Archives: Agenda

• Patient-led research organizations are evolving from advocacy groups into development partners, creating registries, natural history studies, and trial-readiness infrastructure.
• Rare disease evidence is increasingly generated across interconnected data ecosystems rather than within a single clinical trial.
• Sponsors are redefining CRO success from trial execution to orchestration of data, partnerships, and evidence across the disease community

• Implementing successful rare disease programs requires a different approach than traditional drug development because of small patient populations, heterogeneous disease presentation, limited natural history data, and the critical role of patient communities.
• In this presentation, we will cover 10 lessons learned around operationally executing rare disease studies and how to succeed in this space.

In common disease, competitive intelligence informs commercial strategy; in rare disease it determines whether your trial enrolls at all!

When a condition has a few hundred addressable patients concentrated across a handful of centres of excellence, every competing programme is drawing from the same pool, courting the same investigators, and often working with the same advocacy organisations.

This interactive session applies a competitive intelligence discipline to a problem clinical teams usually treat as operational. We will look at how to map the competitive pressure on an indication before feasibility is locked, which external signals reliably predict enrolment difficulty, and how sponsors can position their programme so that patients, sites and advocacy partners choose it.

Participants will work through a live mapping exercise on a rare indication and leave with a practical framework they can apply to their own portfolio.

• Understanding how site burden directly affects the patient and caregiver experience in rare disease trials
• Preparing sites for successful study delivery through targeted training, logistical support, and first-patient-visit readiness
• Integrating patient, caregiver, and site support to reduce operational friction and strengthen retention
• Using patient-informed feasibility to select sites with genuine patient access and the capability to execute the protocol effectively

• The real-world barriers families face when participating in rare disease trials
• Practical ways sponsors and trial designers can reduce burden in protocol design
• The role of patient advocacy organizations in helping improve recruitment and retention
• Lessons learned from the Angelman syndrome community as multiple clinical trials have launched simultaneously

• Understanding the current FDA regulatory landscape and its impact on rare diseases clinical development
• Identifying common regulatory pitfalls that delay approvals and strategies to mitigate them early
• Exploring innovative trial design that shorten timelines whilst keeping compliant with changing regulation

Moderator Robert Loll, SVP, Business Development, Praxis