Pharmaceutical Product Development (PPD) has established a Rare Disease and Pediatric Center of Excellence to oversee all rare disease and pediatric-related drug development activities. This new center of excellence provides pharmaceutical and biotechnology clients with a dedicated team of professionals focused on the design and execution of clinical trials that address the unique strategic, operational, medical and scientific challenges of clinical studies in rare disease and pediatric patient populations.
PPD announced the new center of excellence on Rare Disease Day, which is observed on the last day of February each year to raise awareness of rare diseases. A disease is designated as rare in the United States when it affects fewer than 200,000 people, in Europe when it affects no more than five in 10,000 people and in Japan when it affects fewer than 50,000 people. In the U.S. and Europe alone, as many as 60 million individuals are affected by one of the more than 6,000 identified rare diseases.
“Our new center of excellence builds on PPD’s longstanding expertise and experience to address the special drug development considerations of rare diseases and pediatrics,” said Karen Kaucic, M.D., PPD senior vice president and head of the center of excellence. “With a great majority of rare diseases touching children, we believe our approach will enable us to be a more connected and active partner. Our clients can depend on us for the latest trial design innovations, the ability to navigate complex trial logistics and our keen understanding of customized patient access strategies in low-prevalence, widely dispersed patient populations. We felt today was an appropriate time to introduce this new venture, as we join with the drug development community in recognizing Rare Disease Day.”
Dr. Kaucic will leverage her leadership experience in rare diseases and pediatrics to drive the implementation of PPD’s center of excellence, leading a dedicated team with a wealth of expertise in rare disease trial design and execution in such disciplines as product development, clinical operations, commercial strategy and early engagement.
The PPD team members in the center of excellence are seasoned pharmaceutical and clinical research professionals who have hands-on experience with rare diseases and pediatrics. The team is focused on addressing the specific challenges presented by the small, widely dispersed populations of rare disease and pediatric patients. This group of functional experts has been assembled to support end-to-end clinical trial solutions in such areas as feasibility strategy, clinical information, biostatistics, pharmacovigilance, lab operations and business analytics.
PPD’s particular expertise in rare diseases stems from having conducted more than 220 studies with more than 50,000 patients over the past five years. PPD also is a leading clinical researcher in pediatric drug development, having conducted more than 400 clinical trials during the past five years, enrolling more than 97,000 patients across a wide range of therapeutic indications, including infectious diseases, respiratory, cardiology, oncology and immunology.
PPD leverages a pediatric investigator network that further enhances its pediatric clinical development capabilities by providing clients with faster trial startup, more predictable patient enrollment and higher-quality data. For both rare disease and pediatric trials, PPD has deployed a purpose-built framework that ensures the application of best practices in study development and enables the company to develop a customized approach to address the unique needs of these trials.