Accelerate Timelines in
Rare Diseases
Study Design:
Two phase III studies (1 open-label safety, 1 DB cross-over, comparative) to evaluate a product for a rare disease. Required approximately 50 patients including 15 treatment-naïve patients. Treatment regimen required weekly 1-hour infusions administered within 3-6 hours of reconstitution. Serial pK draws and safety labs were also required. Treatment duration was 24 weeks. 8 U.S. sites and 5 European sites planned to participate.
Issues:
Limited patient population. Multiple lots of study drug; each patient had to receive a single lot. Complex reconstitution and blinding procedures for infusion therapy.
Solution:
CRN’s homecare nursing and pharmacy service compounded, dispersed, delivered and administered study drug for 20 of the required 24 infusions. RNs used to collect pK and safety labs at selected visits and time points. Call center to review patient eligibility, assign randomization, lot assignments and master inventory.
Results:
All required patients were enrolled and completed 8 months ahead of schedule. All patients, including treatment-naïve patients, were identified and enrolled in the U.S. as a result of the convenience of CRN’s services.
