Access to new therapies: FDA accelerated approvals and the corresponding Canadian regulatory and funding decisions.

e19066 Background: Accelerated approval (AA) by the FDA enables earlier access to promising new therapies while evidence generation is ongoing. Health Canada (HC) has a similar process with a Notice of Compliance conditional (NOCc) before full approval (NOC). Canada implemented health technology assessment (HTA) for determination of funding through the pan Canadian Oncology Drug Review (pCODR) in 2011. This study evaluated timelines and decisions from AA approval to HC NOC/c to HTA approval and formulary listing. Methods: FDA AA malignant hematology and oncology approvals from Jan 1 2000-Dec 31 2019 were reviewed. HC decisions were reviewed to determine submission/approval status and dates of NOC/c. pCODR decisions were reviewed to determine submission/approval status and dates of decision. First date of provincial formulary listing was collected. Results: In the 20 year time frame, there were 97 AA by the FDA. Current FDA status: 48 full approval, 44 pending verification and 5 withdrawn. Of the 92 AA that remain approved, HC status: 44 received NOC, 24 NOCc, 24 were not submitted for review, 2 currently under review. Of the 5 AA that were withdrawn; 3 were submitted to HC and received NOC/c and all were subsequently withdrawn. From 2011, 31 of 45 HC approved indications were reviewed at pCODR: 17 received a positive recommendation conditional on cost-effectiveness, 9 not recommended, 3 withdrawn by company, 2 pending final decision. Of the 10 not recommended/withdrawn, 6 were subsequently re-submitted and approved. Time from AA to NOC/c was 9.4 m, time from NOC/c to pCODR decision 15.0 m and time from pCODR decision to first formulary listing 18.4 m. Conclusions: Despite significant timeline differences between AA and HC NOC/c all indications that received AA submitted to Canada were granted regulatory approval. Since 2011, 74% of HC approved therapies submitted to pCODR were recommended and added to formularies. Collaboration between FDA and HC, independent regulatory agencies, through innovative programs such as FDA Project Orbis, may improve time from AA to NOC/c. Stringent criteria for HTA recommendation results in lower approval rates however, provision of additional data at re-submission may enable subsequent approval and adoption of treatment.