- Use of RWD in driving drug discovery in rare diseases
- Leveraging therapeutics for rare disease to larger, multiple global applications
- 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
- 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
- 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
- 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
