Selection of appropriate endpoints for rare disease clinical studies can be challenging but is critical to assessing the efficacy of new medicines. Engaging with patient communities to determine endpoints ensures appropriate measurements of success for all stakeholders and de-risks recruitment and retention strategies. Post-approval, appropriately set clinical research endpoints result in meaningful treatment assessment and delivery by clinicians and patients.
In this white paper, you’ll learn:
- How to determine valid clinical research endpoints
- How to engage with patient advocacy organizations to support product development
- How to select endpoints relevant to all stakeholders including patients, caregivers, and practitioners
RARE DISEASES NEWSLETTER Volume 10, July 2018
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
Exploring the Puzzling World of Rare Diseases
COVID-19 Impact on Rare Disease Clinical Trials
Rare disease patient organizations are coping with a broad range of other organizational challenges, including reduced funding, canceled fundraising…