Clinical trials are an essential part in the development process of new drugs and treatments. Before a new medicine can be made available, it must first be proven safe and effective by undergoing a carefully observed clinical research process in humans. This clinical research is carried out in healthy participants and participants suspected or confirmed of having the illness. However, before clinical research is performed on human volunteers, the researchers must conduct laboratory research to examine a new drugs potential effect on human cells or animals. The new drugs and treatments which provide the most promising results progress to human clinical trials.
Clinical trials are grouped into different phases (Phase I to Phase IV). Phase I involves a small number of participants, normally between 6-10 volunteers and allows scientists to understand what effects the new drug has in humans. Participants are monitored for the occurrence of any side effects.
Once the initial safety of the study drug has been confirmed in Phase I trials, Phase II trials are performed on larger groups of patients, generally 20-100 (depending on the type of disease). Participants are given various doses of the drug and closely monitored to compare the effects and determine the safest and optimum dosage.
Phase III studies are carried out on patient groups of 100 or more and are designed to confirm the safety and efficacy of a treatment and may involve one or more randomised trials, which allow for the safety and effectiveness of the new drug to be compared to other available treatments.
During the final phase (also known as Post-Marketing Surveillance Trials) the treatment has been released to the public and is being used by over 200 people. It is still being monitored by the researchers and the data collected by them can be used to examine the long-term effects of the treatment. After a clinical trial is completed, the research team carefully analyses information collected during the study to make decisions about the results and then they can determine if there’s any need for further testing.
This blog post was written by Alison Hyland. Alison lives with Recessive Dystrophic EB and is about to begin her second year of university in DCU where she studies Genetics and Cell Biology. We were delighted to welcome Alison to DEBRA Ireland to work as a Research Assistant over the summer months this year.
The contribution Alison has made to the Research team’s work has been invaluable as they have been able to gain the perspective of someone living with EB throughout the work that they do – one of the huge benefits of PPI. We believe that Alison, and everyone who lives with EB or cares for someone with EB, are the real experts of EB as they have to live with it every single day. We are hopeful that going forward we can involve our families in research more and make sure that their voice is heard throughout all of our research activities. We will be looking for volunteers to join our PPI panel, known as the ‘EB Expert Panel’, over the next few months so please get in touch if you would like more information.
If you would like to learn more about having your voice heard in research then please contact our Research Officer, Sarah: [email protected]
If you would like to read more about Alison’s story then please click here.