The document discusses different types of experimental study designs used to evaluate medical treatments, including randomized controlled trials, non-controlled experimental studies, and controlled experimental studies using parallel, crossover, and time series (before-after) designs. It explains key aspects of each design such as use of control groups, randomization, blinding, advantages, and disadvantages. An example is provided for each type of design to illustrate its application.
The document discusses clinical trials and their various phases. It begins by defining a clinical trial and noting they systematically study new drugs in human subjects to determine safety and efficacy. It then describes the various phases of clinical trials - phase I examines safety and dosing in small groups, phase II assesses efficacy and further evaluates safety in larger groups, and phase III tests effectiveness compared to standard treatments in large patient populations. The document provides details on trial designs, methods to reduce bias, and goals and considerations of each phase of clinical drug testing.
This document provides an overview of clinical trials. It defines a clinical trial and explains that they are conducted under controlled conditions to evaluate potential therapies. It describes the different phases of clinical trials from early safety testing to post-marketing studies. Key aspects of clinical trial design are discussed, including randomization, blinding, controls and study populations. Reasons for terminating a trial early are also mentioned.
The document discusses randomized controlled trials (RCTs), which are considered the gold standard for evaluating causal relationships. It describes key aspects of RCTs such as randomization methods, blinding, allocation concealment, study populations, interventions, follow-up, and outcome assessment. RCTs follow a strict protocol and involve randomly allocating participants into study and control groups to receive different interventions/exposures. The results are then compared to determine the effectiveness of the new treatment or exposure being tested.
Clinical trials follow a phased process to evaluate new treatments. Phase I trials test safety in small groups. Phase II trials assess efficacy in larger groups. Phase III trials compare new treatments head-to-head with standard treatments in large randomized controlled trials. Higher levels of evidence come from systematic reviews and meta-analyses of multiple randomized controlled trials, while lower levels of evidence derive from expert opinions and single descriptive studies.
Get Research Paper Assignment help sample solution by Phd level experts for Free. contact us 24/7 Live chat, free downloadable solution.
http://www.helpwithassignment.com/admin/filemanager/downloads/Research%20Paper%20Critique%20-%20sample.pdf
The document discusses key aspects of writing a study protocol, including:
- Developing the introduction, research question, objectives and hypothesis.
- Detailing the methodology, including study type, sample size calculation, inclusion/exclusion criteria, randomization process, and statistical analysis plan.
- Common statistical tests for normal and non-normal data distribution are described.
- Key concepts in research design are explained, such as the null hypothesis, randomized clinical trials, longitudinal and cross-sectional studies, and calculating appropriate sample sizes.
Clinical trials are conducted to test new drugs, treatments or medical devices in humans to assess their safety and efficacy. There are four main phases of clinical trials:
Phase I trials involve small groups of people to determine basic safety and dosing requirements. Phase II trials expand the testing to more people to determine efficacy and further evaluate safety. Phase III trials involve large groups of people to confirm effectiveness, monitor side effects, compare to commonly used treatments and collect information to allow safe use of the intervention. Phase IV trials occur after the intervention has been marketed to gather information on effects in various populations and any long-term side effects.
Assignment BriefExcelsior College PBH 321 .docxssuser562afc1
Assignment Brief
Excelsior College PBH 321
Page 1
PRO MISING NEW D RUG FOR ARTH RITIS P AIN – OR SAME OLD STORY?
Use this commentary as an example for how you are expected to respond to questions critiquing the article.
Side notes from the instructor to you, which are simply informational, are in [* ].
Q: What were the study’s rationale/hypothesis and objectives?
This study aimed to test the safety, efficacy, and possible side effects of a new drug for treatment of
osteoarthritis of the knee. Given the limited existing treatment options for the condition, and a small Phase 1
trial which suggested possible benefits of tanezumab, the investigators aimed to compare treatment of
arthritis pain with different injected doses of the drug to treatment with a placebo injection.
Q: What were the assigned treatment and control groups, and how were they defined?
Individuals were randomly allocated to treatment with either 10, 25, 50, 100 or 200 ug of tanezumab
via injection, or a placebo (control group), also injected.
Q: Was there any way that the blinding of treatment status could have been compromised (in other words,
if the investigator somehow figures out treatment status)? If so, what might have been its potential impact?
The pharmacist preparing the dosage (either the drug or placebo) was aware of the subject’s
randomization to treatment or placebo. It is unlikely the pharmacist would have (accidentally or purposefully)
changed someone’s treatment from their randomly assigned one, and since the pharmacist presumably played
no part in the analysis of study data, their awareness of treatment status did not likely affect the study’s
findings. Similarly, the statistician’s knowledge would not be expected to have an impact, barring unethical
research practices from this individual. An opportunity for un-blinding may have been presented if individuals
assigned to placebo experienced significantly more knee pain, or if individuals assigned to tanezumab
experienced many side effects – but this is really only likely to occur when the investigator ascertains the
outcome directly (such as by interviewing or examining the patient). Because participants self-reported all of
their outcome information (i.e., instead of being directly assessed by a physician) throughout the study, and
this information was summarized later by the investigators, compromise of the investigator’s blinding to
subject treatment status was unlikely and probably did not constitute a major source of bias in this study.
