Table 2. Trial Characteristics and Clinical Benefits of Cancer Drugs Recommended for Reimbursement by Pan-Canadian Oncology Drug Review vs Cancer Drugs Approved by the US Food and Drug Administrationa.
| Characteristic | Current findings for drugs recommended by pCODR | Previous findings for drugs approved by the FDA | FDA data source |
|---|---|---|---|
| Percent of submissions that received a positive recommendation, No./total No. (%) | 78/104 (75.0) | Data unavailable | NA |
| Period of assessment | 2011-2020 | 2002-2014 | Fojo et al,3 2014 |
| 2009-2018 | Gyawali et al,19 2019 | ||
| 2009-2013 | Mailankody and Prasad,20 2015 | ||
| 2006-2016 | Tibau et al,21 2018; Tibau et al,22 2018 | ||
| Randomized trial design, No./total No. (%) | 72/78 (92.3) | 116/153 (75.8) | Gyawali et al,19 2019 |
| Improved OS | 39/78 (50.0) | 19/63 (30.2) | Mailankody and Prasad,20 2015 |
| Approved with RR | 5/78 (6.4) | 22/63 (34.9) | Mailankody and Prasad,20 2015 |
| Median gains in OS, mo | 3.7 | 2.1 | Fojo et al,3 2014 |
| 2.9 | Gyawali et al,19 2019 | ||
| Median gains in PFS, mo | 4.7 | 2.5 | Fojo et al,3 2014 |
| Substantial clinical benefit by the ESMO-MCBS, No./total No. (%) | 48/78 (61.5) | 46/105 (43.8), RCT | Tibau et al,21 2018 |
| 45/133 (33.8), single-arm trial | Tibau et al,22 2018 |
Abbreviations: ESMO-MCBS, European Society for Medical Oncology-Magnitude of Clinical Benefit Scale; FDA, US Food and Drug Administration; NA, not applicable; OS, overall survival; pCODR, pan-Canadian Oncology Drug Review; PFS, progression-free survival; RCT, randomized clinical trial; RR, response rate.
All 26 submissions that received a negative recommendation from the pCODR have been approved by the FDA, whereas no drugs that received a positive recommendation from the pCODR were not approved by the FDA. The 26 submissions that received a negative recommendation from the pCODR but were approved by the FDA included crizotinib (lung), pazopanib hydrochloride (sarcoma), regorafenib (colorectal), lapatinib ditosylate (breast), cetuximab (colorectal), aflibercept (colorectal), pertuzumab (neoadjuvant breast), sorafenib tosylate (thyroid), regorafenib (colorectal resubmission), ceritinib (lung), olaparib (ovary), trabectedin (sarcoma), alectinib hydrochloride (lung), dabrafenib mesylate plus trametinib dimethyl sulfoxide (lung), panitumumab (colorectal), TAS-102 (colorectal), pertuzumab plus trastuzumab (breast adjuvant), nivolumab (liver), lenvatinib mesylate (renal), brigatinib (lung), TAS-102 (colorectal resubmission), pembrolizumab (urothelial), atezolizumab (small cell lung), larotrectinib sulfate (histology agnostic), neratinib maleate (breast adjuvant), and lorlatinib (lung). A complete list of all drugs and indications is provided in eTable 3 in the Supplement. A submission that previously received a negative recommendation could have been resubmitted and later received a positive recommendation.