Hemophilia gene therapy: first, do no harm.

The introduction of adeno-associated virus-mediated, liver-directed gene therapy into the hemophilia treatment landscape brings not only great promise but also considerable uncertainty to a community that has a history punctuated by the devastating effects of HIV and hepatitis C virus. These infections were introduced into people with hemophilia through the innovation of factor concentrates in the 1970s and 1980s. Concentrates, heralded as a major advance in treatment at the time, brought devastation and death to the community already challenged by the complications of bleeding into joints, vital organs, and the brain. Over the past 5 decades, considerable advances in hemophilia treatment have improved the survival, quality of life, and participation of people with hemophilia, although challenges remain and health equity with their unaffected peers has not yet been achieved. The decision to take a gene therapy product is one in which an informed, holistic, and shared decision-making approach must be employed. Bias on the part of health care professionals and people with hemophilia must be addressed and minimized. Here, we review data leading to the regulatory authorization of valoctocogene roxaparvovec, an adeno-associated virus 5 gene therapy, in Europe to treat hemophilia A and etranacogene dezaparvovec-drlb in the United States and Europe to treat hemophilia B. We also provide an overview of the decision-making process and recommend steps that should be taken by the hemophilia community to ensure the safety of and optimal outcomes for people with hemophilia who choose to receive a gene therapy product.

Services for seniors in small-town Canada: the paradox of community.

There is a prevailing argument that what small towns lack in formal services they make up for in close ties among rural people and a shared understanding of the notion of community. Drawing on research undertaken in 9 small towns across Canada, the authors examine how the concept of community operates with respect to the provision ofin-home and community care for seniors. The analysis is based on interviews with 55 key informants from local governments, health and social care agencies, voluntary sector organizations, and community groups. The findings reveal the paradox of the conventional belief that rural communities can compensate for lack of services for seniors while failing to take into account the uncertain coping ability of the local informal sectors. The authors challenge rural health policy decision-makers, researchers, and providers to debunk assumptions about services for seniors in rural Canada.

Managing competition in the countryside: Non-profit and for-profit perceptions of long-term care in rural Ontario.

This paper contributes to the current debates surrounding private delivery of health care services by addressing the distinctive challenges, constraints and opportunities facing for-profit and non-profit providers of long-term care in rural and small town settings. It focuses on the empirical case of Ontario, Canada where extensive restructuring of long-term care, under the rubric of managed competition, has been underway since the mid-1990s. In-depth interviews with 72 representatives from local governments, public health institutions and authorities, for-profit and non-profit organisations, and community groups during July 2003 to December 2003 form the platform for a qualitative analysis of the implications of managed competition as it relates to the provision of long-term care in the countryside. The results suggest that the introduction and implementation of managed competition has accentuated the problems of service provision in rural communities, and that the long-standing issues of caregiving in rural situations transcend the differences, perceived or otherwise, between for-profit and non-profit provision. Understanding the implications of market-oriented long-term care restructuring initiatives for providers, and their clients, in rural situations requires a re-focussing of research beyond the for- versus non-profit dichotomy.