Clinical Trials
LAST EDIT – 23.05.2025
EDITOR – PV THORAT
• After preclinical testing, an Investigational New Drug (IND) application is filed with
regulatory authorities for permission to test the drug in humans.
• Testing the drug in humans for safety and efficacy before therapeutic use is called a
clinical trial.
• Clinical trials must be conducted with strict adherence to bioethics principles:
o Autonomy — respecting participant’s informed choice
o Beneficence — maximizing benefits
o Nonmaleficence — avoiding harm
o Justice — fairness in participant selection and treatment
• Clinical trials are conducted in four phases (I–IV).
• Data from each phase is analyzed before progressing to the next phase.
Phase I Clinical Trial — Key Points
• First time testing in humans.
• Participants: ~10–100, usually healthy volunteers.
o Exception: Toxic drugs (e.g., anticancer agents) tested in patients with the
disease.
• Main goals:
o Assess safety.
o Determine maximum tolerated dose.
o Collect pharmacokinetic and pharmacodynamic data.
• Conducted by: Clinical pharmacologists.
• Study design: Open-label (no blinding).
Phase II Clinical Trial (Therapeutic Exploratory Study)
• First testing in patients with the target disease
• Conducted in 50–500 patients, usually across 3–4 centers
• Main objectives:
o Assess effectiveness of the drug
o Determine effective dose range
o Further evaluate safety and pharmacokinetics
• Study design is randomized and controlled, may be blinded
Phase III Clinical Trial (Therapeutic Confirmation Trial)
• Confirms efficacy of the drug in a large patient population (both sexes)
• Conducted at multiple centers
• Usually a randomized, double-blind, comparative trial
• Additional data on pharmacokinetics and drug interactions collected
• Marketing permission granted after successful completion
Phase IV Clinical Trial (Postmarketing Surveillance)
• Conducted after drug approval and marketing
• Collects additional data on long-term benefits and risks in a large patient population
• Provides info on:
o Adverse reactions (ADRs)
o Drug interactions
o New indications
o Different formulations
• Helps to:
o Estimate incidence of ADRs
o Detect previously unknown ADRs
o Identify risk factors for ADRs
• ADR monitoring centres exist nationwide for reporting observed ADRs
• Drug companies must submit postmarketing data regularly to regulatory agencies to
maintain drug approval
• Other clinical study types include case control, cohort, and meta-analysis
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CLINICAL TRIALS BEST MCQS FOR PRACTICE
1. What is the purpose of filing an Investigational New Drug (IND) application?
A) To begin preclinical testing of the drug
B) To obtain permission for testing the drug in humans
C) To market the drug directly to consumers
D) To conduct animal toxicity studies
Answer: B
2. Which of the following is NOT a principle of bioethics upheld during clinical trials?
A) Autonomy
B) Beneficence
C) Profitability
D) Justice
Answer: C
3. Clinical trials are conducted in how many phases?
A) 2
B) 3
C) 4
D) 5
Answer: C
4. Before proceeding to the next phase of a clinical trial, what is usually done?
A) The drug is marketed to the public
B) Information from the current phase is analyzed
C) The drug undergoes preclinical testing again
D) The drug is given to healthy volunteers only
Answer: B
5. What is the main goal of clinical trials?
A) To test the drug for scientific curiosity
B) To ensure the drug is safe and effective in humans
C) To improve the market value of the company
D) To complete paperwork for regulatory agencies
Answer: B
6. Which phase of clinical trials primarily focuses on assessing safety in humans?
A) Phase I
B) Phase II
C) Phase III
D) Phase IV
Answer: A
7. Which ethical principle means "do no harm" during clinical trials?
A) Autonomy
B) Beneficence
C) Nonmaleficence
D) Justice
Answer: C
1. How many participants are usually involved in Phase I clinical trials?
A) 1,000 – 5,000
B) 100 – 500
C) 10 – 100
D) Over 10,000
Answer: C
2. Phase I trials are usually conducted in:
A) Patients with the disease the drug is meant to treat
B) Healthy volunteers
C) Elderly patients only
D) Children only
Answer: B
3. For drugs with potential toxicity, such as anticancer drugs, Phase I trials are conducted
in:
A) Healthy volunteers
B) Cancer patients
C) Animal models
D) Any random population
Answer: B
4. The primary objective of Phase I clinical trials is to:
A) Assess drug efficacy
B) Determine drug safety and maximum tolerated dose
C) Compare the new drug with standard treatments
D) Market the drug
Answer: B
5. Which type of data is obtained during Phase I trials?
A) Pharmacokinetic and pharmacodynamic data
B) Marketing data
C) Genetic data
D) Environmental data
Answer: A
6. Phase I trials are usually conducted by:
A) Pharmacists
B) Clinical pharmacologists
C) Nurses
D) Patients themselves
Answer: B
7. What type of study design is typically used in Phase I trials?
A) Double-blind
B) Single-blind
C) Open label (no blinding)
D) Placebo-controlled
Answer: C
1. Phase II clinical trials are conducted in:
A) Healthy volunteers
B) Patients with the target disease
C) Animals
D) Random individuals without disease
Answer: B
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2. Approximately how many patients participate in Phase II trials?
