Rendre évident : une approche symétrique de la réglementation des produits thérapeutiques

Dans le champ des études sociales de la science, les régimes de régulation du risque appréhendés selon une approche technocratique sont critiqués pour leur attention insuffisante au « public », point de vue épistémologique socialement situé qui pourtant est davantage en mesure d’articuler les préoccupations du monde réel aux implications (collectives) du risque. Il semble qu’on ait répondu à cette critique dans plusieurs pays en modernisant les réglementations et en déployant des efforts stratégiques pour susciter l’ouverture, permettre un examen approfondi et encourager un large éventail de citoyens à s’impliquer dans le processus décisionnel. Tout en reconnaissant que le régime de preuves dans la réglementation du risque aurait grandement avantage à prendre en compte une plus grande diversité de types de savoir (notamment les connaissances qui ont été historiquement négligées), nous soutenons que ce ne sont pas seulement les savoirs publics « profanes », mais aussi les savoirs scientifiques « experts » qui tendent à être négligés dans les régimes modernes de réglementation du risque. Le présent article part d’un cas spécifique — la modernisation de la régulation des produits de santé canadiens — pour rendre compte de l’évolution de la nature et du rôle de l’évidence médicale. En nous appuyant sur une recherche ethnographique et sur la littérature consacrée (science du risque et de la régulation, travaux autour des frontières et anthropologie symétrique), nous proposons une approche symétrique de la réglementation du risque basée sur la production d’un régime de preuves.Within the social studies of science, risk regulation regimes dominated by a technocratic approach are critiqued for neglecting public, socially situated epistemological standpoints, which, it is argued, are more capable of articulating real world concerns and implications of risk. This critique appears to have been tackled through regulatory modernization in several nations, where strategic efforts are being advanced to open up, enable scrutiny and solicit input into decision-making from a broad range of citizens. While we agree that the evidence for risk regulation could benefit from accommodating more ways of knowing (particularly types of knowledge that have a history of being neglected), we argue that it is not only ‘lay’ public knowledges, but also ‘expert’ scientific ones which become neglected in modern risk regulation regimes. In this paper, we draw upon a specific case — modernization in the Canadian health products regulator — to examine the evolving role and nature of evidence. We draw from our own ethnographic research and the relevant literature (risk and regulatory science, boundary work, and symmetrical anthropology) to suggest a symmetrical approach to evidence-based risk regulation.En el campo de los estudios sociales de las ciencias, los regímenes de regulación del riesgo identificados según un enfoque tecnocrático, son criticados por su insuficiente atención a lo “público”, punto de vista epistemológico socialmente situado que, no obstante, está más en condiciones de articular las preocupaciones del mundo real a las implicaciones (colectivas) del riesgo. Pareciera que en varios países se hubiese respondido a esta crítica modernizando las reglamentaciones y realizando esfuerzos estratégicos para suscitar la apertura, permitir un examen profundo y promover la participación de un amplio sector de ciudadanos en el proceso decisional. Reconociendo que el régimen de pruebas en la reglamentación del riesgo tendría considerables ventajas al considerar una mayor diversidad de tipos de saberes (principalmente los conocimientos que históricamente han sido descuidados), nosotros sostenemos que en los regímenes modernos de reglamentación del riesgo no son solamente descuidados los saberes públicos “profanos”, sino además los saberes científicos “expertos”. El presente artículo parte de un caso específico —la modernización de la regulación de los productos de salud canadienses— para dar cuenta de la evolución de la naturaleza y del papel de la evidencia médica. Apoyándonos en una investigación etnográfica y en la literatura consagrada (ciencias del riesgo y de la regulación, trabajos alrededor de las fronteras y antropología simétrica), proponemos un enfoque simétrico de la reglamentación del riesgo basado en la producción de un régimen de pruebas

Tissue-specific regulation of sirtuin and nicotinamide adenine dinucleotide biosynthetic pathways identified in C57Bl/6 mice in response to high-fat feeding

Funding: The Scottish Government's Rural and Environment Science and Analytical Services Division.Peer reviewedPostprin

