Decentralising clinical trials sounds fantastic – people can take part in clinical research from the comfort of their own homes. Decentralisation lowers the barriers to taking part in clinical trials, meaning that more people can participate which could boost recruitment and improve the diversity of clinical trials. And by decentralising clinical trials, the costs of trials can be lowered, which means savings can be made and potentially more trials can be carried out.
However, decentralisation is also not as simple as it sounds. In the heavily regulated sector of medical research, new approaches like decentralisation must be clinically validated. Trials@Home began to explore how decentralisation could be achieved and what research needed to be carried out. In the context of the COVID-19 pandemic, this work became even more important.
By the end of the project, Trials@Home had compiled a robust set of recommendations to help clinical researchers design trials that participants can engage in from their own homes.
The core recommendations were as follows:
- Engaging stakeholders during trial design is essential in order to identify which activities to decentralise. The choice of activities that are decentralised may vary in line with the research question that each specific trial asks.
- A thorough and clear assessment of what is expected from participants must be carried out and the results communicated properly to would-be participants before the trial starts. For instance, expectations should cover what activities need to be performed, the data that needs to be collected, how digitally literate a participant needs to be and the amount of time that will be spent on the study.
- Assessment of decentralised elements is necessary regarding risks and benefits for patients, and ethical and regulatory acceptability must also be ensured. The Trials@Home project found that regulations differed depending on the Member State involved, and local variations were also a challenge. The advice from the project is to engage with regulators early on before starting a decentralised clinical trial, to ensure that it will comply with the correct rules.
- What previous experience does a site have with decentralised clinical trials? The capacity of any site must be assessed to identify any gaps that should be addressed before the trial gets underway, including any training. To help with the contracts of the trial sites, customisation of regional or national trial agreements might be needed. Fair site compensation for decentralised efforts should be a part of this customisation.
- Test and select technologies early. Practical measures like a bring-your-own-device (BYOD) validation, real-world pilot phases and support systems like helpdesks can help to ensure that the trial starts off on the right foot.
- Responsive support. When trial participants are responsible for data recording, using digital devices and other tasks related to data collection, they must receive additional support as needed. Trials@Home recommend that there must be the possibility for participants to have a real-time dialogue with someone who can help to answer their questions, in addition to other types of support. Trials@Home found that prioritising responsive empathetic and technology-enabled support empowers participants to carry out their trial activities confidently and effectively.
- Before starting the project, any new operational or logistical processes should be reviewed so potential risks can be identified and managed early. This helps everyone involved respond quickly if problems arise. For example, choosing several study sites within one country can reduce the risk of not recruiting enough participants or having sites that underperform. Logistics should also be carefully planned. Responsibilities for importing and exporting materials should be clearly defined, and full practice runs of the entire process should be carried out before the trial begins. These steps help spot and fix logistical weaknesses in advance.
- Clearly define who is responsible for what before the trial begins. In some cases, data collection, biological sample collection, and other activities may happen in less traditional ways and may involve participants themselves or third-party vendors. Because of this, roles and responsibilities need to be described in detail. Study sites should also have access to all the data they need to properly oversee the trial and carry out their responsibilities. Finally, communication channels between all parties should be clearly established ahead of time so that information can be shared quickly and efficiently during the trial.
- Strong data protection measures and secure digital infrastructures should be put in place from the beginning. This includes carrying out early risk assessments, clearly defining the responsibilities of any vendors involved, setting up secure ways for data to be transferred, and following relevant regulations such as the General Data Protection Regulation (GDPR). Decentralised clinical trials (DCTs) often rely more on digital tools, remote data collection, and third-party technologies than traditional site-based trials. Because of this, extra attention needs to be paid to protecting participants’ data and ensuring security. Particular care is needed when participants use their own devices or when data is stored in cloud-based systems, so that confidentiality, data integrity, and proper record-keeping are maintained throughout the entire trial.
For Mira Zuidgeest, the project coordinator of Trials@Home from UMC Utrecht, Trials@Home has moved beyond theory to practice by testing several decentralised approaches in a proof-of-concept model in 6 European countries. She outlined how the public-private partnership has been a crucial part of the project’s success.
“If we want to move to better evidence through more impactful, feasible clinical trials in Europe we need to work together with all actors in the ecosystem,” she said. “By co-developing a proof-of-concept trial, leveraging the quality, regulatory and clinical trial operations expertise and requirements from the private partners and challenging each other’s ‘normal way of working’ we made sure that the learnings and recommendations of Trials@Home are relevant and directly adoptable by both public and private trial sponsors.”
Decentralised clinical trials are only going to become more important in the future – the global decentralised clinical trials market size is expected to be worth USD 33 billion by 2033. The recommendations of Trials@Home will help to ensure that decentralised clinical trials are rolled out with maximum success in accordance with scientific research and the established best practices.
Although the project has come to a close, the work of Trials@Home will continue via the Trials@Home Interest Network. This network has been established to advance the field of decentralised clinical trials and it provides a forum for discussing questions and sharing best practises about how to implement decentralised clinical trials in the real world. The aim of the network is to stimulate the short term and long-term uptake of the recommendations and to leverage the network the expertise and the experience gained during the project. The network is voluntary and anyone who wishes to join the network can apply here.
Trials@Home is supported by the Innovative Medicines Initiative, a partnership between the European Union and the European pharmaceutical industry.