Impact of Public-Private Partnerships in Regulatory Science, Drug Development and Regulatory Decision-Making

Description: Critical unmet needs in neurodegenerative, metabolic, and rare diseases cannot be efficiently and effectively addressed by individual organizations. As the Critical Path Initiative identified in 2004, public-private partnerships (PPPs) are a mechanism to meet these challenges through developing a regulatory framework for product development and generating solutions that can optimize trial design and address such critical unmet needs. In this session, we will describe how a neutral convener organization such as the Critical Path Institute (C-Path) coordinates such PPPs. C-Path has established core competencies in data management and standards, biomarker and safety science, regulatory science, clinical outcome assessments, and modeling and analytics, that support 25+ disease areas including neurologic diseases, rare/orphan diseases, infectious diseases, safety biomarkers, and pediatric conditions.

C-Path PPPs have generated solutions such as (1) disease-progression models and clinical trial simulation tools used in trial design for new therapeutic candidates; (2) innovative solutions such as the creation of a data and analytics platform for interrogation of shared data, which were used to generate supportive evidence for approval of novel therapies; and (3) new patient-reported outcome measures, in irritable bowel syndrome with constipation, used as primary endpoints in studies leading to labeling changes of linaclotide.

In addition, glutamate dehydrogenase has been qualified as a safety biomarker to detect drug-induced kidney and liver injuries and is being expanded in bridging studies for safety across different ethnic populations. Other C-Path PPPs are using real-world data, digital technologies, and electronic health records to address drug development in rare diseases, infectious diseases, and vulnerable populations. Participants will learn, through case examples, about the function, value and impact of PPPs and how regulatory affairs professionals can lead, contribute, collaborate, and generate tangible solutions to address critical unmet medical needs.