New Analysis Examines Role of Conditional Approvals on Access to Promising Oncology Therapies

Health Canada’s Notice of Compliance with conditions (NOC/c) policy has been effective in providing people in Canada with faster access to new oncology drugs based on promising data. A retrospective cohort analysis published in the Canadian Journal of Health Technologies substantiates this by reviewing the NOC/c approvals with a focus on the confirmatory study and time to confirmatory evidence submission, as well as how many of these therapies have become standard of care.

This report was produced by the Real-World Evidence (RWE) Oncology Working Group under the auspice of the RWE Steering Committee, which is chaired by Health Canada and Canada’s Drug Agency (CDA-AMC).

The results of this collaborative effort provide an opportunity to understand trends related to Canada’s NOC/c policy and consider options to help address uncertainties or gaps in evidence identified in conditional regulatory approvals. The establishment of the time-limited reimbursement recommendation category for drugs with NOC/c approval, launched in 2023, is 1 example of responses at CDA-AMC to changes in drug development and regulation.

About the NOC/c Policy

Health Canada introduced the NOC/c policy in 1998 to help provide people in Canada with accelerated access to potentially life-saving drugs for serious conditions. Under the policy, Health Canada can issue market approval of a drug based on promising evidence with conditions that a drug sponsor undertake additional studies to confirm the drug’s clinical benefit. Drugs eligible for authorization under this policy can be for rare conditions (frequently, rare cancer indications).

Key Findings

Some key findings in the analysis include the following:

• There is an increasing trend of NOC/c authorizations issued for oncology drugs (11% of the NOC/c issued from 1998 to 2001 were for oncology drugs versus 94% from 2018 to 2021).  
• More than half of the NOC/c authorizations for oncology drugs had all of their conditions lifted within 5 years of the NOC/c issuance. The majority (85.7%) of oncology drugs issued an NOC/c between 2018 and 2021 have become integrated into standard of care guidelines for their respective indications in Canada, showing the important benefit of this regulatory pathway. 
• The analysis highlights some potential approaches to further enhance this regulatory pathway, such as strategies to reduce the time to completion of confirmatory trials and greater consideration of complementary RWE to address evidence gaps.  

In Canada and globally, health technology assessment (HTA) bodies are also working on timely ways to address decision-makers' information needs concerning drugs that regulators approve through expedited pathways. For drugs that receive an NOC/c authorization from Health Canada and meet other specified criteria, CDA-AMC may issue a time-limited reimbursement recommendation. Also, to minimize delays between a regulatory decision and a reimbursement recommendation, drug sponsors can use a parallel review process between Health Canada and CDA-AMC. Through this process, CDA-AMC will accept a drug submission up to 180 days before Health Canada’s anticipated regulatory decision.

These efforts help to ensure that HTA and regulatory pathways are working in parallel and support earlier access to promising new drugs while creating mechanisms to revisit the clinical and economic evidence as it evolves. 

What’s Next

A subsequent and complementary project by the RWE Steering Committee’s Non-Oncology Working Group explores the NOC/c policy in relation to non-oncology drugs. This report will be published later in 2024.