Archives: Agenda
Alleviating data challenges in rare disease research when data sets are limited
- Optimal ways to battle with data quality issues to ensure responsible data collection, analysis, and interpretation
- Capturing data effectively to make the most of your limited data
PATIENT ADVOCATE PERSPECTIVE: The Impact of Patient Networks on EPP Care: Insights from the International Porphyria Patient Network and EPP Austria
- Role of patient organisations in EPP and rare diseases
- Why early and meaningful engagement matters
- Common challenges (power imbalance, tokenistic involvement)
- Best practice principles for collaboration (transparency, co-creation, fair compensation)
- Case examples: IPPN and EPP Austria influencing clinical pathways and policy
- Key recommendations for pharma partnerships
Morning refreshments and networking
PANEL DISCUSSION: Overcoming sites’ reluctance to run trials in rare disease indications
- When resources are tight, how can rare disease trials beat the coemption and win sites over
- Incentivising sites to run orphan trials
- Balancing patient, site, CRO and investor priorities in your trial design
- How can CROs help the phenomenon, and enable rare disease patients to access the appropriate sites
- Proving to sites the ROI in running rare disease trials
Designing appropriate study protocols with limited patient numbers and heterogeneity in rare diseases
- Finding innovative ways to demonstrate efficacy and safety with small sample sizes
- Strategies for embedding protocol flexibility in a rare disease environment
- Collaborating with rare disease CROs to employ statistical methods and help select appropriate endpoints and enhance trial’s chance of success
- Realistically assessing inclusion and exclusion criteria to ensure adequate enrolment
KEYNOTE: Funding, investment and budgeting for clinical trials: Sharing best practices to ensure trial success for small/ medium pharma and biotechs
- Ensuring your trial is both cost-conscious and efficient without compromising on quality and patient safety
- Why financial accruals for clinical and R&D processes are still so challenging despite years of work to alleviate these obstacles
- Strategies for cost-effective budget allocation to maintain long-term financial health and positive ROI
- Avoiding common mistakes to keep your clinical trial budget on track
- Supporting startups: Tackling investor’s lack of willingness to invest in smaller biotechs
The AI-First Protocol in Rare Diseases: Accelerating Research for the Few Who Need It Most
Rare-disease trials face unique challenges – small patient populations, dispersed sites, and limited data. This session explores how AI-first protocol design transforms feasibility and speed in rare-disease research, enabling smarter, patient-centric, and regulator-ready studies from day one. Key Takeaways:
- How AI-first protocols reduce amendments and time-to-first-patient in small-population studies
- Using real-world and registry data to simulate feasibility and optimize site selection
- Building adaptive, patient-centric designs aligned with EMA and FDA expectations
- Integrating AI into cross-functional workflows — from clinical to regulatory teams
- Case examples showing measurable acceleration and cost savings in rare-disease trials