Wednesday, November 5
8:00 am Registration & Morning Coffee
8:50 am Chairs Opening Remarks
Driving Early Diagnosis & Rapid Recruitment to Connect Patients with Options Faster
9:00 am Bridging Socioeconomic & Geographic Gaps to Broaden Trial Participation
Synopsis
- Building trust with underserved communities through culturally sensitive education and transparent communication
- Partnering with local health centres, advocacy groups, and community leaders to raise awareness and improve trial visibility
- Offering practical support: transportation, childcare, flexible scheduling, and financial assistance to reduce participation burdens
9:30 am Breakout Roundtable: Overcoming Trial Access & Enrollment Challenges for Biotech & Large Pharma
Synopsis
No matter your company size, this interactive roundtable will help you tackle the unique challenges of patient access and trial enrollment in rare disease research. Split into two groups – small/mid-sized biotech and large pharma – you’ll collaborate with peers facing similar hurdles to share insights and co-create practical solutions tailored to your scale and resources.
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Synopsis
- How can smaller teams creatively overcome resource constraints to accelerate patient recruitment?
- What grassroots or community-driven strategies work best to build trust with rare disease patients?
- How can smaller biotechs effectively collaborate with advocacy groups and providers to enhance trial visibility?
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Synopsis
- How do you coordinate multi-country trial sites to ensure consistent patient retention and engagement?
- What advanced technologies and data analytics have proven most effective in optimizing large-scale recruitment?
- How can large pharma embed diversity, equity, and inclusion meaningfully across complex trial ecosystems?
10:00 am Accelerating Early Diagnosis to Expand the Pool of Eligible Patients
Synopsis
- Launching targeted awareness campaigns that trigger earlier referrals and diagnoses
- Training primary care and frontline providers to flag rare disease warning signs sooner
- Partnering with patient advocacy groups to help families recognize symptoms and seek evaluation
- Leveraging digital tools for broader patient outreach
10:30 am Morning Break & Speed Networking
Synopsis
As the rare disease research community comes together, this valuable session will ensure you reconnect with existing colleagues and forge new relationships. This structured networking opportunity will pair you with fellow attendees for several 3-minute introductions, allowing you to connect with industry professionals, patient advocates, and clinical operations experts in a dynamic and focused setting!
Reach, Recruit, Retain: Digital Tools Powering Rare Disease Studies
11:30 am Harnessing AI-Powered Patient Identification to Accelerate Trial Enrollment
Synopsis
- Rapidly identifying eligible rare disease patients using AI to analyze medical records (EMR), claims databases, genetic testing partnerships and registries, cutting recruitment timelines by months
- Improving enrollment accuracy by targeting patients who precisely fit trial criteria (both interventional and natural history trials), lowering screen failure rates and cutting costs
- Enhancing recruitment efficiency with data-driven patient targeting to maximize trial enrollment success
12:00 pm AI-Powered Federated Networks: Advancing Patient Discovery Through Secure Data Collaboration
Synopsis
- Enabling secure, privacy-preserving data collaboration between institutions to expand rare disease patient discovery beyond individual silos
- Unlocking insights from decentralized datasets through federated learning, enhancing recruitment reach without centralizing sensitive patient data
- Delivering personalized, real-time educational content to identified patients, increasing awareness, trust, and willingness to participate in trials
12:30 pm Session Reserved for myTomorrows
1:00 pm Lunch Break & Networking
Driving Early Patient Engagement Through Trust, Education & Community Collaboration
2:00 pm Panel Discussion: From Patient Voice to Scientific Value: How Patient Engagement Sparks Innovation
Synopsis
- Redefining engagement by clarifying what meaningful patient involvement in R&D requires structurally, culturally, and scientifically
- Shifting power by enabling true partnership that redistributes influence across trial design, evidence generation, and development priorities
- Integrating patient input early in development to improve trial design, accelerate enrollment, and generate real-world relevant evidence
- Centering equity in engagement strategies to create more relevant science, inclusive access, and lasting trust
2:45 pm Starting with Advocacy to Build Trust & Accelerate Trial Readiness
Synopsis
- Building authentic relationships with patient organizations to align on unmet needs and trial goals before protocol is set
- Collaborating to co-create education materials, FAQs, and outreach strategies that reflect real patient priorities and language
- Avoiding recruitment delays and boosting trust by involving advocates from day one, not just when enrollment begins
Enhancing Strategic Partnerships Across Functions to Drive Patient Access
4:00 pm Fireside Chat: Strengthening Collaboration Between Procurement & PRR
Synopsis
A discussion between Procurement and PRR to help frame the service area, how these two groups can better partner as well as how these groups fit into the organization.
Topics include:
- Clarifying what is and isn’t within PRR’s scope
- Identifying opportunities for team and process development
- Driving new ideas and innovative approaches
- Partnering more strategically with vendors
- Aligning with evolving industry standards and expectations
4:30 pm Driving Market Access by Leveraging HEOR & RWE
Synopsis
- Highlighting how to build and implement direct-to-patient PERFORM registries for rare diseases
- Using real-world evidence to strengthen market access and reimbursement strategies
- Optimizing and streamlining the appeals process with data-driven insights
- Generating the patient-centric data needed to demonstrate value and support medical affairs