Canadian Agency For Drugs And Technologies In Health Research Articles

Timeliness of Health Technology Assessments and Price Negotiations for Oncology Drugs in Canada.

To evaluate whether time targets for Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement reviews and pan-Canadian Pharmaceutical Alliance (pCPA) price negotiations are being achieved for oncology drugs. Recommendations, dates of submission and publication, and indications for oncology medicines issued between January 2014 and December 2023 were recorded from CADTH's reimbursement reports webpage. The date any negotiation began and the date it was completed (successfully or not), or when a decision was made not to pursue negotiation was extracted from the pCPA's webpage. The duration of each CADTH review and pCPA negotiation was calculated, together with time between CADTH's recommendation and start of the pCPA negotiation or a decision not to negotiate. Percentages of reviews completed within CADTH's target and of times taken by the pCPA to decide whether to negotiate and by its price negotiations completed within the relevant targets were calculated. CADTH achieved its 270-days target in 88.2% to 100% of reviews issued between 2015 and 2019 but only in 65.9% to 73.1% of reviews issued in the last three years of the decade. CADTH's "typical timeline" of 180 days was achieved in under 40% of reviews issued in 2015 and not attained in any review in 2021, 2022 or 2023. The pCPA's target of 60 days for deciding whether to negotiate was achieved for all recommendations issued in 2014 but dropped below 40% for the last seven years of the decade; its target of 130 days for negotiations was achieved for over 85% of the recommendations in 2014 but decreased to only 14.3% in 2016 and then gradually increased to 61.5% in 2023. CADTH's "typical timeline" and the pCPA's targets were not met sufficiently to be meaningful. Their processes take too long for cancer drugs.

Cost-effectiveness of the top 100 drugs by public spending in Canada, 2015–2021: a repeated cross-sectional study

ObjectivesTo assess the distribution and spending by cost-effectiveness category among those drugs with the highest public spending levels in Canada.DesignRepeated cross-sectional study.SettingThe Canadian provinces of Manitoba, Ontario, New Brunswick, Nova...

An Exploratory Analysis of the Cost-Effectiveness of a Multi-cancer Early Detection Blood Test Compared with Standard of Care Screening in Ontario, Canada.

Determining whether multi-cancer early detection (MCED) tests are cost effective is important in deciding whether they should be included in the clinical path of cancer care, especially for cancers where screening tools do not exist. The main objective of this study is to determine the cost effectiveness of including a MCED screening regimen together with existing provincial screening protocols for selected cancers that are prevalent in Ontario, Canada, among average risk persons aged 50-75 years. The selected cancers include breast, colorectal, lung, esophageal, liver, pancreatic, stomach, and ovarian. Cost effectiveness was estimated from a provincial Ministry of Health perspective. A state-transition Markov model representing the decision path of both the proposed and existing screening strategies along the natural history of the selected types of cancers was implemented. The incremental cost-effectiveness ratio (ICER) was calculated using data from available literature and the guidelines published by the Canadian Agency for Drugs and Technologies in Health (CADTH) for conducting a cost-effectiveness analysis, which included a discount rate of 1.5% applied to all costs and outcomes. Costs were also converted to 2022 Canadian dollars. To test the robustness of the model, both univariate and probabilistic sensitivity analyses were conducted. MCED screening resulted in more diagnosed cases of each type of cancer, even at an earlier stage of disease. This was also associated with fewer related deaths compared with standard of care. Notwithstanding, the analysis revealed that the MCED intervention was not cost effective [ICER: CAD$143,369 per quality-adjusted life year (QALY)], given a willingness to pay (WTP) threshold of $100,000 per QALY. The probabilistic sensitivity analyses revealed that the MCED intervention strategy was preferred to standard of care no more than 2% of the time at this WTP for both males and females. The model was most sensitive to the cost of MCED screening, and the levels of specificity of the MCED and colorectal cancer screening tests. The main contribution of the study is to present and execute a methodological approach that can be adopted to test the cost effectiveness of an MCED tool in the Canadian setting. The model is also sufficiently generic that it could be adapted to other jurisdictions, and with consideration for increasing the WTP threshold beyond the common $100,000 per QALY limit, given the life-threatening nature of cancer, to ensure that MCED interventions are cost-effective.

