- The UAE as a prominent hub for research and clinical trials, driven by a focus on healthcare innovation and patient-centric approaches
- The UAE’s comprehensive regulatory framework that supports and facilitates clinical research
- Training and upskilling local researchers and clinicians in research methodologies
- Prioritizing patient-centric approach, engaging patient communities and advocacy groups
Archives: Agenda
Diversity Action Plans: preparing, submitting, and receiving regulatory feedback
- An overview of recent US legislation around diversity in clinical trials
- Understanding requirements around DAP approval and submission: what should you include?
- Preparing documentation to demonstrate diversity in your clinical trial
- Experience with DAPs in view of the received FDA feedback
Lunch and networking break
Patient-guided by design: a framework for successful study start-up, informed protocol design and better outcomes
- Optimizing study-start up for improved timelines and site performance
- Integrating patient input into protocol design and the impact of patient motivation assessments
- Case studies: patient-informed protocol design for successful outcomes
PANEL: How to lay the groundwork for a successful rare disease trial through early patient engagement
- Working closely with patients, caregivers and advocacy groups in clinical trial design
- The importance of building a close relationship with patients for trials where patients are the expert in their disease
- Reducing the financial burden of clinical trial participation on patients
- How to make trial participation easier on patients: where are the main hurdles impacting enrolment?
- Building long term relationships with patients post clinical trials
Beyond trial participation: engaging rare disease patients in the long term
- Biopharma’s role in supporting patients with their disease once no longer study participants
- Working with advocacy groups on long term patient engagement
- Where and how can trial sponsors step in to provide long term support for rare disease patients?
- Bridging the scientific advice gap for patients with rare diseases
Morning refreshments and networking break
Overcoming the challenges of enrollment in the changing landscape of a rare disease
This presentation delves into dynamics of enrollment challenges and underscores the significance of fostering strong site relationships in rare diseases, which often prove instrumental in driving successful outcomes. Beginning with a compelling case study, we navigate key drivers influencing sponsor decision-making processes, creating innovative strategies that positively impact the study. Emphasizing the paramount importance of timelines, we discuss how sponsor decisions are strategically molded to align with project deadlines. Through the lens of the “one team, one approach” philosophy, we highlight collaborative strategies that surmounted our enrollment hurdles effectively. Key takeaways offer actionable insights for stakeholders navigating the complex terrain of rare disease research, paving the way for enhanced enrollment outcomes.
CASE STUDY: Improving patient and site experience for rare disease trials
- Sponsors’ roles in supporting sites in order to increase trial efficiency and decrease overall timelines
- Where can clinical trial sponsors better support patients with rare diseases both during trials and in the long term?
- Lessons learned from Moderna’s experience of site and patient engagement during clinical trials
PANEL: How minimizing participant burden leads to on-time enrollment and completion
- Patient reimbursement: how to relieve financial pressures on patients participating in trials
- Where are the main hurdles patients face and how can these be removed?
- Addressing low levels of health literacy in the patient population: how to improve communication
- Engaging patients and advocacy groups early on in the clinical trial design process