Global challenges of implementing human papillomavirus vaccines

Human Papillomavirus vaccines are widely hailed as a sweeping pharmaceutical innovation for the universal benefit of all women. The implementation of the vaccines, however, is far from universal or equitable. Socio-economically marginalized women in emerging and developing, and many advanced economies alike, suffer a disproportionately large burden of cervical cancer. Despite the marketing of Human Papillomavirus vaccines as the solution to cervical cancer, the market authorization (licensing) of the vaccines has not translated into universal equitable access. Vaccine implementation for vulnerable girls and women faces multiple barriers that include high vaccine costs, inadequate delivery infrastructure, and lack of community engagement to generate awareness about cervical cancer and early screening tools. For Human Papillomavirus vaccines to work as a public health solution, the quality-assured delivery of cheaper vaccines must be integrated with strengthened capacity for community-based health education and screening

From Discovery to Delivery: Public Sector Development of the rVSV-ZEBOV Ebola Vaccine

The discovery and development of the Ebola rVSV-ZEBOV vaccine challenge the common assumption that the research and development for innovative therapeutic products and vaccines is best carried out by the private sector. Using internal government documents obtained through an access to information request, we analyze the development of rVSV-ZEBOV by researchers at Canada’s National Microbiology Laboratory beyond its patenting and licensing to a biotech company in the United States in 2010. According to government documentation, the company failed to make any progress toward a phase 1 clinical trial until after the WHO Public Health Emergency of International Concern freed substantial donor and public funds for the vaccine’s further development. The development of rVSV-ZEBOV, from sponsoring early stage research through to carrying out clinical trials during the epidemic, was instead the result of the combined efforts of the Canadian government, its researchers, and other publicly funded institutions. This case study of rVSV-ZEBOV underscores the significant public contribution to the R&D of vaccines even under conditions of precarity, and suggests that an alternative approach to generating knowledge and developing interventions, such as open science, is required in order to fully realize the public sector’s contribution to improved global health. Note: Funding Statement: The research for this paper was funded by the Canadian Institutes of Health Research (CIHR EOG 123678, CIHR PJT 156256, and CIHR PJT 148908), the PACEOMICS project funded by Genome Canada, Genome Alberta, Genome Quebec, the Canadian Institutes for Health Research, Alberta Innovates—Health Solutions, and the Social Sciences and Humanities Research Council. The authors would also like to acknowledge the contributions of several research assistants, including Heather Webster, MarcusMcLeod, Michael Gardener and Alexandra Ghelerter.Declaration of Interests: We have the following interests to declare: MH is a member of Canada’s Patented Medicine Prices Review Board (PMPRB). He receives honoraria for his work as a PMPRB member. MH has no other financial relationships with government agencies, non-governmental organizations, or private corporations. RG has no conflict of interests to declare. JG has no conflict of interests to declare

Impact of caring for someone with a rare rheumatic condition, views from patients and informal carers – the need for cat-like vigilance

Objective ANCA-associated vasculitis (AAV) is a rare multisystem disease. Modern therapeutic protocols have turned AAV from an acute frequently fatal disease into a chronic disease requiring long-term immunosuppression. Patients must often manage substantial burdens related to chronic illness and treatment-related side effects, requiring help from informal carers. This study aimed to explore the experience of patients and of informal carers of patients with AAV about the impact of managing a rare rheumatic condition. Methods A qualitative approach using semi-structured interviews was used. Interviews were conducted with a purposeful sample of 18 pairs of patients with AAV and their informal carers. The interviews were used to explore the participants’ experience and effects of caring. The interviews were recorded and transcribed as verbatim text and analysed using the framework technique. Results 18 patients (seven female) [disease: ten granulomatosis with polyangiitis (GPA); four microscopic polyangiitis (MPA); four eosinophilic granulomatosis with polyangiitis (EGPA), age range 34-78, disease duration 1- 20 years. Caregiver and patient perspectives were shared. The emerging themes were the physical and psychological impact of the disease, the need for constant vigilance and fear of the future. Conclusion Both patients and carers faced a range of challenges in managing a rare condition, from the seriousness of the illness, dealing with the emotional toll and knowing what to expect. This study offers insight into the experiences of patients and informal carers, and health care professionals should address individuals’ fears and expectations for recovery

