Data Strategy for Research Development – What Do Advocates Need to Know

*Recommended for Patients/Advocates

The Role of Patient Data from Basic Research to Approval and Access

• Understanding the role of patient data from preclinical to approval to reimbursements – the importance of planning ahead
• Differentiating study/non study data Evolutions in Natural History Studies and Registries
• Building a registry or natural history study with intent
• Ensuring that data is clearly defined with a goal in mind to make sure it is measurable, actionable and of value to patients and researchers

Measurement Tools: Making them Work in Rare

• Addressing challenges in measuring patient data in rare disease research and limitations of current tools for patient-reported outcomes
• Opportunities to build better measurement tools to help researchers and patients