And why platform trials are the only way to move it forward
This is the first article of a brand-new series inspired by EU-PEARL (EUropean-Patient-cEntric clinicAl tRial pLatforms). The EU-PEARL project aims to shape the future of clinical trials, creating a framework for platform trials.
I am a psychiatrist. And after a few years of clinical practice in a variety of facilities, I have decided to dedicate myself to research.
I often discuss the status of psychiatric research in both formal and informal contexts. What strikes me every time is how psychiatry is perceived as something different from the rest of the medical world.
“Why are you doing it?” “Do you believe in it?” “Does it work?” are only some of the questions I am typically asked.
In this blog, I will talk about the status of psychiatric research, its unsolved issues, and the potential direction for the future.
A different medicine?
Psychiatry is the medical field focusing on the mind, as traditionally opposed to the other medical fields which are focusing on the body.
This historical separation between physical and mental health has often gained a negative connotation for research.
We could sum up the dilemma as: “If something is not in the body, it does not exist”.
Of course, this is a dangerous misconception, but this is where prejudicial beliefs take shape and become rooted.
These biased ideas are not unusual even among clinicians, healthcare professionals, and scientists.
This really hits a nerve and always comes to me as a huge source of personal frustration.
But it makes me question why the stigma around psychiatry is still there.
The main issue with psychiatry is probably that it still lacks a piece of hard evidence.
If we think about other diseases, blood tests or other exams are routinely used to confirm a diagnosis. In infectious diseases, we can usually measure the presence of a specific pathogen (the organism causing disease). For example, we all experienced that a throat/nose swab could detect the presence of the COVID-19 virus.
For most types of cancer, we have a combination of blood tests, scans (different images of the body), and biopsies (small samples of body tissues to analyse under a microscope). We can use this evidence for the diagnosis, to define the staging (how extended the cancer is), and the response to treatments.
In psychiatry we still do not have this.
For example, there is no psychiatric disorder for which we can perform a blood test or do a brain scan to confirm the diagnosis (yet).
Significant research has already produced promising results, but they still need further validation.
But, the fact that we don’t have this evidence now doesn’t mean that we will never have it, or that this evidence doesn’t even exist.
Let me give you an example of gastric ulcer, a break in the stomach lining, and a frequent cause of stomach pain. The shared idea was that stress or food could cause this condition. In 1984, two scientists demonstrated that it was actually a bacterium causing it. This discovery drastically shifted the approach for the management of this condition. We can now treat most cases of gastric ulcer with antibiotics.
I am not saying that I expect something similar will happen in psychiatry, at least not in the near future (as much as I wish).
In mental health, we are dealing with complex diseases. There may be numerous factors with a small effect, rather than a single cause with a large effect (such as in the gastric ulcer case).
Upsides and future directions
These challenges should not divert attention from the upsides of current psychiatric research.
We have validated instruments to conduct rigorous scientific research. These are interviews and scales, which are a structured list of targeted questions. We can use them to diagnose a disorder, or to assess the severity of symptoms and their change over time.
We also have effective treatments, either pharmacological or non-pharmacological, such as psychotherapy. This concept is important; the fact that they do not always work, does not mean they do not work at all.
Now, what can we do to produce better research in psychiatry and fill the gaps?
The answer to this question could be not only related to psychiatry; what if it encompasses the way we are conducting overall medical research?
Let me try to explain.
Clinical trials and platform trials
Currently, medical research is mainly carried out through clinical trials. These are, at their simplest, studies in which a specific intervention is tested in a specific medical condition. For example, a clinical trial in psychiatry could test how effective a pharmacological (drug) or psychotherapeutic (therapy) treatment is in a specific psychiatric disorder.
Therefore, clinical trials usually examine one single treatment in a single population at a time. This often results in long cycles before having significant results (either positive or negative).
In addition, different clinical trials on the same disease are likely to compete to find participants. At the same time, people who are suffering struggle to find the most suitable treatment for them.
A novel concept developed in the past few years aims to overcome these issues: the platform trials.
These are a platform to carry on different clinical trials without the time and space limitations of a traditional clinical trial. For example, we can assess different treatments at the same time for a given disease. We can include new treatments as they develop, and remove ineffective ones.
As a consequence, we will have a more efficient process to get significant results in less time and with fewer participants.
Almost three years ago, as part of a large European consortium, we launched a new project, EU-PEARL (EUropean-Patient-cEntric clinicAl tRial pLatforms). The main aim was to create an infrastructure to plan and complete platform trials in four different diseases. These included depression, a common psychiatric disorder. You can find more information in this piece, written by my colleague Courtney Worrell at the beginning of the project.
Since then, many things have changed.
We faced an unexpected pandemic. And can you guess which was one of the strategies employed to identify potential treatments?
That’s right, platform trials.
According to UK government data, platform trials helped in identifying eight effective treatments for COVID-19 so far (and managed to rule out ten ineffective ones). This is at a speed which may not have been possible by testing each treatment in a single clinical trial.
From this, we understand the advantages of platform trials, as well as the potential benefits for people suffering. And I could mention other promising examples, mainly from cancer research.
Advances in medical research are shifting the way we treat diseases towards a more tailored approach. This personalised medicine requires novel infrastructures such as platform trials.
We are now approaching the completion of EU-PEARL. This means we are gathering all the hard work of the past three years, and we will soon have the infrastructure ready to perform clinical trials in depression.
This is a critical first step for psychiatry to finally fill the gaps with other medical fields.
I believe as a society we are gradually overcoming the mind and body separation and recognising mental health as part and parcel of the whole individual’s health.
Platform trials are a golden opportunity to transform the way we conduct psychiatric research, and (perhaps) the only way to move it forward.
DISCLAIMER: This article reflects the author’s view. Neither Innovative Medicines Initiative (IMI) nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained therein. The EU-PEARL Project has received funding from the IMI 2 Joint Undertaking (JU) under grant agreement No 853966. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and CHILDREN’S TUMOR FOUNDATION, GLOBAL ALLIANCE FOR TB DRUG DEVELOPMENT NON PROFIT ORGANISATION, SPRINGWORKS THERAPEUTICS INC.