Excelsior College PBH 321
Page 2
Q: What was the outcome of interest for this study? How was it measured? Do you see any problems with
the way the outcome of interest was measured? If so, suggest some alternatives.
Pain while walking and overall knee pain was recorded by the patient in a daily diary. The patient’s
overall assessment of their pain during the trial (called “global ass ...
The document discusses clinical trials and their various phases. It begins by defining a clinical trial and noting they systematically study new drugs in human subjects to determine safety and efficacy. It then describes the various phases of clinical trials - phase I examines safety and dosing in small groups, phase II assesses efficacy and further evaluates safety in larger groups, and phase III tests effectiveness compared to standard treatments in large patient populations. The document provides details on trial designs, methods to reduce bias, and goals and considerations of each phase of clinical drug testing.
This document provides an overview of clinical trials. It defines a clinical trial and explains that they are conducted under controlled conditions to evaluate potential therapies. It describes the different phases of clinical trials from early safety testing to post-marketing studies. Key aspects of clinical trial design are discussed, including randomization, blinding, controls and study populations. Reasons for terminating a trial early are also mentioned.
The document discusses randomized controlled trials (RCTs), which are considered the gold standard for evaluating causal relationships. It describes key aspects of RCTs such as randomization methods, blinding, allocation concealment, study populations, interventions, follow-up, and outcome assessment. RCTs follow a strict protocol and involve randomly allocating participants into study and control groups to receive different interventions/exposures. The results are then compared to determine the effectiveness of the new treatment or exposure being tested.
Clinical trials follow a phased process to evaluate new treatments. Phase I trials test safety in small groups. Phase II trials assess efficacy in larger groups. Phase III trials compare new treatments head-to-head with standard treatments in large randomized controlled trials. Higher levels of evidence come from systematic reviews and meta-analyses of multiple randomized controlled trials, while lower levels of evidence derive from expert opinions and single descriptive studies.
Get Research Paper Assignment help sample solution by Phd level experts for Free. contact us 24/7 Live chat, free downloadable solution.
http://www.helpwithassignment.com/admin/filemanager/downloads/Research%20Paper%20Critique%20-%20sample.pdf
The document discusses key aspects of writing a study protocol, including:
- Developing the introduction, research question, objectives and hypothesis.
- Detailing the methodology, including study type, sample size calculation, inclusion/exclusion criteria, randomization process, and statistical analysis plan.
- Common statistical tests for normal and non-normal data distribution are described.
- Key concepts in research design are explained, such as the null hypothesis, randomized clinical trials, longitudinal and cross-sectional studies, and calculating appropriate sample sizes.
Clinical trials are conducted to test new drugs, treatments or medical devices in humans to assess their safety and efficacy. There are four main phases of clinical trials:
Phase I trials involve small groups of people to determine basic safety and dosing requirements. Phase II trials expand the testing to more people to determine efficacy and further evaluate safety. Phase III trials involve large groups of people to confirm effectiveness, monitor side effects, compare to commonly used treatments and collect information to allow safe use of the intervention. Phase IV trials occur after the intervention has been marketed to gather information on effects in various populations and any long-term side effects.
Assignment BriefExcelsior College PBH 321 .docxssuser562afc1
Assignment Brief
Excelsior College PBH 321
Page 1
PRO MISING NEW D RUG FOR ARTH RITIS P AIN – OR SAME OLD STORY?
Use this commentary as an example for how you are expected to respond to questions critiquing the article.
Side notes from the instructor to you, which are simply informational, are in [* ].
Q: What were the study’s rationale/hypothesis and objectives?
This study aimed to test the safety, efficacy, and possible side effects of a new drug for treatment of
osteoarthritis of the knee. Given the limited existing treatment options for the condition, and a small Phase 1
trial which suggested possible benefits of tanezumab, the investigators aimed to compare treatment of
arthritis pain with different injected doses of the drug to treatment with a placebo injection.
Q: What were the assigned treatment and control groups, and how were they defined?
Individuals were randomly allocated to treatment with either 10, 25, 50, 100 or 200 ug of tanezumab
via injection, or a placebo (control group), also injected.
Q: Was there any way that the blinding of treatment status could have been compromised (in other words,
if the investigator somehow figures out treatment status)? If so, what might have been its potential impact?
The pharmacist preparing the dosage (either the drug or placebo) was aware of the subject’s
randomization to treatment or placebo. It is unlikely the pharmacist would have (accidentally or purposefully)
changed someone’s treatment from their randomly assigned one, and since the pharmacist presumably played
no part in the analysis of study data, their awareness of treatment status did not likely affect the study’s
findings. Similarly, the statistician’s knowledge would not be expected to have an impact, barring unethical
research practices from this individual. An opportunity for un-blinding may have been presented if individuals
assigned to placebo experienced significantly more knee pain, or if individuals assigned to tanezumab
experienced many side effects – but this is really only likely to occur when the investigator ascertains the
outcome directly (such as by interviewing or examining the patient). Because participants self-reported all of
their outcome information (i.e., instead of being directly assessed by a physician) throughout the study, and
this information was summarized later by the investigators, compromise of the investigator’s blinding to
subject treatment status was unlikely and probably did not constitute a major source of bias in this study.
