Implementing precision medicine trial designs to overcome the challenge of small and heterogeneous patient populations in rare diseases
24 Jun 2024
Salon C
Clinical Studies
- In cases where the natural history of a rare disease is poorly understood, what strategies can be employed to enhance our knowledge and facilitate more informed research?
- Given the limitations posed by small populations, how can researchers expand study design options to ensure meaningful outcomes in rare disease studies?
- Considering the common lack of essential drug development tools such as outcome measures and biomarkers in rare diseases, what approaches can be taken to address this gap and enhance the drug development process?
- How can a partnership with Patient Advocacy and KOLs help?
