Objectives
Regulatory agencies may approve promising oncology therapies in the absence of phase III trials, resulting in potential challenges for HTA agencies tasked with developing reimbursement recommendations. This study investigated: 1) Trends in CADTH recommendations for oncology products submitted without phase III data; 2) Agreement between CADTH and NICE recommendations for these products.
Methods
CADTH pan-Canadian Oncology Drug Review (pCODR) reports were reviewed from pCODR inception to May 2021 to identify all submissions lacking phase III data for which a final recommendation was issued. NICE recommendations for the same product/indication pairs were identified, if available. Descriptive statistics were generated for the pCODR submissions. Agreement between pCODR and NICE was assessed with respect to recommendations and associated conditions.
Results
35 of 219 (16%) pCODR submissions were submitted without phase III data, with an increasing trend over time. Nearly 40% of submissions were for hematological cancers. 19 of 35 submissions (54%) were recommended for reimbursement, the majority with conditions. For submissions receiving negative recommendations, key criticisms related to the clinical evidence included: lack of data on key outcomes (OS, PFS); comparative data unavailable; no improvement in quality of life; and phase III trials were considered feasible. Corresponding NICE recommendations were identified for 22 of the 35 pCODR recommendations (63%); 86% of NICE recommendations were positive, all with conditions. The majority (80%) of products with positive CADTH recommendations also received a positive NICE recommendation. Among negative CADTH recommendations, 87% received a positive NICE recommendation.
Conclusions
Just over half of oncology submissions to CADTH without phase III data received a positive reimbursement recommendation, versus 86% of corresponding NICE submissions. Notably, most of the products/indications issued negative recommendations by CADTH were recommended by NICE. Hence, CADTH appears to have a lower tolerance for the uncertainty in clinical evidence when phase III data are lacking.
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