Taking part in clinical research
Hopkins C, Surda P, Walker A, Wolf A, Speth M, Jacques T, et al. EPOS 4 patients. Rhinology. 2021 Suppl. 30: 1-57.
Patients are an essential part of clinical research, and not just as trial participants but increasingly are involved with designing clinical studies and helping to implement their findings.
Helping with the design of clinical research studies
In the same way that we are trying to involve patients in all choices regarding their care, by providing all information regarding risks and benefits and reaching shared decisions, researchers are increasingly involving patients in the design of research projects and trials. This helps to ensure that the treatment or test being investigated is something that patients want, need and would accept as a treatment, that the study is performed in a way that patients would feel comfortable taking part in, and that the answers provided by the research project address issues most important for patients. Put simply, it helps ensure that research is worthwhile from a patient point of view.
How can patients get involved in research?
Patient involvement in research design can involve lots of different methods, for example;
Being interviewed about how your illness affects your quality of life in what ways current treatment to identify topics for future research
Looking at a list of research ideas and saying which ones are the most important
Being shown examples of different trials and telling an interviewer your thoughts – whether you would be willing to participate, any concerns or questions you have about the study design
Filling out questionnaires to help decide what outcomes (questions) should be measured in a study
Reading patient information sheets to make sure that they are easy to understand
Taking part in helping to design research studies usually involves giving up some of your time, and sometimes travel but do not involve undergoing treatment. They are usually voluntary, although costs of travel are often refunded, and you will often be given refreshments if you are spending a long time at a meeting. In some cases, you might also be paid for your time.
You might be asked to take part in small group workshops, to take part in telephone interviews or fill in written or email
surveys – the team will explain how they will do this so you can
decide if you are comfortable in that setting. The team will likely be able to make adjustments for any special needs and will also understand that for many patients it can perhaps be a little intimidating sharing your views in a group, and will be there to make everyone feel comfortable and to give everyone time to have their say if they want.
You are of course able to say no if you are asked to take part – there is usually no direct benefit to yourself, but many patients enjoy the chance to help change future research, and some go on to play a large role in research projects – most big trials now have a patient representative on the trial team, and a patient panel that feeds into the trail on a regular basis.
It is important to remember that when you are giving your opinions on study design, there are no wrong answers. Your experience as a patient is actually often far more useful than 20 or 30 years experience as a researcher – and it’s important that you feel able, to be honest. Patient input can really help save researchers from making mistakes that would make their study worthless, as patients often quickly spot problems that researchers have overlooked, so they will really want to hear what you think, good or bad - not what you think they want you to tell them!
Taking part in a clinical trial
There are many ongoing research projects that aim to develop new treatments for CRS or to work out how to improve the effectiveness of current treatments and your doctor may ask if you wish to join a study.
There are different types of trials and your team will carefully explain what is involved, provide you with written information to take away to read through with friends and family, and will give you time to consider carefully if you want to participate before making a decision. Many patients are keen to take part in trials but not everyone will want to do so. There is no obligation to take part and your medical care would not be affected if you say no.
Observational studies follow patients over time, often to study how a disease changes over time. Patients will often be asked to complete questionnaires or investigations on a regular basis. There is usually very little risk in taking part in observational studies, other than giving up your time, and often patients benefit from being monitored more closely than normal.
Clinical trials are usually run to test new treatments or to find out which of two or more treatments are more effective. Trials are performed when there is no pre-existing evidence that one treatment option is better or worse than another, or in the setting of new treatments, no evidence that it is better or worse than doing nothing or continuing with standard care.
Patients are often divided into 2 different groups who receive different treatments, and the allocation to a treatment group is usually randomised (often by a computer that ‘tosses a coin’ to decide). Studies may be placebo-controlled, where one group gets a new treatment, but the other group gets an inactive version, and in these trials, both the doctors and patients may not know which treatment they are getting (this is called a blinded study). In most trials the numbers in each treatment group are the same – that is the chance of getting one group or the other would be 50:50 but sometimes there is a higher chance of getting one treatment than another, or there may be more than 2 different groups.
Keeping patients safe during research
Clinical trials must comply with very high standards of care to maintain the safety of patients taking part. Trials are very
carefully assessed before they can start to recruit patients and steps are put in place to minimise any risks. They are closely and regularly monitored. There will always be a contact person who can be reached in case of any problems, and in blinded studies, it is possible to find out what treatment has been given in case of emergencies.
Patients may withdraw from a study at any time if they suffer any side effects if they feel that their condition is not improving, or simply if they feel they no longer want to take part. Some trials will allow patients to move between treatment groups, while others will allow patients to receive active treatment after the trial is completed if they have received a placebo drug. Your doctor will be able to tell you all about this in advance. Your doctors first priority is always your well-being and safety, and your care will not be affected if you withdraw from a trial.
What are the benefits of taking part in a clinical trial?
You might be given access to new treatments that are not otherwise available
You will often receive more frequent check-ups, either by phone or in-person – and this means that patients taking part in trials often get better improvements in their health even if they don't receive the investigational treatment
The trial may lead to the development of new treatments that you and other patients may benefit from in the future
You may sometimes be rewarded for taking part – although most trials are voluntary.
What are the risks?
The new treatment may not be effective and in some cases may cause side effects. Known side effects will be discussed during the process of informed consent, but with new drugs, there may be some unknown risks
You may not receive any treatment (watchful waiting) or in other cases receive a placebo drug – an inactive drug designed to look the same as the investigation treatment.
You may have more visits to the hospital than would be required if having the standard care.
What happens next?
