Methodological Issues in Economic Evaluations Submitted to the Pan-Canadian Oncology Drug Review (pCODR)

Masucci, Lisa; Beca, Jaclyn; Sabharwal, Mona; Hoch, Jeffrey S.

doi:10.1007/s41669-017-0018-3

Cited by 18 publications

(22 citation statements)

References 29 publications

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“…In addition to the majority of studies not including original data on HRQOL, our results indicate that the data used to calculate QALYs were a frequent concern for the EGP and motivated re‐analysis for approximately 52% of the submissions. Our findings are consistent with previous evidence suggesting that 44% of submissions to pCODR between 2011 and 2014 had issues with utility estimates contained in the submissions . This result was corroborated by research reporting that there was often little confidence in the quality of economic evaluations submitted to pCODR, frequently because of how HRQOL benefits were incorporated into the analysis .…”

Section: Discussionsupporting

confidence: 90%

“…Our findings are consistent with previous evidence suggesting that 44% of submissions to pCODR between 2011 and 2014 had issues with utility estimates contained in the submissions. 9 This result was corroborated by research reporting that there was often little confidence in the quality of economic evaluations submitted to pCODR, frequently because of how HRQOL benefits were incorporated into the analysis. 10 There are 2 important implications of not using specific data on HRQOL and utility to make QALY calculations.…”

Section: Tablementioning

confidence: 89%

“…Beyond the collection of HRQOL data in cancer clinical trials, there are additional issues with drug submissions that require attention. For example, although most submissions adhere to the CADTH guidelines for economic evaluation, previous studies have highlighted many of the modeling issues that arise in manufacturers' submissions to pCODR, including problems with the model structure, misspecification of the time horizon, and extrapolation methods of the drug treatment effect . Because these trials are used to inform submissions in other jurisdictions as well, similar issues have been reported .…”

Section: Discussionmentioning

confidence: 99%

“…For example, although most submissions adhere to the CADTH guidelines for economic evaluation, 3 previous studies have highlighted many of the modeling issues that arise in manufacturers' submissions to pCODR, including problems with the model structure, misspecification of the time horizon, and extrapolation methods of the drug treatment effect. 9,11 Because these trials are used to inform submissions in other jurisdictions as well, similar issues have been reported. 12 Simultaneously, trials for cancer drugs are increasingly using surrogate outcomes such as progression-free survival (PFS) as their endpoint, but recent evidence reported by Kovic et al 13 has suggested that there is no association between PFS and HRQOL.…”

Section: Tablementioning

confidence: 94%

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Health‐related quality of life in oncology drug reimbursement submissions in Canada: A review of submissions to the pan‐Canadian Oncology Drug Review

Raymakers

Regier

Peacock

2019

Cancer

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Background In Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH) evaluates and makes recommendations for the reimbursement of cancer drugs. One component of its recommendation is based on an economic evaluation, which typically takes the form of a cost‐utility analysis. A cost‐utility analysis measures the effects of competing therapies with quality‐adjusted life‐years (QALYs). The data for this calculation typically come from generic, preference‐based measures of health‐related quality of life (HRQOL). The objective of this review is to determine the frequency at which HRQOL data are collected alongside cancer drug trials and used in the cost‐utility analysis submitted to the CADTH pan‐Canadian Oncology Drug Review (pCODR). Methods Submissions between 2015 and 2018 to pCODR, the group charged with evaluating cancer drug submissions at CADTH, were reviewed. All pCODR submissions, either in progress or completed, were publicly available online. The search was restricted to completed evaluations. Results Forty‐three submissions met the inclusion criteria. The incremental gain in QALYs in most submissions from the new technology was small (median incremental gain, 0.86; interquartile range, 0.6‐1.39). More than half of the submissions (56%) did not include original data on HRQOL, with most relying on previous studies of variable relevance and quality. Re‐analyses by pCODR based on concerns over HRQOL data used in the submitted model were common (52%). Conclusions Drug manufacturers do not consistently collect data on HRQOL alongside clinical trials and instead rely on evidence generated in previous studies to inform cost‐utility analyses. These findings should induce manufacturers to collect original HRQOL data that are simultaneously relevant to patients and decision makers.

