Clinical Trial Phases & Design of Clinical Trials
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slide1. Clinical Trial Phases & Design of Clinical Trials Presenting:<br>
slide2. This document is marked with a dated date and speaks only as of that dated date. Readers are cautioned not to assume that any information has been updated beyond the dated date except as to any portion of the document that expressly states that it constitutes an update concerning specific recent events occurring after the dated date of the document. Any information contained in the portion of the document indicated to concern recent events speaks only as of its date. TEXAS HEALTH RESOURCES (“THR”) expressly disclaims any duty to provide an update of any information contained in this document. The information contained in this document may include "forward looking statements" by using forward-looking words such as "may," "will," "should," "expects," "believes," "anticipates," "estimates," or others. You are cautioned that forward-looking statements are subject to a variety of uncertainties that could cause actual results to differ from the projected results. Those risks and uncertainties include general economic and business conditions, receipt of funding grants, and various other factors which are beyond our control. Because we cannot predict all factors that may affect future decisions, actions, events, or financial circumstances, what actually happens may be different from what THR includes in forward-looking statements. Information contained in the presentation is confidential and proprietary and its use is expressly limited to the S&P Global Ratings’ update presentation.
INFORMATION CONTAINED IN THE PRESENTATION CAN NOT BE USED OR DISTRIBUTED TO ANY THIRD PARTY OR USED IN ANY REPORT WITHOUT THE WRITTEN CONSENT OF TEXAS HEALTH RESOURCES. 2<br>
slide3. Clinical Trial Phases & Design of Clinical Trials What is a Clinical Trial?
Any interventional investigation in human participants intended to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of an investigational product(s); and/or to identify any adverse reactions to an investigational product(s); and/or to study absorption, distribution, metabolism and excretion of an investigational product(s) with the object of ascertaining its safety and/or efficacy.
ICH E6 R3 1/2025 3<br>
slide4. In drug development, clinical trials are categorized into phases. According to the FDA, these phases are described as follows:
Phase 0—an exploratory stage with minimal human exposure to the drug, without aiming for therapeutic or diagnostic outcomes (e.g., screening studies and micro-dose studies).
Phase 1—conducted mostly with healthy participants, focusing on safety. The objective is to identify the most common and serious side effects of the drug and understand its metabolism and excretion.
Phase 2—aims to collect initial data on the drug's effectiveness in people with a specific disease or condition. Participants receiving the drug might be compared to those receiving a placebo or another treatment. Safety is still assessed, and short-term side effects are monitored.
Phase 3—aims to gather more extensive data on safety and effectiveness by examining the drug's effects on different populations, using various dosages, and combining it with other medications.
Phase 4—conducted after the FDA has approved the drug for market release, these postmarketing studies may be mandated or agreed upon by the sponsor. They collect further information about the drug's safety, effectiveness, and optimal use in the wider population. 4 Phases of Clinical Trials<br>
slide5. There are Broadly two main types of Clinical Trials Interventional (or clinical) trials
Observational studies
Within these, there are various categories like treatment, prevention, screening, diagnostic, genetic, and quality-of-life trials which we will see in the next slides. 5<br>
slide6. Interventional (Clinical) Trials: These trials involve researchers actively intervening by assigning participants to specific interventions, such as new drugs, therapies, or devices.
Participants are assigned to different groups, and the researchers compare the outcomes of these groups to assess the intervention's effectiveness and safety. This type of trial is often used to test new treatments, therapies, or preventative measures. 6<br>
slide7. Observational Studies: In observational studies, researchers observe and collect data on participants without actively intervening.
They might study the natural course of a disease, track participants over time, or analyze data from existing records.
Observational studies can be used to identify risk factors, understand disease progression, or evaluate the impact of different factors on health outcomes. 7<br>
slide8. Types of Clinical Trials/Interventional trials: Treatment trials:
Focus on testing new treatments, like drugs, medical devices, or therapies.
