We understand that nothing is standard when developing a therapy for a rare or ultra-rare disease. Every step of the clinical development process is more complicated. From regulatory pathway selection to patient recruitment challenges to navigating the astronomical volume of data to collect and clean - a rare disease therapy requires an extraordinary amount of coordination. At Veristat, we have assembled a scientific-minded team of experts who have supported nearly 600 clinical programs and prepared >70 marketing applications for rare disease treatments and cures.
Rare Disease Trials Are Challenging
Rare disease trials present additional unique challenges and opportunities. We help determine if your study qualifies for an expedited regulatory approval pathway – such as Orphan Drug Designation, Fast Track designation, or Priority Review – and we will represent you at US Food & Drug Administration (FDA) and European Medicines Agency (EMA) meetings.
Veristat ensures that your clinical trial or program design supports your regulatory strategy, disease progression analytical models, or previous adult trials. Our experienced teams are poised to plan and implement these efforts quickly, providing:
- Development of regulatory strategy, expedited pathways, and regulatory agency interactions
- Clinical program planning, inclusive of statistical planning and analysis
- Agile patient recruitment strategies to support virtual trial and hybrid approaches
- Rapid deployment of clinical trial databases
- Virtual and remote clinical and medical site monitoring
- Data analysis and migration into CDISC formats
- Writing of clinical trial, safety, and regulatory documents
- Preparation and defense of Marketing Applications – NDAs, BLAs, NDSs, MAAs, jNDAs, etc.
- Post-Marketing Pharmacovigilance and Safety Monitoring to ensure patient safety beyond regulatory approval