Last October 2021 EFPIA organised a multistakeholder workshop on "Accelerating Adoption of Complex Clinical Trials in Europe and Beyond"  with the ambitious aim to identify solutions to some of the challenges seen today when conducting complex clinical trials (CCTs). The objective for increasing understanding of CCTs across stakeholders was achieved and very useful feedback was given by the different stakeholders to potential solutions and next steps, which are currently being considered.

This year, our objective is to organise a workshop that builds on that success and facilitates a multistakeholder dialogue on the use of Digital Health Technologies in clinical trials, including digital endpoints, and to identify opportunities for optimising regulatory pathways and bringing clarity to some of the challenges faced so far.

Modernising Clinical Trials in Europe

It is obvious that there is currently in Europe a number of interesting initiatives relating to the use of new technologies and innovative clinical trial designs in drug development, run either by IMI consortia or by the EU commission and regulators. Not only there is the need for science driven answers to support the use and acceptance by regulatory authorities and other stakeholders of innovative approaches, but also there is value to ‘connect the dots’ and find synergies. EFPIA welcomes the launch of the EMA Accelerating Clinical Trial Initiative (ACT EU) and of the Regulatory Science Research Needs Initiative by EMA and the Commission with the release of a list of regulatory science topics that need further research. These efforts will be key to close gaps and improve medicine development and evaluation to enable access to innovative medicines for patients.

Through the Innovative Medicine Initiative (IMI), some public-private consortia, such as MOBILISE-D and IDEA-FAST, are looking at developing and qualifying digital outcome measures in various disease areas; another ambitious IMI project is Trials@Home which explores the potential of digital technologies for use in decentralised clinical trials. All these projects should dramatically improve the efficiency of clinical trials, facilitate a patient-centric approach in drug development, and ultimately speed up the development of new treatments for the benefit of patients. Similar to IMI, its successor – the Innovative Health Initiative (IHI) - is also dedicated to advancing research and innovation but working across a broader range of sectors, to support truly cross-sectoral projects involving the biopharmaceutical, biotechnology and medical technology sectors, including companies active in the digital area.

Digital Health Technologies in drug development: many opportunities and a few challenges

For over 20 years, we have been advancing the digitalisation of clinical research. The current increase in the array of Digital Health Technologies (DHT) used offers many additional opportunities: they can facilitate the clinical trial experience for patients, allowing patients to participate in trials from their own home; they can improve our ability to detect meaningful change through objective, granular, longitudinal sensor-based measurements; they can help measure the patients’ lived experience as patients can be monitored and measured while performing daily activities; they can help address “unmet measurement needs”; and they can potentially reduce trial duration and size, by inferring more precise and sensitive outcome measures from data collected through DHTs.

Nevertheless, to exploit the true potential of DHTs in drug development we need to find common solutions to some of the challenges we face: current regulatory frameworks are not fit-for-purpose, and are very lengthy and resource intensive; evidentiary requirements are not always clear for innovative DHTs and for digital measures that do not clearly fit under the standard Clinical Outcome Assessment or Biomarker categories. Furthermore, there is lack of Health Authority (HA) harmonisation on the evaluation of DHTs and Digital outcome measures that needs closer HA collaboration and alignment; including alignment between HAs and Notified Bodies, and between regulators across regions. Finally, we do not have enough platforms for multistakeholder collaborations beyond IMI and IHI.

As shown by IMI, partnership in drug development is more than ever crucial, as individual sponsors cannot solve the challenges they faced on their own. Indeed, as Dr Pierre Meulien (the Executive Director of IHI) said, ‘IMI is a partnering machine that has transformed the biomedical ecosystem in Europe.’ This is why the Sub-Group on Digital Endpoints (jointly led by the EFPIA Clinical Research Expert Group –CREG- and the EFPIA Digital Health Working Group –DH WG) is organising a multistakeholder workshop on 8&9 November 2022. The program committee work will kick off in May, to identify the agenda of the workshop.

Stay tuned, as the agenda and registration will be shared shortly. We hope, you will join the workshop, share experiences, and have interactive and in-depth discussions on some of the key challenges when using DHTs in drug development. But most importantly, we look forward to identifying solutions to these challenges and laying out a roadmap for the next 2-5 years to accelerate the momentum in Europe, TOGETHER.