Prevention trials:
Aim to find ways to prevent diseases or their recurrence, potentially using medications, vaccines, or lifestyle changes.
Screening trials:
Investigate the best ways to detect diseases early, often through new screening methods or technologies. 8<br>
slide9. Types of Clinical Trials/Interventional trials: Diagnostic trials:
Study the accuracy and effectiveness of diagnostic tests or procedures for specific diseases.
Genetic studies:
Explore the role of genes in disease development and progression, often to identify genetic markers or potential targets for therapy.
Quality-of-life trials:
Focus on improving the well-being and quality of life for individuals with chronic illnesses or condition. 9<br>
slide10. Other Types of Clinical Trials: Accuracy Studies
In certain cases involving medical devices, studies might be conducted to evaluate how well a new device performs in comparison to either an existing FDA-cleared device (known as a predicate device) or a reference method regarded as the gold standard.
The objective of these studies is not to prove that the new method is superior, but rather to establish agreement between the new method and either the reference method or the predicate device.
Such studies are usually carried out to support a traditional 510(k) application, where the requirement for clearance is to show "substantial equivalence" to an already cleared product (a predicate device). 10<br>
slide11. The Interventional Trial you will see often and Why: The Randomized Controlled Trial (RCT) is considered the highest standard for interventional studies.
RCTs evaluate how effective an intervention is on a patient population while minimizing bias.
The study involves comparing a group of patients who receive the treatment or intervention (the treatment group) with another group that usually does not receive it (the placebo group). This trial design is frequently used in pharmaceutical research and is also applicable to certain medical device studies. 11<br>
slide12. Clinical Trial Design The study design describes the strategy by which interventions or therapies are assigned to participants in a clinical study.
There are several types of intervention models, including:
Single group design
Parallel design
Crossover design
Factorial design 12<br>
slide13. Interventional Study Designs explained Single Group:
Single Group design describes a clinical research study in which all subjects receive the same intervention throughout their participation in the study.
Parallel:
Parallel design describes a study in which two or more subject groups receive different interventions at the same time. 13<br>
slide14. Interventional Study Designs explained Crossover:
In a crossover study design, subjects are given one treatment and then “crossover” to another treatment.
Factorial:
Factorial design describes a clinical study in which groups of participants receive one of several combinations of interventions. During the study, all possible combinations of the two drugs, A and B, and placebo are given to different groups of participants. 14<br>
slide15. Blinding is a key mechanism to minimize the risk of bias. Depending on the degree of blinding, the following types are distinguished:
Open-label trials: Both patients and investigators know which product each participant receives. Used mainly when blinding is impossible or unethical.
Single-blind trials: Only the investigator knows whether the patient is receiving the experimental product or the standard therapy/placebo. Reduces placebo effect and patient expectation bias.
Double-blind trials: Neither the investigator nor the patient knows the group allocation. Ensures the highest level of objectivity in evaluating outcomes.
Triple-blind trials: Additionally, data analysts are unaware of group assignments during data analysis. 15 Ways of Eliminating Bias in Clinical Trials<br>
slide16. Clinical Trial Phases & Design of Clinical Trials . 16<br>
slide17. For more information on Clinical Trials: Resources
ClinicalTrials.gov is the official website of the US Department of Health and Human Services, National institutes of Health, National Library of Medicine, and National Center for Biotechnology Information. It is a place to learn about clinical studies from around the world. https://clinicaltrials.gov:Home
US Food and Drug Administration (FDA) The FDA publishes numerous guidance documents that provide detailed information on specific topics related to clinical trials. The FDA has regulations that govern clinical trials ensuring compliance with ethical and scientific standards. https://www.fda.gov
To Access the THRE Webpage for more information on clinical research visit https://www.texashealth.org/research.
