9:00 am Coffee & Networking

8:55 am Chair’s Opening Remarks

  • Jessica Klein Director, Global Rare Disease Awareness, Ipsen Pharma

Overcoming Recruitment & Participation Barriers

9:00 am Optimizing Clinical Operations to Overcome Participation Barriers

Synopsis

  • Identifying key operational hurdles that affect patient retention
  • Implementing solutions to simplify patient visits, such as centralized scheduling systems, coordinated multi-site appointments, and improved site accessibility
  • Incorporating wearable technology for continuous data collection and monitoring, reducing the frequency of in-person visits

9:30 am Enhancing Awareness & Accessibility of Rare Disease Clinical Trials

  • Bethany Firem Associate Director - Global Patient Affairs, BioMarin Pharmaceutical

Synopsis

  • Navigating the challenges patients face in accessing relevant clinical trial information in rare disease communities
  • Highlight successful examples where centralized platforms have effectively improved patient access to clinical trials, leading to increased participation and engagement

10:15 am Session Reserved for Medical Research Network

Synopsis

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11:00 am Morning Break & Networking

11:30 am Exploring Creative Solutions for Minimizing Participation Challenges in Rare Disease Trials

  • Samantha Botts Global Recruitment & Retention Lead, Sanofi
  • Belle Estee Dante Patient Recruitment & Retention Global Lead, Rare Disease, Rare Blood Disorders, Neurology, Sanofi

Synopsis

  • Identifying and discussing common hurdles to patient retention
  • Introducing innovative solutions to minimize these burdens
  • Highlighting the role of technology in facilitating easier participation

Streamlining Patient Recruitment & Enhancing Education in Rare Disease Trials

12:00 pm Developing & Executing Education & Resource Strategies for Rare Disease Communities

Synopsis

  • Presenting innovative educational strategies aimed at small, rare disease populations
  • Utilizing patient feedback to improve and adapt educational programs and resources
  • Navigating the challenges patients face in accessing relevant clinical trial information in rare disease communities

12:30 pm Strategies for Cost-Effective Patient Recruitment in Rare Disease Trials

  • Jessica Klein Director, Global Rare Disease Awareness, Ipsen Pharma

Synopsis

  • Analyzing success rates and cost savings achieved through a range of patient recruitment strategies
  • Implementing disease state awareness campaigns that efficiently educate both patients and healthcare professionals to reduce diagnosis time and streamline recruitment
  • Demonstrating how targeted awareness efforts lead to more efficient patient recruitment

1:00 pm Lunch Break

Boosting Patient Retention During Rare Disease Trials

2:00 pm Roundtable:Ensuring Ethical & Compliant Communication Throughout Rare Disease Trials

Synopsis

  • Introducing approaches for sharing less favourable data ethically and compliantly
  • Maintaining transparency with patient organizations during challenging updates
  • Managing global communication effectively, considering diverse regulatory environments

2:30 pm Optimizing Patient Engagement Throughout the Trial Process

  • Amanda Rohrig Director, Patient Advocacy, Bridge Bioscience Corp

Synopsis

  • Understand the specific needs and lifestyles of different disease communities and how customized engagement plans can optimize clinical trial participation
  • Explore barriers to recruitment, engagement and retention in rare disease trials
  • Present examples of successful engagement strategies for different patient communities throughout the clinical trial development cycle
  • Introduce methods for regulatory gathering patient feedback to ensure retention

3:00 pm Closing Remarks

3:15 pm End of Summit