Excelsior College PBH 321
Page 2
Q: What was the outcome of interest for this study? How was it measured? Do you see any problems with
the way the outcome of interest was measured? If so, suggest some alternatives.
Pain while walking and overall knee pain was recorded by the patient in a daily diary. The patient’s
overall assessment of their pain during the trial (called “global ass ...
The document discusses various clinical trial designs, including parallel, crossover, dose-response, factorial, non-inferiority, and sequential parallel trials. It covers factors to consider when choosing a design like the questions being addressed and disease characteristics. Examples are provided for each design type to illustrate how they can be applied to evaluate different research questions. Issues related to active control and non-inferiority trials are also discussed.
Randomized controlled trials (RCTs) provide the highest level of evidence in clinical research. RCTs involve randomly assigning subjects to experimental and control groups to test clinical interventions. Key aspects of RCTs include formulating a research question, randomization to eliminate confounding factors, blinding of subjects and researchers, monitoring outcomes in both groups, and presenting results including relative risk, efficacy and number needed to treat. Common RCT designs are parallel, cross-over and factorial designs. RCTs aim to discover safety and efficacy of clinical interventions.
The document discusses different types of research designs, including explanatory trials which aim to study efficacy under ideal conditions, pragmatic trials which aim to study effectiveness in regular clinical practice, and cost benefit studies which aim to study efficiency in relation to resources consumed. It also discusses analytical studies, experimental trials using control groups, randomized controlled trials including group comparative and cross-over designs, non-randomized controlled trials, uncontrolled clinical trials, cohort studies, cross-sectional studies, and case control studies. The key aspects of each design such as whether they are prospective or retrospective and examples are provided.
Excelsior College PBH 321 Page 1 EXPERI MENTAL E.docxgitagrimston
Excelsior College PBH 321
Page 1
EXPERI MENTAL E PIDE MIOLOGICAL STUDIE S
Epidemiologic studies are either observational or experimental. Observational studies, including ecologic,
cross-sectional, cohort, and case-control designs, are considered “natural” experiments, but experimental
studies are considered true experiments. We will spend the next 2 modules discussing these designs.
Before we begin to discuss study designs, we need a brief introduction to a concept that we will spend more
time discussing in later modules -- bias. The definition of bias is:
“Deviation of results or inferences from the truth, or processes leading to such deviation. Any trend in the
collection, analysis, interpretation, publication, or review of data that can lead to conclusions that are
systematically different from the truth.” (Last, J.M., A Dictionary of Epidemiology, 4th ed.)
Epidemiologists are naturally concerned whether the results of an epidemiologic study are biased, since many
important public health decisions are often drawn from epidemiologic research. The severity of the bias, that
is - how much it influences or distorts the results, is related to the study design as well as how information is
analyzed.
Experimental Studies
The defining feature of experimental studies is that the investigator assigns exposure to the study subjects.
Experimental studies most closely resemble controlled laboratory experiments and serve as models for the
conduct of observational studies, thus they are the “gold standard” of epidemiologic research. Experimental
studies have high validity (i.e., less bias), and can identify even very small effects. The most well known type of
experimental study is a randomized trial (sometimes referred to as a randomized controlled trial), where the
investigator randomly assigns exposure to the study subjects. In this type of study, the only expected
difference between the experimental and control groups is the outcome variable being studied.
Experimental designs like the randomized trial can assess both preventive interventions, where a prophylactic
agent is given to healthy or high-risk individual to prevent disease, or can assess effects of therapeutic
treatment, such as those given to diseased individuals to reduce their risk of disease recurrence, or to improve
their survival or quality of life.
Preventive intervention: Does tamoxifen lower the incidence of breast cancer in women with high risk profile
compared to high risk women not given tamoxifen?
Therapeutic intervention: Do combinations of two or three antiretroviral drugs prolong survival of AIDS
patients as well as regimens of single drugs?
The investigator can assign exposures (or allocate interventions) to either individuals or to an entire
community.
Individual-level assignment: Do women with stage I breast cancer given a lumpectomy alone survive as long
without recurrence of disease as women given a lumpec ...
This document summarizes the different types of clinical studies, including clinical trials, cohort studies, and case control studies. It then provides detailed descriptions of clinical trials, including phases of clinical trials from pre-clinical animal studies to post-marketing surveillance. Clinical trials aim to evaluate safety and efficacy of new drugs and are conducted in a phased manner from small healthy volunteer studies to large multicenter studies in patients. Rigorous ethical and scientific standards are followed to ensure safety and quality of clinical research.
This document provides an overview of clinical research and clinical trials. It defines clinical research and clinical trials, discusses the importance of research. It describes the different types and phases of clinical trials, from phase 0 to phase IV. It outlines the key players involved in clinical trials and provides an overview of the clinical trial process from study design to statistical analysis and reporting.
This document discusses different types of study designs used in clinical research. It describes experimental designs like randomized clinical trials (RCTs) which are considered the highest level of evidence. It also covers observational study designs including cohort studies, case-control studies, cross-sectional studies, case series, and case reports. For each design it provides details on what it is, strengths, weaknesses and examples. It concludes with a brief overview of meta-analyses which systematically review and combine results from multiple studies.
