Shannon Gibson

Why the Shift? Taking a Closer Look at the Growing Interest in Niche Markets and Personalized Medicine

Pharmaceutical research and development is increasingly focused on niche markets, most notably treatments for rare diseases and “personalized” medicine. Drawing on the results of a qualitative study of 34 key Canadian stakeholders (including drug regulators, funders, scientists, policy experts, pharmaceutical industry representatives, and patient advocates), we explore the major trends that are reportedly contributing to the growing interest of the pharmaceutical industry in niche markets. Informed by both these key informant interviews and a review of the relevant literature, our paper provides a critical analysis of the many different—and sometimes conflicting—views on the reasons for and extent of the shift toward niche markets. We consider some of the potential advantages to industry, as well the important implications and risks that arise from the increasing pursuit of niche markets and pharmacogenomics. While there are many potential benefits associated with targeted therapies and drug development for historically neglected rare diseases, niche market therapies also present evidentiary challenges (e.g., smaller clinical trials and enrichment strategies) that can make approval decisions difficult, and uncertainties remain around the true benefits of many therapies.

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.

Transparency of Biobank Access in Canada: An Assessment of Industry Access and the Availability of Information on Access Policies and Resulting Research

A key issue impacting public trust in biobanks is how these resources are utilized, including who is given access to biobank data and samples. To assess the conditions under which researchers are given access to Canadian biobanks, we reviewed websites and contacted Canadian biobanks to determine the availability of information on access policies and procedures; research resulting from access biobank data and samples; and conditions on private industry access to biobanks. We also conducted expert interviews with key Canadian stakeholders (n = 11) to obtain their perspectives on biobank transparency and access policies. Among 21 Canadian biobanks, there was wide variation in the access information made publicly available, and the majority of these allowed access by industry applicants. Biobanks should be governed by the principles of transparency, accountability, and accessibility, and attention must be given to the conditions around the commercialization of biobank-based research.

The Role of the Internet in Expanding Consumer-Driven Adverse Drug Reaction Reporting

The reporting of adverse drug reactions (ADR) plays a critical role in helping to ensure the safety and efficacy of prescription drugs approved for sale in Canada. Although physicians have typically comprised the most important source of ADR reports, consumers themselves are now playing an increasingly active role in sharing information about their experiences with drug products. Due to the limitations on the ADR reporting systems maintained by governments, some privately developed databases are stepping in to meet growing consumer demand for the ability to both report and access information on ADRs. Concurrently, social media and new interactive technologies have transformed how consumers access health information online. In particular, many consumers participate in online patient communities that are aimed specifically at connecting patients with similar health experiences and concerns. Monitoring online consumer discussion of health conditions and medication use may help to highlight potential trends and problems early on. However, despite the promise of using interactive online technologies as a source of data on ADRs, critics are sceptical of the quality of such information and its ultimate utility. While there is clearly a need for caution, the move towards greater disclosure of adverse events information appears to be inevitable.