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Section: Discussionsupporting

confidence: 90%

Section: Tablementioning

confidence: 89%

Section: Discussionmentioning

confidence: 99%

Section: Tablementioning

confidence: 94%

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Health‐related quality of life in oncology drug reimbursement submissions in Canada: A review of submissions to the pan‐Canadian Oncology Drug Review

Raymakers

Regier

Peacock

2019

Cancer

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“…Previous studies have looked at methodological issues and consistencies in economic evaluations by pCODR. 6,7 Almost all services related to cancer treatment are publicly funded in Canada, and cancer drugs are actively price-negotiated. This poses unique challenges and opportunities, with drug approvals that could vary systematically from private healthcare systems.…”

Section: New Cancer Drug Approvals From the Perspective Of A Universamentioning

confidence: 99%

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

Niraula¹,

Nugent²

2018

J Natl Compr Canc Netw

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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.

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Evaluation of the Clinical Benefit of Cancer Drugs Submitted for Reimbursement Recommendation Decisions in Canada

Meyers

Jenei

Chisamore³

et al. 2021

JAMA Intern Med

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IMPORTANCE Cancer drugs approved by the US Food and Drug Administration have come under scrutiny for marginal clinical benefits; however, the clinical benefits of cancer drugs recommended for reimbursement in Canada have not been adequately studied.OBJECTIVE To assess the differences in the clinical evidence and benefit of cancer drugs that received a positive vs a negative recommendation for provincial reimbursement in Canada. DESIGN, SETTING, AND PARTICIPANTSThis cohort study obtained publicly available regulatory documents from the pan-Canadian Oncology Drug Review (pCODR) and corresponding clinical trial documentation. All cancer drugs with a solid tumor indication that were submitted from the inception of the pCODR (July 2011) to February 2020 were evaluated. To be included, submissions had to have a final reimbursement recommendation; submissions that were incomplete, were withdrawn, or had a pending decision were excluded. EXPOSURES A completed reimbursement recommendation decision from the pCODR. MAIN OUTCOMES AND MEASURES Final reimbursement recommendation (positive vs negative); trial characteristics; and relevant clinical outcomes (ie, overall survival [OS] and progression-free survival [PFS]), including the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS) scores available at the time of pCODR assessment.RESULTS Between 2011 and 2020, the pCODR issued 104 reimbursement recommendation decisions for cancer drugs with a solid tumor indication. Among these drug submissions, 78 (75.0%) received a positive recommendation, of which 72 (92.3%) were conditional. Drugs that received a positive recommendation compared with those with a negative recommendation were more likely to have phase 3 randomized clinical trial design (92.3% [72 of 78] vs 53.8% [14 of 26]; P < .001) and have substantial benefit according to the ESMO-MCBS scores (61.5% [48 of 78] vs 19.2% [5 of 26]; P < .001). The most common primary end points associated with the successful submissions were PFS (53.9%) and OS (32.1%). Overall, 39 of 78 submissions (50.0%) that received a positive recommendation had shown OS benefit, with median (interquartile range) OS gains of 3.7 (2.7-6.5) months.CONCLUSIONS AND RELEVANCE This cohort study found that, although the pCODR takes into account the magnitude of clinical benefit, only half of the cancer drugs that received a positive recommendation had evidence of improved OS and the survival gains were usually modest. These results suggest that, although the pCODR helps filter out some cancer drugs with low quality of evidence and low magnitude of benefit, cancer drugs without meaningful patient benefit continue to enter the Canadian market; these findings are important for making reimbursement policy decisions globally.

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Methodological Issues in Economic Evaluations Submitted to the Pan-Canadian Oncology Drug Review (pCODR)

Cited by 18 publications

References 29 publications

Health‐related quality of life in oncology drug reimbursement submissions in Canada: A review of submissions to the pan‐Canadian Oncology Drug Review

Health‐related quality of life in oncology drug reimbursement submissions in Canada: A review of submissions to the pan‐Canadian Oncology Drug Review

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

Evaluation of the Clinical Benefit of Cancer Drugs Submitted for Reimbursement Recommendation Decisions in Canada

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