Risk of sexual transmission of HIV in the context of viral load suppression.

In 2018, the Public Health Agency of Canada (PHAC) published a systematic review to calculate the risk of sexual transmission of human immunodeficiency virus (HIV) in the context of antiretroviral therapy (ART). In 2022, PHAC commissioned the Canadian Agency for Drugs and Technologies in Health (CADTH) to conduct a rapid review of evidence published since 2017. We undertook a meta-analysis of relevant studies from these two reviews. Studies from the rapid review that adequately assessed exposure (HIV viral load) and outcome (HIV seroconversion) were included and assessed for risk of bias (RoB) and certainty of evidence. Results were pooled to estimate the risk of HIV transmission per 100 person-years. Three studies from the rapid review were eligible for inclusion and one was excluded after RoB assessment. In the remaining studies examining risk among people living with HIV who take ART and maintain a suppressed viral load (fewer than 200 copies/mL, measured every 4-6 months), no sexual transmissions of HIV were observed. The pooled incidence estimate based on these studies, and one from the 2018 PHAC review, was zero transmissions/100 person-years (95% CI: 0.00-0.10). No studies in the rapid review provided data on the risk of sexual transmission of HIV in situations of varying levels of viral load. This update highlights the consistency of evidence since the 2018 PHAC review. There remains no evidence of HIV transmission to sexual partners when a person living with HIV is on ART and maintains a suppressed viral load.

Evaluating the impact of novel oncology drug coverage through patient assistance programs in British Columbia (BC), Canada.

13 Background: Despite a universal public healthcare system, Canadian oncology patients often enroll in patient assistance programs (PAPs) through drug manufacturers to access oncology drugs that are awaiting funding decisions by national and provincial regulatory bodies. Our multicenter study evaluated the pharmacoeconomic and clinical impact of PAPs for cancer patients in BC. Methods: Eligible patients were diagnosed with cancer and enrolled in a PAP for an oncology drug between January 1, 2016 - December 31, 2019 in 3 BC centres. Charts were reviewed for treatment details and survival data. We referenced median overall survival (mOS) or median progression-free survival (mPFS) data from phase III trials. mPFS data was used if mOS was unavailable or not statistically significant. For each drug indication, the hazard ratio from trial data was multiplied by the mOS or mPFS of our cohort to estimate the mOS or mPFS if the drug was not received. Life-years gained (LYG) was defined as the difference between the actual mOS or mPFS in our cohort and the estimated mOS or mPFS if the drug was not received. Mean OS was used if median OS was not reached by the cut-off date January 1, 2021. Quality-adjusted life years (QALY) and cost per drug were obtained from the Canadian Agency for Drugs and Technologies in Health (CADTH). QALY gained per drug was calculated as QALY multiplied by LYG. Assuming $100,000 Canadian dollars (CAD) for 1 QALY gained (based on prior studies), the economic value of QALY gained was calculated as $100,000 CAD multiplied by QALY gained per drug. Results: Our cohort consisted of 1025 patients that accessed 40 oncology drugs via PAP. By the cut-off date, 290 patients continued on treatment while 735 had stopped, most often due to disease progression (69%) or toxicity (14%). 74 patients then accessed another drug in their second exposure to PAP, and 5 patients accessed drugs in their third exposure to PAP. Median time from Health Canada approval to public funding was 2.04 years (IQR 1.53 – 2.34). QALY gained per drug ranged from 0.007-2.997 years for OS and 0.001-1.339 years for PFS. This translated to a total of 268.3 QALY gained in OS and 117.6 QALY gained in PFS. In the first exposure group, total economic value gained from PAP was $26,788,512 for those with OS benefits, $11,763,571 for those with PFS benefits, and total drug costs were $94,150,774. In the second exposure group, total economic value gained was $566,253 for those with OS benefits, $947,134 for those with PFS benefits, and total costs were $4,917,017. In the third exposure group, $44,754 total economic value was gained and total costs were $931,207. Conclusions: Nearly $100 million CAD of non-public funding was required to bridge gaps between regulatory approval and public funding. The economic value and QALY gained from PAPs are substantial. Future studies should focus on strategies to optimize the funding process for novel cancer therapies.