Kandi V, Vadakedath S. Clinical Trials and Clinical Research: A Comprehensive Review. Cureus. 2023 Feb 16;15(2):e35077. doi: 10.7759/cureus.35077. PMID: 36938261; PMCID: PMC10023071. 17<br>
slide2. This document is marked with a dated date and speaks only as of that dated date. Readers are cautioned not to assume that any information has been updated beyond the dated date except as to any portion of the document that expressly states that it constitutes an update concerning specific recent events occurring after the dated date of the document. Any information contained in the portion of the document indicated to concern recent events speaks only as of its date. TEXAS HEALTH RESOURCES (“THR”) expressly disclaims any duty to provide an update of any information contained in this document. The information contained in this document may include "forward looking statements" by using forward-looking words such as "may," "will," "should," "expects," "believes," "anticipates," "estimates," or others. You are cautioned that forward-looking statements are subject to a variety of uncertainties that could cause actual results to differ from the projected results. Those risks and uncertainties include general economic and business conditions, receipt of funding grants, and various other factors which are beyond our control. Because we cannot predict all factors that may affect future decisions, actions, events, or financial circumstances, what actually happens may be different from what THR includes in forward-looking statements. Information contained in the presentation is confidential and proprietary and its use is expressly limited to the S&P Global Ratings’ update presentation.
INFORMATION CONTAINED IN THE PRESENTATION CAN NOT BE USED OR DISTRIBUTED TO ANY THIRD PARTY OR USED IN ANY REPORT WITHOUT THE WRITTEN CONSENT OF TEXAS HEALTH RESOURCES. 2<br>
slide3. Clinical Trial Phases & Design of Clinical Trials What is a Clinical Trial?
Any interventional investigation in human participants intended to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of an investigational product(s); and/or to identify any adverse reactions to an investigational product(s); and/or to study absorption, distribution, metabolism and excretion of an investigational product(s) with the object of ascertaining its safety and/or efficacy.
ICH E6 R3 1/2025 3<br>
slide4. In drug development, clinical trials are categorized into phases. According to the FDA, these phases are described as follows:
Phase 0—an exploratory stage with minimal human exposure to the drug, without aiming for therapeutic or diagnostic outcomes (e.g., screening studies and micro-dose studies).
Phase 1—conducted mostly with healthy participants, focusing on safety. The objective is to identify the most common and serious side effects of the drug and understand its metabolism and excretion.
Phase 2—aims to collect initial data on the drug's effectiveness in people with a specific disease or condition. Participants receiving the drug might be compared to those receiving a placebo or another treatment. Safety is still assessed, and short-term side effects are monitored.
Phase 3—aims to gather more extensive data on safety and effectiveness by examining the drug's effects on different populations, using various dosages, and combining it with other medications.
Phase 4—conducted after the FDA has approved the drug for market release, these postmarketing studies may be mandated or agreed upon by the sponsor. They collect further information about the drug's safety, effectiveness, and optimal use in the wider population. 4 Phases of Clinical Trials<br>
slide5. There are Broadly two main types of Clinical Trials Interventional (or clinical) trials
Observational studies
Within these, there are various categories like treatment, prevention, screening, diagnostic, genetic, and quality-of-life trials which we will see in the next slides. 5<br>
slide6. Interventional (Clinical) Trials: These trials involve researchers actively intervening by assigning participants to specific interventions, such as new drugs, therapies, or devices.
Participants are assigned to different groups, and the researchers compare the outcomes of these groups to assess the intervention's effectiveness and safety. This type of trial is often used to test new treatments, therapies, or preventative measures. 6<br>
slide7. Observational Studies: In observational studies, researchers observe and collect data on participants without actively intervening.
They might study the natural course of a disease, track participants over time, or analyze data from existing records.
Observational studies can be used to identify risk factors, understand disease progression, or evaluate the impact of different factors on health outcomes. 7<br>
slide8. Types of Clinical Trials/Interventional trials: Treatment trials:
Focus on testing new treatments, like drugs, medical devices, or therapies.
Prevention trials:
Aim to find ways to prevent diseases or their recurrence, potentially using medications, vaccines, or lifestyle changes.
