Blog – Two Worlds of Clinical Trials

If we put to one side self-driven research projects, the two main groups of clinical trials in which you might be asked to participate are those funded by charities or NHS-related funders such as the Chief Scientists Office in Scotland. These studies are usually led by senior academics within the UK. On the other hand there are studies funded by the pharmaceutical industry and often led by internationally recognised academics, based either in the UK or abroad, albeit there can be some overlap between how trials are classified.

There are, of course, some common features between these two groups of trials. Each will have carefully designed protocols clearly defining the intervention, the duration of exposure to that intervention, including a drug, and criteria to be met if someone is either to be included or excluded from the trial. If you choose to participate, there will be a target for the number of people you are expected to recruit and retain until the intervention has been completed.

Both commercial and academic sponsors tend to use a recruitment funnel to try and establish the feasibility of a local centre being able to meet the expected recruitment target.

Targets generated in this way are often wholly unrealistic.

I can recall on one occasion being approached by a senior researcher who told me that based on my local population and the expected number of people with dementia I should have no difficulty recruiting 400 to 500 people to a drug trial within a year. 
Notwithstanding the fact that my staffing levels would not support this magnitude of recruitment, this number exceeded the total recruitment to that trial from multiple centres and over a much longer period. A better measure is to look at how many people you recruited to a similar study in the past or to look for recruitment levels at other similarly-sized centres across the country.

The likelihood is that academically-led and commercially-sponsored studies will have different goals for their research. In the case of drug trials, academically-led trials are likely to concentrate on drugs already in use and try to address puzzling clinical problems, such as, for instance, which antidepressant might be more effective in the treatment of depression associated with dementia. Alternatively academically-led studies might look at the potential for re-purposing an existing medication, which might have been used in other conditions, but is no longer subject to a patent.

Commercial trials, on the other hand, are likely to be assessing the efficacy and safety of drugs which are not currently licensed for treatment. 
These studies might be examining the effectiveness and safety of different doses of the drug on the condition in question, the potential benefits and problems associated with longer term use or delayed start of the drug under investigation or even be the first use in humans, though those trials are usually carried out in specialised centres.


The use of a placebo is almost uniform in commercially-sponsored studies and not infrequent in academically-led studies and you must always discuss this openly with potential participants.

There is typically a considerable difference in the intensity associated with each type of trial. The duration of a clinical trial is likely to be much longer if commercially-sponsored and you should be prepared to support someone’s participation over a long period, even if deterioration is apparent.  Criteria for entry into commercial studies often includes very detailed assessment of the potential participant, including perhaps MRI and/or PET scan sometimes repeated on several occasions. Cognitive and other assessments used in commercial trials are often less familiar to those who primarily work in the clinical domain. 
Many of these assessments require intensive training and benchmarking against established standards. Although this might be anxiety-provoking, developing these new skills is rewarding and one must remember that in a clinica trial your role is to administer the tests and not interpret the results in the way that might be done by a neuropsychologist if used in clinical practice. In many trials, interpretation is often done remotely from your trial centre. Obviously, if there are issues of patient safety or immediate therapeutic importance these results can be utilised and become very useful, given that the test may not have been carried out had the participant not been in the clinical trial.

In academically-led studies the intervention is usually much shorter and perhaps more relevant to a current clinical problem. There may still be multiple assessments carried out but many of these are more likely to be familiar to those working in the clinical domain. MRI or PET scans might be involved depending on the clinical question and on the level of funding, but are much less likely to be required repetitively throughout the trial.

In either type of study it is extremely useful to liaise with other participating centres. Not only does this allow a forum for the discussion of problems which have arisen in your own centre, but the general level of camaraderie is always appreciated. Naturally, a little competitiveness over meeting recruitment targets is permissible.

Funding is of course also quite different in scale. The fees payable to your centre from the pharmaceutical industry will certainly help to support the level of staffing you require to undertake their study, but these staff invariably must be available at the start of the study and throughout, which can be a not insignificant risk to your organisation. You should also be aware that not only are the assessments more detailed but also the monitoring of your performance, your notes and your interpretation of findings, where appropriate, is going to be subject to monitoring at almost forensic levels by external companies. 
While essential for the running of the trials, some researchers find the intensity of this monitoring highly stressful. Obviously, monitoring and quality control is also in place in academically-led studies to ensure that data is robust and the trial conducted to high standards, but somehow the pressure does not seem so great.

Ultimately, both types of trials are essential if we are to expand the range of interventions we can offer to our patients and participation in each type of study can be very enjoyable and rewarding. If you are asked to participate, certainly think of doing so, but do talk to others who have experienced the pros and cons of participation before you decide to do so.

Dr Peter Connelly

Author

Dr Peter Connelly is a retired Old Age Psychiatrist who spent much of his career in Tayside, helping to establish clinical trials for dementia and neuroprogressive disorders in Scotland. Now working with the Scottish Neuroprogressive and Dementia Network, he combines professional insight with personal experience as a former carer. In retirement, he enjoys music, golf, and time with his grandchildren.

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