MSR28 The Power of One: Examining the Impact of Single Arm Trials on Oncology Drug Approvals by NICE and CADTH

Single-arm trials (SATs) are widely used to evaluate treatments for rare and orphan diseases, and for advanced and refractory cancer where conducting randomized controlled trials is not feasible or ethical. This study aims to evaluate the impact of SATs on treatment recommendations by the National Institute for Health and Care Excellence (NICE) in the UK and the Canadian Agency for Drugs and Technologies in Health (CADTH) in Canada.

HTA43 Do Complexities of Combination Oncology Submissions Impact Reimbursement Outcomes: An Evaluation of Multi-Therapy Oncology Regimens in Canada

The increasing number of oncology combination regimens has highlighted the complexity of multiple manufacturer evaluations. This study aims to assess the dynamics of combination oncology regimen reviews with the Canadian Agency for Drugs and Technologies in Health (CADTH) and the subsequent pricing negotiations through the pan-Canadian Pharmaceutical Alliance (pCPA).

EE262 Use of Single-Arm Trials in Cadth Reviews of Oncology Drugs

Single-arm trials (SAT) have been increasingly used to support oncology reimbursement submissions worldwide. In a previous study, the authors assessed the use of SAT-based oncology technical appraisals by the National Institute for Health and Care Excellence (NICE) from 2017 to 2022, approach to generate comparative evidence, and recommendations from committee reviews. The objective of this study was to assess the use of SATs in Canadian Agency for Drugs and Technologies in Health (CADTH) reviews of oncology drugs.

EE511 Modeling Approaches in the Cost-Effectiveness Analysis of Cancer Treatments: A Review of Submissions Made to the Canadian Agency for Drugs and Technologies in Health

Partitioned Survival Model (PSM) and State Transition Models are the most frequently used approaches to estimate the cost-effectiveness of treatments. However, each of these approaches have their own limitations in their application in oncology. Our objective was to review the reimbursement submissions of oncology treatments made to Canadian Agency for Drugs and Technologies in Health (CADTH) between 2018 and 2022 to examine the modeling approaches considered by manufacturers.

Conditional Funding Recommendations for Drugs in Canada: A Cross-Sectional Analysis.

Before listing by drug plans, new drugs that receive regulatory bodies' approval are assessed by the Health Technology Assessment agencies, including the Canadian Agency for Drugs and Technologies in Health (CADTH). Considering the impact of CADTH recommendations on payers' listing decisions, the growing number of high-cost drugs, and conditional listings, conditional recommendations have increasingly been of interest to the users of CADTH recommendations. This study aims to review the conditional reimbursement recommendations issued by CADTH and explores the reasons for those recommendations. All final drug reimbursement recommendations posted on the CADTH website since 1 January, 2003 were reviewed. Recommendations were included if accepted for review by 1 May, 2021 and completed before 1 January, 2022. The complete records excluding 'requests for advice' were categorized into 'reimburse,' 'do not reimburse,' and 'reimburse with criteria/condition(s)'. The identified variables were extracted and analyzed descriptively. Of the 889 submissions, 785 (88.6%) completed recommendations were consideredrelevant to the study purpose. Of 750 recommendations, excluding 35 (4.5%) 'requests for advice,' 500 (66.6%) were 'reimburse with criteria/condition(s)'. 'Clinical criteria' at 481 (96.2%), 'cost-effectiveness improvement to an acceptable level' at 132 (26.4%), and 'the treatment cost not to exceed the cost of the alternative option(s)' at 118 (23.6%) were the most frequently reported conditions. The most frequent reasons for conditional reimbursement were 'clinical benefit and economic evaluation' at 304 (60.8%). Of conditional recommendations, 468 (93.6%) included more than one reason. CADTH has facilitated patient access to innovative drugs even with limited evidence by conditional recommendations. The clinical criteria are the cornerstone of conditional recommendations. CADTH has developed the assessment process over time.

