Towards greater digitization in clinical trials?
Clinical trials are used to ensure the efficacy and safety of new drugs and study new uses of drugs already available on the market. The clinical trials phase represents, on average, about 57.1% of the costs of developing new drugs. It is estimated that these costs have been increasing in recent decades due, among other reasons, to the type of drugs under study and increased regulatory compliance scrutiny [1,2]. Furthermore, the current pandemic has imposed additional challenges to the conduct of clinical trials already underway and to the start of new studies. In addition, the rigid social distancing rules and the fact that clinical trial participants are generally part of populations more vulnerable to COVID-19 caused many of these trials to be temporarily interrupted [3,4].
To mitigate the disruptions caused by the pandemic and allow the continuation (or the start) of clinical trials related to coronavirus or rare diseases and serious diseases, without adequate treatment alternatives, the European Commission (EC) issued guidelines that include some flexibility in the trials. However, it does not compromise the safety of the participants nor the quality of the studies. For clinical trials in progress, for example, the possibility of in-person visits to patients being replaced by telephone or video call follow-up was created. Similarly, test quality monitoring activities, traditionally done on-site, can be carried out remotely. However, the EC clarifies that this relaxation is temporary and will cease once the pandemic has passed. Additionally, the European Union Regulation (No. 536/2014) on clinical trials of medicinal products for human use - which repeals Directive 2001/20/EC regarding the simplification and harmonization of clinical trials in the EU - does not deal with issues related to the digitization of clinical studies. Based on these guidelines and regulations, we could deduce that the future of (partially) remote clinical trials in the EU would be compromised.
The recent guideline of the European Medicines Agency (EMA) on the use of computer systems and electronic data in clinical trials, which is in public consultation from June 18th to December 17th, 2021, shows, however, that there is an apparent effort to modernize the clinical trials paradigm in the EU. In this document, the EMA provides guidelines that should be followed to ensure the quality of data collected in the trials and to ensure the privacy, safety, and well-being of study participants. Electronic informed consent and data collection through wearable devices are some procedures that can revolutionize clinical trials and which are mentioned in the guideline.
The benefits of digitization in clinical trials are vast. As an illustration, the digital recruitment of trial participants increases the ability to reach populations traditionally underrepresented in studies, contributing to greater robustness of the results [5,6]. In turn, if they are adequately tested for the purpose, wearable devices offer the opportunity to remotely aggregate a large amount of data that would not be collected in the occasional face-to-face follow-up visits. In theory, these data provide a better understanding of the heterogeneity of responses to the treatments under study, increase the efficiency of clinical trials and reduce costs .
However, it should be noted that despite all the optimism placed on digitizing and conducting remote clinical trials, more evidence is still needed to understand its actual impact on trial costs and efficiency . On the other hand, something that will undoubtedly help to reduce costs and encourage the conduct of international clinical trials in the EU is the harmonization provided for in the European Union Regulation (No. 536/2014), and the need to submit a single application for authorization for one trial - instead of multiple similar applications in different EU countries. It is hoped that, with this simplification, new drugs will reach those who need them more quickly. However, it remains to be seen how the potential reduction in the costs of clinical trials, and therefore the cost of developing new drugs, will affect drug prices in the EU.
(The Nova SBE Notes column contributes to the reflection in the health area by the members of the research center Nova SBE Health Economics and Management Knowledge Center. These are opinion articles under the sole responsibility of the authors.)