Thursday, November 6
8:15 am Registration & Morning Coffee
8:50 am Chair’s Opening Remarks
Comprehensive Strategies for Paediatric Rare Disease Trials: Engagement, Retention, & Transition Solutions
9:00 am Putting Youth at the Center: Best Practices for Pediatric Engagement
Synopsis
- Learning how to authentically engage pediatric patients as active contributors in clinical research and innovation
- Discovering strategies to create inclusive, age-appropriate materials, consent processes, and engagement tools that reflect young people's needs and voices
- Understanding how a structured youth feedback loop, including iCAN’s Seal of Approval, ensures how materials and initiatives meet pediatric standards and expectations
9:30 am Improving Paediatric Trial Compliance & Retention by Supporting Caregivers
Synopsis
- Targeting and engaging parents effectively as primary decision-makers to boost awareness and trust in paediatric rare disease trials
- Providing tailored support to manage caregiver time constraints while ensuring critical safety monitoring without added burden
- Highlighting effective caregiver support programs that reduce dropout rates and improve patient retention
10:00 am Session Reserved for Medical Research Network
10:30 am Morning Break & Networking
Innovating Trial Design: Enhancing Patient Inclusion & Flexibility in Rare Disease Trials
11:15 am Partnering with Patients: Co-Creating Clinical Trials to Shape Smarter Trial
Synopsis
- Engaging patients and caregivers from the outset to co-develop protocols, endpoints, and eligibility criteria
- Integrating patient insights to drive more inclusive designs, increase trial relevance, and boost enrolment
- Avoiding misalignment by validating assumptions early and embedding the patient experience in study planning
11:45 am Operationalizing Strategies for Inclusive & Representative Clinical Trials in Rare Disease Research
12:15 pm Lunch Break & Networking
1:15 pm Decentralized Trials in Rare Disease: What’s Working & What’s Not?
Synopsis
- Which aspects of decentralized trials (e.g. home nursing, remote monitoring, eConsent) are proving most viable in rare indications?
- Where do challenges remain – such as diagnostics, sample collection, or maintaining data integrity?
- How can we strike the right balance between virtual convenience and medical oversight?
Boosting Trial Completion Rates with Fair Compensation & Patient-Centric Support Strategies
1:45 pm Specialised Retention Strategies for Rare Disease Clinical Trials
Synopsis
- Exploring key barriers to long-term engagement and how to address emotional, logistical, and clinical challenges unique to rare disease populations
- Examine successful models of decentralisation, flexibility, and patient support that reduce burden and prevent drop-out
2:15 pm Afternoon Break
2:45 pm Planning for Retention: Embedding Reimbursement & Support Strategies into Rare Disease Trials from the Start
Synopsis
- Embedding patient-friendly reimbursement workflows into trial design from day one to avoid costly mid-study changes
- Managing cross-border reimbursement challenges, including banking limitations and regional regulations
- Evaluating the role of concierge services and home nursing to support retention, and the complexities of adding these services mid-trial
- Reducing stress for patients and families by streamlining communications and minimizing administrative burden
3:15 pm Designing Fair & Flexible Compensation Models to Maximize Trial Participation
Synopsis
- Assessing patient financial burdens beyond travel, childcare, lost wages, and more
- The voice of the patient and caregiver as critical factor in shaping clinical meaningful endpoint selection
- Balancing ethical considerations with practical incentives to encourage participation
- Collaborating with payers and regulators to ensure compliance, transparency, and meaningful outcomes