New Cancer Drug Approvals From the Perspective of a Universal Healthcare System: Analyses of the Pan-Canadian Oncology Drug Review Recommendations

Abstract

Background: FDA approvals do not consider cost, but they set the tone for regulatory approvals worldwide, including in countries with universal healthcare where cost-effectiveness, utility, and adoption feasibility are considered rigorously. Methods: Data from the pan-Canadian Oncology Drug Review (pCODR), a national drug review system that makes evidence-based funding recommendations to Canada's provinces and territories, were collected. Our objectives were to assess (1) temporal trends in cost and efficacy of drugs reviewed, (2) correlations among magnitude of benefits, cost, and pCODR decisions, and (3) predictors of approvals. Results: A total of 60 drugs for 91 indications were reviewed by pCODR from January 2012 to January 2018. Of the 91 reviews (approved previously by FDA), 18 received negative recommendations on the grounds of inadequate clinical benefits; 87% (64/73) of those approved were conditional on improvement in cost. Surrogate outcomes were used to support approvals in 83% of the reviews, which were not correlated with overall survival (r Spearman = +0.16; P=.24). Median cost/quality-adjusted life years (QALY) increased by 36% per annum (quantile regression, P=.0029), although benefits in overall and progression-free survival were stable (P=.21 and .65, respectively). Median-based incremental cost-effectiveness ratio (ICER) of new drugs was $186,403 CAD (range, $7,200 to $2.1 million). Higher ICER was a strong predictor of a negative pCODR recommendation (P<.01). Conclusions: A substantial number of cancer drugs that are FDA approved for public use do not meet Canadian standards for efficacy. Cost of cancer drugs increases by a third annually in Canada, but the benefits-measured mostly with surrogates that did not correlate with survival-are stable. With finite resources to share among multiple societal priorities, such as education and preventive health, cancer drug cost may be unsustainable despite price regulation.

A systematic literature review of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment

“…The majority of studies ( n = 7) (21–23;25–27;29) only investigated decisions in a single agency, except that Pinto et al (28) study researched the recommendations in two agencies in a separate analysis, and Maynou Pujolras and Cairns (24) study pooled the decisions from six European agencies. A total of six agencies were studied independently, including Pharmaceutical Benefits Advisory Committee (PBAC) in Australia (21), Commission of Reimbursement of Medicines (CRM) in Belgium (27), Health Insurance Review and Assessment Service (HIRA) in South Korea (22), National Authority for Health (HAS) in France (23), NICE in the UK (28), and pCODR in Canada (25;26;28;29). The number of HTA decisions ranged from 17 to 393 (median = 75).…”

Cost‐effectiveness analysis of cabozantinib as second‐line therapy in advanced hepatocellular carcinoma

“…The cost‐effectiveness of a proposed treatment is not a legislative mandate in the USA. The FDA does not consider potential costs when making regulatory decisions on marketing applications . Based on 30 drugs approved for cancer indications in 2015‐2017, gaps persist as to their financial harm compared with the related clinical benefit, although they are being routinely applied in a large‐scale fashion .…”

Strategizing health technology assessment for containment of cancer drug costs in a universal health care system: Case of the pan‐Canadian Oncology Drug Review

“…1 Hospital costs, drug costs, and physician costs represent approximately 28.3%, 16.4%, and 15.4%, respectively, of total health spending, but the growth in drug costs has outpaced that of hospitals and physicians in recent years. 2 Canadian provinces made independent decisions regarding cancer drug funding for many years until a joint national body, the pan-Canadian Oncology Drug Review (pCODR), was established in 2010 as part of the Canadian Agency for Drugs and Technologies in Health (CADTH) to "bring consistency and clarity" to the assessment of cancer drugs. 2 Canadian provinces made independent decisions regarding cancer drug funding for many years until a joint national body, the pan-Canadian Oncology Drug Review (pCODR), was established in 2010 as part of the Canadian Agency for Drugs and Technologies in Health (CADTH) to "bring consistency and clarity" to the assessment of cancer drugs.…”

“…1 With an increase in drug cost per quality-adjusted life-year of 36% per year, trends in cancer drug prices arguably have become the most disquieting cancer health policy issue today. 2 Canadian provinces made independent decisions regarding cancer drug funding for many years until a joint national body, the pan-Canadian Oncology Drug Review (pCODR), was established in 2010 as part of the Canadian Agency for Drugs and Technologies in Health (CADTH) to "bring consistency and clarity" to the assessment of cancer drugs. 3 Based on an objective evaluation of cancer drugs under 4 components of a deliberative framework (clinical benefits, economic analysis, patient-based values, and adoption feasibility), the pCODR provides reimbursement recommendations and advice to provincial and territorial public drug plans and provincial cancer agencies (with the exception of Quebec).…”