Access to Neoadjuvant Pertuzumab for HER2 Positive Breast Cancer in Canada: A Dilemma Increasingly Difficult to Explain

The addition of pertuzumab to neoadjuvant trastuzumab and chemotherapy for women with early-stage, high-risk, HER2+ breast cancer has been observed to lead to higher pathologic complete response rates (pCR), and improved event-free survival compared to trastuzumab and chemotherapy alone. Based on available data, neoadjuvant pertuzumab is recommended by ESMO, ASCO, and NICE as well as by a Canadian Consensus Guideline Group. We discuss the implications for Canadian patients with HER2+ early breast cancer due to a second and final negative funding decision by the Canadian Agency for Drugs and Technologies in Health (CADTH) related to neoadjuvant pertuzumab. This decision will have adverse impacts for up to 1 in 6 women receiving neoadjuvant therapy for high-risk HER2+ breast cancer, due to suboptimal pCR rates and higher risks of invasive breast cancer recurrent events, resulting in the need for more toxic adjuvant therapy.

OP49 A Systematic Review Of The Activities Of Early Advice, Early Dialogue, Scientific Advice By HTA Doers

IntroductionThere is a range of activities that health technology assessment (HTA) doers have started to improve the process of generation of required evidence for new technologies, and the alignment of regulatory and reimbursement processes that retard the access to patients to them. Different organizations call those processes early advice, early dialogue, or scientific advice to those activities.MethodsWe performed a systematic review of the activities named scientific advice (SA), early advice (EA) and early dialogue (ED). Major databases and HTA organizations were explored. The protocol and search strategy were published in PROSPERO. The selection of final articles and documents was done in pairs, and when discrepancies were found a third person resolved with the consensus of the others. A matrix was used to define the commonalities and differences of the described processes.ResultsWe initially retrieved 949 documents, after the analysis of duplications and the full text reading of the selected ones, we finally selected 39 documents and described: the type of technologies, the process, the stakeholders, the duration, the costs, and the impact. Big HTA agencies such as the Canadian Agency for Drugs and Technologies in Health (CADTH) or the National Institute for Health and Care Excellence (NICE) included EA or SA among their portfolio of activities as well as networks (European Network for HTA (EUnetHTA) or smaller agencies such as HTA Wales or Basque Office for HTA (Osteba) among others. The type of activity, the process, duration, purpose and costs differ among HTA doers.ConclusionsThere is a need to define what we meant when we are talking about SA, ED, and EA. In fact, regulators used the same processes with different purposes. Our systematic review and the lessons learnt from the European-funded SAFENMEDTECH project will propose a detailed framework that can be useful to better understanding the needs of each of the involved parties and how to make the processes involved more efficient.

HTA2 Conditional Recommendations by the Canadian Agency for Drugs and Technologies in Health (CADTH)

HTA2 Conditional Recommendations by the Canadian Agency for Drugs and Technologies in Health (CADTH)

Appraisals by Health Technology Assessment Agencies of Economic Evaluations Submitted as Part of Reimbursement Dossiers for Oncology Treatments: Evidence from Canada, the UK, and Australia.

Publicly funded healthcare systems, including those in Canada, the United Kingdom (UK), and Australia, often use health technology assessment (HTA) to inform drug reimbursement decision-making, based on dossiers submitted by manufacturers, and HTA agencies issue publicly available reports to support funding recommendations. However, the level of information reported by HTA agencies in these reports may vary. To provide insights on this issue, we describe and assess the reporting of economic methods in recent oncology HTA recommendations from the Canadian Agency for Drugs and Technologies in Health (CADTH), National Institute for Health and Care Excellence (NICE), and Pharmaceutical Benefits Advisory Committee (PBAC). Publicly available HTA recommendations and reports for oncology drugs issued by CADTH over a 2-year period, 2019–2020, were identified and compared with the corresponding HTA documents from NICE and the PBAC. Reporting of key model characteristics and attributes, survival analysis methods, methodological criticisms, and re-assessment of the economic results were characterized using descriptive statistics. Dichotomous differences in the methodological criticisms observed between the three agencies were assessed using Cochran’s Q tests and substantiated using pairwise McNemar tests. Chi-squared tests were used to assess the dichotomous differences in the reporting of methods and explore the potential relationships between categorical variables, where appropriate. HTAs published by CADTH, NICE, and the PBAC consistently reported a broad spectrum of descriptive information on the economic models submitted by manufacturers. While common economic evaluation attributes were well-reported across the three HTA agencies, significant differences in the reporting of survival analysis methods and methodological criticisms were observed. NICE consistently reported more comprehensive information, compared to either CADTH or PBAC. Despite these differences, broadly similar recommendation rates were observed between CADTH and NICE. The PBAC was found to be more restrictive. Based on our 2-year sample of oncology, the HTAs published by CADTH matched with the corresponding HTAs from NICE and PBAC; we observed important variations in the reporting of economic evidence, especially technical aspects, such as survival analysis, across the three agencies. In addition to guidelines for HTA submissions by manufacturers, the community of HTA agencies should also have common standards for reporting the results of their assessments, though the information and opinions reported may differ.

