This blog post was co-authored by Mary Bailey, an EB family member.

Clinical trials are how scientists test new methods to diagnose, treat, cure, and prevent health conditions, including rare health conditions such as EB. Clinical trials are a lengthy process usually, as the goal is to ensure this new method is both safe and effective before use.

Clinical trials are carefully designed and reviewed before they can take place. This is to ensure the research is as safe as possible and that the results will be worthwhile. The trial needs to be approved by government regulatory bodies before it can begin. For example, in Ireland, the Health Products Regulatory Authority (HPRA) is responsible for assessing clinical trials using medicinal products.

Before a clinical trial takes place, a research team must engage in laboratory research, known as preclinical research, to discover new drugs and test their potential effect on human cells or animal models. This can take several years to complete, but if the preclinical research results are promising, then the scientists will move forward with a clinical trial to see how well their method will work on humans. For example, in EB research a new oral spray to treat mouth ulcerations has been tested on a small sample of human EB cells in a laboratory to ensure it is not toxic and safe enough to progress to clinical trials.

Clinical trials are split into various phases, as the research progresses to the next phase the number of human participants in the trial grows and so does the likelihood of the treatment becoming available for public use. As you can see from the diagram below, this is a long process with many trials lasting over ten years, which also makes it an extremely expensive process.


The Phases of Clinical Trials

Drug Discovery Phase – This phase takes place in the laboratory, where scientists use various experiments to discover new drugs which may be effective at targeting a specific disease or ailment, such as EB. This research usually takes between 3 to 5 years as the drug needs to be identified before it can be tested.

Preclinical Phase – This phase also takes place in the laboratory, however at this point the treatment has been developed and the researchers are now testing it on either human cells or animal models. Preclinical research usually takes about 1 to 2 years and aims to ensure the treatment is both useful and safe.

Phase I Clinical Trial – This is where the researchers begin to test on humans to understand how the treatment effects them directly. Up to 10 human participants can be tested on at this point and this can take around 1 year.

Phase II Clinical Trial – If the treatment passes phase I clinical trials, it can progress to phase II. This involves testing the treatment on 20 to 50 participants for around 2 years. The aim of this phase is to evaluate the safety of the treatment and how effective it is for human use.

Phase III Clinical Trial – In order to confirm the benefit and safety of the treatment, it must progress to phase III clinical trials. This phase takes 1 to 4 years to complete, and the treatment is tested on 100-200 participants at this point. If the treatment passes this phase it is usually deemed to be safe and beneficial for human use. If the trial is successful, the regulatory authority (such as the HPRA) grant the researchers a license to market the product.

Phase IV Clinical Trial – At this point the treatment has been released to the public and is being used by 200+ people, however it is still being monitored by the researchers. They use data from real-life usage to evaluate the long-term effects of the treatment.


What about EB Clinical Trials?

There are currently 36 EB clinical trials worldwide, either active or being recruited for.* Although EB research does not receive the funding that other common diseases benefit from, we are starting to see a movement of this research from lab to clinical trial phase. This in turn will lead to more EB specific treatments reaching the market in years to come. An example of this is Amryts’s Filsuvez, the first EB wound treatment to successfully complete Phase III clinical trials. It is now being assessed by the regulatory authorities. At DEBRA Ireland, we continuously look into ways we can use our fundraising initiatives to raise much-needed funds for EB research to make EB treatments a reality.

*on 7th September 2021


DEBRA Ireland would like to thank Mary Bailey, who is an EB family member, for her fantastic contribution to this article. We really appreciate it.

We love to see those who have EB or their family members getting involved in research, as no one understands EB better than those who have experienced it.

If you would like to get involved in future blog posts or projects, please contact Sarah: [email protected]

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DEBRA Ireland is a member of the Health Research Charities Ireland group (HRCI). One of the aims of HRCI is to support charities in Ireland to fund healthcare research to assist them in improving the lives of those they support.

The Health Research Board (HRB) has been co-funding the HRCI-HRB Joint Funding Scheme with the HRCI since 2006. The HRB is the lead agency in Ireland supporting patient-centred research linked to health and social care. The HRCI-HRB Joint Funding Scheme allows charities to support patient-focused research of particular interest to specific patient groups. For DEBRA Ireland, this is research to improve the lives of those living with the rare, genetic skin condition, Epidermolysis Bullosa (EB). Through the HRCI-HRB Joint Funding Scheme, the costs of successful patient-focused research projects are shared by the HRB and the sponsoring charity, which must be a member of HRCI.

DEBRA Ireland is participating in the HRCI-HRB Joint Funding Scheme in 2021 based on our current research priorities, which are outlined below. All researchers must submit their applications to DEBRA Ireland directly to be considered for the scheme. The HRB will manage the awards

Expressions of Interest is open now, and will close Friday 20th August 2021 at 5pm.

Opening Date for Applications: 1st September 2021

Closing Date for Applications: 13th October 2021

Level of funding: Funding up to €300,000 (€100,000 p.a.)

Duration of Funding: 12 to 36 months

Focus of the call: We will prioritise applications that will contribute to knowledge of the causes, diagnosis and treatment for Dystrophic EB, Junctional EB, and EB Simplex based on the following areas: wound healing, pain management, gene therapy and medical devices.

For more information or to submit interest in applying, please contact: [email protected]

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Title: Advocacy and Policy Manager

Salary: €50,000 – €55,000 (FTE) / Salary will be commensurate to level of experience, achievements and qualifications.

Hours: Full-time permanent but potential for 4-day week

Reporting to: CEO

Location: 8 Clanwilliam Terrace, Grand Canal Quay, Dublin 2 (* Note: in light of the COVID-19 pandemic and restrictions, this role will be full-time home-based while public health restrictions remain in place)

For more information on the role: DEBRA Ireland Advocacy and Policy Manager spec

To Apply:

Interested candidates should submit a copy of their CV and cover letter to [email protected]

Closing date for applications is the 25th June 2021 at 5pm.

For informal queries please contact [email protected]

DEBRA Ireland is an equal opportunities employer.



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