human micro dose studies. These trials provide pharmacokinetic data or aid in the imaging of certain targets without causing pharmacological effects.
Phase 1: The initial evaluation of a medicine is done in phase I studies, which use fewer healthy human participants.
Phase 1 typically involves 20 to 80 healthy volunteers who have the illness or condition. Patients are often only utilized when a drug's mechanism of action indicates that healthy individuals will not tolerate it.
Nonetheless, researchers carry out Phase 1 studies in patients with that particular form of diabetes if a new medication is recommended for use in diabetic patients.
Phase 2: The purpose of phase II studies, which involve larger patient populations (a few hundreds), is to confirm the results of phase I safety evaluations and examine the drug's efficacy.
These tests are insufficient to determine whether the medicine will be effective in treating patients.
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Phase 3 studies deliver most of the safety data.
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There are numerous observational strategies and assessment patterns used in Phase 4 trials to evaluate the efficacy, cost-effectiveness, and safety of an involvement in real-world settings.
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The Promise: CRISPR offers exciting possibilities:
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VISION
Being proactive
Supporting optimal animal and human health
Exploring ways to reduce overall use of antimicrobials
Using the drugs that prevent and treat disease by killing microscopic organisms in a responsible way
GOAL
to prevent the generation and spread of antimicrobial resistance (AMR). Doing so will preserve the effectiveness of these drugs in animals and humans for years to come.
being to preserve human and animal health and the effectiveness of antimicrobial medications.
to implement a multidisciplinary approach in assembling a stewardship team to include an infectious disease physician, a clinical pharmacist with infectious diseases training, infection preventionist, and a close collaboration with the staff in the clinical microbiology laboratory
to prevent antimicrobial overuse, misuse and abuse.
to minimize the developme
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1. Seminar On
Clinical Trial And Its And Design
PRESENTED BY
Mhaske Sujit Ramesh
Roll No – 10
M.Pharm (Sem -02)
(Pharmacology Department)
GUIDANCE BY
Prof. Pagar Sir
M.Pharm
Pharmacology
DR. VITHALRAO VIKHE PATIL FOUNDATION’S
COLLEGE OF PHARMACY, VILAD GHAT,
AHMEDNAGAR (2022-2023)
1
2. *
1. Introduction
2. Types of clinical trials
3. Design of clinical trials
a) Observational designs
i. Cohort design
ii. Cross sectional design
iii. Case control study design
b) Experimental designs
i. RCT
3. *
*Definition given by WHO
*A clinical trial is any research study that prospectively
assigns human participants or groups of humans to one or
more health-related interventions to evaluate the effects on
health outcomes.
*Interventions include but are not restricted to drugs, cells and
other biological products, surgical procedures, radiological
procedures, devices, behavioural treatments, process-of-care
changes, preventive care, etc.
5. Phase 0 Phase I Phase II Phase III
Design
features:
• Single, ascending
dose tiers
• Unblinded
• uncontrolled
• Placebo controlled
comparisons
• active controlled
comparisons
• well-defined entry
criteria
• Randomized
• Controlled
• 2-3 treatment arms
• broader eligibility
criteria
• Uncontrolled
• observational
Duration Up to 1 month Several months Several years Ongoing (following
FDA approval)
Population: Healthy volunteers or
individuals with the
target disease (such as
cancer or HIV)
Individuals with target
disease
Individuals with
target disease
Individuals with
target disease, as
well as new age
groups, genders, etc.
Sample size: 20 to 80 200 to 300 Hundreds to
thousands
Thousands
Example: • Study of a single dose
of drug X in normal
subjects
• Double-blind study
evaluating safety
and efficacy of drug
X vs. Placebo in
patients with
hypertension
• Study of drug X vs.
Standard treatment
in hypertension
study
• Study of
economic benefit
of newly-
approved drug X
vs. Standard
treatment for
hypertension
8. *
Population
(Sample)
Exposed
(smoking)
*A population at risk for the disease events is followed over time for the
occurrence of disease and events.
*This study used to estimate how often disease or life events happen in a
certain population.