Types of clinical trials designs were discussed including parallel designs, crossover designs, factorial designs, cluster designs, and adaptive designs. The key factors in choosing a clinical trial design are treatment duration and chronology of events, trial cost, and subject convenience. Commonly used designs include parallel, crossover, factorial, and equivalence/non-inferiority designs. The randomized, double-blind, placebo-controlled, parallel design is often considered the best to determine efficacy. Different designs can answer different therapeutic questions.
This document describes a doubly randomized delayed-start design for clinical trials. The design consists of two periods. In the first period, patients are randomized to receive either a new drug or placebo. In the second period, patients who received placebo in the first period and meet certain enrichment criteria can be rerandomized to receive either the new drug or continue on placebo. The design aims to reduce bias from high placebo responses and more efficiently study maintenance effects. It is naturally adaptive as aspects of the second period can be modified based on interim analysis of the first period. Efficacy data from both periods are combined for the overall analysis. This design offers greater efficiency for clinical development compared to traditional parallel designs.
This document discusses uncontrolled clinical trials, which evaluate new medical treatments without a control group for comparison. Uncontrolled trials have limitations since they lack a control group, but they can provide preliminary insights into treatment safety and efficacy. They help generate hypotheses for further research and justify larger controlled trials. The document provides examples of uncontrolled trials in various phases of clinical research. While uncontrolled trials are not as rigorous as controlled trials, they still play an important role in the early evaluation of novel medical therapies.
This document provides an overview of clinical research and the clinical trial process. It discusses the various phases of clinical trials from phase 1 to phase 4. Phase 1 trials assess safety in a small group of participants, while phase 2 trials provide preliminary efficacy and safety data in patients. Phase 3 trials further evaluate efficacy and monitor safety in a larger group of patients. Phase 4 trials collect additional safety and efficacy data after marketing approval. The document outlines the objectives and requirements of each phase of clinical trials and the overall goal of generating evidence about new treatments to improve human health.
This document discusses different types of experimental research designs, including their advantages and disadvantages. It covers true experimental designs like pretest-posttest and Solomon four-group designs. It also discusses quasi-experimental designs like nonequivalent control group and time series designs, as well as pre-experimental designs. Threats to internal and external validity are explained for different designs.
This document discusses key considerations for clinical trial design, size, and study population. It outlines common trial designs like parallel group, crossover, and factorial designs. Appropriate study design and adequate sample size are important to achieve study objectives and answer key questions. Sample size calculations should account for the primary endpoint, expected treatment effect, variability, type I and II errors. Selection of subjects and controls also impacts trial validity. An independent data monitoring committee provides trial oversight.
The document discusses bioequivalence studies and their protocol. It defines bioequivalence as drug substances from two dosage forms reaching systemic circulation at the same rate and to the same extent. The objective is for drug bioavailability from test and reference products to not be statistically different when administered under similar conditions. Study designs commonly used include crossover, parallel group and replicate designs. Key aspects covered are analytical methods, subject selection, drug administration, sampling, data evaluation, and applications of pharmacokinetics in drug development and novel drug delivery systems.
This document discusses randomised controlled trials (RCTs) and their use in evaluating health care interventions. It provides background on James Lind, who in 1747 conducted one of the earliest known clinical trials to test treatments for scurvy. The document outlines key aspects of RCTs such as defining the research question, population, interventions, and outcomes, as well as the importance of randomization and blinding to reduce bias. It distinguishes between explanatory and pragmatic trials and notes RCTs are generally considered the gold standard for evaluating health care technologies.
This document discusses various types of clinical trial designs. It begins by defining clinical trials and describing key elements like the PICO framework. It then covers ways to reduce bias through randomization and blinding. The document categorizes clinical trials based on factors like number of centers, control groups, randomization, and blinding. It provides details on traditional study designs like parallel group designs and crossover designs. It also discusses special designs for small populations and miscellaneous designs. Overall, the document provides an overview of different clinical trial designs, methods to reduce bias, and ways to categorize trial types.
This document provides an overview of the methodology used in clinical trials. It defines key terms like randomized controlled trials, control groups, randomization, and blinding. It describes the various phases of clinical trials including phases 1-3 and post-marketing studies. Phase 1 trials test safety in healthy volunteers while phases 2 and 3 test efficacy in larger patient populations. The goals of each phase are explained as well as important demographic information. The document also outlines the drug development process from preclinical research through regulatory approval and commercialization.
This document discusses various research designs used in nursing research. It defines research design as the plan or blueprint for conducting a study. Experimental designs aim to identify cause-effect relationships through manipulation of independent variables and use of control groups. True experiments allow the highest level of control but quasi-experiments and pre-experimental designs are also used when true experiments are not possible. Non-experimental designs observe variables without manipulation and are used when variables cannot be manipulated or experiments would be unethical.
Clinical trials involve testing new medical treatments on people to determine their safety and effectiveness. They are divided into phases, with early phases focusing on safety with small groups and later phases testing effectiveness against existing treatments with larger groups. There are different types of clinical trials including prevention, screening, diagnostic, treatment, and supportive care trials. Trials may also be classified as fixed or adaptive based on whether modifications can be made during the trial.