275 Impact of caring for someone with a rare rheumatic condition: views from patients and informal carers

Background: ANCA-associated vasculitis is a rare multisystem disease. Modern therapeutic protocols have turned ANCA-associated vasculitis from an acute frequently fatal disease into a chronic disease requiring long-term immunosuppression. Patients must often manage substantial burdens related to chronic illness and treatment-related side effects. Patients often need help and support to manage their disease. The aim of this study was to explore the experience of patients and of informal carers of patients about the impact of managing a rare rheumatic condition. Methods: A qualitative approach using semi-structured interviews was used. Interviews were conducted with a purposeful sample of 18 pairs of patients with ANCA-associated vasculitis and their informal carers. The interviews were used to explore the participants experience and affects of caring. The interviews were recorded and transcribed as verbatim text and analysed using the framework technique. Results: 18 patients (seven female) [disease: ten granulomatosis with polyangiitis (GPA); four microscopic polyangiitis (MPA); four eosinophilic granulomatosis with polyangiitis (EGPA), age range 34-78, disease duration 1- 20 years. Caregiver and patient perspectives were shared. The emerging themes were the physical and psychological impact of the disease, the need for constant vigilance and fear of the future. Conclusion: Both patients and carers faced a range of challenges in managing a rare condition. From the seriousness of the illness, dealing with the emotional toll and knowing what to expect. This study offers insight into the experiences of patients and informal carers and health care professionals should address individuals’ fears and expectations for recovery. Disclosures: J.M. honoraria; Abbvie, Bristol Myres Squibb. K.G. and R.A.W. have declared no conflicts of interest

From Sandbox to Pandemic: Agile Reform of Canadian Drug Regulation

Public health urgency for emerging COVID-19 treatments and vaccines challenges regulators worldwide to ensure safety and efficacy while expediting approval. In Canada, legislative amendments by 2019 Om- nibus Bill C-97 created a new agile licensing framework known as the Advanced Therapeutic Pathway (ATPathway) and modernized the regulation of clinical trials of drugs, vaccines, and medical devices. Bill C-97′s amendments are worthy of attention in Canada and globally, as health product regulation bends to COVID-19. The amendments follow reforms elsewhere to accommodate health product innovation, how- ever, the Canadian ATPathway is broader and more flexible than its counterparts in other jurisdictions. In addition, Bill C-97 informed Canada’s COVID-19 response in important ways, particularly in relation to clinical trials. The measures adopted by the drug regulatory authority, Health Canada (HC) during COVID- 19 may become the new norm in Canadian regulatory practice insofar as they help achieve the amend- ments introduced by Bill C-97. Finally, despite government rhetoric of transparency, the agenda-setting, formulation, and implementation of the amendments have occurred with little opportunity for scrutiny or public engagement

Global vaccine safety blueprint: the landscape analysis (survey of regulators)

The perspectives of regulatory licensing authorities were assessed in countries that produce, procure, and both produce and procure vaccines. A web-based survey was developed to explore regulators’ knowledge, attitudes and practices concerning their national vaccine safety system. Additionally, their opinions about what would be needed to ensure future capacity and capabilities for a global vaccine safety system were probed, along with models of collaboration for regulatory authorities and the private sector that address public safety issues

Regulators, Pivotal Clinical Trials, and Drug Regulation in the Age of COVID-19

Medicine regulators rely on pivotal clinical trials to make decisions about approving a new drug, but little is known about how they judge whether pivotal trials justify the approval of new drugs. We explore this issue by looking at the positions of 3 major regulators: the European Medicines Agency, Food and Drug Administration, and Health Canada. Here we report their views and the implications of those views for the approval process. On various points, the 3 regulators are ambiguous, consistent, and demonstrate flexibility. The range of views may well reflect different regulatory cultures. Although clinical trial information from pivotal trials is becoming more available, regulators are still reluctant to provide detailed information about how that information is interpreted. As medicines and vaccines come up for approval for treatment of COVID-19, transparency in how pivotal trials are interpreted will be critical in determining how these treatments should be used

Regulators, Pivotal Clinical Trials, and Drug Regulation in the Age of COVID-19

Medicine regulators rely on pivotal clinical trials to make decisions about approving a new drug, but little is known about how they judge whether pivotal trials justify the approval of new drugs. We explore this issue by looking at the positions of 3 major regulators: the European Medicines Agency, Food and Drug Administration, and Health Canada. Here we report their views and the implications of those views for the approval process. On various points, the 3 regulators are ambiguous, consistent, and demonstrate flexibility. The range of views may well reflect different regulatory cultures. Although clinical trial information from pivotal trials is becoming more available, regulators are still reluctant to provide detailed information about how that information is interpreted. As medicines and vaccines come up for approval for treatment of COVID-19, transparency in how pivotal trials are interpreted will be critical in determining how these treatments should be used