*These are the best method for determining the incidence and natural
history of a condition.
Disease
(Lung cancer)
No disease
Unexposed
Disease
(Lung cancer)
No disease
9. *
• A group of people is chosen who do not
have the outcome of interest (for
example, myocardial infarction).
• The investigator then measures a variety
of Variables that might be relevant to the
Development of the condition.
Prospective
• These use data already collected for other
purposes. The methodology is the same
but the study is performed.
• Outcome is already developed.
Retrospective
10. *
*Prospective
*Studies carried out from present time to future
*Can be tailored to collect specific exposure rate
*But long wait for events to occur
*Expensive
*Prone to high dropout rates
* Retrospective
* Look at medical events from past to present
* Information is available immediately
* Difficulty in tracing subjects and doubt on quality of recorded
information
11. *
*Is a type of observational study that are primarily used to determine
prevalence.
*Prevalence equals the number of cases in a population at a given
point in time.
*All the measurements on each person are made at one point in time.
Cohort Cross sectional
Study group Population at risk Entire population
Common measures Risks and rates Prevalence
12. *
*An observational study that compares patients who have a disease or
outcome of interest (cases) with patients who do not have the disease or
outcome (controls), and looks back retrospectively to compare how
frequently the exposure to a risk factor is present in each group
Study base
Cases
50
exposed
40
Controls
50
unexposed exposed
10 15
unexposed
35
Odds ratio
(no of exposed cases)/(no of unexposed cases)
(no of exposed control)/(no of unexposed controls)
13. *
• Efficient- saves time and energy
• Used for rare diseases, small sample sizes
• Can generate hypothesis for future study
*
• Susceptible to bias-recall, reporting
• Prone to methodological errors
• Selection of an appropriate comparison group may be difficult
14. *
*Interventional study (clinical trial) A type of clinical study in
which participants are assigned to groups that receive one or
more intervention/treatment (or no intervention) so that researchers
can evaluate the effects of the interventions on health-related
outcomes.
Interventional study
Quasi-experimental designs
True experimental designs
15. *
Also called nonrandomized interventional study design
*In non randomly assigned control group studies, at least two separate
groups are evaluated—
*One of which receives the intervention of interest and another that
serves as a control or comparison group.
That means it is used to estimate the causal impact of an intervention
on its target population without random assignment.
16. *
• One group pre-test post-test design
• Non equivalent control group design
• Interrupted time series design
17. *
* An epidemiological experiment in which subjects in a population
are randomly allocated into groups, usually called study and control
groups, to receive or not receive an experimental preventive or
therapeutic
*Patients are randomly assigned to the study all groups that help in
avoiding bias in patient
Study
population
Intervention
group
Control group
Outcome
No
outcome
Outcome
No
outcome
(Randomized Clinical Trial)
19. Randomized Controlled Clinical Trial includes Diagnostic,
Therapeutic, Prophylactic
e.g. Evaluation of nitrates in reducing cardiovascular mortality
Randomized Controlled Field Trial: It is similar to an Randomized
Controlled Clinical Trial except that the intervention is preventive
and not therapeutic.
e.g. In this, the efficacy of a preventive intervention such as a
new vaccine is tested in one study group and the other group
receives a placebo or standard
Preventive Trial: Trial of primary preventive measures
e.g. Vaccines
Risk Factor Trial: Investigator intervenes to interrupt the usual
sequence in the development of disease for those individuals who
have risk factor for developing the disease
e.g. Primary prevention of CHD using simvastatin to lower
serum cholesterol
20. *
*Any systematic deviation from true results.
*It may be any error in the design, conduct or analysis of a study that
results in distortion of truth.
Experiment
Untruth
Untruth
Truth
22. *
*Mann, C.J., 2003. Observational research methods. Research
design II: cohort, cross sectional, and case-control
studies. Emergency medicine journal, 20(1), pp.54-60.
*Grimes, D.A. and Schulz, K.F., 2002. Bias and causal
associations in observational research. The Lancet, 359(9302),
pp.248-252.
*www.who.int/topics/clinical_trials/en