CROSSOVER STUDY DESIGN, DESIGN OF PHARMACOKINETIC STUDIES, FACTORS INFLUENCING BIOAVAILABILITY STUDIES, STUDY DESIGN, PARALLEL DESIGN, CROSS-OVER STUDIES, LATIN SQUARE DESIN, TWO-PERIOD CROSSOVER STUDY DESIGN, BALANCED INCOMPLETE BLOCK DESIGN (BIBD), REPLICATE CROSSOVER STUDY DESIGN , DIFFERENCE BETWEEN PARALLEL AND CROSSOVER STUDY DESIGN.
The document discusses various clinical trial designs, including parallel, crossover, dose-response, factorial, non-inferiority, and sequential parallel trials. It covers factors to consider when choosing a design like the questions being addressed and disease characteristics. Examples are provided for each design type to illustrate how they can be applied to evaluate different research questions. Issues related to active control and non-inferiority trials are also discussed.
Randomized controlled trials (RCTs) provide the highest level of evidence in clinical research. RCTs involve randomly assigning subjects to experimental and control groups to test clinical interventions. Key aspects of RCTs include formulating a research question, randomization to eliminate confounding factors, blinding of subjects and researchers, monitoring outcomes in both groups, and presenting results including relative risk, efficacy and number needed to treat. Common RCT designs are parallel, cross-over and factorial designs. RCTs aim to discover safety and efficacy of clinical interventions.
The document discusses different types of research designs, including explanatory trials which aim to study efficacy under ideal conditions, pragmatic trials which aim to study effectiveness in regular clinical practice, and cost benefit studies which aim to study efficiency in relation to resources consumed. It also discusses analytical studies, experimental trials using control groups, randomized controlled trials including group comparative and cross-over designs, non-randomized controlled trials, uncontrolled clinical trials, cohort studies, cross-sectional studies, and case control studies. The key aspects of each design such as whether they are prospective or retrospective and examples are provided.
Excelsior College PBH 321 Page 1 EXPERI MENTAL E.docxgitagrimston
Excelsior College PBH 321
Page 1
EXPERI MENTAL E PIDE MIOLOGICAL STUDIE S
Epidemiologic studies are either observational or experimental. Observational studies, including ecologic,
cross-sectional, cohort, and case-control designs, are considered “natural” experiments, but experimental
studies are considered true experiments. We will spend the next 2 modules discussing these designs.
Before we begin to discuss study designs, we need a brief introduction to a concept that we will spend more
time discussing in later modules -- bias. The definition of bias is:
“Deviation of results or inferences from the truth, or processes leading to such deviation. Any trend in the
collection, analysis, interpretation, publication, or review of data that can lead to conclusions that are
systematically different from the truth.” (Last, J.M., A Dictionary of Epidemiology, 4th ed.)
Epidemiologists are naturally concerned whether the results of an epidemiologic study are biased, since many
important public health decisions are often drawn from epidemiologic research. The severity of the bias, that
is - how much it influences or distorts the results, is related to the study design as well as how information is
analyzed.
Experimental Studies
The defining feature of experimental studies is that the investigator assigns exposure to the study subjects.
Experimental studies most closely resemble controlled laboratory experiments and serve as models for the
conduct of observational studies, thus they are the “gold standard” of epidemiologic research. Experimental
studies have high validity (i.e., less bias), and can identify even very small effects. The most well known type of
experimental study is a randomized trial (sometimes referred to as a randomized controlled trial), where the
investigator randomly assigns exposure to the study subjects. In this type of study, the only expected
difference between the experimental and control groups is the outcome variable being studied.
Experimental designs like the randomized trial can assess both preventive interventions, where a prophylactic
agent is given to healthy or high-risk individual to prevent disease, or can assess effects of therapeutic
treatment, such as those given to diseased individuals to reduce their risk of disease recurrence, or to improve
their survival or quality of life.
Preventive intervention: Does tamoxifen lower the incidence of breast cancer in women with high risk profile
compared to high risk women not given tamoxifen?
Therapeutic intervention: Do combinations of two or three antiretroviral drugs prolong survival of AIDS
patients as well as regimens of single drugs?
The investigator can assign exposures (or allocate interventions) to either individuals or to an entire
community.
Individual-level assignment: Do women with stage I breast cancer given a lumpectomy alone survive as long
without recurrence of disease as women given a lumpec ...
This document summarizes the different types of clinical studies, including clinical trials, cohort studies, and case control studies. It then provides detailed descriptions of clinical trials, including phases of clinical trials from pre-clinical animal studies to post-marketing surveillance. Clinical trials aim to evaluate safety and efficacy of new drugs and are conducted in a phased manner from small healthy volunteer studies to large multicenter studies in patients. Rigorous ethical and scientific standards are followed to ensure safety and quality of clinical research.
This document provides an overview of clinical research and clinical trials. It defines clinical research and clinical trials, discusses the importance of research. It describes the different types and phases of clinical trials, from phase 0 to phase IV. It outlines the key players involved in clinical trials and provides an overview of the clinical trial process from study design to statistical analysis and reporting.
