Developing interactions with industry in rare diseases: lessons learned and continuing challenges.

ABSTRACT: The National Institutes of Health (NIH) established the Rare Diseases Clinical Research Network to address the unique challenges of performing research on rare diseases. The Urea Cycle Disorders Consortium (UCDC) was one of the original ten consortia established. The UCDC represents a unique partnership among clinicians, patients, and the NIH with a primary goal of increasing the development of therapeutics that improve patient outcomes for persons affected with a UCD. Based in part on financial incentives associated with the Orphan Drug Act biopharmaceutical and investment entities have an intense interest in engaging with research consortia like the UCDC, which have compiled potentially valuable longitudinal data characterizing outcomes in a relatively large number of affected individual

SUBMITTER: Berry SA 

PROVIDER: S-EPMC6944635 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature