ABSTRACT
This three-day scientific conference; “C-Path Scientific Breakthrough Conference: Addressing unmet
needs and challenges in underserved drug development areas through collaborative partnerships” will
bring together representatives from an interdisciplinary team of industry and academic researchers,
patient groups, and regulatory agencies – to discuss drug development challenges in neonatal
medicine, alpha-1 antitrypsin deficiency (AATD), and lysosomal diseases (LD), and brainstorm how
pre-competitive public private partnerships (PPPs) can help advance drug development in these
underserved populations. Each year in the U.S., 10% of neonates are born preterm and there is an
urgent unmet need to improve survival and outcome in this vulnerable population. In 2015 the FDA
collaborated with C-Path to create International Neonatal Consortium (INC), a PPP of industry leaders,
academic researchers, regulatory agencies, families, and nurses to advance medical product
development tools for endorsement by FDA and other global regulatory agencies. AATD is a clinically
under-recognized disease associated with an increased risk of chronic liver disease in adults and
children and is the leading cause of chronic obstructive pulmonary disease (COPD) in adults, outside
of smoking. Unmet needs in AATD pertain to both diagnosis, evaluation, and treatment of AATD – for
both hepatic and pulmonary manifestations. To overcome the lack of tools that provide both
comprehensive and objective assessment of outcomes in AATD, the Critical Path for AATD (CPA-1)
has involved stakeholders from industry, academia, patient groups, and regulatory agencies, with the
objective to achieve actionable solutions or tools for AATD drug development. Lysosomal diseases
(LDs) are a group of inherited metabolic disorders caused by mutations in genes that code for
enzymes involved in the breakdown of macromolecules in lysosomes. Challenges related to this group
of rare diseases relate to early detection, lack of effective diagnostic tools and screening programs,
and lack of effective treatments. Critical Path for Lysosomal Diseases (CPLD) is a PPP comprised of
stakeholders from pharmaceutical companies, academic scientists with an interest in LD drug
development, patient groups, and regulatory representatives. This conference will showcase the work
done by INC, CPA-1, and CPLD and capitalize on strong existing pre-competitive relationships
between the various stakeholders in the drug development communities to advance efforts to produce
publicly available drug development tools aiming at improving the efficiency and safety of medical
product development.
