The collection of high-quality endpoint data was paramount to the success of this Phase II program. Because this was the first clinical trial ever done for this disease, it was necessary to develop non- disease-specific clinical assessments, in collaboration with Key Opinion Leaders (KOLs), for use as endpoints. The newness of these tools presented challenges to site selection and training, as well as risks to uniformity in patient recruitment and consistency in data collection.
The Patient Voice: Engaging Rare Disease Patients Improves Clinical Trial Enrollment & Retention
Clinical trials in rare diseases are complex and present unique challenges. However, patients in these trials are very vocal, with caretakers, family…
Risk-Based Clinical Development
PRA Health Sciences’ risk-based, comprehensive approach to clinical development and clinical study risk management includes centralized monitoring…
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