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
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Cross-Border Enrollment of Rare Disease Patients
Clinical trials in rare diseases present unique challenges unseen in trials for more common conditions. Cross-border enrollment can be key to…