The COVID-19 pandemic accelerated several digital developments in health care, such as remote patient monitoring and electronic informed consent, both critical facilitators in what seems to be the next step in drug development: decentralised clinical trials.
Since January 2022, the European Commission, the European Medicines Agency (EMA) and the Heads of Medicines Agencies group (HMA) are united in the ACT EU initiative.
Through this initiative, these partners intend to promote the EU as a place for clinical trials and transform the way trials are conducted. This transformation may happen through decentralisation – particularly relevant for rare diseases, as clinical trials for rare diseases are notoriously difficult with low numbers of patients in any one country.
The main idea behind decentralising is that clinical trials no longer need to be in one place. Instead of travelling to a central location, patients will receive treatment in their local hospital – or self-administer at home, with drugs shipped to patients directly. Decentralising clinical trials makes particular sense in the EU as it connects 27 distinct healthcare cultures with their own requirements and paperwork.
Apart from attracting pharmaceutical innovation to the EU, the hope is also to reach those demographics of patients unwilling or unable to travel to a centralised location, as well as reduce the drop-out rates of those who participate (Taylor, 2022).
The question, of course, is how to do successfully achieve this objective. How to best manage the data, monitor the trial, arrange the informed consent, anonymise the data, etc.? ACT EU has recently published guidance on this, acknowledging that many clinical trials already encompass digital elements and providing future directions (ACT EU, 2022).
Most of this guidance focuses on privacy issues, which is indeed critical. However, ‘place’ is an often-ignored factor in data collection. It is also easy to see how it may affect patient experience and even trial outcomes. For instance, patients can stay close to their families when they don’t have to travel and stay in an uncomfortable hotel. Patients may also be less inclined to finish a course of a particular medicine when at home, and the care received from their doctor on the other side of the screen may be of a different quality.
In a way, decentralising clinical trials might better predict medicine effectiveness in the real world. As the place of data generation is now so much more ‘realistic’, this begs the question: how different is this from what is known as real-world evidence, generally a term reserved for data collected post-trials?
This novel way of organising data generation and collection clearly blurs boundaries between research and care, like many social pharmaceutical innovations do. It will be vital to see how place affects data and how these data get valued in decision-making.
ACT EU (2022), RECOMMENDATION PAPER ON DECENTRALISED ELEMENTS IN CLINICAL TRIALS. Accessed 7 February 2023. https://health.ec.europa.eu/system/files/2022-12/mp_decentralised-elements_clinical-trials_rec_en.pdf
Taylor, P. (2022). EU regulators issue guidance on decentralised trials. Pharmaphorum. Accessed 7 February 2023. https://pharmaphorum.com/news/eu-regulators-issue-guidance-on-decentralised-trials/?utm_source=pharmaphorum+Daily+Newsletter&utm_campaign=32bff41654-EMAIL_CAMPAIGN_2019_09_24_10_10_COPY_01&utm_medium=email&utm_term=0_a54496134b-32bff41654-443823458
Written by: Tineke Kleinhout-Vliek, PhD