You will usually be given a detailed explanation and written information sheet, and then some time to carefully consider whether you wish to join the study. The research team will need to check that you meet the inclusion criteria for a study and that you don't meet any of the exclusion criteria – for example, studies may be looking at only certain types of CRS, and may exclude patients who are already taking certain medications or have other associated conditions.
If you meet the criteria and wish to take part you will be asked to sign a consent form, and will then be allocated to treatment. Your team will let you know what this involves, how often treatments and study visits are required, and how long the study continues. They will also let you know what happens at the end of the study and will usually keep you up to date with the results of the study
What do you need to know before you make your decision? The Patient Information Sheet should include all the information you need in order to decide whether to participate. It should cover all of the following questions, but you may want to ask your doctor/research nurse if you still have any questions
What is this study trying to find out?
What treatment or tests will I have as part of the study and will they hurt? Will you give me the test or lab results?
What are the chances I will get the experimental treatment or the placebo, and will I know what treatment I am having?
How will this be decided?
What are the possible risks and benefits of the study treatment compared with my current treatment?
How will I know if the treatment is working, and what happens if it does not work?
How long will the clinical trial last?
Where will the study take place?
Will you provide a way for me to get to the study site if I need it?
Will being in the study cost me anything? If so, will I be paid back for expenses such as travel or parking?
Will I receive any other payments for taking part in the study
How will the study affect my everyday life?
What steps are taken to ensure my privacy?
How will you protect my health while I am in the study?
What happens if my CRS gets worse during the study?
Does the study allow me to change the treatment group?
Can I take my regular medicines while in the trial?
Can I take emergency medicines while in the trial such as antibiotics?
Who will be in charge of my care while I am in study?
How will you keep my doctor informed about my participation in the trial?
If I have to withdraw, will this affect my normal care?
Will you follow up on my health after the end of the study?
Will you tell me the results of the study?
Whom do I contact if I have more questions?
Patients’ experience of both taking part in a clinical trial and involvement in trial design
Patient 1 reports:
“My first personal experience of clinical research was when I was diagnosed with bladder cancer more than ten years ago. I underwent surgery and it was then explained by my specialist nurse that a course of treatment stretching over the following three years would be needed, involving some 24 visits to the clinic. Added to this was a mention of a research project which was looking at the effectiveness of additional medication in the form of specific vitamin and mineral supplements, and would I like to take part. Being recently retired and no longer under time pressures, I agreed and was given an information sheet about the work.
A meeting was arranged with the research nurse who took me through the background to the study and that the trial involved taking either the medication or a visual placebo which neither I nor she would know which I received. I signed the consent form and was given a diary in which to record the taking of the medication and a prescription for the first set of medication and sent me off to the hospital pharmacy. This was not a good start since it involved more than an hour wait to receive it! I also quickly found that the daily diary rapidly became a chore, since it consisted of box-ticking for taking the medication and experience or absence of a range of symptoms, which were almost invariably absent. However, at the next hospital visit, I was told that the diary couldn’t be changed, but on the positive side, I would receive further medication directly from the research nurse. Some further discussion enabled aligning future appointments with those for my mainstream treatment, hence saving extra journeys into the hospital. My participation continued for over twelve months, but then I received a phone call from the nurse to tell me that the trial was being halted early and I should stop taking the medication. Rather to my surprise, I heard nothing more.
This may sound like a somewhat negative experience, but it provided some fruitful learning for when I became a patient representative on a team designing a CRS treatment research project. I was fully involved in designing the trial to give the best patient experience, including its organisation, information leaflets, diaries and communication plan, and that too provided some further learning. For instance, by chance I underwent one of the procedures involved in the trial at the time the patient diary for it was being finalised, so was able to test it first hand and provide user feedback. Clearly, any opportunity to “road test” documentation and diaries on actual patients during planning for a trial should be explored and implemented. Another learn- ing experience was in the compilation of the patient information leaflets. These followed standard templates required by the sponsoring research centre, which, from my experience of standardisation in an industry I fully supported. However, the need for some degree of flexibility to accommodate different types of trial and minimise repetition and irrelevant information was clearly needed in order to produce the most concise and digestible document. All learning which can feed into and improve the next trial.”
It’s clear that this patient’s first experience of taking part in a trial, over 10 years ago was not a very positive experience. The involvement of patients in trial design now helps to ensure that trial visits are kept to a minimum, and that completing diaries and other questionnaires is made as simple as possible – usually with options to reply by text, by email or using paper diaries, as everyone is different. Sometimes trials allow access to new treatments, but rarely, as in this case, trials are stopped ahead of schedule if it becomes clear that the treatment either works very well or doesn't work at all. Nowadays, patients will be kept informed at all stages of the trial if there are any changes and will be sent a report after the study has finished if they would like to know the findings. The participant below has had a much more positive experience and we certainly hope that this is now what can be expected by anyone taking part in the research.
Please remember that the decision as to whether you wish to take part is yours and it will not affect your care if you choose not to or change your mind at any point. However, it can be rewarding and is an essential step to developing better treatments that you might benefit from in the future.
Patient 2 reports:
I was asked to take part in a clinical trial in 2017. I was very apprehensive at first as I had very little knowledge of clinical trials. From start to finish I felt very supported by my consultant and the research nurse, who was at the end of the phone whenever I had any queries and was very attentive throughout the whole clinical trial.
I was given an information pack that clearly outlined when I would be required to take the injection, what dates I’d be seen in the clinic for tests etc and what the plan was for the whole duration of the clinical trial.
A slight disadvantage is not knowing whether you are receiving the drug or the placebo. However, through undertaking clinical trials you do your bit for science and improve the chances of having access to a drug that will help you and others in the future. I had an excellent experience overall and would highly support and encourage others to take part in clinical trials.