HTA decision-making for drugs for rare diseases: comparison of processes across countries.

INTRODUCTION: Drugs for rare diseases (DRDs) offer important health benefits, but challenge traditional health technology assessment, reimbursement, and pricing processes due to limited effectiveness evidence. Recently, modified processes to address these challenges while improving patient access have been proposed in Canada. This review examined processes in 12 jurisdictions to develop recommendations for consideration during formal government-led multi-sectoral discussions currently taking place in Canada. METHODS: (i) A scoping review of DRD reimbursement processes, (ii) key informant interviews, (iii) a case study of evaluations for and the reimbursement status of a set of 7 DRDs, and (iv) a virtual, multi-stakeholder consultation retreat were conducted. RESULTS: Only NHS England has a process specifically for DRDs, while Italy, Scotland, and Australia have modified processes for eligible DRDs. Almost all consider economic evaluations, budget impact analyses, and patient-reported outcomes; but less than half accept surrogate measures. Disease severity, lack of alternatives, therapeutic value, quality of evidence, and value for money are factors used in all decision-making process; only NICE England uses a cost-effectiveness threshold. Budget impact is considered in all jurisdictions except Sweden. In Italy, France, Germany, Spain, and the United Kingdom, specific factors are considered for DRDs. However, in all jurisdictions opportunities for clinician/patient input are the same as those for other drugs. Of the 7 DRDs included in the case study, the number that received a positive reimbursement recommendation was highest in Germany and France, followed by Spain and Italy. No relationship between recommendation type and specific elements of the pricing and reimbursement process was found. CONCLUSIONS: Based on the collective findings from all components of the project, seven recommendations for possible action in Canada are proposed. These focus on defining "appropriate access", determining when a "full" HTA may not be needed, improving coordination among stakeholder groups, developing a Canadian framework for Managed Access Plans, creating a pan-Canadian DRD/rare disease data infrastructure, genuine and continued engagement of patient groups and clinicians, and further research on different decision and financing options, including MAPs.

Closing the Gaps to Timely Patient Access: Perspectives on Conditional Funding Models.

The Canadian system for approval of new cancer drugs is complex with multiple steps. Health Canada grants a license for a drug to be marketed and prescribed. The Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d'excellence en santé et services sociaux (INESSS) make recommendations by way of health technology assessments (HTA). If positive, the latter then lead to confidential price negotiations at the pan-Canadian pharmaceutical alliance (pCPA), after which individual provinces and territories make a listing decision. Delays can occur at each stage, but post-HTA delays can be lengthy and unpredictable, denying or impeding access to an effective drug with the potential for devastating clinical outcomes. Conditional funding models have been adopted in a number of European countries with the goal of providing timely access to new medications in areas of unmet need, in advance of further steps in the reimbursement process. This manuscript discusses different stakeholder perspectives on conditional funding agreements-including a recent successful example of such a process in the UK-based on a panel discussion at the 2021 Canadian Association of Population Therapeutics (CAPT) Conference.

Sensor-Based Technology: Bringing Value to People with Diabetes and the Healthcare System in an Evolving World.

PURPOSE: Evidence demonstrates that glucose-sensing technologies have enabled effective glycemic control for adults and children with type 1 diabetes (T1DM) or adults with type 2 diabetes (T2DM) on insulin therapy or non-insulin therapy. Here, we report on the wider value of glucose-sensing technology from the perspectives of person living with diabetes (PWD), healthcare providers (HCPs), and healthcare policy stakeholders. METHODOLOGY: Literature searches were conducted to identify published records and analysis, including across various healthcare organizations and agencies, of the impact of the FreeStyle Libre® flash glucose monitoring system in diabetes. These findings were combined with the outcomes of three healthcare attitudes surveys among PWD and diabetes healthcare professionals in Canada, including two commissioned for this purpose. RESULTS: Clinical trials data and real-world evidence have proven the benefits of the FreeStyle Libre system on limiting hypoglycemia, lowering HbA1c, optimizing metrics of glucose control and reducing hospital admissions. These benefits are accompanied by improvements in patients' quality of life, work productivity, and savings to the health system. The FreeStyle Libre system has created an opportunity to change the organization and delivery of care, including during COVID-19 restrictions on access to standard care, thus generating system-wide benefits in addition to those accrued by patients and HCPs. CONCLUSION: Evidence-based improvements in glucose control for PWD using flash glucose monitoring are accompanied by increased treatment satisfaction and quality of life. Telemedicine with such remote monitoring systems increases the opportunities for simultaneous review of glucose data with HCPs and shared decision-making, thus encouraging adherence with treatment.