Burden of Atopic Dermatitis in Adults and Adolescents: a Systematic Literature Review.

Although previously regarded as a children's disease, it is clear that atopic dermatitis (AD) is also highly prevalent in adults. Because AD is not associated with mortality, it is usually neglected compared with other, fatal diseases. However, several studies have highlighted that AD burden is significant due to its substantial humanistic burden and psychosocial effects. This study aims to summarize and quantify the clinical, economic, and humanistic burden of AD in adults and adolescents. A systematic literature search was performed in PubMed, Scopus, Cochrane, Centre for Reviews and Dissemination (CRD), EconPapers, The Professional Society for Health Economics and Outcomes Research (ISPOR), The National Institute for Health and Care Excellence (NICE), and The Canadian Agency for Drugs and Technologies in Health (CADTH). Studies were included if they reported clinical, economic, or humanistic effects of AD on adults or adolescents, from January 2011 to December 2020. The Grading of Recommendations Assessment tool was used to assess risk of bias for the included studies. Regression models were used to explain the correlation between factors such as disease severity and quality of life (QoL). Among 3400 identified records, 233 studies were included. Itch, depression, sleep disturbance, and anxiety were the most frequently reported parameters related to the clinical and humanistic burden of AD. The average utility value in studies not stratifying patients by severity was 0.779. The average direct cost of AD was 4411 USD, while the average indirect cost was 9068 USD annually. The burden of AD is significant. The hidden disease burden is reflected in its high indirect costs and the psychological effect on QoL. The magnitude of the burden is affected by the severity level. The main limitation of this study is the heterogeneity of different studies in terms of data reporting, which led to the exclusion of potentially relevant data points from the summary statistics.

Time to potential for listing of new drugs on public and private formularies in Canada: a cross-sectional study.

Information about the timing involved in various stages of making new drugs available to Canadians is important for understanding how a national pharmacare plan will affect timely access to new drugs. I explored the timing of the various steps between receiving a Notice of Compliance and a decision by the pan-Canadian Pharmaceutical Alliance (pCPA). I gathered data from various databases (Canadian and other) about new drugs approved between 2011 and 2020, including generic names, date of application for approval (New Drug Submission [NDS]), date of Notice of Compliance, date of marketing, dates when a submission was made to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the pCPA, and when these agencies made a decision. Marketing dates were available for 301 of the 337 new drugs approved. The median time from NDS to marketing was less than the time to a positive pCPA decision for all years between 2011 and 2020. There was no significant change in the difference between the 2 periods over time (p = 0.2). Additional therapeutic value did not make a difference in the delay (p = 0.3) and companies did not take full advantage of the opportunity to file early submissions with CADTH. The delay between when drugs could be listed on private compared with public formularies was at least 1 year. If a national pharmacare plan is instituted, one of the priorities should be to concentrate on consolidating and working to shorten the CADTH and pCPA processes.

