Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada

Advances in pharmacogenomic research and increasing industry interest in personalized medicine have important implications for the way that orphan drug policies are interpreted and applied. Concerns have been raised about the potential impact of pharmacogenomics and new genomic technologies on our understanding of how disease categories are delineated, and subsequently, how the concept of rare disease should be defined for the purposes of orphan drug policies. This article considers whether orphan drug legislation can be drafted in a way that will maximize benefits and minimize concerns relating to the impact of pharmacogenomics on orphan drug research and development. After reviewing the issues that may arise at the intersection of orphan drug policies and pharmacogenomics, this article will discuss the potential impact of pharmacogenomics at two critical points: orphan designation and approval of the drug product. At each of these points, the relevant aspects of current US orphan drug legislation are examined, focusing on the extent to which recent amendments may address concerns that have been raised previously. This analysis will then provide the foundation for a critical review and recommendations regarding the proposed new Canadian orphan drug framework.     

Limits of knowing or the consequences of difficult-access problems for multi-method research and public policy

How can scientists acquire an adequate level of knowledge on phenomena whose actors actively conceal their activities? Social phenomena such as terrorism, sects, corruption, Mafia organizations, drug dealers, or government intelligence agencies actively guard their secrecy, conceal their activities, decide who is allowed (not) to know, and have no interest in being observed or understood by others. The article discusses the consequences of researching difficult-access problems for doing multi-method research. In an ideal?Ctypical approach, we distinguish between social problems that can be easily accessed and those that are difficult to access or non-accessible. To distinguish the two, we define characteristics that eventually lead to the conclusion that scientific inquiry that follows the conventional paradigm of professionalism, transparency, and replicable research reaches its limits when confronted with the active resistance of phenomena that do not like to be observed, understood, or critically approached. From this flows, the necessity to think about interdisciplinary, collaborative, and investigative modes of research that come with various prices.