Drug discovery.

Contents:

• How do clinical trials work?
• Who is involved in clinical trials?
• What makes a 'good' clinical trial?
• What treatments are currently undergoing clinical trial?

DEBRA UK funds medical research for clinical trials on treatments being developed, to repurpose existing treatments and basic science.

 

How do clinical trials work? 

Clinical trials are carried out to demonstrate whether a treatment, medical strategy or device is safe and whether it is as effective to treat or prevent symptoms as the approaches already in use.

Clinical trials recruit a certain number of people with particular symptoms and offer treatment for a specific length of time. Patients usually join a clinical trial through their regular visits to see a doctor or nurse rather than all starting together. This means it could take a year to get twenty people to complete a three-month trial.

Clinical trial process.

Initially a trial may only be carried out on a small number of people in case there are unforeseen side effects. If a phase 1 trial shows a new treatment is safe, it can be trialled on people with symptoms in a phase 2 trial. This stage can be skipped if a treatment already in use for another condition is being considered as it will already have been shown to be safe to use. This is called drug repurposing and is an important part of our research strategy as it has the potential to provide effective treatments more quickly to families living with EB. 

Drug repurposing process.

If a phase 2 trial shows the treatment appears to be safe and effective in a small number of people with EB, a phase 3 trial can be carried out on a larger number of individuals to show that this new treatment will work better than what is currently being used. This phase can also provide information about the safety and effectiveness of different doses or frequency of treatment and discover more about the risks of potential side effects.

When a treatment passes a phase 3 trial successfully it can be approved and licensed for use and manufacture in different countries. This process can take a few more months but helps to ensure that the treatments available are genuinely safe and effective.

Once a treatment is in use more widely, phase 4 trials can continue as more and more people start using it. This allows researchers to fine tune what is known about a treatment and adjust the way it is used to make it even more effective.

You can read more about how different types of clinical trial work in this article.

 

Who is involved in a clinical trial?

To carry out a clinical trial, a lot of funding is required to support a research team including many different experts such as doctors, nurses, social workers, health care professionals, scientists, data managers, and clinical trial coordinators. It also requires willing volunteers to participate in a trial and this can involve both risks and benefits. Participants will always be asked to give their “informed consent” before joining a trial. This means that they aren’t just agreeing because they trust their healthcare provider but have fully understood what their involvement will mean before they agree. It is also important for participants to know that they can choose to withdraw from a clinical trial at any point without giving any reason. At the end of a clinical trial, the number of participants who didn’t choose to complete it can give an idea of how difficult the treatment was to tolerate. The progress of the clinical trial will be monitored, and some are stopped early if the risks appear to outweigh the benefits but the nature of medical research studies means that some risks are unavoidable. 

The results of clinical trials add to medical knowledge and provide reliable information to assist in health care decision-making and better guidelines for treatment.

 

What makes a 'good' clinical trial? 

Some clinical trials provide stronger evidence that a treatment works than others because they take account of the parts of human nature that can cause false results. They do this using certain well-established research techniques:

A placebo

A placebo is a treatment that looks, feels, smells and/or tastes just like the new medicine being tested… but doesn’t actually have the active ingredient of the new medicine in it. You could make a pill that looks like an ibuprofen but that didn’t have any actual ibuprofen in it. This is important because when people take a pill, expecting to feel better and wanting to feel better, they often do feel a bit better. The Placebo Effect is a very real, very strong effect so researchers must take account of it when designing their clinical trial.

Randomisation

This means the doctors and nurses can’t choose who gets the new treatment and who gets the placebo. Randomisation removes all biases, even those that the researchers might not know they have.

Blinding

If you know you are getting a new, potentially fantastic, ground-breaking and expensive treatment, you are likely to feel pretty good about it and this could mean you report feeling a bit better or even do actually get a bit better. In a ‘single-blind’ clinical trial, the people receiving the treatment don’t know whether they are getting the new treatment or the placebo and this helps to make the results of the trial more reliable. If the doctor or nurse knows someone is receiving the new treatment, they may subconsciously allow more time in appointments and make more careful documentation of changes in symptoms. Researchers are only human after all, and everyone wants a new treatment to work. So, to provide strong evidence, a clinical trial will be ‘double-blind’ so that neither the people receiving the treatment nor the doctors and nurses giving it to them know whether their treatment is the placebo.

Statistical significance

When a group of people with symptoms are given a treatment, some of them will get better simply because they were getting better anyway. To provide strong evidence that people are getting better because of the treatment, researchers use maths. They can count how many people got better when given a placebo and compare to how many people got better when given the new treatment. The more people there are in a clinical trial, the more reliable the results can be. Sometimes a trial will report that there was an improvement in symptoms, but it was not ‘statistically significant’. This means the researchers cannot confidently say that it was unlikely to be due to chance.

Strong evidence from well-designed clinical trials is vital to support licensing and NHS funding decisions.

 

What treatments for EB are currently undergoing clinical trial? 

There are various treatments being trialled for EB. These include topical treatments that are applied to wounds and systemic treatments that are taken by mouth as tablets or are transfused directly into the blood. There are new treatments involving genetic engineering developed specifically for EB and medicines that have been used in other conditions that affect the skin and may help reduce EB symptoms. Some are just beginning the journey at phase 1 while others are closer to approval.

Here is a list of some of the potential treatments for EB that are currently undergoing clinical trials.

Clinical trials involving EB are listed here  by the US National Library of Medicine if you would like more details on any of these studies.

To find out about specific research studies that you could be involved in, please talk to your specialist doctor or nurse. They will be able to find out if you could participate in research local to you. 

 

Image credit: Two Smarties candies (49095075672), by Dan Keck. Licensed under the Creative Commons CC0 1.0 Universal Public Domain Dedication.