Taking part in PCD research
PCD is a rare disease with very little evidence for its management. Currently, management guidelines are mainly based on research from other respiratory conditions and expert opinion. To improve understanding of the condition and improve the outcomes and the quality of life for people with PCD, it is important that we design and carry out research with PCD patients.
There are four PCD centres in England that make up the PCD National Diagnostic and Management Services. The whole service works together as one National Service and collaborates internationally as part of BEAT-PCD. This means we can consider the most important questions patients need answering and work together to answer them. You can find out more about BEAT-PCD by following this link http://www.beatpcd.org/
All centres are involved in research and will offer their patients the chance to take part in clinical trials. You may also receive information about research via the PCD Family Support Group. These may be local, national or international studies. This information aims to answer your questions about research and to help you decide whether, or not, you want to be involved.
Shaping PCD Research – PPI how you can help
You can help PCD research by volunteering to be a patient or public representative. This is an important role that will help ensure research is patient-centred and that patients and the public are involved in the design of research studies. Representatives will be able to offer a unique perspective and have a personal understanding that doctors, nurses and researchers may lack. If this is something you feel you may be able to help with, please complete the PCD Patient and Public Involvement Registration Form and email it to PCDClinical@uhs.nhs.uk.
What is PCD medical research?
PCD research aims to:-
- improve our understanding of the causes and development PCD
- Improve diagnostic techniques and reduce time to diagnosis/exclusion of PCD
- Help prevent and reduce severity and frequency that PCD patients become ill with chest or ENT problems
- Look for the best ways to treat infections and improve the quality of life for people living with PCD
- Develop new treatments and medicines
Research undertaken at your local PCD centre hopes to not only improve your care but also improve the care throughout the country and further afield.
Why is research into PCD important?
Without research there would be no new medicines or tests, improved treatments, or better ways of treating people with PCD. A European Respiratory Taskforce for PCD has recently created an evidence based guideline for PCD diagnostics. This will improve the testing for PCD internationally; ensuring centres all around the world are using the best evidence to inform their decisions for diagnosing PCD. This may be further improved in the future as new techniques for diagnosis become available.
What are the different types of PCD research?
There are four main types of research you may be invited to participate in.
- Observational research – most children in England have been invited to share their anonymised data on a local and/or international database. (PCD and the PCD Registry are two international databases that record the diagnostic history and follow up data for adults and children with PCD). As this information is collected we will be able to learn more about PCD as well as learning about the relationship between the genetics and the characteristics of the condition.
- Qualitative research – many studies look to measure objective things such as height, weight, lung function but don’t look at the individual patient experience. Qualitative research is usually conducted through a series of interviews with patients and families to find out how their condition affects them individually so we can have a better idea of how to best care for PCD patients.
- Research involving tissue and cells – cells are basic units that make up the human body to perform all the functions we need. For example, there are cells that line the airways of our lungs that produce mucus and other cells that grow cilia. Together they form the basis of mucociliary clearance to keep our lungs clean. Human cells and tissue are vital for medical research. They can be collected during many routine procedures, including:
- blood tests
- biopsies (tissue samples taken for examination e.g. nasal brushings)
Researchers can use these cells and tissues to learn about how PCD starts and develops, and also how new drugs and tests might work before clinical trials begin.
- Clinical trials – new treatments or medicines need to be tested to see if they are of benefit to patients. The treatments PCD patients undertake are known to be safe but have rarely been tested for efficacy with PCD patients. Many of the treatments have been tested on cystic fibrosis patients or patients with other respiratory conditions but have not been demonstrated to be effective in trials patients with PCD. An example of a clinical trial is the recent randomised control trial looking at the use of the antibiotic Azithromycin in patients with PCD. This was part of the BESTCILIA programme. More details can be found here http://bestcilia.eu/
What will I/my child have to do if they take part?
All research participation is voluntary.
Each study will have its own requirements. It is important you read the patient information sheet and ask any questions you may have before deciding whether you/ your child wants to take part. You may wish to consider:
- Number of visits
- Treatment changes
- Time it will take you
- Tests you will be required to undertake
If you are happy to get involved in a research study you will need to give informed consent, to show that you understand what is being asked of you and to confirm you agree to take part. You should not give your consent if you are unclear about any aspect of the research.
What benefits are there of taking part?
If you take part in a research study, you may have more contact with your PCD team which may improve your understanding of your condition and its management. In many cases, the research will not help you personally, but it may provide vital information that will help people in the future. This will be explained to you.
What are the risks?
In order to eliminate any risks to patients, all research must be approved by a Research Ethics Committee – a panel of health and social care professionals and members of the public with knowledge of, and/or an interest in research. Each study will be different and any known risks will be explained to you.
What if I have concerns?
Each study will have a patient information sheet with a named person for you to contact with any concerns. You can also contact your local Patient Advice and Liaison Service (PALS) with any concerns you may have.