S622 Methodological Quality of Clinical Practice Guidelines for the Management of Antithrombotic Agents in Patients Undergoing GI Endoscopy: A Systematic Critical Appraisal Using the AGREE II Tool

Introduction: Several published clinical practice guidelines (CPGs) exist for the management of antithrombotic agents in patients undergoing GI endoscopy. However, to date, the quality of such CPGs has not been systematically appraised. The goal of this study was to identify and evaluate the quality of CPGs for the management of antithrombotic agents in peri endoscopic period published within last 6 years. Methods: A systematic search of PubMed and Embase database was performed to identify eligible CPGs. Any CPGs published between January 1, 2016 and April 14, 2022 addressing management of antithrombotic agents in patients undergoing GI endoscopy were eligible. The Canadian Agency for Drugs and Technologies in Health (CADTH) broad filter for the CPG terms was used since it is the most sensitive filter. The quality of the CPGs was independently assessed by five reviewers using the Appraisal of Guidelines for Research & Evaluation II (AGREE II) instrument. Domain scores were considered of sufficient quality when >60% and of good quality when >80%. Results: The search yielded 343 citations of which 7 were CPGs and included for the critical appraisal (Table) (one CPG was published in two journals). Five CPGs were published by the gastroenterology associations in Europe (n=2) and Asia (n=3) and two in the United States. The overall median score for the AGREE II domains was 100% for scope and purpose, 72% for stakeholder involvement, 86% for rigor of development, 100% for clarity of presentation, 29% for applicability and 89% for editorial independence. Table presents scores for the AGREE II domains for each included CPG. Conclusion: The findings from our study show that the overall methodological quality of the CPGs for the management of antithrombotic agents in patients undergoing GI endoscopic procedures varies across various domains. Only one CPG included patient representatives in the CPG development process. There is a significant scope for improvement for methodological rigor and the applicability. Table 1. - Clinical practice guidelines included in the critical appraisal using AGREE II instrument. Authors Title Journal AGREE II scores [%] Scope and purpose Stakeholder involvement Rigor of development Clarity of presentation Applicability Editorial independence Overall Acosta et al. The management of antithrombotic agents for patients undergoing GI endoscopy Gastrointest Endosc 2016 Jan;83(1):3-16. doi: 10.1016/j.gie.2015.09.035. 100% 29% 64% 100% 14% 57% 64% Kato et al. Guidelines for Gastroenterological Endoscopy in Patients Undergoing Antithrombotic Treatment: 2017 Appendix on Anticoagulants Including Direct Oral Anticoagulants Dig Endosc. 2018 Jul;30(4):433-440. doi: 10.1111/den.13184. 100% 57% 64% 86% 29% 79% 61% Chan et al. Management of patients on antithrombotic agents undergoing emergency and elective endoscopy: joint Asian Pacific Association of Gastroenterology (APAGE) and Asian Pacific Society for Digestive Endoscopy (APSDE) practice guidelines Gut 2018 Mar;67(3):405-417. doi: 10.1136/gutjnl-2017-315131 100% 86% 79% 100% 14% 100% 73% Farcas et al. Gastrointestinal endoscopy in patients on direct oral anticoagulants. A consensus paper of the Romanian Society of Gastroenterology and Hepatology J Gastrointestin Liver Dis. 2018 Jun;27(2):179-187. doi: 10.15403/jgld.2014.1121.272.end. 100% 57% 71% 71% 29% 86% 61% Lim et al. Clinical Practice Guideline for the Management of Antithrombotic Agents in Patients Undergoing Gastrointestinal Endoscopy Clin Endosc. 2020 Nov;53(6):663-677. doi: 10.5946/ce.2020.192 100% 86% 100% 100% 29% 100% 85% Veitch et al. Endoscopy in patients on antiplatelet or anticoagulant therapy: British Society of Gastroenterology (BSG) and European Society of Gastrointestinal Endoscopy (ESGE) guideline update Endoscopy. 2021 Sep;53(9):947-969. doi: 10.1055/a-1547-2282 100% 100% 100% 100% 43% 100% 90% Abraham et al. American College of Gastroenterology-Canadian Association of Gastroenterology Clinical Practice Guideline: Management of Anticoagulants and Antiplatelets During Acute Gastrointestinal Bleeding and the Periendoscopic Period 1. J Can Assoc Gastroenterol.2022 5(2):100-101. doi: 10.1093/jcag/gwac010.2. Am J Gastroenterol 2022 117(4):542-558. doi:10.14309/ajg.0000000000001627. 100% 100% 100% 100% 57% 93% 83%

HTA4 Applicability of Willingness to Pay Thresholds for Oncology Drugs: A Review of Submissions Made to the Canadian Agency for Drugs and Technologies in Health

Canadian Agency for Drugs and Technologies in Health (CADTH) has no established incremental cost-effectiveness ratio (ICER) threshold for reimbursement purposes. However, a willingness to pay (WTP) threshold of $50,000 per Quality Adjusted Life Year (QALY) is viewed favourably for both oncology and non-oncology products. We aimed to review the submissions of cancer drugs made to CADTH from January 2016 to June 2021, to understand the applicability of willingness to pay thresholds acceptable for CADTH.

