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Until now, there has been no standardized method of performing and presenting budget impact analyses (BIAs) in Canada. Nevertheless, most drug plan managers have been requiring this economic data to inform drug reimbursement decisions. This paper describes the process used to develop the Canadian BIA Guidelines; describes the Guidelines themselves, including the model template; and compares this guidance with other guidance on BIAs. The intended audience includes those who develop, submit or use BIA models, and drug plan managers who evaluate BIA submissions. The Patented Medicine Prices Review Board (PMPRB) initiated the development of the Canadian BIA Guidelines on behalf of the National Prescription Drug Utilisation Information System (NPDUIS). The findings and recommendations from a needs assessment with respect to BIA submissions were reviewed to inform guideline development. In addition, a literature review was performed to identify existing BIA guidance. The detailed guidance was developed on this basis, and with the input of the NPDUIS Advisory Committee, including drug plan managers from multiple provinces in Canada and a representative from the Canadian Agency for Drugs and Technologies in Health. A Microsoft® Excel-based interactive model template was designed to support BIA model development. Input regarding the guidelines and model template was sought from each NPDUIS Advisory Committee member to ensure compatibility with existing drug plan needs. Decisions were made by consensus through multiple rounds of review and discussion. Finally, BIA guidance in Canadian provinces and other countries were compared on the basis of multiple criteria. The BIA guidelines consist of three major sections: Analytic Framework, Inputs and Data Sources, and Reporting Format. The Analytic Framework section contains a discussion of nine general issues surrounding BIAs (model design, analytic perspective, time horizon, target population, costing, scenarios to be compared, the characterisation of uncertainty, discounting, and validation methods). The Inputs and Data Sources section addresses methods for market size estimation, comparator selection, scenario forecasting and drug price estimation. The Reporting Format section describes methods for BIA reporting. The new Canadian BIA Guidelines represent a significant departure from the limited guidance that was previously available from some of the provinces, because they include specific details of the methods of performing BIAs. The Canadian BIA Guidelines differ from the Principles of Good Research Practice for BIAs developed by the International Society for Pharmacoeconomic and Outcomes Research (ISPOR), which provide more general guidance. The Canadian BIA Guidelines and template build upon existing guidance to address the specific requirements of each of the participating drug plans in Canada. Both have been endorsed by the NPDUIS Steering Committee and the PMPRB for the standardization of BIA submissions. [ABSTRACT FROM AUTHOR]

Transformative cell and gene therapies have now launched worldwide, and many potentially curative cell and gene therapies are in development, offering the prospect of significant health gains for patients. Access to these therapies depend on decisions made by health technology assessment (HTA) and payer organizations. We sought to describe the emerging cell and gene therapies market access landscape by analyzing 17 US commercial payer medical policies, and HTA reports from five European countries and Canada. We found that some US health plans applied coverage restrictions more often than others (four plans applied restrictions in all decisions, while four plans applied restrictions in <30% of decisions). The European and Canadian HTA bodies recommend access to fewer therapies than US health plans, reflecting a more stringent approach in the context of limited evidence and high scientific uncertainty that is commonly associated with these treatments. Our findings suggest that patient access to approved cell and gene therapies is restricted in all regions studied, though the nature of these restrictions differs between US health plans and the European/Canada HTA recommendations. Payers, HTA groups, pharmaceutical companies, and other stakeholders should collaborate to more clearly define the "uncertainties" and develop market access policies that balance benefits of early access with ongoing data collection to close evidence gaps over time., (Copyright © 2021. Published by Elsevier B.V.)

Background: Regulatory approval and reimbursement are necessary to bring health technologies to market. As both are distinct processes, the lack of interaction between regulators and payers has been criticized for resulting in health system inefficiencies., Objectives: This study describes the experiences of health system stakeholders on the development and implementation of initiatives to better harmonize HTA-reimbursement and regulatory activities, as well as Canadian perceptions on such initiatives., Methods: Thematic analysis of semi-structured interviews was conducted. Participants represented those whom have published or worked actively in this area or who held positions in HTA and/or reimbursement agencies that gave them insight into the Canadian healthcare system. Interviews were recorded, transcribed and analyzed to identify emerging themes and relationships., Results: Fourteen individuals from twelve organizations participated, including representatives from Canada, US, UK and Netherlands. Harmonization was identified as either a means to: (i) develop economies of scale in the generation of clinical data; and/or (ii) align the lifecycle of a health technology. For such initiatives to be successfully implemented, the following key issues emerged: (i) fostering healthy relationships; (ii) promoting well-intentions; (iii) defining governance and leadership clearly; and (iv) securing organizational infrastructure., Conclusions: Attempts to harmonize reimbursement and regulatory activities is in its infancy; although, much can be learned from current Canadian and international experiences. Within Canada there is much interest, although concerns have been raised on the extent to which harmonization is possible. Successful policy implementation would require inter-agency collaborations, with particular attention towards capacity building and strong leadership.

Should patients in a randomized, pragmatic health economics trial be allowed to switch therapy in mid-trial to that provided in the other arm? Specifically, should patients in the treatment arm (T) be allowed to switch to the therapy of the comparator arm (C) if they need a change of therapy--that is, should TC switches be allowed? Also, should patients in the comparator arm be allowed to switch to the therapy of the treatment arm if they need changes of therapy--should CT switches be allowed? This is a nontrivial issue in study design that has been debated in the clinical trials literature and is currently being handled inconsistently in the health economics literature. In this article, the authors argue that TC switches should always be allowed and that CT switches should be allowed or not depending on the economic question. They further argue that the most common economic question is one that would lead to CT switches not being allowed.

The authors examine a number of ways in which willingness to pay (WTP) can be defined for measurement and use in a cost-benefit analysis (CBA) of a collectively funded health care program. They show how ambiguous specification of the program consequences that respondents should consider in their WTP responses can lead to problems of double counting or zero countingin a subsequent CBA. An example is whether the value of lost time from work because of poor health should be included by a CBA analyst (e.g., valued at the wage rate) as a separate cost item or whether this has already been monetized and included in respondents' WTP data. The authors highlight how differences in assumed or actual institutional structures are often ignored in measures of WTP and the consequences of this for the interpretation of WTP data.

The aging population and the increasing availability of new medical technologies, particularly pharmaceuticals, have led to growing pressure on governments worldwide to contain healthcare costs. Increasingly, economic evaluation is used to aid decisions on the reimbursement and formulary access of drugs, and pharmaceutical companies are often required to demonstrate the cost effectiveness of their products. Canada and the UK are examples of countries that have successfully incorporated mandatory requirements for economic evaluations into the decision-making process in healthcare. Japan faces cost-containment issues for its health and welfare system similar to those seen elsewhere in the world. Despite this, economic assessments are not currently used in the allocation of drug budgets. Reasons why economic evaluations for healthcare have not yet been used routinely in Japan include governmental approaches to healthcare cost containment, the pricing of pharmaceuticals, the organisation of the healthcare system, attitudes of the medical profession, and limited knowledge and expertise. However, small but encouraging steps are now being taken towards the introduction of economic evaluations in Japanese medicine.