Screening trials:
Investigate the best ways to detect diseases early, often through new screening methods or technologies. 8<br>
slide9. Types of Clinical Trials/Interventional trials: Diagnostic trials:
Study the accuracy and effectiveness of diagnostic tests or procedures for specific diseases.
Genetic studies:
Explore the role of genes in disease development and progression, often to identify genetic markers or potential targets for therapy.
Quality-of-life trials:
Focus on improving the well-being and quality of life for individuals with chronic illnesses or condition. 9<br>
slide10. Other Types of Clinical Trials: Accuracy Studies
In certain cases involving medical devices, studies might be conducted to evaluate how well a new device performs in comparison to either an existing FDA-cleared device (known as a predicate device) or a reference method regarded as the gold standard.
The objective of these studies is not to prove that the new method is superior, but rather to establish agreement between the new method and either the reference method or the predicate device.
Such studies are usually carried out to support a traditional 510(k) application, where the requirement for clearance is to show "substantial equivalence" to an already cleared product (a predicate device). 10<br>
slide11. The Interventional Trial you will see often and Why: The Randomized Controlled Trial (RCT) is considered the highest standard for interventional studies.
RCTs evaluate how effective an intervention is on a patient population while minimizing bias.
The study involves comparing a group of patients who receive the treatment or intervention (the treatment group) with another group that usually does not receive it (the placebo group). This trial design is frequently used in pharmaceutical research and is also applicable to certain medical device studies. 11<br>
slide12. Clinical Trial Design The study design describes the strategy by which interventions or therapies are assigned to participants in a clinical study.
There are several types of intervention models, including:
Single group design
Parallel design
Crossover design
Factorial design 12<br>
slide13. Interventional Study Designs explained Single Group:
Single Group design describes a clinical research study in which all subjects receive the same intervention throughout their participation in the study.
Parallel:
Parallel design describes a study in which two or more subject groups receive different interventions at the same time. 13<br>
slide14. Interventional Study Designs explained Crossover:
In a crossover study design, subjects are given one treatment and then “crossover” to another treatment.
Factorial:
Factorial design describes a clinical study in which groups of participants receive one of several combinations of interventions. During the study, all possible combinations of the two drugs, A and B, and placebo are given to different groups of participants. 14<br>
slide15. Blinding is a key mechanism to minimize the risk of bias. Depending on the degree of blinding, the following types are distinguished:
Open-label trials: Both patients and investigators know which product each participant receives. Used mainly when blinding is impossible or unethical.
Single-blind trials: Only the investigator knows whether the patient is receiving the experimental product or the standard therapy/placebo. Reduces placebo effect and patient expectation bias.
Double-blind trials: Neither the investigator nor the patient knows the group allocation. Ensures the highest level of objectivity in evaluating outcomes.
Triple-blind trials: Additionally, data analysts are unaware of group assignments during data analysis. 15 Ways of Eliminating Bias in Clinical Trials<br>
slide16. Clinical Trial Phases & Design of Clinical Trials . 16<br>
slide17. For more information on Clinical Trials: Resources
ClinicalTrials.gov is the official website of the US Department of Health and Human Services, National institutes of Health, National Library of Medicine, and National Center for Biotechnology Information. It is a place to learn about clinical studies from around the world. https://clinicaltrials.gov:Home
US Food and Drug Administration (FDA) The FDA publishes numerous guidance documents that provide detailed information on specific topics related to clinical trials. The FDA has regulations that govern clinical trials ensuring compliance with ethical and scientific standards. https://www.fda.gov
To Access the THRE Webpage for more information on clinical research visit https://www.texashealth.org/research.
Kandi V, Vadakedath S. Clinical Trials and Clinical Research: A Comprehensive Review. Cureus. 2023 Feb 16;15(2):e35077. doi: 10.7759/cureus.35077. PMID: 36938261; PMCID: PMC10023071. 17<br>