Question

Give an example of a fundamental principle in development (and experiment used to demonstrate it) that has been used as the basis for a clinical treatment or potential clinical treatment in humans.

Answer 1

The randomized controlled trial (RCT) is one of the simplest but most powerful tools of research. It is a form of study or scientific experiment in which people are allocated at random to receive one of several clinical interventions. These experiments are most commonly used in testing the safety and efficacy of any therapeutic or health care procedures. Preparing the protocol, specifying the methods of improving the transparency and validity of reporting the results of RCT’s and submitting for ethical review are basic prerequisites. Randomization reduces the risk of serious imbalance in important unknown as well as known factors that could influence the clinical course of the participants. Methods of Randomization include use of a table of random numbers, computer programs and tossing a coin etc.

Randomized Controlled Trial (RCT) is a type of scientific experiment or a form of clinical trial most commonly used in testing the safety and effectiveness of health care services or preventive or therapeutic procedures. [1]

In recent decades, modern medicine has been blessed with a pharmaceutical armamentarium that is much more powerful than it had before. A major factor for such rapid advance has been the development and refinement of the clinical research method known as the RCT. A clinical trial is defined as a prospective scientific experiment that involves human subjects in whom an intervention is initiated for the evaluation of a therapeutic procedure.[2] In an RCT, each patient is assigned to receive a specific treatment intervention by a chance mechanism.

The people who take part in RCTs (the ‘study population’) are called ‘participants’. Participants can be patients, healthy volunteers, relatives of patients, members of the general public, communities, or institutions. The people who design and carry out the study and analyze the results are called the ‘investigators.

RCTs usually try to measure and compare different events called ‘outcomes’ that are present or absent after the participants receive the interventions. RCTs are also considered as comparative studies as they are used to compare two or more intervention. One of the interventions is regarded as a standard of comparison or ‘control’, and the group of participants who receive it is called the ‘control group’

BASIC STEPS OF AN RCT

1. The first step is to prepare the protocol and submit it for ethical review.
2. Choosing the reference population: A reference population may be as broad as mankind or may be limited to specific groups like age, sex, social or occupational groups depending on the type of the study.
3. Selecting the experimental or study population: The study population is derived from the reference population. It is the population which actually participates in the study. The basic characteristics of the experimental population are:
a) They should have the same characteristics as the reference population and must always be randomly selected from the experimental population.
b) They must give informed consent to participate in the study after having been duly informed about the objectives, interventions to be used and possible risks of the trial.
4. Randomization: It is a statistical procedure by which the participants are allocated into study and control groups randomly.
5. Intervention: The next step is to apply the intervention to the study group by administering the new drug or anything as laid down in the protocol. The procedures for performing the trial should be standardized and should be adhered.
6. Follow Up: This Implies examination or observation of both groups at defined intervals of time in a standard manner till final assessment of the outcome.
7. Analysis : The incidence of both positive or negative results /outcome are rigorously compared in both the groups and the differences are tested for statistical significance.