The subject of clinical research was presented by Sarah Venugopal from Raremark, a company connecting families affected by rare diseases with information on the latest research and treatments in the field.
Clinical research, also known as clinical trials, clinical studies or human trials, are conducted to collect data about the safety and effectiveness of a potential new test, drug or device before it is approved and widely used.
The key players in clinical research are:
- The sponsor – organization that funds the clinical trial, can be a pharma company, a charity, a hospital or even an individual
- The ethics review boards – independent groups responsible for the protection of the rights, safety and well-being of people taking part in a trial
- The principal investigator (PI) – person who leads the trial, usually a specialist doctor or researcher
- The study coordinator – person who supports the PI and is in charge of the day-to-day running of the trial, there can be more than one
- The participants – the patients or healthy volunteers taking part in the trial
People take part in clinical trials to help speed up the approval process for new drugs for themselves or others, to access specialist care, to potentially access an experimental treatment and to be monitored closely.
Main types of clinical trial:
- Diagnostic trials – new tests or procedures for diagnosing diseases
- Natural history studies – often done for rare diseases, they generate insights into how diseases might progress naturally over time
- Observational studies – monitor participants without intervening for example whilst they are already on an existing treatment or after surgery
- Screening trials – evaluate new tests, test the best way to detect certain diseases or medical conditions
- Treatment trials – the one people are more familiar with, they evaluate the effectiveness and safety of potential new treatments
Four phases of Clinical trials:
Phase I – Typically a small study (5-100) with healthy volunteers assessing safety usually with a very low dose of the drug. This phase usually lasts months to a year. In rare diseases, sometimes this phase is with patients with the condition so that the drug can be approved faster. 70% of the drugs tested on phase I make it to phase II.
Phase II – Assessing safety and efficacy, larger study with people with the condition, can include a placebo. 33% of the drugs tested on phase II make it to phase III.
Phase III – Larger studies, randomized and controlled, people with the condition, lead to a potential approval, looks at dosing. 70-90% of the drugs tested on phase III make it to phase IV.
Phase IV – Drug is approved for use, monitor long-term safety and effectiveness in the real world.
Usually the entire process lasts about 6 to 10 years.
What happens during a trial?
It starts with the sponsor designing the trial with doctors/investigators, then investigators see patients and collected data, the data is entered into a database, the data is analysed and presented in a report and finally the report is used to support approval.
Most large clinical trials are randomized, double-blind placebo-controlled trials. This is a common way to design a trial as it ensures the data is robust, reliable and reduces bias.
Placebo-controlled – a placebo contains no active ingredients and is given to some of the participants in a trial so that the effectiveness of the drug can be accurately seen
Randomized – participants are randomly assigned to either take the active drug or the placebo
Double-blind – to reduce bias, neither the researchers nor the patients know who is taking the experimental drug or the placebo
What happens after a trial?
If the drug is shown to be safe the results are submitted for approval to regulatory bodies (FDA or EMA), this process takes a very long time and the data is reviewed to make sure is reliable, cost-effective and has a positive impact. Additional data might be needed and it important to continue to monitor drug in real world. Usually there is publication of results on PubMed or clinicaltrials.gov but unfortunately, not all sponsors publish their results.