FDA Accelerated Approval for Malignant Hematology and Oncology Indications in the Canadian Environment.

Cheryl Ho,Howard J Lim,Dean A Regier

doi:10.3390/curroncol29020036

Cheryl Ho, Howard J Lim + Show 1 more

Open Access

https://doi.org/10.3390/curroncol29020036

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Abstract

Accelerated approval (AA) by the FDA enables earlier access to promising new therapies. Health Canada has a similar process. Canada implemented a national health technology assessment (HTA) for reimbursement decisions in 2011. This study evaluated regulatory and funding timelines and decisions for FDA AA cancer therapies in Canada. The FDA’s AA of malignant hematology and oncology from January 2000–December 2019 was reviewed. Dates from Health Canada, HTA decisions and provincial listings were collected. There were 94 FDA AAs, two of which were subsequently withdrawn. Of the 92 AAs, 70 received full (46)/conditional (24) Health Canada approval, and 22 were not filed. Since the introduction of HTA, 31 out of 45 of Health Canada’s approved indications underwent HTA review: 18 received a positive recommendation conditional on cost-effectiveness, 8 were not recommended and 5 were withdrawn/suspended. The median time from the AA to any Health Canada approval is 9.4 months, from any Health Canada approval to HTA decision is 5.8 months and from HTA decision to the first formulary listing is 12.0 months. The access and timeline for the first formulary listing differences were observed between the USA and Canada due to the decision of pharmaceutical companies to submit (or not) to regulatory/HTA bodies, national procedural delays with different healthcare delivery models and submission timelines. This study demonstrates that there is delayed access to promising new therapies in Canada.

Highlights

Received: 26 November 2021Regulatory approval is one of the critical steps in the complex process of drug development that facilitates access to new medications for diseases
This study examines oncology indications authorized under an AA to Health Canada and panCanadian Oncology Drug Review (pCODR) approvals by assessing timelines and Canadian funding decisions
The date the drug met the requirements for Notice of Compliance with Conditions (NOC/c) or Notice of Compliance (NOC) approval was collected

Summary

Introduction

Received: 26 November 2021Regulatory approval is one of the critical steps in the complex process of drug development that facilitates access to new medications for diseases. The rigorous evaluation of submissions in support of new therapies can be lengthy, but this crucial process enables informed decision making regarding the risks and benefits of treatments. In serious or life-threatening diseases such as cancer, there is tension between the standard approval process and clinicians’ and patients’ demand for early market access to promising and often expensive treatments. To address this issue in the USA, the FDA initiated expedited pathways, including the Accelerated Approval (AA) process [1]. AA enables the approval of therapies for serious illnesses on the basis of a surrogate or intermediate clinical endpoint or in light of non-randomized data while additional evidence is being generated. Common surrogate or intermediate clinical endpoints that have been demonstrated to predict clinical benefit include response rate, duration of response and Accepted: 12 January 2022

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