A) 10 – 100
B) 50 – 500
C) 1,000 – 5,000
D) Over 10,000
Answer: B
3. The main objective of Phase II trials is to:
A) Assess drug safety only
B) Determine effective dose range and assess effectiveness
C) Compare the drug to other marketed drugs
D) Obtain pharmacoeconomic data
Answer: B
4. Phase II trials are usually conducted at:
A) One center only
B) Three to four centers
C) Over 100 centers
D) Only in outpatient clinics
Answer: B
5. In Phase II trials, the study design is typically:
A) Open label and uncontrolled
B) Randomized and controlled, may be blinded
C) Double-blind and uncontrolled
D) Single-subject case study
Answer: B
6. Besides effectiveness, which other aspects are evaluated in Phase II trials?
A) Marketing strategies
B) Safety and pharmacokinetics
C) Drug manufacturing costs
D) Environmental impact
Answer: B
1. Phase II clinical trials are the first to be conducted in:
A) Healthy volunteers
B) Patients with the target disease
C) Animals
D) Volunteers without the disease
Answer: B
2. How many patients typically participate in Phase II trials?
A) 10–100
B) 50–500
C) 1,000–5,000
D) Over 10,000
Answer: B
3. Phase II trials are usually conducted at how many centers?
A) One
B) Two
C) Three to four
D) More than ten
Answer: C
4. The primary objective of Phase II trials is to:
A) Determine the maximum tolerated dose only
B) Assess drug effectiveness and determine effective dose range
C) Market the drug to patients
D) Conduct toxicity studies
Answer: B
5. Which study design characteristic is typical of Phase II trials?
A) Open-label, uncontrolled
B) Randomized and controlled, may be blinded
C) Single-subject case study
D) Non-randomized observational
Answer: B
6. In addition to effectiveness, what other evaluations are done in Phase II trials?
A) Manufacturing cost analysis
B) Safety and pharmacokinetics
C) Advertising impact
D) Environmental risk assessment
Answer: B
1. The main aim of Phase III clinical trials is to:
A) Assess drug safety in healthy volunteers
B) Confirm efficacy of the drug in a large number of patients
C) Conduct animal toxicity studies
D) Market the drug
Answer: B
2. Phase III trials are generally:
A) Open label and uncontrolled
B) Randomized, double-blind, comparative trials
C) Single-subject case studies
D) Non-randomized observational studies
Answer: B
3. Phase III clinical trials are conducted in:
A) A single center
B) Multiple centers
C) Animal labs only
D) Pharmacies
Answer: B
4. After successful completion of Phase III trials, the company may receive:
A) Permission for marketing the drug
B) Approval to start Phase IV trials only
C) Permission to conduct preclinical studies
D) Patent for the drug
Answer: A
5. Additional data obtained during Phase III trials include:
A) Genetic sequencing
B) Pharmacokinetics and drug interactions
C) Marketing strategies
D) Environmental safety
Answer: B
1. Phase IV clinical trials are conducted:
A) Before marketing approval
B) After the drug is approved for marketing
C) Only in healthy volunteers
D) During animal testing
Answer: B
2. The primary purpose of Phase IV studies is to:
A) Determine drug safety and maximum tolerated dose
B) Obtain additional data about benefit and risk during long-term use
C) Test drug effectiveness in a small group of patients
D) Conduct preclinical testing
Answer: B
3. Phase IV studies provide information on:
A) Adverse reactions and drug interactions
B) Manufacturing costs
C) Drug pricing strategies
D) Preclinical toxicology
Answer: A
4. Postmarketing surveillance helps to:
A) Detect previously unknown adverse reactions
B) Market the drug internationally
C) Conduct animal studies
D) Replace Phase I trials
Answer: A
5. Adverse Drug Reactions (ADRs) observed in patients should be reported to:
A) The marketing department
B) ADR monitoring centers
C) The patient's family only
D) No one, it is optional
Answer: B
6. The drug company must submit postmarketing data:
A) Once, at the time of drug approval
B) At regular intervals to regulatory agencies
C) Only if adverse reactions occur
D) Never, after marketing begins
Answer: B
7. Besides clinical trials, which of the following are other types of clinical studies?
A) Case control study, cohort study, meta-analysis
B) Animal studies and lab tests
C) Marketing surveys
D) Manufacturing audits
Answer: A
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