This document discusses different types of study designs used in clinical research. It describes experimental designs like randomized clinical trials (RCTs) which are considered the highest level of evidence. It also covers observational study designs including cohort studies, case-control studies, cross-sectional studies, case series, and case reports. For each design it provides details on what it is, strengths, weaknesses and examples. It concludes with a brief overview of meta-analyses which systematically review and combine results from multiple studies.
Types of clinical trials designs were discussed including parallel designs, crossover designs, factorial designs, cluster designs, and adaptive designs. The key factors in choosing a clinical trial design are treatment duration and chronology of events, trial cost, and subject convenience. Commonly used designs include parallel, crossover, factorial, and equivalence/non-inferiority designs. The randomized, double-blind, placebo-controlled, parallel design is often considered the best to determine efficacy. Different designs can answer different therapeutic questions.
This document describes a doubly randomized delayed-start design for clinical trials. The design consists of two periods. In the first period, patients are randomized to receive either a new drug or placebo. In the second period, patients who received placebo in the first period and meet certain enrichment criteria can be rerandomized to receive either the new drug or continue on placebo. The design aims to reduce bias from high placebo responses and more efficiently study maintenance effects. It is naturally adaptive as aspects of the second period can be modified based on interim analysis of the first period. Efficacy data from both periods are combined for the overall analysis. This design offers greater efficiency for clinical development compared to traditional parallel designs.
This document discusses uncontrolled clinical trials, which evaluate new medical treatments without a control group for comparison. Uncontrolled trials have limitations since they lack a control group, but they can provide preliminary insights into treatment safety and efficacy. They help generate hypotheses for further research and justify larger controlled trials. The document provides examples of uncontrolled trials in various phases of clinical research. While uncontrolled trials are not as rigorous as controlled trials, they still play an important role in the early evaluation of novel medical therapies.
This document provides an overview of clinical research and the clinical trial process. It discusses the various phases of clinical trials from phase 1 to phase 4. Phase 1 trials assess safety in a small group of participants, while phase 2 trials provide preliminary efficacy and safety data in patients. Phase 3 trials further evaluate efficacy and monitor safety in a larger group of patients. Phase 4 trials collect additional safety and efficacy data after marketing approval. The document outlines the objectives and requirements of each phase of clinical trials and the overall goal of generating evidence about new treatments to improve human health.
This document discusses different types of experimental research designs, including their advantages and disadvantages. It covers true experimental designs like pretest-posttest and Solomon four-group designs. It also discusses quasi-experimental designs like nonequivalent control group and time series designs, as well as pre-experimental designs. Threats to internal and external validity are explained for different designs.
This document discusses key considerations for clinical trial design, size, and study population. It outlines common trial designs like parallel group, crossover, and factorial designs. Appropriate study design and adequate sample size are important to achieve study objectives and answer key questions. Sample size calculations should account for the primary endpoint, expected treatment effect, variability, type I and II errors. Selection of subjects and controls also impacts trial validity. An independent data monitoring committee provides trial oversight.
The document discusses bioequivalence studies and their protocol. It defines bioequivalence as drug substances from two dosage forms reaching systemic circulation at the same rate and to the same extent. The objective is for drug bioavailability from test and reference products to not be statistically different when administered under similar conditions. Study designs commonly used include crossover, parallel group and replicate designs. Key aspects covered are analytical methods, subject selection, drug administration, sampling, data evaluation, and applications of pharmacokinetics in drug development and novel drug delivery systems.
This document discusses randomised controlled trials (RCTs) and their use in evaluating health care interventions. It provides background on James Lind, who in 1747 conducted one of the earliest known clinical trials to test treatments for scurvy. The document outlines key aspects of RCTs such as defining the research question, population, interventions, and outcomes, as well as the importance of randomization and blinding to reduce bias. It distinguishes between explanatory and pragmatic trials and notes RCTs are generally considered the gold standard for evaluating health care technologies.
This document discusses various types of clinical trial designs. It begins by defining clinical trials and describing key elements like the PICO framework. It then covers ways to reduce bias through randomization and blinding. The document categorizes clinical trials based on factors like number of centers, control groups, randomization, and blinding. It provides details on traditional study designs like parallel group designs and crossover designs. It also discusses special designs for small populations and miscellaneous designs. Overall, the document provides an overview of different clinical trial designs, methods to reduce bias, and ways to categorize trial types.
This document provides an overview of the methodology used in clinical trials. It defines key terms like randomized controlled trials, control groups, randomization, and blinding. It describes the various phases of clinical trials including phases 1-3 and post-marketing studies. Phase 1 trials test safety in healthy volunteers while phases 2 and 3 test efficacy in larger patient populations. The goals of each phase are explained as well as important demographic information. The document also outlines the drug development process from preclinical research through regulatory approval and commercialization.
This document discusses various research designs used in nursing research. It defines research design as the plan or blueprint for conducting a study. Experimental designs aim to identify cause-effect relationships through manipulation of independent variables and use of control groups. True experiments allow the highest level of control but quasi-experiments and pre-experimental designs are also used when true experiments are not possible. Non-experimental designs observe variables without manipulation and are used when variables cannot be manipulated or experiments would be unethical.
Clinical trials involve testing new medical treatments on people to determine their safety and effectiveness. They are divided into phases, with early phases focusing on safety with small groups and later phases testing effectiveness against existing treatments with larger groups. There are different types of clinical trials including prevention, screening, diagnostic, treatment, and supportive care trials. Trials may also be classified as fixed or adaptive based on whether modifications can be made during the trial.