Medicines pricing and reimbursement in Canada

Objective: overview of Canadian practices for regulating, financing, and funding prescription drugs. Canada provides universal health coverage for hospital and physician services but excludes universal insurance of prescription medicines. Public plans provide 42% of financing, while private drug insurance covers 35% of expenditures and over 60% of Canadians – mainly through their employer. Canada has relatively high out-of-pocket expenditure (19% of spending) and is currently the tenth largest pharmaceutical market, following Brazil. It is wrestling with inequitable coverage, low use of biosimilars, and affordability and sustainability issues driven by rare disease drugs. Both federal and provincial/territorial governments and their agencies have roles in setting policy and regulating drug prices and costs. These include the federal Patented Medicine Prices Review Board (PMPRB) which ensures prices of new patented drugs are not excessive; the pan-Canadian Pharmaceutical Alliance (pCPA) which negotiates lower patented, generic and biosimilar drug prices on behalf of member jurisdictions; and the Canadian Agency for Drugs and Technologies in Health (CADTH) which provides most public drug plans with robust health technology assessment (HTA), including clinical, economic and budget impact analyses of new drugs. Private drug insurers tend to follow government initiatives, including the use of HTA and confidential Product Listing Agreements. Conclusions: Pharmaceutical coverage in Canada is a “patchwork” of more than 100 public drug plans and 100,000 private insurance plans. As such, it creates gaps in coverage which result in inequitable access and high out-of-pocket drug expenses for some Canadians. Canada’s decentralized health system and the absence of universal drug insurance, among other factors, likely contribute to higher per capita drug expenditure relative to comparable nations that have broader, publicly-funded universal health insurance and more rigourous policy and program strategies.

Health technology assessment and price negotiation alignment for rare disorder drugs in Canada: Who benefits?

BackgroundSince 2014, the Canadian Agency for Drugs and Technologies in Health (CADTH), which performs health technology assessments for all federal, provincial and territorial government drug programs (except Quebec’s) and the pan-Canadian Pharmaceutical Alliance (pCPA), which conducts price negotiations with manufacturers for all government drug programs, have been aligning their processes.ObjectiveTo examine trends in CADTH recommendations for non-oncology drugs for rare disorders (DRDs) released between 2014 and 2021, results of pCPA negotiations for the same drugs, and listings in government drug plans to assess who benefits from the alignment.ResultsRecommendations were positive in 87% of the reviews, although all included clinical criteria for use and/or economic conditions. Almost 90% of the DRDs with a positive recommendation had a successful price negotiation and 71% of those with a negative recommendation had no negotiation. Although no recommendation published before mid-2016 had a specified price reduction, almost 95% of those issued afterwards included the price reduction required to achieve a specific low cost-effectiveness threshold. The median time between the DRDs receiving marketing approval and a completed price negotiation was 663 days. Negotiations for DRDs completed after 2017 generally had fewer listings in government drug plans, but there was no distinct trend. The drug’s price likely played a role in listing decisions. When DRDs were listed, drug plans had access criteria consistent with CADTH’s or stronger for all the DRDs.ConclusionsThe governments who own, fund and manage CADTH and the pCPA benefit from their alignment. The alignment is less beneficial for patients waiting for access to the DRDs. The time taken by CADTH and pCPA actions and individual government drug plans to make listing decisions delays access. CADTH’s clinical criteria have become more extensive and are applied rigorously by drug plans which restricts patient access to DRDs. Canadians with rare disorders urgently need their governments to implement a long-overdue, comprehensive rare disease strategy to ensure DRDs are reviewed and reimbursed quickly and equitably to provide adequate health care to all who need them.