Background: We examined if oncology drug indications with high clinical benefit, as measured by the American Society of Clinical Oncology Value Framework (ASCO-VF) and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS), received public reimbursement status faster than those with lower clinical benefit from the time of pan-Canadian Oncology Drug Review (pCODR) recommendation.

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Background: Regulatory approval of oncology drugs is often based on interim data or surrogate endpoints. However, clinically relevant data, such as long-term overall survival and quality of life (QoL), are often reported in subsequent publications. This study evaluated the ASCO-Value Framework (ASCO-VF) net health benefit (NHB) at the time of approval and over time as further evidence arose. Methods: FDA-approved oncology drug indications from January 2006 to December 2016 were reviewed to identify clinical trials scorable using the ASCO-VF. Subsequent publications of clinical trials relevant for scoring were identified (until December 2019). Using ASCO-defined thresholds (≤40 for low and ≥45 for substantial benefit), we assessed changes in classification of benefit at 3 years postapproval. Results: Fifty-five eligible indications were included. At FDA approval, 40.0% were substantial, 10.9% were intermediate, and 49.1% were low benefit. We then identified 90 subsequent publications relevant to scoring, including primary (28.9%) and secondary endpoint updates (47.8%), safety updates (31.1%), and QoL reporting (47.8%). There was a change from initial classification of benefit in 27.3% of trials (10.9% became substantial, 9.1% became low, and 7.3% became intermediate). These changes were mainly due to updated hazard ratios (36.4%), toxicities (56.4%), new tail-of-the-curve bonus (9.1%), palliation bonus (14.5%), or QoL bonus (18.2%). Overall, at 3 years postapproval, 40.0% were substantial, 9.1% were intermediate, and 50.9% were low benefit. Conclusions: Because there were changes in classification for more than one-quarter of indications, in either direction, reassessing the ASCO-VF NHB as more evidence becomes available may be beneficial to inform clinical shared decision-making. On average, there was no overall improvement in the ASCO-VF NHB with longer follow-up and evolution of evidence.

e19360 Background: We examined if publicly reimbursed oncology drug indications with evidence of high clinical benefit, as measured by the American Society of Clinical Oncology Value Framework v2 (ASCO-VF), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale v1.1 (ESMO-MCBS), received reimbursement status faster than those with lower clinical benefit from the time of pCODR recommendation. Methods: Oncology drug indications that received pCODR recommendations between Jan 2012 and July 2018 were identified. Indications that did not receive provincial reimbursement, without notice of compliance, or received a negative pCODR recommendation were excluded. The relationship between clinical benefit, as measured by ASCO-VF and ESMO-MCBS, and the time to reimbursement was evaluated using Spearman correlation coefficient, univariable, and multivariable linear regression analyses. Results: Overall, 84 indications met inclusion criteria yielding 80 ASCO-VF and 66 ESMO-MCBS scores. The mean ASCO-VF and ESMO-MCBS scores were 38.8 (SD = 23.8) and 3.0 (SD = 1.1) respectively. Higher ASCO-VF and ESMO-MCBS scores had low correlation with shorter time to provincial funding, (rho = -0.15, 95%CI -0.24, -0.06) and (rho = -0.25, 95%CI -0.34, -0.16) respectively. Univariable analyses showed that manufacturer reported incremental cost effectiveness ratio (ICER) values, year of pCODR recommendation, province and cancer type were associated with time to public reimbursement (all p < 0.0001). After adjusting for potential confounders in the respective multivariable analysis, ASCO-VF (p = 0.29) and ESMO-MCBS (p = 0.15) scores were not significantly associated with time to public reimbursement. Year of pCODR recommendation remained associated with time to public reimbursement (p < 0.001). Earlier years (2012-2014) had a shorter time to reimbursement (mean = 10.4 months) than later years (2015-2018) (mean = 14.5 months). Other factors that were associated with time to reimbursement in multivariable analysis were province (p < 0.001) and cancer type (p < 0.001). Conclusions: Currently, oncology drug indication with evidence of high clinical benefit do not appear to be funded faster than those with low clinical benefit. This suggests the need to prioritize cancer drug indications based on clinical benefit in order to allow for timely public reimbursement of cancer drugs with higher clinical benefit to patients.

7011 Background: Regulatory approval of oncology drugs are often based on data presented in the primary publication of clinical trials (CT). However, clinically relevant data, such as long-term overall survival (OS) and quality of life (QOL), are often reported in subsequent publications. Therefore, this study aimed to evaluate the ASCO-VF NHB at the time of drug approval and over time as further evidence is published. Methods: All FDA approved oncology drug indications from 01/06-12/16 were reviewed to identify CTs that were scorable using the ASCO-VF version 2. Subsequent publications of included CTs relevant for scoring were identified from Web of Science with a follow-up time of 3 years from approval. Using ASCO-defined threshold scores of ≤40 for low benefit and ≥45 for substantial benefit, changes in classification of benefit were assessed at 3-years post-FDA approval. Results: We identified 57 FDA approved indications (40.4% OS, 59.6% progression-free survival (PFS) as primary endpoints) with scorable ASCO-VF CTs. Among those 57 indications, 36.8% at the time of FDA approval demonstrated substantial benefit, 10.5% demonstrated intermediate benefit, and 52.6% demonstrated low benefit. We then identified 96 subsequent publications relevant to scoring within 3-years of FDA approval, consisting of primary endpoint updates (29.2%; 14.6% OS, 12.5% PFS), secondary endpoint updates (44.8%; 16.7% OS, 7.3% PFS), new reporting of secondary endpoint (4.2% OS), safety updates (28.1%), and QOL reporting (43.8%). Upon reassessment of the NHB in subsequent publications, there was an overall change from initial classification of benefit in 36.8% of trials (17.5% became substantial, 8.8% became low, and 10.5% became intermediate). Changes in scores were mainly the result of an updated hazard ratio (35.1%), change in scoring endpoints from PFS to OS as per ASCO-VF endpoint hierarchy (8.8%), toxicity updates (57.9%), new tail of the curve bonus (12.3%), palliation bonus (14.0%), or QOL bonus (22.8%). Overall, at reassessment at 3 years post-FDA approval, 42.1% were substantial, 10.5% were intermediate, and 47.3% were low benefit. Conclusions: Only a modest proportion of FDA approved drugs have demonstrated substantial NHB at time of approval. As further evidence was published, a substantial proportion of indications have a change in classification of NHB, resulting in a small increase in the overall proportion of indications being deemed to have substantial benefit at 3 years post-approval.