CROSSOVER STUDY DESIGN, DESIGN OF PHARMACOKINETIC STUDIES, FACTORS INFLUENCING BIOAVAILABILITY STUDIES, STUDY DESIGN, PARALLEL DESIGN, CROSS-OVER STUDIES, LATIN SQUARE DESIN, TWO-PERIOD CROSSOVER STUDY DESIGN, BALANCED INCOMPLETE BLOCK DESIGN (BIBD), REPLICATE CROSSOVER STUDY DESIGN , DIFFERENCE BETWEEN PARALLEL AND CROSSOVER STUDY DESIGN.
This document provides an overview of wound healing, its functions, stages, mechanisms, factors affecting it, and complications.
A wound is a break in the integrity of the skin or tissues, which may be associated with disruption of the structure and function.
Healing is the body’s response to injury in an attempt to restore normal structure and functions.
Healing can occur in two ways: Regeneration and Repair
There are 4 phases of wound healing: hemostasis, inflammation, proliferation, and remodeling. This document also describes the mechanism of wound healing. Factors that affect healing include infection, uncontrolled diabetes, poor nutrition, age, anemia, the presence of foreign bodies, etc.
Complications of wound healing like infection, hyperpigmentation of scar, contractures, and keloid formation.
A Visual Guide to 1 Samuel | A Tale of Two HeartsSteve Thomason
These slides walk through the story of 1 Samuel. Samuel is the last judge of Israel. The people reject God and want a king. Saul is anointed as the first king, but he is not a good king. David, the shepherd boy is anointed and Saul is envious of him. David shows honor while Saul continues to self destruct.
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إضغ بين إيديكم من أقوى الملازم التي صممتها
ملزمة تشريح الجهاز الهيكلي (نظري 3)
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تتميز هذهِ الملزمة بعِدة مُميزات :
1- مُترجمة ترجمة تُناسب جميع المستويات
2- تحتوي على 78 رسم توضيحي لكل كلمة موجودة بالملزمة (لكل كلمة !!!!)
#فهم_ماكو_درخ
3- دقة الكتابة والصور عالية جداً جداً جداً
4- هُنالك بعض المعلومات تم توضيحها بشكل تفصيلي جداً (تُعتبر لدى الطالب أو الطالبة بإنها معلومات مُبهمة ومع ذلك تم توضيح هذهِ المعلومات المُبهمة بشكل تفصيلي جداً
5- الملزمة تشرح نفسها ب نفسها بس تكلك تعال اقراني
6- تحتوي الملزمة في اول سلايد على خارطة تتضمن جميع تفرُعات معلومات الجهاز الهيكلي المذكورة في هذهِ الملزمة
واخيراً هذهِ الملزمة حلالٌ عليكم وإتمنى منكم إن تدعولي بالخير والصحة والعافية فقط
كل التوفيق زملائي وزميلاتي ، زميلكم محمد الذهبي 💊💊
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1. Measurement System for Impact
of OBE through Examination
and Evaluation.
Dr Rajendra Talware
2. Experimental Study Design
Best design for determining efficacy of treatment:
Randomized, controlled, double blinded,
experimental designed study.
Experimental study can be controlled or non-
controlled.
Randomized or non-randomized
Double blinded, single blinded or no blinding
A randomized controlled trial (study) may be
described as prospective, longitudinal, follow-up, and
experimental.
3. Randomized Controlled Trial
Patients are assigned to one of two or more groups
that receive a treatment or intervention and are
followed over time for a measured outcome.
Some patients are designated as the control group
which serves as the comparison group and which a
placebo or sugar pill or a comparison drug is given
Both groups are compared on the outcomes and
treatment effectiveness is determined.
4. Randomized Controlled Trial
Advantages
Utilizes a control group (strongest evidence)
Bias is minimized
Extraneous factors being responsible for
outcomes is minimized.
Most reliable technique for evaluating
treatments
Most statistically powerful study design
5. Randomized Controlled Trial
Disadvantages
Expensive
Patient enrollment can be difficult (time length)
Drop out rate is higher because of long term
follow-up (required by FDA)
Ethical problems in testing new therapies in
humans
Time necessary to perform study can be long.
Most complex to interpret.
6. Experimental Controlled Study
Example
To determine whether Retin A cream can
reduce wrinkle formation, investigators
recruited 100 healthy subjects from 65-70 y.o.
and randomized them to receive Retin A or
placebo. They applied the cream 2x daily for
1 year. A dermatologist rated the degree of
facial wrinkles using a scale from 1-10 at the
beginning and end of the study for all
participants and compared the two groups.
7. Randomized Controlled Trial
(RCT) Summary
The RCT design is suitable for most types of
pharmaceutical research.
It is considered to be the most statistically
powerful study design.
The major difference between the clinical trial
and other designs is the ability of the
investigator to actively intervene, rather than
simply observe.
8. Non-controlled experimental Study
Does not utilized a control group.
The drug treatment procedure to be studied is
administered to a single group of patients.
Outcome measures are determined in this
group.
