Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public-private partnerships.

Medicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient, either by stranding in the translational stage or due to suboptimal patient access related to pricing or uncertain effectiveness. Expanding and solidifying the role of the academic in public-private partnerships (PPPs) may present an innovative solution to help overcome these complexities.

Importance of intellectual property generated by biomedical research at universities and academic hospitals.

Biomedical research has many different facets. Researchers and clinicians study disease biology and biochemistry to discover novel therapeutic targets, unravel biochemical pathways and identify biomarkers to improve diagnosis, or devise new approaches to clinically manage diseases more effectively. In all instances, the overall goal of biomedical research is to ensure that results thereof (such as a therapy, a device, or a method which may be broadly referred to as "inventions") are clinically implemented.