Bringing patient centricity to diabetes medication access in Canada.

Canada must become proactive in addressing type 2 diabetes. With the second highest rate of diabetes prevalence in the developed world, the number of Canadians living with diabetes will soon reach epidemic levels. Against international comparisons, Canada also performs poorly with respect to diabetes-related hospitalizations, mortality rates, and access to medications. Diabetes and its comorbidities pose a significant burden on people with diabetes (PWD) and their families, through out-of-pocket expenses for medications, devices, supplies, and the support needed to manage their illness. Rising direct and indirect costs of diabetes will become a drain on Canada's economy and undermine the financial stability of our health care system. Canada's approach to diabetes medication assessment and funding has created a patchwork of medication access across provinces. Access to treatments for those who rely on public programs is highly restricted compared to Canadians with private drug plans, as well in contrast with public payers in other countries. Each person living with diabetes has different needs, so a "patient-centric" approach ensures treatment focused on individual circumstances. Such tailoring is difficult to achieve, with the linear approach required by public payers. We may be undermining optimal care for PWD because of access policies that are not aligned with individualized approaches - and increasing overall health care costs in the process. The scope of Canada's diabetes challenge demands holistic and proactive solutions. Canada needs to get out from "behind the eight ball" and get "ahead of the curve" when it comes to diabetes care. Improving access to medications is one of the tools for getting there. Canada's "call to action" for diabetes starts with effective implementation of existing best practices. A personalized approach to medication access, to meet individual needs and optimize outcomes, is also a key enabler. PWD and prescribers need reimbursement approaches that allow them to use existing tools (ie, medications and supplies) to manage diabetes in a timely manner and to avoid and/or delay major downstream complications.

Evolution of health technology assessment: best practices of the pan-Canadian Oncology Drug Review.

BACKGROUND: In 2007, Canada chose to develop a separate and distinct path for oncology drug health technology assessment (HTA). In 2013, the decision was made to transfer the pan-Canadian Oncology Drug Review (pCODR) to the Canadian Agency for Drugs and Technologies in Health (CADTH), to align the pCODR and CADTH Common Drug Review processes while building on the best practices of both. The objective of this research was to conduct an examination of the best practices established by the pCODR. METHODS: A qualitative research approach was taken to assess the policies, processes, and practices of the pCODR, based on internationally accepted best practice "principles" in HTA, with a particular focus on stakeholder engagement. Publicly available information regarding the approach of the pCODR was used to gauge the agency's performance against these principles. In addition, stakeholder observations and real-world experiences were gathered through key informant interviews to be inclusive of perspectives from patient advocacy groups, provincial and/or cancer agency decision-makers, community and academic oncologists, industry, expert committee members, and health economists. RESULTS: This analysis indicated that, through the pCODR, oncology stakeholders have had a voice in and have come to trust the quality and relevance of oncology HTA as a vital tool to ensure the best decisions for Canadians with cancer and their health care system. It could be expected that adoption of the principles and processes of the pCODR would bring a similar level of engagement and trust to other HTA organizations in Canada and elsewhere. CONCLUSION: The results of this research led to recommendations for improvement and potential extrapolation of these best practices to other HTA organizations worldwide, along with suggestions for continued evolution of the pCODR in conjunction with its integration into the CADTH. It is clear that the transition of the pCODR to CADTH provides an opportunity for practices initiated by the pCODR to become the standard for these newly amalgamated HTA agencies in Canada.

Health-related quality of life and regulatory issues in the US and Canada.

The purpose of this paper is to examine the utilisation of HR-QOL information by US and Canadian federal regulatory bodies, with a focus on pre- and postmarket product review processes. There are a number of challenges facing regulators in using HR-QOL data derived from clinical trials and/or pharmacoeconomic analyses. Some of these challenges are inherent to HR-QOL tools and methodologies, while others relate to their inappropriate application in clinical studies. Regulators also need to take some responsibility for the chasm that exists between the potential and the reality of the contribution of HR-QOL information in decision making. Federal regulators need to develop and promulgate clear guidances that will help improve the quality and validity of studies submitted for review, and greater transparency is needed in terms of how such information is used in the product review process. Advances in technology are sure to increase the volume and complexity of HR-QOL information collected within clinical trials, and regulators need to prepare themselves to address this influx of data. Key actions are proposed to improve both the credibility as well as the uptake of HR-QOL information within the regulatory process so that this important source of information can be used appropriately and to its full potential.