9. Non-controlled experimental Study
Example
A study to determine the efficacy of Zofran in
prevention and treatment of Cisplatin induced nausea
and vomiting (N&V) was done. 50 patients receiving
Cisplatin for ovarian cancer were given Zofran at the
start of Cisplatin therapy. The number of episodes of
nausea and vomiting were recorded. Only 10% rated
the N&V as severe. Investigators concluded that
Zofran is effective in minimizing Cisplatin induced
N&V.
10. Design Types of Controlled
Experimental Studies
Parallel design
Crossover design
Time series design (Before and After)
11. Differences between types of
controlled experimental designs
Different outcomes can be measured more
accurately with different designs.
Different statistical tests are applied to
different study designs.
Different bias risks are inherent in each
design
12. Parallel Design
A parallel design includes independent study groups and each
group receives a different treatment regimen or intervention
Randomized Controlled Trials are often parallel design
Parallel design is more useful for studying conditions which are
prone to change over time (pain, acute exacerbations of a
disease, remissions)
Example:In a study to evaluate the efficacy of beta blockers for
hypertension, 24 patients are randomized into two groups of 12
patients. One group is then treated with a beta blocker and the
other treated with placebo.
13. Crossover Design
A crossover design may have just one study
group that receives all of the treatments (ie.
Drug and placebo).
It is more statistically sensitive and efficient,
using fewer patients.
Fewer patients can lead to a more
homogenous group with less variability in
measurement. (Less variability between groups implies a
measured difference is more likely to be due to treatment effects
instead of interpatient variability.
14. Cross-Over Design Example
In a study to evaluate the efficacy of beta
blockers for hypertension patients, 12
patients would be enrolled into the study and
6 patients would be assigned to treatment
with the beta blocker first, followed by
placebo treatment and the other 6 patients
would receive the same treatments in reverse
order, all having a washout period in-between
treatments.
15. Cross-Over Design
Advantages
Smaller number of patients are required since the same
patient groups receive both treatments
The ability to analyze patients both within groups as well as
between groups
Within groups: baseline factors (age, gender differences)
which could influence the results are eliminated because
patients serve as their own control group.
Between groups: Evaluate the effect of time on the
results (we can see what the patient does during the
placebo time period as well as what he does during
treatment period)
16. Cross-Over Design
Disadvantages
Time involved for a crossover design is longer
than other design types
More drop outs because of time involved.
Study design is very sensitive to drop outs
since small number of patients involved.
Period effects
Sequence effects
Carry over effects
17. Period Effects
Differences between treatment groups over
passages of time.
Period effects occur because patients are
observed at least twice and their condition
may change between the first and second
observation.
Period effects increase within-person
variability, which reduces the power of the
design and decreases the advantage of a
cross-over design study
18. Period Effect Variables
Depressed patients may be less depressed during
the 2nd treatment period simply because depression
tends to improve over time.
Learned effects
Development of tolerance or resistance
Changes in the disease state
Psychological variables: pain syndromes,
exacerbation of exzema, multiple sclerosis acute flare
ups, etc.
19. Sequence Effects
Changes in the effectiveness of the drug
treatment produced by the order in which the
drugs were administered.
Appear statistically as interactions.
Interactions affect the interpretation of the
results because the magnitude of the
treatment differences is not consistent.
20. Carry-over effect
When the effects of the drug given during the
first period persists into the second period.
Carryover effects only affect the treatment
response in the 2nd time period.
Can be eliminated by using a washout period
between treatments. This allows the patient to
return to baseline levels before the 2nd
treatment is started.
21. Carry-over effects
The ability to remove the influence of carryover
effects through the use of a washout period
differentiates carry-over and period effects.
Period effects represent long term or permanent
changes in the subject that are unlikely to be
eliminated with a washout period.
Carry-over effects represent temporary changes
secondary to continued presence of the drug in the
system, such as for a drug with a long half-life
persisting into the 2nd treatment period, but the
effects of the drug with a short half-life not persisting
22. Cross-Over Design
One risk is that this design is not powerful enough to
detect a clinically important interaction of period,
sequence or carry-over effect.
If the interaction is clinically significant, then you must
transform the crossover study into a parallel design to
do the statistical analysis. (defeats purpose of using
less subjects and saving money)
Types of studies good for cross-over design are:
bioavailability studies (interactions would be less likely
since the patients serve as their own controls)
23. Time Series (Before and After)
Design
Patients are studied before the experimental
drug is given. After the drug is given for a
certain amount of time, the patients are
evaluated again to determine the effects of
the drug.
More than one drug can be tested with this
type of study design by continuing to
administer drugs in sequence.
24. Times Series (Before and After)
Advantages
Certain factors which could influence the study
are eliminated (age, gender differences, etc)
Patients serve as their own controls so smaller
number of patients are needed (as compared
to parallel design)
25. Times Series (Before and After)
Disadvantages
The disease/condition being treated can change
over time, unrelated to the drug treatment.
Carry-over effects could occur.
Things that cause a carry-over effect
Drugs with a long elimination half-life
Drugs with active metabolites (esp. active
metabolites with long half-lives)
Drugs whose effects on the disease state being
treated persist after the drugs themselves are
eliminated from the body (lipophillic drugs)