Rescue of a Global Phase III Orphan Drug Study for Hypoparathyroidism
Investigator identification and patient recruitment are critical in an orphan drug clinical trial and this is magnified when atrial is failing. This complex Phase III trial for hypoparathyroidism was being conducted across Central/Eastern Europe and North America. The incumbent international CRO was unable to meet the enrolment timelines and costs were escalating. Of the target patient enrolment, approximately 95% had yet to be identified when TFS assumed full-service responsibility for this study.
- Enrolment timelines at risk — over reliance on KOL enrolment
- Only 60% of sites identified — only 5% of patients enrolled
- Sites underperforming & lagging site initiation
- Vendor contracts held by incumbent CRO
- Incumbent CRO retained on-going data management & biostatistics responsibilities
- TFS developed a 65-day detailed transition plan from the incumbent CRO and was fully operational within 45 days — 20 days early
- TFS introduced Optimal Site Referral Identification & Patient Advocacy Group
- Initiated Social Media and patient education/ websites
- Identified 300+ investigators treating 1,500+ unique hypoparathyroid patients meeting the specified guidelines
- Identified 2,688 potential referral physicians
- Initiated 6 additional sites: 4 met/exceeded average enrolment rate; 1 doubled the
- enrolment rate
- Vendor contracts renegotiated with no disruption to service or timelines
- TFS developed & maintained a positive relationship with the incumbent CRO throughout the study to closure
TFS’ agility, global footprint, and ‘out of the box’ solutions for site & patient enrolment rescued this study. Patient enrolment timelines were met. The orphan drug produced positive top-line results and the sponsor has filed for FDA approval.
TFS were awarded the customer's next trial.