How do clinical trials play an important role in research?

Clinical trials play a vital role in trialling the development of new treatments for conditions such as age-related macular degeneration (AMD) and diabetic macular oedema (DMO). But, how do they work? And, why is participation in these trials so important? 

In a recent webinar, Sarah Clinton and Geraldine Hoad from our research team gave an overview of clinical trials, how they work, why they matter, and what someone can expect if they decide to take part.

What are clinical trials?

“Clinical trials are really important studies to test whether new treatments are safe and effective,” Sarah said. The trials are necessary before any treatment can be considered for general use. In the UK, approval must be granted by the Medicines and Healthcare products Regulatory Agency (MHRA), followed by further assessment by NICE, which considers whether a treatment is both effective and cost-efficient enough to be offered on the NHS.

Clinical trials can involve drugs such as tablets or injections, but may also include surgery, non-surgical approaches, or new technologies. “Trials don’t always have to be testing new treatments. They can also be testing new tools to help improve quality of life,” she said.

Why consider joining a trial?

“Taking part gives you early access to the latest treatments and drugs,” Sarah explained. However, she added that there is “no guarantee that the treatment will be successful or that you will be put into the treatment arm.” Some participants may receive a placebo or standard care, and often they won’t know which until after the trial ends.

Sarah added: “In clinical trials, treatments are typically tested against placebo or any available treatments.

“So, for trials where there's already an approved treatment, trials will typically be compared against standard care, which compares whether the new treatment is better or as good as the treatments currently available.

She added: “However if we don't have a standard care or a treatment already available, this is when we use a control arm where some participants may receive a placebo or sham treatment. This is because there's something called the placebo effect, where people may experience improvement in symptoms when taking a treatment even if that treatment is a sham or a fake treatment such as a sugar pill. So, this placebo group allows researchers to be sure that any changes that are seen in participants are due to the treatment rather than the placebo effect.”

But, whether you receive the new drug or a placebo, there are still benefits to taking part in the trial. For example, patients may benefit from more frequent monitoring and treatment, which can be helpful for those with conditions such as dry AMD who may not currently be seeing a specialist about their condition.

Importantly, every trial contributes to the progress being made.

“Every trial, whether it’s successful or not, helps us to learn more about macular disease and brings us closer to finding these new treatments. It can also allow you to take an active role in your care, to understand your condition and to get more frequent monitoring.”

Are clinical trials safe

Patients are often concerned about the risks of taking part in a trial and if they are safe. Sarah said: “We often get asked whether clinical trials are safe and if they're safe to start in clinical trials.

“Any possible new treatments that start in clinical trials have to go through rigorous laboratory testing to ensure that they are safe for humans. So, these treatments can be going through years of preclinical testing to ensure that any side effects are not harmful to those who are taking part in a clinical trial.”

All drugs go through a several phases of clinical trials in order to be approved. These phases clinical trials get larger and more thorough as they advance. Not every treatment will make it through all phases, all three phases. If the results for a previous trial are unsuccessful, companies may decide not to move forward with research into that treatment.

What to expect from the process

“Most trials have a very strict criteria of who’s suitable to join,” Sarah explained. These rules are designed to ensure the results are reliable and that it is safe for someone to take part.

If you are invited to join, you’ll have a chance to ask questions, review the details of what is involved, and take your time to decide. “This is referred to as ‘consenting’ and you’ll go through a patient information sheet detailing everything that’s involved,” she said. Patients are encouraged to speak with family or friends before deciding on whether to take part.

Sarah added: “It’s really important to be aware that there should be no pressure to join a clinical trial.” You should also be able to leave the trial at any time, and staff are expected to support that decision.

More than clinical trials

Clinical trials are only one of the wider research picture. Observational studies are also important. These do not involve giving participants a new treatment, but instead follow people over time to see how disease develops and changes.

“This is often really important for rare conditions where less is known of how and why disease progresses,” Sarah said. In AMD, observing patterns of progression can guide future research and help decide what kinds of treatments should be developed.

Focus groups and patient surveys also play a vital role. “It’s really important that we hear directly from people living with a condition to understand their experience and their hopes for future treatments,” Sarah said. This helps researchers focus on what patients actually find useful.

Finding a cure 

More and more research is looking into macular disease and possible new treatments, using a range of approaches including artificial intelligence. Taking part in a trial not only contributes to that work but also helps ensure that the patient’s voice is part of the conversation.

Sarah said: “Taking part in clinical trials ensures that the patient’s point of view is heard when research is being planned and carried out and reported.”