A Patient-Centered Approach to Site Support
In the challenging landscape of rare disease clinical trials, success often hinges not only on robust science and patient-centered protocol design, but also on precise operational execution at the site level. Complex trial designs, sporadic patient enrollment, and specialized care requirements can create significant hurdles for research sites conducting these critical studies.
Unique challenges of rare disease trials
Rare disease trials face distinct operational challenges including:
- Requirements for investigative staff support. Site staff may face steep learning curves with complex protocols. Since enrollment is often sporadic, resources need to be allocated toward retaining knowledge, minimizing loss of interest, and mitigating variability between patients. Over time, staff turnover and transitions further complicate continuity.
- Need for patient and care network support. Patients with rare diseases and their care networks have often navigated a long journey to diagnosis with few, if any, treatment options. In the context of a clinical trial, it is critical to communicate study expectations clearly and to provide support throughout the study experience.
- Management of multidisciplinary teams. Multiple touchpoints across different specialists and care level transitions require careful, customized coordination and comprehensive training.
- Setup of academic or research hospital settings. These settings may have internal standard operating procedures (SOPs) and policies that do not align to support the conduct of clinical trial protocols. In addition, the physical layout of these hospitals may mean that study activities are dispersed across different locations.
- Need for inpatient or complex care. Studies requiring inpatient stays significantly impact patients and their care networks, so minimizing participant burden and the number of “unknowns” associated with the clinical trial journey is critical. Further, implementing research protocols within standard care practices can often present logistical challenges.
These challenges are compounded by practical issues such as restrictions on access to imaging, specific guidelines for advanced therapy medicinal products (ATMPs), scheduling constraints for operating room or procedure spaces, and the need to align study requirements with standard hospital procedures.
Supporting sites so sites can support patients
Addressing these multifaceted challenges requires a comprehensive approach to site support that places the patient at the center, surrounded by three interlocking strategies (see Figure 1):
Site Support Management Team
At Ergomed, our Site Support Management Team is a group of dedicated research specialists focused on providing patient-centric and real-time support to investigational sites. Key activities of this team include:
- Supporting sites in first patient visit preparation and scheduling
- Developing site-specific study execution maps (SSEMs) aligned to the schedule of assessments and any associated non-protocol-driven activities that could significantly impact clinical trial conduct
- Supporting sample processing compliance
- Overseeing visit schedule compliance, treatment adherence, and caregiver diary completion
- Assisting study site coordinators (SSCs) with patient and care network support
- Tracking supplies and re-supply needs
- Facilitating and tracking sample shipments
- Consulting on recruitment progress, patient retention, study procedure timelines, and data quality
- Supporting management of the investigational medicinal product (IMP)
- Assisting with implementation of patient engagement initiatives
- Notifying key stakeholders of any site issues detected and initiating corrective actions when necessary
The assigned Lead Site Manager develops a site management plan (SMP) outlining study-specific site management activities, with an initial emphasis on first patient and first treatment visits at each site. Further activities will be dynamically adjusted from site-to-site based on individual input from each site regarding what additional support and oversight would be beneficial.
Country or regional site managers are accountable for executing the SMP and enhancing both positive site relationships and overall site compliance. These site managers work hand-in-hand with clinical research associates (CRAs), sharing common objectives of study data quality and compliance with Good Clinical Practice (GCP).
This continuous administrative and logistical support minimizes site burden and maintains ongoing site motivation, allowing research staff to focus on patient care and protocol implementation.
Study Physician Support
The study physician support component establishes valuable medic-to-medic relationships that inform and facilitate trial execution through:
- Peer-to-peer dialogue on patient populations and inclusion/exclusion criteria, study challenges, and medical issues
- Sharing of experiences to improve protocol design and implementation
- Training on medical issues for the project manager and study site teams
- Motivational impact on investigators
This collaboration helps bridge the gap between the study protocol and practical clinical implementation.
Patient Support Services
Patient support services focus directly on minimizing study burden and enhancing the experience of patients and their care networks. These services run the gamut from providing clear, comprehensive information and reminders about the study and visit logistics to arranging home nursing support, facilitating travel arrangements and timely reimbursement, and creating welcoming environments.
These services acknowledge that patient retention in rare disease trials depends heavily on minimizing participation barriers and creating a supportive research atmosphere.
Impact on trial performance and patient experience
When implemented effectively, this comprehensive site support model delivers tangible benefits:
- Improved patient recruitment through direct access for site staff with questions
- Enhanced retention through comprehensive support services
- More evaluable subjects through improved protocol adherence
- Optimized investigator performance through peer-to-peer support
- Proactive sharing of best practices across research sites
- Early identification and mitigation of site-specific risks
Most importantly, by placing the patient at the center, this approach recognizes that successful rare disease trials must account for the human experience of participation, not just the scientific and regulatory requirements.
Key Takeaway
Complex rare disease studies require differentiated approaches that go beyond traditional monitoring and oversight. Deploying a comprehensive model that addresses the study- and site-specific challenges of these trials by providing tailored strategies to optimize the performance and motivation of sites, study physicians, and patients and their care networks can significantly improve execution and outcomes.