Innovating Paediatric Clinical Trials: Enabling Participation of Children with Complex Needs Through ondemand and mobilehealth.

The Challenge

mdgroup supported a groundbreaking initiative in paediatric clinical trials, focusing on children with SCN2A-DEE and SCN8A-DEE, which causes a range of neurological conditions, including severe early-onset epilepsies, encephalopathies, autism spectrum disorder and movement disorders. As a result, the study faced unique challenges due to the complex medical needs of the participants. Many with this indication rely on wheelchairs and ventilation equipment, require continuous feeding via PEG tubes and have poor access for blood draws, all of which required managing along with logistical hurdles associated with participating in a decentralized clinical trial.

The trial started with ondemand (travel and reimbursement) services and mobile health at some visits. As the client became familiar with the challenges of travel for many families due to the participants’ condition, they requested a pivot to a fully decentralized clinical trial (DCT) with the option for all visits to take place remote from the site. This required mdgroup coordination of telehealth calls with the site at each visit, direct-to-participant (DTP) shipment of IMP from the study site to participant locations, in-visit activation of eDiaries shipped from the site and study visits lasting up to 8 hours with a RN and MD. Procedures required to be conducted remotely included vital signs, weight and height, ECGs, collection and processing of safety and lab samples (blood and urine), physician-led physical and neurological examinations, oversight of telehealth calls, eDiary set-up, questionnaires, IMP dosing and observation.

The use of ondemand services was still requested for some of the participants whose family either chose to attend the site or for whom visits could not be conducted in their home location. This required special considerations when travelling, given the medical challenges for the participants. Furthermore, as many participants are young children, they often would be travelling with their whole family and other siblings (sometimes even a pet).

The Solution

mdgroup implemented a range of innovative solutions to meet the client and participants’ needs.

The study team worked very closely with the sites to determine the individualized process that worked best for their site-required procedures. The Clinical Managers and study RNs were in close contact with the caregivers of the participants and the HCPs to determine any scheduling preference and the Navigator – Project Coordinator worked with the courier to schedule sample collection and IP delivery and return to the site for accountability. This ensured all visits had efficient scheduling and quality execution of study procedures for participants.

The IMP was monitored with real time GPS tracking that includes temperature and humidity monitoring to ensure usable IP or repeat shipments to mitigate any possible delays in IP administration.

Through mdgroup’s internal resourcing division seacolehealth, the team created a global network and found specialized registered nurses (RNs) and medical doctors (MDs) in nine countries. The resourcing was tailored to the specific needs of each participant taking into account the information provided to the mdgroup team by the site and the caregivers. This ensured that participants worldwide got access to the expert care they needed, no matter where they were.

Equipment and ancillary supplies for this study were tailored to the study population and requirements and were all sourced and distributed by our in-house supply and logistics division horreum. Equipment for challenging blood draws included vein finders, BUZZY® and additional paediatric supplies to assist with the most difficult blood draw challenges for the nurses.

Two families required travel from the UK to a suitable location in the EU due to client ethics approvals, to enable the visit to be conducted remotely. Although travel was still required, the travel was significantly less extensive than if the participant travelled to the site, and every step was taken to reduce the burden on the families as much as possible. They then required mobilehealth providers sourced in the area they travelled to, instead of in their home country. The mdgroup team provided seamless travel, accommodation, and equipment needs for these families. Additionally, the team supported the relocation of an entire family from their home country to the site. This required the coordination of long-term accommodation that met the family’s needs. Along with ensuring that the child had access to all of their specialized equipment and medications by working closely with the study site.

The operational and commercial teams partnered with the client to look at the unique set up of this study (3 sites in the US and Spain drawing participants from 10 countries) on an evolving basis to determine the most efficient way for change orders to occur, while ensuring costs are covered but there is flexibility in scope. This model will help future projects be set up in a way that is beneficial to both mdgroup and the client. The model will consider which services need to be done proactively to scope a new country and which can be done under a “just in time” agile project management model.

The team put approval processes in place internally and externally to approve unscheduled visits as soon as possible, to not delay eligibility and enrolment decisions. This was a crucial factor, as participants were required to come off their anti-seizure medications for study eligibility – this often leads to an increase in seizures during this period and therefore it is critical to keep this time to a minimum.

The Outcome

The implementation of these innovative solutions led to significant achievements. The team has gone above and beyond to meet the challenges on this project. Each participant and visit were treated as a unique challenge and the cross-divisional communication has been instrumental in providing the white glove service that mdgroup is known for.

With this complex study successfully implemented, mdgroup facilitated decentralised visits in 9 countries and 6 US states, enabling maximum participation and retention in this key trial, and providing a safe and effective option to address the significant travel restrictions, whilst ensuring the collected data was of the highest possible quality. Although this study came with many challenges, we are proud of the results. The client praised the pivotal role mdgroup’s services played in trial enrolment and expressed interest in future collaborations, along with expanding services for these participants.

When the study started, many of the identified participants were not going to be able to participate due to the requirement to travel to the site. The ability for mdgroup to pivot to a DCT option has allowed children to participate in this important trial. The team has onboarded some amazing HCPs (Health Care Professionals) who have been able to use their specialized skillset to further this research. This allowed children with complex medical needs to participate in the trial, contributing to advancements in paediatric research.

This project exemplifies our commitment to innovation and participant-centricity in clinical trial management. By leveraging specialized expertise, agile project management, and technological solutions, we successfully addressed complex challenges and facilitated the conduct of a pioneering paediatric clinical trial.

 

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