What is a clinical trial and how does a trial work?

Question 1

Define the phases of clinical trials

Answer 1

1) Phase 1 Trials:

• These are the first stage of testing in humans
• They typically involve a small number of healthy volunteers (20-100) and aim to evaluate safety, determine a safe dosage range, and identify side effects
• This phase helps to ascertain the treatment’s safety profile and the appropriate dosage for further testing

2) Phase 2 Trials:

• involve a larger group of people (100-300) who have the condition the new treatment is intended to treat
• This phase aims to determine whether the treatment is effective for its intended use, and to further evaluate its safety

3) Phase 3 Trials:

• large patient groups (1,000-3,000) and aim to confirm effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely
• This is the phase before the treatment is submitted for market approval

4) Phase 4 Trials:

• These trials, also known as post-marketing surveillance trials, are conducted after a treatment has been approved for consumer use
• They involve thousands of participants and aim to monitor effectiveness and side effects in large, diverse populations over longer time periods, and to identify any rare or long-term side effects

Question 2

Discuss the role of different parties in clinical trial

Answer 2

1) Investigators: These are the researchers who conduct the study. Their role is to design the trial, recruit participants, administer treatments, monitor patient health, and collect data. The lead investigator, often called the principal investigator, takes overall responsibility for the trial.

2) Participants: These are the individuals who volunteer to take part in the study. Their role is to follow the protocol and provide information and data through their experiences of the treatment.

3) Sponsors: These can be individuals, institutions, companies, or organizations that take responsibility for the initiation, management, and financing of the clinical trial.

4) Clinical Research Coordinators: These individuals organize the trial on the ground. They help with participant recruitment, informed consent, data collection, and ensuring adherence to the trial protocol and regulatory requirements.

5) Institutional Review Boards (IRBs) / Ethics Committees: These independent groups review, approve, and monitor the trial to ensure it is ethically acceptable and protects the rights and welfare of the participants.

6) Regulatory Authorities: These are governmental bodies, such as the Food and Drug Administration (FDA) in the U.S. or the European Medicines Agency (EMA) in the EU, that oversee the approval and monitoring of clinical trials in their jurisdiction. They also review and approve the market release of successful treatments.

7) Contract Research Organizations (CROs): These are companies hired by the sponsor to manage and administer the clinical trial. They provide various services, such as data management, statistical analysis, and regulatory submission.

8) Data Safety and Monitoring Boards (DSMBs): These independent groups review interim data for participant safety, treatment effectiveness, and trial validity. They can recommend continuing, modifying, or stopping a trial based on these reviews.

9) Biostatisticians: These experts design the study’s statistical plan, help with sample size determination, and conduct the final analysis of the trial data.

10) Clinical Trial Nurses / Research Nurses: These are registered nurses with specialized training in administering clinical trials. They are typically involved in participant care, education, and sometimes recruitment and data collection.

Question 3

Compare and contrast between different study designs

Answer 3

Randomised Controlled Trials:

Question 4

Describe step by step procedures of running an RCT

Question 5

Discuss the history of development of human research protections