COMMITTED TO RARE DISEASE RESEARCH
Lack of widespread clinical knowledge, regulatory precedents and consensus on endpoints all contribute to the complexity of developing long-awaited therapies for patients and families dealing with rare diseases. Couple these challenges with the need to access often geographically dispersed patients and to develop research-naïve sites, and it becomes clear why you require a development partner with distinct expertise and a proven approach.
That’s where PPD comes in.
PPD’S EXPERIENCE IN THE PAST FIVE YEARS:
Rare Disease and Pediatric Center of Excellence
PPD’s Rare Disease and Pediatric Center of Excellence is an experienced and dedicated cross-functional center of excellence bringing together development and operational expertise from all therapeutic areas and critical functions within PPD for the success of your research programming. Our Rare Disease and Pediatric Center of Excellence team addresses strategic, operational, medical and scientific challenges presented by the studies in all rare indications.
PPD’s RARE DISEASE FRAMEWORK
Our medical, operational, regulatory and real-world experts design and operationalize studies with a customized approach.
- Focus on patients and caregivers
- Specialized workforce and rare disease operating model
- Dedicated general rare disease and indication-specific training for our research teams
- Massive investment in data and novel technologies bringing trials closer to patients
- Capitalize on our global footprint allowing for immediate updates on regulatory enviroments and the inclusion of broad, in-country and local language capabilties
We generate evidence to optimize market access for rare disease therapies including:
- Market access strategies and value stories supported by strong evidence
- Inclusion of the patient voice across the drug development life cycle
- Comprehensive real-world evidence generation and dissemination
- Interventional studies to bridge the gap between pre- and post-approval evidence requirements