For pharmaceutical companies to obtain marketing licenses and make new drugs available to the general public, the safety, quality and efficacy of all potential new drugs must be demonstrated through a series of rigorous clinical trials, also referred to as clinical studies, research studies or medical research.
Clinical trials are generally carried out in a hospital or a medical research center and involve healthy volunteers or patients. Volunteers or patients are not generally required to stay at the hospital or center throughout the trial, but the effects of the drug will be closely observed and monitored throughout by highly qualified personnel.
Different phases of a clinical trial
Clinical trials can be classified according to four different phases. The first three are conducted before a license is granted and the last as a post-licensing phase. Each phase varies in size, character and focus.
Phase I - to assess drug safety, determine drug side effects and evaluate how the drug is administered
Phase II - to assess drug short-term efficacy and safety and determine optimum dosage range
Phase III - to confirm results of Phase II trials and to compare drug performance
Phase IV - to obtain more information from a larger group of patients
A customized protocol is developed for each trial. This is a study plan which is designed to answer specific research questions and also has the safety of volunteers and patients in mind. Protocols determine, among other things, who is eligible to participate (inclusion and exclusion criteria), the drug tested and, if any, the comparator (another drug for the same indication or a placebo), the dosages, the kind of tests and their schedules, and the trial duration.
Clinical investigators or medical researchers conduct clinical trials and follow study protocol. They are usually doctors, nurses, pharmacists or other health care professionals. In accordance with Good Clinical Practice guidelines the clinical investigator(s) will be qualified by education, training and experience to assume responsibility for the proper conduct of the trial, will meet all the qualifications specified by the applicable regulatory requirement(s), and will provide evidence of such qualifications through up-to-date curriculum vitae and/or other relevant documentation requested by the sponsor, the institutional review board and independent ethics committee.
Once the study protocol is approved, the hospital or medical research center will start to recruit healthy volunteers or patients to participate in the trial. The informed consent process is in place to ensure that all recruits are fully briefed about the trial and the nature of their participation, in both oral and written form. The informed consent document in written form contains:
- Information about the trial (purpose, duration, placebo or other comparator)
- Trial procedures
- Potential benefits and potential risks of participating in this study
- Explanation of how study results and patient medical information will be used
Volunteers or patients will be given time to read the informed consent document at home, to discuss their participation with family or close relatives and take time to make their decisions.
If a decision is made to join the clinical trial, the patient will be asked to sign the informed consent document.
Most trials will involve some sort of comparison with the new drug being tested. This means that in many clinical trials, while one group of volunteers or patients will be given the product under investigation, a control group is given either an existing standard treatment for the condition being treated or a placebo, a dose that looks like the medicine being tested but in fact contains no medical ingredients.
Regulation and Monitoring
All trials must be performed in line with Good Clinical Practice (GCP) guidelines or they will be rejected by the regulators.
Good Clinical Practice guidelines is the standard for the design, conduct, performance, monitoring, auditing, recording, analyses, and reporting of clinical trials that provides assurance the data and reported results are credible and accurate, and the rights, integrity and confidentiality of trial subjects are protected.
Every effort is made to ensure volunteer and patient safety throughout the course of any clinical trial. We do this through a series of tests that may include blood tests, physical examinations, procedures such as CT scans or ECGs and by listening to the patient. The UCB safety team meets once a month to examine trends and side effects across all study volunteers and patients. However, it must be noted that all clinical trials run the risk of side effects as mentioned in the Frequently Asked Questions (FAQs).