Clinical Trials Flashcards
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What are clinical trials?
Clinical trials are human experiments that involve medical interventions to test the safety and efficacy of new treatments or procedures. These trials may involve testing biological products, surgical procedures, radiological procedures, medical devices, behavioral treatments, process of care changes, or preventative care. Clinical trials are conducted to obtain approval from regulatory bodies for new drugs or procedures and to determine whether a medicine or intervention is safe and effective.
What types of medical interventions are tested in clinical trials?
Clinical trials test a wide range of medical interventions, including:
Biological products
Surgical procedures
Radiological procedures
Medical devices
Behavioral treatments
Changes in the process of care
Preventative care and more.
What is the purpose of an Investigational New Drug Application (IND)?
The Investigational New Drug (IND) application is a request to regulatory bodies to begin clinical trials with a new drug. The IND must include detailed information such as:
Animal pharmacology and toxicology studies
Preclinical data to assess safety for human testing
Chemistry and manufacturing details
Clinical protocols describing study design and safety measures
An investigator’s brochure, which contains essential information for conducting the trial safely.
What happens during Phase 1 of a clinical trial?
In Phase 1, a new drug is given to healthy volunteers to assess its safety and determine the maximum tolerated dose. This phase involves gradually increasing the drug dosage to identify side effects. It also examines how the drug is absorbed, broken down, and excreted by the body. Phase 1 trials may last several months or up to two years, and drug-food interactions are tested to prepare for progression to Phase 2.
What is the focus of Phase 2 in clinical trials?
Phase 2 focuses on studying the safety and efficacy of the drug in treating a specific disease. In this phase, the drug is tested on participants who have the condition of interest. Trials can be randomized or non-randomized and often use blinding methods, such as double-blind trials, to minimize bias. Phase 2 trials can last months to years, and around 25% of drugs successfully complete this phase.
Phase 2a (Proof of Concept) involves pilot trials in a small group of patients to refine dosing and assess safety and efficacy.
Phase 2b (Pivotal Trials) aims to rigorously demonstrate the drug’s efficacy and select the appropriate dose for Phase 3 trials.
What is the purpose of Phase 3 in clinical trials?
Phase 3 trials provide the most comprehensive data on the drug’s effectiveness and possible adverse effects. These trials often involve multiple well-designed studies with diverse patient populations that reflect the broader target community. Phase 3 trials are typically double-blinded and randomized. This phase is designed to demonstrate long-term safety and efficacy and is used to seek regulatory approval for the drug’s market release.
What occurs during Phase 4 of a clinical trial?
Phase 4, also known as post-marketing surveillance, occurs after a drug has been approved for market use. It involves monitoring the drug’s long-term effectiveness, comparing it to other drugs on the market, and assessing its cost-effectiveness. Phase 4 studies also help identify rare or long-term side effects. Findings from Phase 4 can result in the drug being withdrawn or restricted from the market if necessary.
What is the strongest study design in clinical trials?
The double-blind, randomized controlled clinical trial (RCT) is considered the strongest study design. This design helps eliminate selection bias and reduces confounding factors by randomly assigning participants to different groups, with neither the participants nor the researchers knowing who is receiving the treatment or placebo (double-blind).
What are inclusion and exclusion criteria in clinical trials?
Inclusion and exclusion criteria define who can and cannot participate in a clinical trial. These criteria ensure that participants have the appropriate medical condition, stage, and age for the trial and help control for other comorbidities or medications that might affect the study’s results. It’s also important to have strong justification for exclusion criteria to avoid bias and ensure the trial includes representative groups, including underrepresented populations such as older adults, pregnant women, children, and minorities.
What is informed consent in clinical trials?
Informed consent is a critical process in clinical trials where participants are provided with detailed information about the trial, including its purpose, procedures, potential risks, and benefits. Participants must give their consent voluntarily after understanding the trial details, ensuring they can make an informed decision about whether to participate.
What are the key considerations when designing a clinical trial?
When designing a clinical trial, the primary focus is on safety and minimizing bias. The trial must:
Include measurable and accepted endpoints.
Be designed for statistical analysis, determining the expected clinical effects and the required sample size.
Use randomization to eliminate selection bias and reduce confounding factors, both known and unknown.
Ensure that the trial design is robust and can provide reliable results.
What is the role of a sponsor in a clinical trial?
A sponsor is an individual, company, institution, or organization responsible for initiating, managing, and financing a clinical trial. It is a legal requirement for any clinical trial involving an investigational medicinal product (CTIMP) to have a sponsor. The sponsor ensures that the trial is conducted according to regulatory requirements and ethical standards.
Why is randomization important in clinical trials?
Randomization is crucial in clinical trials because it helps eliminate selection bias and ensures that participants are assigned to different treatment groups in a way that is free from human bias. It also reduces the influence of confounding variables, both known and unknown, and ensures that the results are more likely to be attributed to the treatment rather than other factors.
What is an adaptive platform trial design?
An adaptive platform trial design allows for flexible and dynamic adjustments during the trial based on interim results. This design can adapt by adding or removing treatments, adjusting dosage, or changing participant allocation based on ongoing findings. This approach is particularly useful for clinical trials involving underrepresented populations or evolving treatments.
Why are underrepresented populations important in clinical trials?
Underrepresented populations, including older adults, pregnant women, children, and minorities, are important to include in clinical trials to ensure that the results are generalizable to the broader population. Clinical trials that fail to include these groups may not accurately reflect how a drug or intervention works across different demographics. It’s essential to develop clinical trial strategies that ensure fair inclusion and report trends in the representation of these populations.
